Macular Degeneration - Pipeline Assessment and Market Forecasts to 2017 is an essential source of information and analysis on the global Macular Degeneration market. The report identifies the key trends shaping and driving the global Macular Degeneration market. The report also provides insights on the prevalent competitive landscape and the emerging players expected to significantly alter the market positioning of the current market leaders. Most importantly, the report provides valuable insights on the pipeline products within the global Macular Degeneration sector. This report is built using data and information sourced from proprietary databases, primary and secondary research and in-house analysis by a team of industry experts.
The authors valued the global macular degeneration therapeutics market at $1.3 billion in 2009. It is expected to grow to $3.0 billion with a Compound Annual Growth Rate (CAGR) of 10.4% by 2017. This growth is primarily attributed to the increase in the aged population across the world. Macular degeneration is a common disease that affects people above 50 years of age. Incidence is high in patients who are above 70 years. The growth will be supported by increased competition between the traditionally used off label products and the newly approved drugs and is expected to continue in the future. In 2009, Lucentis (ranibizumab), a blockbuster drug from Genentech, was the leading player in the global macular degeneration therapeutics market. Avastin is one of Genentech's blockbuster drugs, approved for various types of cancer. Avastin was widely used as an off-label drug by patients with macular degeneration before the approval of Lucentis.
Competition in the Macular Degeneration Therapeutics Market is Expected to Intensify The leading companies in the global macular degeneration therapeutics market are Genentech, Osi Eyetech Inc. and QLT Ltd. Together, these companies accounted for more than 95% of the global macular degeneration therapeutics market in 2009.
Genentech, with its blockbuster drug Lucentis, was the market leader with a share of more than 90%, followed by Visudyne (verteporfin). However, companies such as Novartis AG, Bayer/Regeneron Pharma, Adeona Pharma, Ophthotech Corp, Alcon Inc, Pfizer Inc, Acucela Inc, Alimera Sciences, Neurotech Pharma and Allergan Inc have the most technologically advanced products in their pipeline portfolios and are expected to attract most of the investors attention. The global macular degeneration therapeutics market has seen some intense competition in recent times, which is expected to further intensify over the coming years.
GlobalData, the industry analysis specialist, has released its new report, Macular Degeneration - Pipeline Assessment and Market Forecasts to 2017. The report is an essential source of information and analysis on the global Macular Degeneration market. The report identifies the key trends shaping and driving the global Macular Degeneration market. The report also provides insights on the prevalent competitive landscape and the emerging players expected to significantly alter the market positioning of the current market leaders. Most importantly, the report provides valuable insights on the pipeline products within the global Macular Degeneration sector. This report is built using data and information sourced from proprietary databases, primary and secondary research and in-house analysis by Our team of industry experts.
GlobalData valued the global macular degeneration therapeutics market at $1.3 billion in 2009. It is expected to grow to $3.0 billion with a Compound Annual Growth Rate (CAGR) of 10.4% by 2017. This growth is primarily attributed to the increase in the aged population across the world. Macular degeneration is a common disease that affects people above 50 years of age. Incidence is high in patients who are above 70 years. The growth will be supported by increased competition between the traditionally used off label products and the newly approved drugs and is expected to continue in the future. In 2009, Lucentis (ranibizumab), a blockbuster drug from Genentech, was the leading player in the global macular degeneration therapeutics market. Avastin is one of Genentechs blockbuster drugs, approved for various types of cancer. Avastin was widely used as an off-label drug by patients with macular degeneration before the approval of Lucentis.
Scope The report provides information on the key drivers and challenges of the Macular Degeneration market. Its scope includes: Annualized global Macular Degeneration market revenues data from 2001 to 2009, forecast for eight years to 2017. Pipeline analysis data providing a split across the different phases, mechanisms of action being developed and emerging trends. Pipeline candidates fall under major therapeutic classes such as VEGF inhibitors, angiogenesis inhibitors, C5a receptor antagonists, antioxidants, tyrosine kinase inhibitosr, clotting factor inhibitors, cyclooxygenase inhibitors, RPE inhibitors, antioxidants, PDGF inhibitors, chemokine inhibitors, IL-2 inhibitors, retinol protein binders, a51 integrin antagonist, protein C3 inhibitors and mRNA targeting agents. Analysis of the current and future competition in the global Macular Degeneration market. Key market players covered are Novartis AG, Bayer Pharma, Adeona Pharma, Ophthotech Corp., Alcon Inc, Pfizer Inc, Acucela Inc, Alimera Sciences, Neurotech Pharma, and Allergan Inc. Insightful review of the key industry drivers, restraints and challenges.
Each trend is independently researched to provide a qualitative analysis of its implications. Key topics covered include strategic competitor assessment, market characterization, unmet needs and the implications for the Macular Degeneration therapeutics market.
Reasons to buy The report will enhance your decision making capability. It will allow you to: Develop and design your in-licensing and out-licensing strategies through a review of pipeline products and technologies and by identifying the companies with the most robust pipeline. Develop business strategies by understanding the trends shaping and driving the global Macular Degeneration market. Drive revenues by understanding the key trends, innovative products and technologies, market segments and companies likely to impact the global Macular Degeneration market in future. Formulate effective sales and marketing strategies by understanding the competitive landscape and by analyzing the performance of various competitors. Identify emerging players with potentially strong product portfolios and create effective counter-strategies to gain a competitive advantage. Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments and strategic partnerships. What's the next big thing in the global Macular Degeneration market landscape? Identify, understand and capitalize.
Key Topics Covered: 1 Summary 2 Macular Degeneration Therapeutics: Disease Overview 3 Macular Degeneration Therapeutics: Market Characterization 4 Macular Degeneration Therapeutics: Competitive Assessment 5 Macular Degeneration Therapeutics: Pipeline Assessment 6 Global Macular Degeneration Therapeutics: Implications for Future Market Competition 7 Macular Degeneration Therapeutics: Future Players in the Macular Degeneration Disease Market 8 Macular Degeneration Therapeutics: Appendix Companies Mentioned: Alcon Inc. Novartis AG Ophthotech Corporation Pfizer Inc Acucela Inc. Regeneron Pharmaceuticals, Inc.
Showing posts with label Macular Degeneration. Show all posts
Showing posts with label Macular Degeneration. Show all posts
Saturday, January 29, 2011
Sunday, January 23, 2011
Age-related macular degeneration is growing with the graying American population
By LESLIE BARKER GARCIA
DALLAS — Jennifer Tyler was driving down a country road in Oklahoma a couple of years ago when she noticed something odd. The telephone poles all had kinks in them.
"I thought, 'Wow, what's all that about?'" says Tyler, a Dallas fundraising consultant. "They all had distinctive bends in the middle."She pulled off the road and called a friend. "I knew something bad was happening," she says. "It was nothing I could ignore."Her friend referred Tyler to Texas Retina Associates. Tyler made an appointment and was diagnosed as having age-related macular degeneration.
AMD is a disease that causes significant vision loss in 1.75 million Americans. More than 10 million suffer from various forms of this potentially sight-stealing disease, more than glaucoma and cataracts combined, according to the American Macular Degeneration Foundation.By 2020, as the population ages, that number is expected to double.In addition, says Dr. Karl Csaky (pronounced like chalky) of the Retina Foundation of the Southwest, "more severe forms will be more of an issue."
"People who are in their 70s and 80s are extremely active," says Csaky, head of the foundation's Harrington Molecular Laboratory and an ophthalmologist with Texas Retina Associates. "If you had even a relatively small disturbance in vision, that could be extremely devastating in terms of what you're used to doing."Some vision disturbances, such as difficulty reading small print, are inherent with aging. The lenses of our eyes change and can no longer focus close up, so we rely on reading glasses.
Macular degeneration goes beyond the normal aging process. It affects the center of the retina; specifically, the 0.1 percent that's responsible for 99.9 percent of our fine vision, Csaky says."Unfortunately," Csaky says, "the central part is what gives you quality of life, the ability to read, drive, play golf, to discern faces."
He uses the analogy of a camera to explain the difference between normal eye aging and macular degeneration."The lens of the eye is like the lens of a camera," he says. "Cataracts are when the lens of the camera gets cloudy and the picture gets fuzzy. The solution? Change your lens, which is what cataract surgery is."In macular degeneration, your film has gotten old. We can change lenses, put in a special lens, and still have a bad picture because the film is starting to decay."
Macular degeneration has no cure, but those involved in studying and treating it point out glimmers of hope:Though it may cause legal blindness, rendering some victims unable to drive or read, it won't cause total blindness.Most kinds never progress past the initial stage. Diagnosis is not "a death sentence for vision," Csaky says.
Treatments can slow down and sometimes reverse the more severe cases of the condition. Diagnostic tools can catch it in its early stages.To check for it, "We dilate your eyes, use a lens and look for yellow spots (drusen) or pigmentation changes or bleeding," says Dr. Yuguang He, associate professor of ophthalmology at the University of Texas Southwestern Medical Center and a specialist in age-related macular degeneration. "It's not like it takes a blood test or X-ray to diagnose. We just look at it."
The sooner it's found, the better, he says. "There is a window in which we can do treatments."Most treatments are geared toward the more advanced form called wet macular degeneration. The early stage is called dry, which can progress to late-severe dry and sometimes lead to wet.Progression from the dry form to wet can be rapid. Tyler went to a doctor when she first had symptoms, so he was able to diagnose the disease early. Thus, she's been able to receive a relatively new and often successful treatment: a monthly shot directly into each of her eyes."It's psychologically and emotionally very draining," says Tyler, whose father has the dry variety of the disease.
The shot, explains Csaky, her doctor, inhibits a protein responsible for blood vessels growing and bleeding under the retina."We're not curing the disease," he says. "We're inhibiting, with a drug, a protein that's constantly being made, and with the injection it disappears for four to six weeks. The blood vessels are still there in a dormant state. If you don't treat them, they start to grow back."It's not comfortable, but patients tolerate it well, he says, though occasionally "we have a loved one faint who's watching. We use a topical anesthetic and a very, very small needle."
Although there is no surefire way to prevent the disease, studies have shown that eating fish two or more times per week helps reduce the risk, he says. "I take fish oil. I'm 54 years old and take it not only to prevent AMD but also to protect my heart, help my cholesterol. I tell patients, something good for your heart has to be good for your eyes."He also points out the Age-Related Eye Disease study of the National Eye Institution. Participants took large quantities of vitamins A and C, as well as zinc, copper and beta carotene. The regimen neither prevents the disease nor, in the study, did it have any effect on the dry form, but it "slowed the progression of the wet variety by 20 percent," he says.
Tyler, who declined to give her age, manages her ailment as best she can, getting injections and participating in a study. She is constantly aware of her surroundings, on the lookout for any line in a grid that appears curvy or wavy. She checks the spindles on her staircase, "whatever's around me," for possible distortion."It was never even in my mind, as driven and focused as I am in my career, that I wouldn't be responsive to it," she says. "It's your vision for heaven's sake. How scary is that?
"You can work with it and make it a positive thing, or it can devastate you. You make these choices in life. What am I going to do about it? Is it going to define me, or am I going to help define it?"Age-related macular degeneration (AMD) is the No. 1 cause of irreversible vision loss among senior citizens, according to the Retina Foundation of the Southwest. Some risk factors:
Caucasians are more likely to contract AMD than other races."If you're a Caucasian older than 65, there's a 20 percent chance you'll get it," says Dr. Yuguang He, associate professor of ophthalmology at UT Southwestern Medical Center. "If you're Chinese or Japanese, less than 5 percent. African-Americans have it much less than Caucasians."Yet, "as the Japanese adopted our diet, their incidence has gone up," says Dr. Karl Csaky of the Retina Foundation of the Southwest and Texas Retina Associates.
Smoking increases incidence."Cigarette smokers are 20 times more likely to develop AMD than nonsmokers," he says. "The chances are very, very high."
If one eye is affected, the other eye will be, too.It's important to periodically cover up the eye that has it to see whether the other eye is seeing distortions, too.Genetics play a factor."If your parents have it, there's an increased risk you'll get it, but it's not a guarantee," he says. "We're still struggling with the practical implications of these genetic associations other than scaring people."
DALLAS — Jennifer Tyler was driving down a country road in Oklahoma a couple of years ago when she noticed something odd. The telephone poles all had kinks in them.
"I thought, 'Wow, what's all that about?'" says Tyler, a Dallas fundraising consultant. "They all had distinctive bends in the middle."She pulled off the road and called a friend. "I knew something bad was happening," she says. "It was nothing I could ignore."Her friend referred Tyler to Texas Retina Associates. Tyler made an appointment and was diagnosed as having age-related macular degeneration.
AMD is a disease that causes significant vision loss in 1.75 million Americans. More than 10 million suffer from various forms of this potentially sight-stealing disease, more than glaucoma and cataracts combined, according to the American Macular Degeneration Foundation.By 2020, as the population ages, that number is expected to double.In addition, says Dr. Karl Csaky (pronounced like chalky) of the Retina Foundation of the Southwest, "more severe forms will be more of an issue."
"People who are in their 70s and 80s are extremely active," says Csaky, head of the foundation's Harrington Molecular Laboratory and an ophthalmologist with Texas Retina Associates. "If you had even a relatively small disturbance in vision, that could be extremely devastating in terms of what you're used to doing."Some vision disturbances, such as difficulty reading small print, are inherent with aging. The lenses of our eyes change and can no longer focus close up, so we rely on reading glasses.
Macular degeneration goes beyond the normal aging process. It affects the center of the retina; specifically, the 0.1 percent that's responsible for 99.9 percent of our fine vision, Csaky says."Unfortunately," Csaky says, "the central part is what gives you quality of life, the ability to read, drive, play golf, to discern faces."
He uses the analogy of a camera to explain the difference between normal eye aging and macular degeneration."The lens of the eye is like the lens of a camera," he says. "Cataracts are when the lens of the camera gets cloudy and the picture gets fuzzy. The solution? Change your lens, which is what cataract surgery is."In macular degeneration, your film has gotten old. We can change lenses, put in a special lens, and still have a bad picture because the film is starting to decay."
Macular degeneration has no cure, but those involved in studying and treating it point out glimmers of hope:Though it may cause legal blindness, rendering some victims unable to drive or read, it won't cause total blindness.Most kinds never progress past the initial stage. Diagnosis is not "a death sentence for vision," Csaky says.
Treatments can slow down and sometimes reverse the more severe cases of the condition. Diagnostic tools can catch it in its early stages.To check for it, "We dilate your eyes, use a lens and look for yellow spots (drusen) or pigmentation changes or bleeding," says Dr. Yuguang He, associate professor of ophthalmology at the University of Texas Southwestern Medical Center and a specialist in age-related macular degeneration. "It's not like it takes a blood test or X-ray to diagnose. We just look at it."
The sooner it's found, the better, he says. "There is a window in which we can do treatments."Most treatments are geared toward the more advanced form called wet macular degeneration. The early stage is called dry, which can progress to late-severe dry and sometimes lead to wet.Progression from the dry form to wet can be rapid. Tyler went to a doctor when she first had symptoms, so he was able to diagnose the disease early. Thus, she's been able to receive a relatively new and often successful treatment: a monthly shot directly into each of her eyes."It's psychologically and emotionally very draining," says Tyler, whose father has the dry variety of the disease.
The shot, explains Csaky, her doctor, inhibits a protein responsible for blood vessels growing and bleeding under the retina."We're not curing the disease," he says. "We're inhibiting, with a drug, a protein that's constantly being made, and with the injection it disappears for four to six weeks. The blood vessels are still there in a dormant state. If you don't treat them, they start to grow back."It's not comfortable, but patients tolerate it well, he says, though occasionally "we have a loved one faint who's watching. We use a topical anesthetic and a very, very small needle."
Although there is no surefire way to prevent the disease, studies have shown that eating fish two or more times per week helps reduce the risk, he says. "I take fish oil. I'm 54 years old and take it not only to prevent AMD but also to protect my heart, help my cholesterol. I tell patients, something good for your heart has to be good for your eyes."He also points out the Age-Related Eye Disease study of the National Eye Institution. Participants took large quantities of vitamins A and C, as well as zinc, copper and beta carotene. The regimen neither prevents the disease nor, in the study, did it have any effect on the dry form, but it "slowed the progression of the wet variety by 20 percent," he says.
Tyler, who declined to give her age, manages her ailment as best she can, getting injections and participating in a study. She is constantly aware of her surroundings, on the lookout for any line in a grid that appears curvy or wavy. She checks the spindles on her staircase, "whatever's around me," for possible distortion."It was never even in my mind, as driven and focused as I am in my career, that I wouldn't be responsive to it," she says. "It's your vision for heaven's sake. How scary is that?
"You can work with it and make it a positive thing, or it can devastate you. You make these choices in life. What am I going to do about it? Is it going to define me, or am I going to help define it?"Age-related macular degeneration (AMD) is the No. 1 cause of irreversible vision loss among senior citizens, according to the Retina Foundation of the Southwest. Some risk factors:
Caucasians are more likely to contract AMD than other races."If you're a Caucasian older than 65, there's a 20 percent chance you'll get it," says Dr. Yuguang He, associate professor of ophthalmology at UT Southwestern Medical Center. "If you're Chinese or Japanese, less than 5 percent. African-Americans have it much less than Caucasians."Yet, "as the Japanese adopted our diet, their incidence has gone up," says Dr. Karl Csaky of the Retina Foundation of the Southwest and Texas Retina Associates.
Smoking increases incidence."Cigarette smokers are 20 times more likely to develop AMD than nonsmokers," he says. "The chances are very, very high."
If one eye is affected, the other eye will be, too.It's important to periodically cover up the eye that has it to see whether the other eye is seeing distortions, too.Genetics play a factor."If your parents have it, there's an increased risk you'll get it, but it's not a guarantee," he says. "We're still struggling with the practical implications of these genetic associations other than scaring people."
Saturday, January 15, 2011
Aarkstore Enterprise Macular Degeneration, Pipeline Review
by: Pipeline Review,Q4
Aarkstore announce a new report “Macular Degeneration – Pipeline Review, Q4 2010 ” through its vast collection of market research report.
“Macular Degeneration Pipeline Review, Q4 2010”, provides an overview of the Macular Degeneration therapeutic pipeline. This report provides information on the therapeutic development for Macular Degeneration, complete with latest updates, and special features on late-stage and discontinued projects. It also reviews key players involved in the therapeutic development for Macular Degeneration. “Macular Degeneration-Pipeline Review 2010, Q4 2010” is built using data and information sourced from proprietary databases, Company/University websites, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources, put together.
Scope
- A snapshot of the global therapeutic scenario for Macular Degeneration.
- A review of the Macular Degeneration products under development by companies and universities/research institutes based on information derived from company and industry-specific sources.
- Coverage of products based on various stages of development ranging from discovery till registration stages.
- A feature on pipeline projects on the basis of monotherapy and combined therapeutics.
- Coverage of the Macular Degeneration pipeline on the basis of therapeutic class, route of administration and molecule type.
- Profiles of late-stage pipeline products featuring sections on product description, mechanism of action and research & development progress.
- Key discontinued pipeline projects.
- Latest news and deals relating to the products.
- Identify and understand important and diverse types of therapeutics under development for Macular Degeneration.
- Identify emerging players with potentially strong product portfolio and design effective counter-strategies to gain competitive advantage.
- Plan mergers and acquisitions effectively by identifying players with the most promising pipeline.
- Devise corrective measures for pipeline projects by understanding Macular Degeneration pipeline depth and focus of Macular Degeneration therapeutics.
- Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope.
- Modify the therapeutic portfolio by identifying discontinued projects and understanding the factors that drove them from pipeline., provides an overview of the Macular Degeneration therapeutic pipeline. This report provides information on the therapeutic development for Macular Degeneration, complete with latest updates, and special features on late-stage and discontinued projects. It also reviews key players involved in the therapeutic development for Macular Degeneration. “Macular Degeneration-Pipeline Review 2010, Q4 2010” is built using data and information sourced from proprietary databases, Company/University websites, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources, put together.
Aarkstore announce a new report “Macular Degeneration – Pipeline Review, Q4 2010 ” through its vast collection of market research report.
“Macular Degeneration Pipeline Review, Q4 2010”, provides an overview of the Macular Degeneration therapeutic pipeline. This report provides information on the therapeutic development for Macular Degeneration, complete with latest updates, and special features on late-stage and discontinued projects. It also reviews key players involved in the therapeutic development for Macular Degeneration. “Macular Degeneration-Pipeline Review 2010, Q4 2010” is built using data and information sourced from proprietary databases, Company/University websites, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources, put together.
Scope
- A snapshot of the global therapeutic scenario for Macular Degeneration.
- A review of the Macular Degeneration products under development by companies and universities/research institutes based on information derived from company and industry-specific sources.
- Coverage of products based on various stages of development ranging from discovery till registration stages.
- A feature on pipeline projects on the basis of monotherapy and combined therapeutics.
- Coverage of the Macular Degeneration pipeline on the basis of therapeutic class, route of administration and molecule type.
- Profiles of late-stage pipeline products featuring sections on product description, mechanism of action and research & development progress.
- Key discontinued pipeline projects.
- Latest news and deals relating to the products.
- Identify and understand important and diverse types of therapeutics under development for Macular Degeneration.
- Identify emerging players with potentially strong product portfolio and design effective counter-strategies to gain competitive advantage.
- Plan mergers and acquisitions effectively by identifying players with the most promising pipeline.
- Devise corrective measures for pipeline projects by understanding Macular Degeneration pipeline depth and focus of Macular Degeneration therapeutics.
- Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope.
- Modify the therapeutic portfolio by identifying discontinued projects and understanding the factors that drove them from pipeline., provides an overview of the Macular Degeneration therapeutic pipeline. This report provides information on the therapeutic development for Macular Degeneration, complete with latest updates, and special features on late-stage and discontinued projects. It also reviews key players involved in the therapeutic development for Macular Degeneration. “Macular Degeneration-Pipeline Review 2010, Q4 2010” is built using data and information sourced from proprietary databases, Company/University websites, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources, put together.
Sunday, January 9, 2011
FDA Approves Company's Clinical Trial Of hESCs- based Treatment for AMD
by: Advanced Cell Technology, Inc.
Advanced Cell Technology, Inc. (OTCBB: ACTC), a regenerative medicine company based in Marlborough, Mass., announced on January 3 that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application to treat dry age-related macular degeneration (AMD) using retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs).
ACT is now permitted to launch a Phase 1-2 multicenter clinical trial to treat patients with dry AMD, the most common form of macular degeneration in the world.
There are no treatments available for this prevalent disease of an aging global population. According to the company, dry AMD, representing a substantial global market opportunity and afflicts between 10-15 million Americans.
Age-Related Macular Degeneration has two predominant forms, wet and dry. Dry AMD is the most common form, accounting for almost 90 percent of all cases. The progress of dry AMD includes a breakdown or thinning of the layer of RPE cells in the patient’s macula, the region at the center of the retina responsible for high acuity vision. Over time, the progressive loss of RPE cells and accompanying loss of photoreceptors can cause severe vision loss and even blindness.
“ACT is now the first company to receive FDA clearance for two hESC trials, and is now a true translational leader in the field of regenerative medicine,” said interim CEO Gary Rabin. “It marks a major step forward, not just within the stem cell sector, but, potentially for modern healthcare techniques. We plan to proceed into the clinic with both of our hESC-based programs as quickly as possible.”
The Phase 1-2 trial will be a prospective, open-label study that is designed to determine the safety and tolerability of the RPE cells following sub-retinal transplantation into patients with dry AMD. Twelve patients will be enrolled in the study at multiple clinical sites. Sites currently under consideration are the Jules Stein Eye Institute at UCLA, and the Ophthalmology Department at Stanford University School of Medicine. Additional sites may be considered.
“Dry AMD is the leading cause of blindness in individuals over the age of 55,” said Robert Lanza, M.D., chief scientific officer. “As the population ages, the incidence of AMD is expected to double over the next 20 years, further exacerbating this unmet medical need. Using our clinical-grade hESC lines, we are able to generate a virtually unlimited and reproducible supply of healthy RPE cells. Because only a small number of cells (50-200K) are needed to treat each patient, manufacturing and distribution of the therapeutic product is scalable with many similarities to the drug businesses that pharmaceutical companies understand well. Based on our animal model studies, we are very excited about the opportunity to treat patients. In a rat model of macular degeneration, we have seen a remarkable improvement in visual performance over untreated animals, without any adverse effects. We have also maintained near-normal function in a mouse model of Stargardt’s Disease, a form of juvenile macular degeneration. In addition to this trial, we plan to concurrently use our RPE cells in our Phase I/II Clinical Trial for Stargardt’s Disease, which received the green light from the FDA in November. We hope to see a similar benefit in both Stargardt’s Disease and Dry AMD patients.”
ACT’s dry AMD therapeutic program uses RPE cells derived from hESCs to replace the lost RPE cells in the patient’s eyes. ACT’s proprietary RPE cell manufacturing process is protected by a number of broad patents, as is the use of hESC-derived RPE cells for treating macular degeneration. While the initial portion of the clinical trial will focus on safety, in subsequent clinical trials the company hopes to demonstrate that the RPE cells injected into the retinal space will be capable of slowing or halting progression of the disease, and potentially even restoring some visual acuity to patients.
“It is estimated that over ten million Europeans suffer from age-related macular degeneration, representing a vast unmet need and a significant market opportunity,” commented Edmund Mickunas, vice president of regulatory affairs. “We are moving ahead aggressively to seek regulatory clearance from the European Medicines Agency to conduct clinical trials in Europe.”
Advanced Cell Technology, Inc. (OTCBB: ACTC), a regenerative medicine company based in Marlborough, Mass., announced on January 3 that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application to treat dry age-related macular degeneration (AMD) using retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs).
ACT is now permitted to launch a Phase 1-2 multicenter clinical trial to treat patients with dry AMD, the most common form of macular degeneration in the world.
There are no treatments available for this prevalent disease of an aging global population. According to the company, dry AMD, representing a substantial global market opportunity and afflicts between 10-15 million Americans.
Age-Related Macular Degeneration has two predominant forms, wet and dry. Dry AMD is the most common form, accounting for almost 90 percent of all cases. The progress of dry AMD includes a breakdown or thinning of the layer of RPE cells in the patient’s macula, the region at the center of the retina responsible for high acuity vision. Over time, the progressive loss of RPE cells and accompanying loss of photoreceptors can cause severe vision loss and even blindness.
“ACT is now the first company to receive FDA clearance for two hESC trials, and is now a true translational leader in the field of regenerative medicine,” said interim CEO Gary Rabin. “It marks a major step forward, not just within the stem cell sector, but, potentially for modern healthcare techniques. We plan to proceed into the clinic with both of our hESC-based programs as quickly as possible.”
The Phase 1-2 trial will be a prospective, open-label study that is designed to determine the safety and tolerability of the RPE cells following sub-retinal transplantation into patients with dry AMD. Twelve patients will be enrolled in the study at multiple clinical sites. Sites currently under consideration are the Jules Stein Eye Institute at UCLA, and the Ophthalmology Department at Stanford University School of Medicine. Additional sites may be considered.
“Dry AMD is the leading cause of blindness in individuals over the age of 55,” said Robert Lanza, M.D., chief scientific officer. “As the population ages, the incidence of AMD is expected to double over the next 20 years, further exacerbating this unmet medical need. Using our clinical-grade hESC lines, we are able to generate a virtually unlimited and reproducible supply of healthy RPE cells. Because only a small number of cells (50-200K) are needed to treat each patient, manufacturing and distribution of the therapeutic product is scalable with many similarities to the drug businesses that pharmaceutical companies understand well. Based on our animal model studies, we are very excited about the opportunity to treat patients. In a rat model of macular degeneration, we have seen a remarkable improvement in visual performance over untreated animals, without any adverse effects. We have also maintained near-normal function in a mouse model of Stargardt’s Disease, a form of juvenile macular degeneration. In addition to this trial, we plan to concurrently use our RPE cells in our Phase I/II Clinical Trial for Stargardt’s Disease, which received the green light from the FDA in November. We hope to see a similar benefit in both Stargardt’s Disease and Dry AMD patients.”
ACT’s dry AMD therapeutic program uses RPE cells derived from hESCs to replace the lost RPE cells in the patient’s eyes. ACT’s proprietary RPE cell manufacturing process is protected by a number of broad patents, as is the use of hESC-derived RPE cells for treating macular degeneration. While the initial portion of the clinical trial will focus on safety, in subsequent clinical trials the company hopes to demonstrate that the RPE cells injected into the retinal space will be capable of slowing or halting progression of the disease, and potentially even restoring some visual acuity to patients.
“It is estimated that over ten million Europeans suffer from age-related macular degeneration, representing a vast unmet need and a significant market opportunity,” commented Edmund Mickunas, vice president of regulatory affairs. “We are moving ahead aggressively to seek regulatory clearance from the European Medicines Agency to conduct clinical trials in Europe.”
Sunday, January 2, 2011
Case Western Reserve receives $10 million to study Retina Disease
by: Jessica Studeny
The Departments of Pharmacology and Ophthalmology and Visual Sciences at Case Western Reserve University School of Medicine have been awarded a $10.1 million grant from the National Eye Institute (NEI) to research and develop new treatments for diseases of the retina, a leading cause of blindness.
"The grant strongly positions the School of Medicine and collaborating organizations to play a significant role in advancing the treatment of retinal diseases in order to restore quality of life to countless patients," said Jonathan H. Lass, MD, professor and chair of the Department of Ophthalmology and Visual Sciences at Case Western Reserve School of Medicine and director of the University Hospitals Eye Institute. "It is the largest grant of its kind ever awarded to the university by the National Eye Institute, a tremendous achievement."
The NEI, part of the National Institutes of Health (NIH), will award the grant over five years, funding the work of researchers in the Departments of Pharmacology, Ophthalmology, and Biomedical Engineering at the School of Medicine who are working in collaboration with the Retinal Therapeutics Study Group. This interdisciplinary consortium of investigators is screening FDA-approved drugs for their potential application to the treatment of eye diseases affecting the retina.
The combined group, which also includes researchers from the Cincinnati Drug Discovery Center, the University of Pennsylvania and Washington University, aims to accelerate the rate at which basic science discoveries are used to develop new therapies for complex retinal disorders and diseases.
Conditions affecting the retina, the tissue in back of the eye responsible for vision, are a primary cause of blindness in adults in the United States. Such diseases include age-related macular degeneration (AMD), the main cause of blindness in adults over the age of 55. More than 1.3 million people in the U.S. are legally blind and 8 to 10 million aging individuals show signs of developing AMD, an incurable eye disease characterized by damage to the retina and the loss of central daylight vision.
"The research being funded by the NIH is critical to availing patients of new, more effective treatments, particularly for diseases like AMD, for which there is currently no cure," says Krzysztof Palczewski, PhD, John H. Hord Professor, chair of the Department of Pharmacology, and principal investigator and director of the research funded by the new NEI grant. "Our goal is to develop new drugs based on the screening of FDA-approved drugs to evaluate their effectiveness in treating retinal diseases."
Researchers will employ a range of scientific expertise and state-of-the-art physiological, chemical and analytical imaging technologies to test potential retinal disease therapies in basic research models. A non-invasive imaging technology developed at Case Western Reserve by Dr. Palczewski's research group will facilitate monitoring of the retina to detect molecular changes, defects, or harmful toxins in the retina.
FDA-approved drugs will be screened for their potential application in research models engineered to mimic conditions such as AMD, Stargardt's disease (a form of inherited juvenile macular degeneration) and retinitis pigmentosa (a group of inherited retinal disorders characterized by progressive peripheral vision loss). The drug-screening process is designed to accelerate the process of drug development before clinical testing in humans.
Dr. Palczewski is working with researcher Akiko Maeda, MD, PhD, senior instructor in the Departments of Ophthalmology and Pharmacology; and Zheng-Rong Lu, PhD, professor of biomedical engineering; to hone in on existing drugs with chemical properties and initial research results that suggest they may provide a basis for developing new drugs for treating retinal diseases like AMD.
The research builds upon previous work in Dr. Palczewski's laboratory which identified mechanisms in the eye responsible for metabolizing vitamin A, an essential step in triggering the nerve signals sent to the brain to enable vision. Researchers determined that in healthy patients, this visual cycle operates rapidly. However, in older patients and those with AMD and Stargardt-like diseases, one of the critical biochemical reactions in the series that recycle vitamin A is slowed. This allows a toxic byproduct produced by the breakdown of vitamin A to accumulate, which damages the retina over time, probably contributing to the development of AMD and/or impairing vision.
The combined teams in Ophthalmology and Pharmacology are now looking for drugs that can target the mechanism that captures toxic vitamin A metabolites, to neutralize and counter the build-up of any visual cycle toxic byproduct, as a means of preventing or controlling retinal diseases. "Current treatments for AMD focus on management of the late stages of the disease. These studies could result in treatments at the earlier stages and save more vision as a result" says Dr. Lass.
In addition to testing existing FDA-approved drugs for their ability to reduce toxic substances within the retina, priorities for the NIH-funded research also include evaluating existing FDA-approved drugs as potential lead compounds for retinal diseases because these drugs have already been proven safe and effective in basic research and clinical studies for other indications. Researchers will likewise assess the ability of potential compounds to penetrate and remain in the eye without negatively affecting vision and they will explore and develop new drug delivery systems to achieve and maintain therapeutic drug concentrations in the eye.
"We have an idea as to what drugs could be most effective for our purposes," said Dr. Maeda, who relocated from Japan to the United States to work with Dr. Palczewski. She is the study's co-principal investigator and a group leader who is responsible for basic research models. "During my clinical practice in ophthalmology, I was very frustrated with the lack of treatment options for many patients with retinal degenerative diseases, and I became determined to dedicate myself to developing new treatments for these patients through basic science research. I am very excited to develop our ideas for treating currently incurable retinal diseases."
Dr. Palczewski and his team have already examined 24 FDA-approved drugs, from antibiotics to drugs fighting cancer and infectious diseases, for their ability to attack the buildup of harmful toxins in the retina. These studies were done with mice that were genetically engineered to mimic Stargardt's disease. At least 16 of the drugs tested have already demonstrated the potential to limit the progression of retinal diseases. The resulting data provided the basis for funding the $10 million NIH grant request.
About Case Western Reserve University School of Medicine
Founded in 1843, Case Western Reserve University School of Medicine is the largest medical research institution in Ohio and is among the nation's top medical schools for research funding from the National Institutes of Health. The School of Medicine is recognized throughout the international medical community for outstanding achievements in teaching. The School's innovative and pioneering Western Reserve2 curriculum interweaves four themes--research and scholarship, clinical mastery, leadership, and civic professionalism--to prepare students for the practice of evidence-based medicine in the rapidly changing health care environment of the 21st century. Nine Nobel Laureates have been affiliated with the school of medicine.
Annually, the School of Medicine trains more than 800 MD and MD/PhD students and ranks in the top 20 among U.S. research-oriented medical schools as designated by U.S. News & World Report "Guide to Graduate Education."
The School of Medicine's primary affiliate is University Hospitals Case Medical Center and is additionally affiliated with MetroHealth Medical Center, the Louis Stokes Cleveland Department of Veterans Affairs Medical Center, and the Cleveland Clinic, with which it established the Cleveland Clinic Lerner College of Medicine of Case Western Reserve University in 2002.
The Departments of Pharmacology and Ophthalmology and Visual Sciences at Case Western Reserve University School of Medicine have been awarded a $10.1 million grant from the National Eye Institute (NEI) to research and develop new treatments for diseases of the retina, a leading cause of blindness.
"The grant strongly positions the School of Medicine and collaborating organizations to play a significant role in advancing the treatment of retinal diseases in order to restore quality of life to countless patients," said Jonathan H. Lass, MD, professor and chair of the Department of Ophthalmology and Visual Sciences at Case Western Reserve School of Medicine and director of the University Hospitals Eye Institute. "It is the largest grant of its kind ever awarded to the university by the National Eye Institute, a tremendous achievement."
The NEI, part of the National Institutes of Health (NIH), will award the grant over five years, funding the work of researchers in the Departments of Pharmacology, Ophthalmology, and Biomedical Engineering at the School of Medicine who are working in collaboration with the Retinal Therapeutics Study Group. This interdisciplinary consortium of investigators is screening FDA-approved drugs for their potential application to the treatment of eye diseases affecting the retina.
The combined group, which also includes researchers from the Cincinnati Drug Discovery Center, the University of Pennsylvania and Washington University, aims to accelerate the rate at which basic science discoveries are used to develop new therapies for complex retinal disorders and diseases.
Conditions affecting the retina, the tissue in back of the eye responsible for vision, are a primary cause of blindness in adults in the United States. Such diseases include age-related macular degeneration (AMD), the main cause of blindness in adults over the age of 55. More than 1.3 million people in the U.S. are legally blind and 8 to 10 million aging individuals show signs of developing AMD, an incurable eye disease characterized by damage to the retina and the loss of central daylight vision.
"The research being funded by the NIH is critical to availing patients of new, more effective treatments, particularly for diseases like AMD, for which there is currently no cure," says Krzysztof Palczewski, PhD, John H. Hord Professor, chair of the Department of Pharmacology, and principal investigator and director of the research funded by the new NEI grant. "Our goal is to develop new drugs based on the screening of FDA-approved drugs to evaluate their effectiveness in treating retinal diseases."
Researchers will employ a range of scientific expertise and state-of-the-art physiological, chemical and analytical imaging technologies to test potential retinal disease therapies in basic research models. A non-invasive imaging technology developed at Case Western Reserve by Dr. Palczewski's research group will facilitate monitoring of the retina to detect molecular changes, defects, or harmful toxins in the retina.
FDA-approved drugs will be screened for their potential application in research models engineered to mimic conditions such as AMD, Stargardt's disease (a form of inherited juvenile macular degeneration) and retinitis pigmentosa (a group of inherited retinal disorders characterized by progressive peripheral vision loss). The drug-screening process is designed to accelerate the process of drug development before clinical testing in humans.
Dr. Palczewski is working with researcher Akiko Maeda, MD, PhD, senior instructor in the Departments of Ophthalmology and Pharmacology; and Zheng-Rong Lu, PhD, professor of biomedical engineering; to hone in on existing drugs with chemical properties and initial research results that suggest they may provide a basis for developing new drugs for treating retinal diseases like AMD.
The research builds upon previous work in Dr. Palczewski's laboratory which identified mechanisms in the eye responsible for metabolizing vitamin A, an essential step in triggering the nerve signals sent to the brain to enable vision. Researchers determined that in healthy patients, this visual cycle operates rapidly. However, in older patients and those with AMD and Stargardt-like diseases, one of the critical biochemical reactions in the series that recycle vitamin A is slowed. This allows a toxic byproduct produced by the breakdown of vitamin A to accumulate, which damages the retina over time, probably contributing to the development of AMD and/or impairing vision.
The combined teams in Ophthalmology and Pharmacology are now looking for drugs that can target the mechanism that captures toxic vitamin A metabolites, to neutralize and counter the build-up of any visual cycle toxic byproduct, as a means of preventing or controlling retinal diseases. "Current treatments for AMD focus on management of the late stages of the disease. These studies could result in treatments at the earlier stages and save more vision as a result" says Dr. Lass.
In addition to testing existing FDA-approved drugs for their ability to reduce toxic substances within the retina, priorities for the NIH-funded research also include evaluating existing FDA-approved drugs as potential lead compounds for retinal diseases because these drugs have already been proven safe and effective in basic research and clinical studies for other indications. Researchers will likewise assess the ability of potential compounds to penetrate and remain in the eye without negatively affecting vision and they will explore and develop new drug delivery systems to achieve and maintain therapeutic drug concentrations in the eye.
"We have an idea as to what drugs could be most effective for our purposes," said Dr. Maeda, who relocated from Japan to the United States to work with Dr. Palczewski. She is the study's co-principal investigator and a group leader who is responsible for basic research models. "During my clinical practice in ophthalmology, I was very frustrated with the lack of treatment options for many patients with retinal degenerative diseases, and I became determined to dedicate myself to developing new treatments for these patients through basic science research. I am very excited to develop our ideas for treating currently incurable retinal diseases."
Dr. Palczewski and his team have already examined 24 FDA-approved drugs, from antibiotics to drugs fighting cancer and infectious diseases, for their ability to attack the buildup of harmful toxins in the retina. These studies were done with mice that were genetically engineered to mimic Stargardt's disease. At least 16 of the drugs tested have already demonstrated the potential to limit the progression of retinal diseases. The resulting data provided the basis for funding the $10 million NIH grant request.
About Case Western Reserve University School of Medicine
Founded in 1843, Case Western Reserve University School of Medicine is the largest medical research institution in Ohio and is among the nation's top medical schools for research funding from the National Institutes of Health. The School of Medicine is recognized throughout the international medical community for outstanding achievements in teaching. The School's innovative and pioneering Western Reserve2 curriculum interweaves four themes--research and scholarship, clinical mastery, leadership, and civic professionalism--to prepare students for the practice of evidence-based medicine in the rapidly changing health care environment of the 21st century. Nine Nobel Laureates have been affiliated with the school of medicine.
Annually, the School of Medicine trains more than 800 MD and MD/PhD students and ranks in the top 20 among U.S. research-oriented medical schools as designated by U.S. News & World Report "Guide to Graduate Education."
The School of Medicine's primary affiliate is University Hospitals Case Medical Center and is additionally affiliated with MetroHealth Medical Center, the Louis Stokes Cleveland Department of Veterans Affairs Medical Center, and the Cleveland Clinic, with which it established the Cleveland Clinic Lerner College of Medicine of Case Western Reserve University in 2002.
Monday, December 27, 2010
Age-Related Macular Degeneration Research from University of Bristol
by: University
"Neovascular age-related macular degeneration (nvAMD) is a chronic, progressive disease of the central retina, and its prevalence is expected to rise with the aging population. Using a bottom-up approach based on retrospective data, this cross-sectional study estimated average annual direct costs of nvAMD to be A 4,047 pound, and average annual indirect costs to be A 449 pound," scientists in Bristol, the United Kingdom report.
"An attempt to measure intangible costs through willingness-to-pay yielded a lower response rate and estimated intangible costs to be 11.5% of monthly income. Direct costs were significantly higher for male participants, for those who have mild or moderate visual impairment in both eyes, and for those who have been diagnosed for a shorter time," wrote K.M. Ke and colleagues, University of Bristol.
The researchers concluded: "The findings of this study suggest that the availability of early diagnosis, effective treatment, support services, and sustained research into the management of nvAMD may reduce the burden of visual impairment caused by nvAMD to affected individuals and the state."
Ke and colleagues published their study in European Journal of Health Economics (The direct, indirect and intangible costs of visual impairment caused by neovascular age-related macular degeneration. European Journal of Health Economics, 2010;11(6):525-531).
For additional information, contact K.M. Ke, University of Bristol, Bristol Dental School, Dept. of Oral & Dental Science, Lower Maudlin St., Bristol BS1 2LY, Avon, UK.
The publisher's contact information for the European Journal of Health Economics is: Springer, 233 Spring St., New York, NY 10013, USA.
Keywords: City:Bristol, Country:United Kingdom, Age-Related Macular Degeneration, Retinal Degeneration, Retinal Diseases
"Neovascular age-related macular degeneration (nvAMD) is a chronic, progressive disease of the central retina, and its prevalence is expected to rise with the aging population. Using a bottom-up approach based on retrospective data, this cross-sectional study estimated average annual direct costs of nvAMD to be A 4,047 pound, and average annual indirect costs to be A 449 pound," scientists in Bristol, the United Kingdom report.
"An attempt to measure intangible costs through willingness-to-pay yielded a lower response rate and estimated intangible costs to be 11.5% of monthly income. Direct costs were significantly higher for male participants, for those who have mild or moderate visual impairment in both eyes, and for those who have been diagnosed for a shorter time," wrote K.M. Ke and colleagues, University of Bristol.
The researchers concluded: "The findings of this study suggest that the availability of early diagnosis, effective treatment, support services, and sustained research into the management of nvAMD may reduce the burden of visual impairment caused by nvAMD to affected individuals and the state."
Ke and colleagues published their study in European Journal of Health Economics (The direct, indirect and intangible costs of visual impairment caused by neovascular age-related macular degeneration. European Journal of Health Economics, 2010;11(6):525-531).
For additional information, contact K.M. Ke, University of Bristol, Bristol Dental School, Dept. of Oral & Dental Science, Lower Maudlin St., Bristol BS1 2LY, Avon, UK.
The publisher's contact information for the European Journal of Health Economics is: Springer, 233 Spring St., New York, NY 10013, USA.
Keywords: City:Bristol, Country:United Kingdom, Age-Related Macular Degeneration, Retinal Degeneration, Retinal Diseases
Monday, December 20, 2010
Genetic Testing for AMD is here Today
by:Diana Shechtman OD FAAO & Steven Ferrucci OD FAAO
Age-Related macular degeneration (AMD) is a progressive disease and the leading cause of vision loss among the elderly, affecting central vision required for daily activities such as driving and reading. There are a number of factors affecting AMD, such as advanced age, smoking, UV exposure, overall health (that contribute to high blood pressure, obesity, diet) and family history. Many factors may be modified and yet others like genetics cannot. Although AMD may seem to be hereditary in some families and not others, genetics have been shown to contribute significantly to the disease. Multiple twin and sibling studies have collaborated to the familial nature of the disease. First-degree relatives of patients with AMD are at a significantly increased risk for the disease. Furthermore, large epidemiological studies have suggested a strong genetic risk factor for AMD. In fact, the risk of developing AMD increases 4 fold among patients with a positive family history. In 2005 a breakthrough occurred in the area of genetic research and AMD; Klein and associates discovered a strong link between AMD and certain genetic variants. Similarly, numerous other genes have been implicated in AMD, which may increase the risk of AMD up to 70%.
Until recently there was no test to help determined patient’s inherited risk for AMD. Today, Macula Risk (ArcticDX, Toronto, Ontario) is a genetic test specifically designed to determine genetic predisposition to AMD and vision loss attributed to the more advanced stage of the disease.
Macula Risk genetic test separates individuals into one of 5 macula risk (MR) categories, with MR 3 through 5 representing an increased risk for the more advance stage of the disease. This accounts for approximately 20% of the general population. MR1 has less than a 5% risk of the advanced stage of the disease, while MR 5 carries greater than 55% risk. These results can aid the doctor in devising a specific management plan and follow-up protocol in order to reassure early intervention to prevent vision loss.
The test only requires a simple in-office cheek swab, which is sent directly to the genetic lab. The report includes test results and written genetic support information (including access to genetic counseling). Macula Risk genetic testing is covered by most insurance providers, including Medicare, as long as the specific diagnosis (ICD-9) are identified by the doctor. The prognostic genetic test is intended for patients who have a diagnosis of early or intermediate AMD. Thus, the “at risk patient,” would have to pay a fee for the test.
Despite our best efforts and new treatment options available today, many people are still losing vision from AMD. New advancements in the area of AMD are becoming an integral part in preventing future visual deterioration. AMD is affected by both environmental and genetic factors that interact with one another to determine prevalence and progression of the disease. Furthermore, at this time we do not know all of the genes linked to AMD. Hence, genetic testing in the area of AMD is only a risk indicator and cannot predict “without a shadow of a doubt,” which patients will and which will not develop the disease. However, this test provides a genetic profile screening to help identify those at risk as well as aids in tailoring a distinct management approach for those with the disease. With various researches devoted to treatment options for AMD, genetic testing in the area of AMD is at the frontier of providing crucial information.
REFERENCES
1. Swaroop, Branham KE, Chen W, Abecasis G. Genetic susceptibility to age-related macular degeneration: a paradigm for dissecting complex disease traits. Hum Mol Genet 2007; 16: 174-82.
2. Klein ML, Mauldin WM, Stoumbos VD. Heredity and age-related macular degeneration. Observations in monozygotic twins. Arch Ophthalmol. 1994; 112: 932-7.
3. Meyers SM, Greene T, Gutman FA. A twin study of age-related macular degeneration. Am J Ophthalmol. 1995; 120: 757-66.
4. Heiba IM, Elston RC, Klein BE, et al. Sibling correlations and segregation analysis of age-related maculopathy: The Beaver Dam Eye Study. Genet Epidemiol. 1994; 11: 51-67.
5. Klaver CC, Wolfs RC, Assink JJ, et al. Genetic risk of age-related maculopathy. Population-based familial aggregation study. Arch Ophthalmol. 1998; 116: 1646-51.
6. Seddon JM, Ajani UA, Mitchell BD. Familial aggregation of age-related maculopathy. Am J Ophthalmol. 1997; 123: 199-206.
7. Klein RJ, Zeiss C, Chew EY, et al. Complement Factor H Polymorphism in Age-Related Macular Degeneration. Science 2005; 308: 385-389.
8. Seddon JM, Reynolds R, Maller J, Fagerness JA, Daly MJ, Rosner B. Prediction model for prevalence and incidence of advanced age-related macular degeneration based on genetic, demographic, and environmental variables. Invest Ophthalmol Vis Sci 2009; 50 (65): 2044-53.
9. www.macularisk.com/en/physicians/order.html (accessed Dec. 14th , 2010)
Age-Related macular degeneration (AMD) is a progressive disease and the leading cause of vision loss among the elderly, affecting central vision required for daily activities such as driving and reading. There are a number of factors affecting AMD, such as advanced age, smoking, UV exposure, overall health (that contribute to high blood pressure, obesity, diet) and family history. Many factors may be modified and yet others like genetics cannot. Although AMD may seem to be hereditary in some families and not others, genetics have been shown to contribute significantly to the disease. Multiple twin and sibling studies have collaborated to the familial nature of the disease. First-degree relatives of patients with AMD are at a significantly increased risk for the disease. Furthermore, large epidemiological studies have suggested a strong genetic risk factor for AMD. In fact, the risk of developing AMD increases 4 fold among patients with a positive family history. In 2005 a breakthrough occurred in the area of genetic research and AMD; Klein and associates discovered a strong link between AMD and certain genetic variants. Similarly, numerous other genes have been implicated in AMD, which may increase the risk of AMD up to 70%.
Until recently there was no test to help determined patient’s inherited risk for AMD. Today, Macula Risk (ArcticDX, Toronto, Ontario) is a genetic test specifically designed to determine genetic predisposition to AMD and vision loss attributed to the more advanced stage of the disease.
Macula Risk genetic test separates individuals into one of 5 macula risk (MR) categories, with MR 3 through 5 representing an increased risk for the more advance stage of the disease. This accounts for approximately 20% of the general population. MR1 has less than a 5% risk of the advanced stage of the disease, while MR 5 carries greater than 55% risk. These results can aid the doctor in devising a specific management plan and follow-up protocol in order to reassure early intervention to prevent vision loss.
The test only requires a simple in-office cheek swab, which is sent directly to the genetic lab. The report includes test results and written genetic support information (including access to genetic counseling). Macula Risk genetic testing is covered by most insurance providers, including Medicare, as long as the specific diagnosis (ICD-9) are identified by the doctor. The prognostic genetic test is intended for patients who have a diagnosis of early or intermediate AMD. Thus, the “at risk patient,” would have to pay a fee for the test.
Despite our best efforts and new treatment options available today, many people are still losing vision from AMD. New advancements in the area of AMD are becoming an integral part in preventing future visual deterioration. AMD is affected by both environmental and genetic factors that interact with one another to determine prevalence and progression of the disease. Furthermore, at this time we do not know all of the genes linked to AMD. Hence, genetic testing in the area of AMD is only a risk indicator and cannot predict “without a shadow of a doubt,” which patients will and which will not develop the disease. However, this test provides a genetic profile screening to help identify those at risk as well as aids in tailoring a distinct management approach for those with the disease. With various researches devoted to treatment options for AMD, genetic testing in the area of AMD is at the frontier of providing crucial information.
REFERENCES
1. Swaroop, Branham KE, Chen W, Abecasis G. Genetic susceptibility to age-related macular degeneration: a paradigm for dissecting complex disease traits. Hum Mol Genet 2007; 16: 174-82.
2. Klein ML, Mauldin WM, Stoumbos VD. Heredity and age-related macular degeneration. Observations in monozygotic twins. Arch Ophthalmol. 1994; 112: 932-7.
3. Meyers SM, Greene T, Gutman FA. A twin study of age-related macular degeneration. Am J Ophthalmol. 1995; 120: 757-66.
4. Heiba IM, Elston RC, Klein BE, et al. Sibling correlations and segregation analysis of age-related maculopathy: The Beaver Dam Eye Study. Genet Epidemiol. 1994; 11: 51-67.
5. Klaver CC, Wolfs RC, Assink JJ, et al. Genetic risk of age-related maculopathy. Population-based familial aggregation study. Arch Ophthalmol. 1998; 116: 1646-51.
6. Seddon JM, Ajani UA, Mitchell BD. Familial aggregation of age-related maculopathy. Am J Ophthalmol. 1997; 123: 199-206.
7. Klein RJ, Zeiss C, Chew EY, et al. Complement Factor H Polymorphism in Age-Related Macular Degeneration. Science 2005; 308: 385-389.
8. Seddon JM, Reynolds R, Maller J, Fagerness JA, Daly MJ, Rosner B. Prediction model for prevalence and incidence of advanced age-related macular degeneration based on genetic, demographic, and environmental variables. Invest Ophthalmol Vis Sci 2009; 50 (65): 2044-53.
9. www.macularisk.com/en/physicians/order.html (accessed Dec. 14th , 2010)
Saturday, December 11, 2010
The link Between Age-Related Macular Degeneration and Cardiovascular Disease
by:Johns Hopkins Health Alert
Several studies have found that people with age-related macular degeneration (AMD) are more likely to have heart disease than those without age-related macular degeneration. A U.S. Medicare study, for example, found that elderly people with age-related macular degeneration were 20% more likely to have a heart attack than their counterparts who didn't have age-related macular degeneration. And a large Australian study, reported in the British Journal of Ophthalmology, suggests that age-related macular degeneration increases the risk of dying from coronary heart disease or stroke.
The researchers used data from nearly 3,000 participants, age 49 and older, in the Blue Mountains Eye Study -- none with a history of coronary heart disease or stroke at enrollment. They found that people younger than age 75 who had early signs of age-related macular degeneration at the study's start were twice as likely to die of coronary heart disease as their counterparts who did not have early signs. However, there was no increased risk of dying of a stroke in this group.
People with late age-related macular degeneration at the beginning of the study who were under age 75 had five times the risk of dying of coronary heart disease and 10 times the risk of dying of a stroke. The link between late age-related macular degeneration and risk of death from cardiovascular disease should be interpreted cautiously, warn the researchers, because there were so few people with late age-related macular degeneration at study entry.
Surprisingly, the researchers found no increased risk of death from cardiovascular disease among people over age 75 with age-related macular degeneration. They speculate that past 75, patients died of other serious health conditions that overshadowed the connection.
Take-away message: What's behind the link between age-related macular degeneration and cardiovascular disease? Some researchers suspect that age-related macular degeneration and cardiovascular diseases may share a common genesis: for instance, atherosclerosis, inflammation, and oxidative stress (the cell damage caused by free radicals) are known to affect both conditions. Another alternative: age-related macular degeneration may simply be a disease of aging.
Medical Disclaimer: This information is not intended to be substituted for the advice of a physician. Johns Hopkins Health Alerts
Several studies have found that people with age-related macular degeneration (AMD) are more likely to have heart disease than those without age-related macular degeneration. A U.S. Medicare study, for example, found that elderly people with age-related macular degeneration were 20% more likely to have a heart attack than their counterparts who didn't have age-related macular degeneration. And a large Australian study, reported in the British Journal of Ophthalmology, suggests that age-related macular degeneration increases the risk of dying from coronary heart disease or stroke.
The researchers used data from nearly 3,000 participants, age 49 and older, in the Blue Mountains Eye Study -- none with a history of coronary heart disease or stroke at enrollment. They found that people younger than age 75 who had early signs of age-related macular degeneration at the study's start were twice as likely to die of coronary heart disease as their counterparts who did not have early signs. However, there was no increased risk of dying of a stroke in this group.
People with late age-related macular degeneration at the beginning of the study who were under age 75 had five times the risk of dying of coronary heart disease and 10 times the risk of dying of a stroke. The link between late age-related macular degeneration and risk of death from cardiovascular disease should be interpreted cautiously, warn the researchers, because there were so few people with late age-related macular degeneration at study entry.
Surprisingly, the researchers found no increased risk of death from cardiovascular disease among people over age 75 with age-related macular degeneration. They speculate that past 75, patients died of other serious health conditions that overshadowed the connection.
Take-away message: What's behind the link between age-related macular degeneration and cardiovascular disease? Some researchers suspect that age-related macular degeneration and cardiovascular diseases may share a common genesis: for instance, atherosclerosis, inflammation, and oxidative stress (the cell damage caused by free radicals) are known to affect both conditions. Another alternative: age-related macular degeneration may simply be a disease of aging.
Medical Disclaimer: This information is not intended to be substituted for the advice of a physician. Johns Hopkins Health Alerts
Tuesday, December 7, 2010
SiemCells, Inc. Expands SC Proven(R) Product Portfolio With launch of Proprietary Human Cell Detection Antibodies
Posted by: StemCells,Inc.
StemCells, Inc. (Nasdaq: STEM | PowerRating) announced today the launch of STEM101(TM), STEM121(TM) and STEM123(TM), three new antibody reagents that significantly improve the visualization of human cells, including human stem cells and their progeny. These high potency antibodies are the latest additions to the Company's growing SC Proven(R) portfolio of research products, and provide powerful, cost-effective tools for the detection, tracking and characterization of human cells both in vitro and when transplanted into animal models of human diseases.
"The commercial launch of these proprietary antibodies demonstrates our ongoing commitment to broaden our portfolio of innovative research products and to take advantage of the growing market for research-grade cells, media and reagents," said Stewart Craig, Senior Vice President, Development and Operations at StemCells, Inc. "Their utility has been proven by our scientists and by independent academic collaborators in the conduct of their research and development activities. These antibodies have also become the cornerstone of our extensive preclinical studies, which have allowed us to successfully advance our stem cell therapeutic candidates into multiple clinical trials. There is considerable demand for these reagents, so we are pleased to now make them available to the scientific community at large."
STEM101, STEM121 and STEM123 are human-specific mouse monoclonal antibodies that have been extensively used to detect the engraftment, migration and differentiation of human neural stem cells and human liver engrafting cells (hLEC(TM)) transplanted into rodents.1,2,3,4,5 These antibodies can be used for immunohistochemistry and immunofluorescence applications such as:
-- Quantifying the location and number of engrafted cells
-- Tracking the migration pattern of engrafted cells
-- Determining the nature of engrafted cells
-- Identifying specific differentiated human-derived cells such as
astrocytes
STEM101 recognizes the Ku80 protein found in human nuclei, STEM121 recognizes a cytoplasmic protein of human cells, and STEM123 recognizes human glial fibrillary acidic protein (GFAP).
About SC Proven Products
The SC Proven product portfolio comprises a range of specialty cell culture products that enable the standardized and reproducible production and propagation of highly purified stem cells and their differentiated progeny, as well as reagents for cell detection, isolation and characterization.
References
-- Kelly S, et al. Transplanted human fetal neural stem cells survive,
migrate, and differentiate in ischemic rat cerebral cortex. PNAS (2004)
101:11839-11844
-- Cummings B.J., et al. Human neural stem cells differentiate and promote
locomotor recovery in spinal cord-injured mice. PNAS (2005) 102:
14069-14074
-- Tamaki S.J., et al., Neuroprotection of Host Cells by Human Central
Nervous System Stem Cells in a Mouse Model of Infantile Neuronal Ceroid
Lipofuscinosis. Cell Stem Cell (2009) 5:310-319
-- Kallur T., et al. Human Fetal Cortical and Striatal Neural Stem Cells
Generate Region-Specific Neurons In Vitro and Differentiate Extensively
to Neurons After Intrastriatal Transplantation in Neonatal Rats. J
Neurosci Res. (2006) 84:1630-1644
-- Salazar D.L., et al., Human Neural Stem Cells Differentiate and Promote
Locomotor Recovery in an Early Chronic Spinal Cord Injury NOD-scid Mouse
Model. PLoS ONE (2010) 5: e12272
About StemCells, Inc.
StemCells, Inc. is engaged in the research, development, and commercialization of cell-based therapeutics and tools for use in stem cell-based research and drug discovery. In its therapeutic product development programs, StemCells is targeting disorders of the central nervous system and the liver. StemCells' lead product candidate, HuCNS-SC(R) cells (purified human neural stem cells), is currently in clinical development for the treatment of two fatal neurodegenerative disorders in children, and in preclinical development for spinal cord injury and retinal disorders such as age-related macular degeneration. StemCells also markets research products, including media and reagents, under the SC Proven(R)brand, and is developing stem cell-based assay platforms for use in pharmaceutical research, drug discovery and drug development. Further information about StemCells is available at www.stemcellsinc.com.
The StemCells, Inc. logo is available at http://www.globenewswire.com/newsroom/prs/?pkgid=7014
Apart from statements of historical fact, the text of this press release constitutes forward-looking statements within the meaning of the U.S. securities laws, and is subject to the safe harbors created therein. These statements include, but are not limited to, statements regarding the ability of STEM101, STEM121 and STEM123 to enable and improve the visualization, detection, tracking and characterization of human cells both in vitro and when transplanted into animal models of human diseases; the clinical development of the Company's HuCNS-SC cells; the prospects for the Company to pursue non-therapeutic applications of its cell-based technologies; and the future business operations of the Company. These forward-looking statements speak only as of the date of this news release. The Company does not undertake to update any of these forward-looking statements to reflect events or circumstances that occur after the date hereof. Such statements reflect management's current views and are based on certain assumptions that may or may not ultimately prove valid. The Company's actual results may vary materially from those contemplated in such forward-looking statements due to risks and uncertainties to which the Company is subject, including those described under the heading "Risk Factors" in the Company's Annual Report
on Form 10-K for the year ended December 31, 2009, and in its subsequent reports on Form 10-Q and Form 8-K.
StemCells, Inc. (Nasdaq: STEM | PowerRating) announced today the launch of STEM101(TM), STEM121(TM) and STEM123(TM), three new antibody reagents that significantly improve the visualization of human cells, including human stem cells and their progeny. These high potency antibodies are the latest additions to the Company's growing SC Proven(R) portfolio of research products, and provide powerful, cost-effective tools for the detection, tracking and characterization of human cells both in vitro and when transplanted into animal models of human diseases.
"The commercial launch of these proprietary antibodies demonstrates our ongoing commitment to broaden our portfolio of innovative research products and to take advantage of the growing market for research-grade cells, media and reagents," said Stewart Craig, Senior Vice President, Development and Operations at StemCells, Inc. "Their utility has been proven by our scientists and by independent academic collaborators in the conduct of their research and development activities. These antibodies have also become the cornerstone of our extensive preclinical studies, which have allowed us to successfully advance our stem cell therapeutic candidates into multiple clinical trials. There is considerable demand for these reagents, so we are pleased to now make them available to the scientific community at large."
STEM101, STEM121 and STEM123 are human-specific mouse monoclonal antibodies that have been extensively used to detect the engraftment, migration and differentiation of human neural stem cells and human liver engrafting cells (hLEC(TM)) transplanted into rodents.1,2,3,4,5 These antibodies can be used for immunohistochemistry and immunofluorescence applications such as:
-- Quantifying the location and number of engrafted cells
-- Tracking the migration pattern of engrafted cells
-- Determining the nature of engrafted cells
-- Identifying specific differentiated human-derived cells such as
astrocytes
STEM101 recognizes the Ku80 protein found in human nuclei, STEM121 recognizes a cytoplasmic protein of human cells, and STEM123 recognizes human glial fibrillary acidic protein (GFAP).
About SC Proven Products
The SC Proven product portfolio comprises a range of specialty cell culture products that enable the standardized and reproducible production and propagation of highly purified stem cells and their differentiated progeny, as well as reagents for cell detection, isolation and characterization.
References
-- Kelly S, et al. Transplanted human fetal neural stem cells survive,
migrate, and differentiate in ischemic rat cerebral cortex. PNAS (2004)
101:11839-11844
-- Cummings B.J., et al. Human neural stem cells differentiate and promote
locomotor recovery in spinal cord-injured mice. PNAS (2005) 102:
14069-14074
-- Tamaki S.J., et al., Neuroprotection of Host Cells by Human Central
Nervous System Stem Cells in a Mouse Model of Infantile Neuronal Ceroid
Lipofuscinosis. Cell Stem Cell (2009) 5:310-319
-- Kallur T., et al. Human Fetal Cortical and Striatal Neural Stem Cells
Generate Region-Specific Neurons In Vitro and Differentiate Extensively
to Neurons After Intrastriatal Transplantation in Neonatal Rats. J
Neurosci Res. (2006) 84:1630-1644
-- Salazar D.L., et al., Human Neural Stem Cells Differentiate and Promote
Locomotor Recovery in an Early Chronic Spinal Cord Injury NOD-scid Mouse
Model. PLoS ONE (2010) 5: e12272
About StemCells, Inc.
StemCells, Inc. is engaged in the research, development, and commercialization of cell-based therapeutics and tools for use in stem cell-based research and drug discovery. In its therapeutic product development programs, StemCells is targeting disorders of the central nervous system and the liver. StemCells' lead product candidate, HuCNS-SC(R) cells (purified human neural stem cells), is currently in clinical development for the treatment of two fatal neurodegenerative disorders in children, and in preclinical development for spinal cord injury and retinal disorders such as age-related macular degeneration. StemCells also markets research products, including media and reagents, under the SC Proven(R)brand, and is developing stem cell-based assay platforms for use in pharmaceutical research, drug discovery and drug development. Further information about StemCells is available at www.stemcellsinc.com.
The StemCells, Inc. logo is available at http://www.globenewswire.com/newsroom/prs/?pkgid=7014
Apart from statements of historical fact, the text of this press release constitutes forward-looking statements within the meaning of the U.S. securities laws, and is subject to the safe harbors created therein. These statements include, but are not limited to, statements regarding the ability of STEM101, STEM121 and STEM123 to enable and improve the visualization, detection, tracking and characterization of human cells both in vitro and when transplanted into animal models of human diseases; the clinical development of the Company's HuCNS-SC cells; the prospects for the Company to pursue non-therapeutic applications of its cell-based technologies; and the future business operations of the Company. These forward-looking statements speak only as of the date of this news release. The Company does not undertake to update any of these forward-looking statements to reflect events or circumstances that occur after the date hereof. Such statements reflect management's current views and are based on certain assumptions that may or may not ultimately prove valid. The Company's actual results may vary materially from those contemplated in such forward-looking statements due to risks and uncertainties to which the Company is subject, including those described under the heading "Risk Factors" in the Company's Annual Report
on Form 10-K for the year ended December 31, 2009, and in its subsequent reports on Form 10-Q and Form 8-K.
Sunday, November 28, 2010
DeGette Hails Approval of Second Embryonic Stem Cell Human Trial
by:PoliticalNews
Congresswoman Diana DeGette (CO-01) hailed the approval of the second human treatment trial using human embryonic stem cells. The test, just approved by the FDA, will be conducted by Advanced Cell Technology (ACT) and will focus on Stagart disease. Medical professionals believe a successful human trial will open the doors to the treatment of other, more common eye diseases such as macular degeneration. DeGette has been the leading Congressional advocate for federal funding of ethical embryonic stem cell research, as these investments drive breakthrough trials like the one announced this week.
“Embryonic stem cell research holds enormous promise for countless diseases,” said DeGette, “and the approval of this test is yet another step towards the breakthroughs it can bring for millions of Americans. I am excited to see the results of this, and other trials, and encourage my colleagues to recognize the enormous benefits of ethical embryonic stem cell research.”
Stagart’s disease affects central vision – e.g., reading and facial recognition – and can cause its victims to lose peripheral vision, only be able to see various levels of light, or ultimately go blind. This test will treat Stagart’s patients with healthy cells, created from embryonic stem cells, to replace the “scavenger cells” that deteriorate their vision.
The test is the second trial using embryonic stem cells to be approved in the United States. Last month, the Geron Corporation was permitted to conduct a trial involving embryonic stem cells to treat spinal cord injuries. Preliminary testing is still ongoing in that trial. Like the Geron trial, this latest trial will focus primarily on the safety and viability of embryonic stem cell use in the treatment of this condition. The Stagart’s trial will likely start early next year and 12 subjects will be treated.
“The approval of these two tests in such close order demonstrates how the investment by the federal government in ethical stem cell research is beginning to bear fruit for the millions of Americans facing debilitating diseases and conditions,” said DeGette. “The breakthroughs of these discoveries underscore the critical importance of finally codifying ethical stem cell research regulations, so our scientists and their critical work can no longer be subject to political whims.”
Congresswoman Diana DeGette (CO-01) hailed the approval of the second human treatment trial using human embryonic stem cells. The test, just approved by the FDA, will be conducted by Advanced Cell Technology (ACT) and will focus on Stagart disease. Medical professionals believe a successful human trial will open the doors to the treatment of other, more common eye diseases such as macular degeneration. DeGette has been the leading Congressional advocate for federal funding of ethical embryonic stem cell research, as these investments drive breakthrough trials like the one announced this week.
“Embryonic stem cell research holds enormous promise for countless diseases,” said DeGette, “and the approval of this test is yet another step towards the breakthroughs it can bring for millions of Americans. I am excited to see the results of this, and other trials, and encourage my colleagues to recognize the enormous benefits of ethical embryonic stem cell research.”
Stagart’s disease affects central vision – e.g., reading and facial recognition – and can cause its victims to lose peripheral vision, only be able to see various levels of light, or ultimately go blind. This test will treat Stagart’s patients with healthy cells, created from embryonic stem cells, to replace the “scavenger cells” that deteriorate their vision.
The test is the second trial using embryonic stem cells to be approved in the United States. Last month, the Geron Corporation was permitted to conduct a trial involving embryonic stem cells to treat spinal cord injuries. Preliminary testing is still ongoing in that trial. Like the Geron trial, this latest trial will focus primarily on the safety and viability of embryonic stem cell use in the treatment of this condition. The Stagart’s trial will likely start early next year and 12 subjects will be treated.
“The approval of these two tests in such close order demonstrates how the investment by the federal government in ethical stem cell research is beginning to bear fruit for the millions of Americans facing debilitating diseases and conditions,” said DeGette. “The breakthroughs of these discoveries underscore the critical importance of finally codifying ethical stem cell research regulations, so our scientists and their critical work can no longer be subject to political whims.”
Monday, November 22, 2010
Chardan Capital Market Initiates Research Coverage of Stemcells
by GlobeNewswire
PALO ALTO, Calif., - StemCells, Inc. /quotes/comstock/15*!stem/quotes/nls/stem (STEM 1.12, -0.02, -1.75%) announced today that Chardan Capital Markets (Chardan) initiated independent equity research coverage on the Company with a "Buy" recommendation and a 12-month price target of $1.45 per share. The new report, issued yesterday, was authored by Keay Nakae, Senior Analyst at Chardan.
Chardan is the second firm to initiate independent equity research coverage of StemCells, Inc. this year. Headquartered in New York, Chardan is an investment banking and institutional brokerage firm with a focus on micro, small, and mid-cap markets. More information about Chardan is available at www.chardancm.com. StemCells does not endorse or adopt the reports, projections or statements of any analyst.
About StemCells, Inc.
StemCells, Inc. is engaged in the research, development, and commercialization of cell-based therapeutics and tools for use in stem cell-based research and drug discovery. In its therapeutic product development programs, StemCells is targeting disorders of the central nervous system and the liver. StemCells' lead product candidate, HuCNS-SC(R) cells (purified human neural stem cells), is currently in clinical development for the treatment of two fatal neurodegenerative disorders in children, and in preclinical development for spinal cord injury and retinal disorders such as age-related macular degeneration. StemCells also markets stem cell research products, including media and reagents, under the SC Proven(R) brand, and is developing stem cell-based assay platforms for use in pharmaceutical research, drug discovery and drug development. Further information about StemCells is available at www.stemcellsinc.com.
PALO ALTO, Calif., - StemCells, Inc. /quotes/comstock/15*!stem/quotes/nls/stem (STEM 1.12, -0.02, -1.75%) announced today that Chardan Capital Markets (Chardan) initiated independent equity research coverage on the Company with a "Buy" recommendation and a 12-month price target of $1.45 per share. The new report, issued yesterday, was authored by Keay Nakae, Senior Analyst at Chardan.
Chardan is the second firm to initiate independent equity research coverage of StemCells, Inc. this year. Headquartered in New York, Chardan is an investment banking and institutional brokerage firm with a focus on micro, small, and mid-cap markets. More information about Chardan is available at www.chardancm.com. StemCells does not endorse or adopt the reports, projections or statements of any analyst.
About StemCells, Inc.
StemCells, Inc. is engaged in the research, development, and commercialization of cell-based therapeutics and tools for use in stem cell-based research and drug discovery. In its therapeutic product development programs, StemCells is targeting disorders of the central nervous system and the liver. StemCells' lead product candidate, HuCNS-SC(R) cells (purified human neural stem cells), is currently in clinical development for the treatment of two fatal neurodegenerative disorders in children, and in preclinical development for spinal cord injury and retinal disorders such as age-related macular degeneration. StemCells also markets stem cell research products, including media and reagents, under the SC Proven(R) brand, and is developing stem cell-based assay platforms for use in pharmaceutical research, drug discovery and drug development. Further information about StemCells is available at www.stemcellsinc.com.
Monday, November 15, 2010
UC Santa Barbara Part of International Research Collaboration Focusing on Age-Related Macular degeneration Cure
By AScribe Newswire
SANTA BARBARA, Calif., Nov. 15 - An international collaboration between UC Santa Barbara, the Keck School of Medicine of the University of Southern California (USC), and several other research institutions, is bringing together leaders in the fields of stem cell biology, basic science, and ophthalmology to develop a treatment for blindness caused by age-related macular degeneration.
The California Project to Cure Blindness (CPCB) was formed with a $16 million California Institute for Regenerative Medicine (CIRM) "disease team" grant awarded in late 2009 to fund development of a stem cell-based treatment for age-related macular degeneration. As part of the CIRM Disease Team partnership program, an additional $4.1 million from Britain's Medical Research Council funds collaborative work at University College of London.
"UCSB scientists in the Center for Stem Cell Biology and Engineering and the Center for the Study of Macular Degeneration are excited to provide the basic research that will allow translation of stem cell research to the clinic," said Dennis Clegg, professor in UCSB's Department of Molecular, Cellular, and Developmental Biology, and co-director of the UCSB Center for Stem Cell Biology and Engineering. Both centers are part of UCSB's Neuroscience Research Institute. Grant funds totaling $2.5 million for this work were assigned to UCSB through USC.
The overall grant was awarded to principal investigator Mark Humayun, professor of ophthalmology, cell and neurobiology and biomedical engineering at the Keck School, and David R. Hinton, professor of pathology and ophthalmology at the Keck School. Co-investigator is Martin Pera, director of the Eli and Edythe Broad CIRM Center for Regenerative Medicine and Stem Cell Research at USC.
"With this collaboration, we hope to accelerate research on a stem cell-based therapy for age-related macular degeneration," said Humayun. "Age-related macular degeneration is the leading cause of irreversible vision loss, affecting one in three people age 75 or older. The CIRM grant enables us to work with numerous researchers and experts who are dedicated to finding the cure to this devastating medical condition."
The cause of blindness in age-related macular degeneration is the death of retinal pigment epithelial cells, which provide critical support of photoreceptor function and health. The project objective is to replace damaged retinal epithelium with healthy tissue derived from human embryonic stem cells to prevent loss of vision.
Stem cell therapy offers the possibility of a wider range of options for age-related macular degeneration patients, said Keck School Dean Carmen A. Puliafito. "While exciting new pharmaceuticals to treat age-related macular degeneration are now available, these are effective only in a select group of patients, and can be used only during a narrow time window," said Puliafito, an ophthalmologist whose academic focus is macular degeneration. "In contrast, stem cell therapy promises to be broadly applicable. The potential is tremendous."
CIRM President Alan Trounson noted that the disease team approach exemplified by the California Project to Cure Blindness could transform the direction of future research.
"Scientists have talked for years about the need to find ways to speed the pace of discovery," said Trounson. "CIRM, through the Disease Team Award Program, has encouraged applicants to form teams composed of the best researchers from around the world. The partnership between the California Project to Cure Blindness and Britain's Medical Research Council is a great example of CIRM's vision of a new standard for funding translational research."
SANTA BARBARA, Calif., Nov. 15 - An international collaboration between UC Santa Barbara, the Keck School of Medicine of the University of Southern California (USC), and several other research institutions, is bringing together leaders in the fields of stem cell biology, basic science, and ophthalmology to develop a treatment for blindness caused by age-related macular degeneration.
The California Project to Cure Blindness (CPCB) was formed with a $16 million California Institute for Regenerative Medicine (CIRM) "disease team" grant awarded in late 2009 to fund development of a stem cell-based treatment for age-related macular degeneration. As part of the CIRM Disease Team partnership program, an additional $4.1 million from Britain's Medical Research Council funds collaborative work at University College of London.
"UCSB scientists in the Center for Stem Cell Biology and Engineering and the Center for the Study of Macular Degeneration are excited to provide the basic research that will allow translation of stem cell research to the clinic," said Dennis Clegg, professor in UCSB's Department of Molecular, Cellular, and Developmental Biology, and co-director of the UCSB Center for Stem Cell Biology and Engineering. Both centers are part of UCSB's Neuroscience Research Institute. Grant funds totaling $2.5 million for this work were assigned to UCSB through USC.
The overall grant was awarded to principal investigator Mark Humayun, professor of ophthalmology, cell and neurobiology and biomedical engineering at the Keck School, and David R. Hinton, professor of pathology and ophthalmology at the Keck School. Co-investigator is Martin Pera, director of the Eli and Edythe Broad CIRM Center for Regenerative Medicine and Stem Cell Research at USC.
"With this collaboration, we hope to accelerate research on a stem cell-based therapy for age-related macular degeneration," said Humayun. "Age-related macular degeneration is the leading cause of irreversible vision loss, affecting one in three people age 75 or older. The CIRM grant enables us to work with numerous researchers and experts who are dedicated to finding the cure to this devastating medical condition."
The cause of blindness in age-related macular degeneration is the death of retinal pigment epithelial cells, which provide critical support of photoreceptor function and health. The project objective is to replace damaged retinal epithelium with healthy tissue derived from human embryonic stem cells to prevent loss of vision.
Stem cell therapy offers the possibility of a wider range of options for age-related macular degeneration patients, said Keck School Dean Carmen A. Puliafito. "While exciting new pharmaceuticals to treat age-related macular degeneration are now available, these are effective only in a select group of patients, and can be used only during a narrow time window," said Puliafito, an ophthalmologist whose academic focus is macular degeneration. "In contrast, stem cell therapy promises to be broadly applicable. The potential is tremendous."
CIRM President Alan Trounson noted that the disease team approach exemplified by the California Project to Cure Blindness could transform the direction of future research.
"Scientists have talked for years about the need to find ways to speed the pace of discovery," said Trounson. "CIRM, through the Disease Team Award Program, has encouraged applicants to form teams composed of the best researchers from around the world. The partnership between the California Project to Cure Blindness and Britain's Medical Research Council is a great example of CIRM's vision of a new standard for funding translational research."
Thursday, November 4, 2010
Miracle Eye Implant Restores sight to Blind
by Victoria Fletcher
A MAN who was totally blind can now read letters of the alphabet and the time on a clock face with a microchip implanted in his eye, it was revealed yesterday.
Experts said hopes of such a leap forward had previously existed “only in the realm of science fiction”.
But now researchers have shown it is possible to restore vision lost to disease with an electronic eye.
The study, by a team in Germany, will offer hope to the 25,000 Britons who are told they will go blind due to an inherited condition known as retinitis pigmentosa.
But it could also eventually treat the 300,000 who have macular degeneration which also leads to blindness.
The microchip, smaller than the tip of a pen and containing 1,500 tiny light sensors, fits into a natural space beneath the retina.
When an image comes through the lens of the eye it hits the sensors which send an electrical pulse to nerve cells at the back of the eye. These transmit the message to the brain.
Miikka can now tell an apple from a banana and right Professor Robert Maclaren
The device is powered by a thin cable that runs from the eye, out of the side of the skull and is attached to a battery behind the ear.
The new study reveals that three patients have been able to see grainy images of objects and recognise shapes after having the device fitted.
Finn Miikka Terho, 46, was able to walk around a room with ease, read his own name and even tell researchers they had spelt it wrongly.
He could recognise shades of grey, read the time from a clock and pick up an apple and a banana from the table in front of him.
Before the implant he was totally blind apart from being able to detect changes between light and dark.
The pilot study, published in the Proceedings of the Royal Society B Journal, means the technology behind the device works, is safe and is ready to be tested on a larger number of patients in a proper clinical trial.
Although it does not mean patients will ever be able to see normally, it does raise the prospect that the blind will be helped to see enough to regain some independence.
Prof Robert Maclaren, Professor of Ophthalmology at Oxford University, will conduct the next trial.
He said: “This is a big breakthrough, no two ways about it. To take someone who is blind and help them see again is pretty incredible.”
He added: “The successful testing of this electronic implant in Germany is without doubt a truly significant advance.
“One previously blind patient was able to read his own name with the implant switched on. Until now, this concept would have been considered only in the realms of science fiction.”
In recent years, scientists have helped to restore vision using a small camera mounted on spectacles.
This allowed a rough interpretation of an image beamed through a wire into the brain.
But the new device, developed by German scientist Prof Eberhart Zrenner, shows that the damaged light receptor cells in the eye can simply be replaced by a microchip.
The rest of the image is obtained by the natural eye. Prof Zrenner has now set up the technology firm Retinal Implant AG to develop the device.
Around one in 3,000 Britons have retinitis pigmentosa, which destroys light cells in the eye.The condition, triggered by a range of genes, is incurable and untreatable.
More than 10 times as many people have macular degeneration.
It usually affects older adults and leads to a loss of central vision, making it difficult or impossible to read or recognise faces although enough some peripheral vision remains.
Prof Maclaren said he was thrilled he could now tell patients with retinitis pigmentosa that there was hope ahead after years of having to tell them they would be left completely blind. And he said that although the device did not offer an amazing quality of vision, it would be significant for people who had previously been unable to see anything.
David Head, chief executive of the British Retinitis Pigmentosa Society, welcomed the news, calling it “a very significant advance”. But he said he wanted patients to realise that even this breakthrough would not restore their vision.
He added: “The technology is exciting and hopefully this will advance in the next few years but we have to temper it with reality.”
Up to 12 patients are expected to take part in the British study, due to start early next year at King’s College Hospital, London, and the Oxford Eye Hospital.It is hoped that when the device can be left in place for years instead of months, the brain may learn to interpret the grey and white images into far more accurate pictures of what the eye is really seeing.
A MAN who was totally blind can now read letters of the alphabet and the time on a clock face with a microchip implanted in his eye, it was revealed yesterday.
Experts said hopes of such a leap forward had previously existed “only in the realm of science fiction”.
But now researchers have shown it is possible to restore vision lost to disease with an electronic eye.
The study, by a team in Germany, will offer hope to the 25,000 Britons who are told they will go blind due to an inherited condition known as retinitis pigmentosa.
But it could also eventually treat the 300,000 who have macular degeneration which also leads to blindness.
The microchip, smaller than the tip of a pen and containing 1,500 tiny light sensors, fits into a natural space beneath the retina.
When an image comes through the lens of the eye it hits the sensors which send an electrical pulse to nerve cells at the back of the eye. These transmit the message to the brain.
Miikka can now tell an apple from a banana and right Professor Robert Maclaren
The device is powered by a thin cable that runs from the eye, out of the side of the skull and is attached to a battery behind the ear.
The new study reveals that three patients have been able to see grainy images of objects and recognise shapes after having the device fitted.
Finn Miikka Terho, 46, was able to walk around a room with ease, read his own name and even tell researchers they had spelt it wrongly.
He could recognise shades of grey, read the time from a clock and pick up an apple and a banana from the table in front of him.
Before the implant he was totally blind apart from being able to detect changes between light and dark.
The pilot study, published in the Proceedings of the Royal Society B Journal, means the technology behind the device works, is safe and is ready to be tested on a larger number of patients in a proper clinical trial.
Although it does not mean patients will ever be able to see normally, it does raise the prospect that the blind will be helped to see enough to regain some independence.
Prof Robert Maclaren, Professor of Ophthalmology at Oxford University, will conduct the next trial.
He said: “This is a big breakthrough, no two ways about it. To take someone who is blind and help them see again is pretty incredible.”
He added: “The successful testing of this electronic implant in Germany is without doubt a truly significant advance.
“One previously blind patient was able to read his own name with the implant switched on. Until now, this concept would have been considered only in the realms of science fiction.”
In recent years, scientists have helped to restore vision using a small camera mounted on spectacles.
This allowed a rough interpretation of an image beamed through a wire into the brain.
But the new device, developed by German scientist Prof Eberhart Zrenner, shows that the damaged light receptor cells in the eye can simply be replaced by a microchip.
The rest of the image is obtained by the natural eye. Prof Zrenner has now set up the technology firm Retinal Implant AG to develop the device.
Around one in 3,000 Britons have retinitis pigmentosa, which destroys light cells in the eye.The condition, triggered by a range of genes, is incurable and untreatable.
More than 10 times as many people have macular degeneration.
It usually affects older adults and leads to a loss of central vision, making it difficult or impossible to read or recognise faces although enough some peripheral vision remains.
Prof Maclaren said he was thrilled he could now tell patients with retinitis pigmentosa that there was hope ahead after years of having to tell them they would be left completely blind. And he said that although the device did not offer an amazing quality of vision, it would be significant for people who had previously been unable to see anything.
David Head, chief executive of the British Retinitis Pigmentosa Society, welcomed the news, calling it “a very significant advance”. But he said he wanted patients to realise that even this breakthrough would not restore their vision.
He added: “The technology is exciting and hopefully this will advance in the next few years but we have to temper it with reality.”
Up to 12 patients are expected to take part in the British study, due to start early next year at King’s College Hospital, London, and the Oxford Eye Hospital.It is hoped that when the device can be left in place for years instead of months, the brain may learn to interpret the grey and white images into far more accurate pictures of what the eye is really seeing.
Tuesday, November 2, 2010
OGI invests in personalized medicine for age-related macular degeneration
by administrator
Established in 2007 and based in Toronto, ArcticDx has developed a test, Macula Risk®, the first of its kind and specifically designed to determine one's inherited risk for age-related macular degeneration (AMD), the most common form of acquired blindness in the developed world, affecting over 10% of individuals. ArcticDx will use the PBDF investment to undertake studies in support of a planned filing for Food and Drug Administration (FDA) approval for Macula Risk.
Macula Risk detects variations in genetic markers known to predict the progression of early asymptomatic AMD to blindness using a cheek swab sample. The eyesight of individuals who are genetically predisposed to blindness can be saved through enhanced surveillance and early treatment. Macula Risk helps target effective care to those who need it most and relieves others who would otherwise live with uncertainty.
"The investment from OGI will support our filing for FDA approval for Macula Risk," commented Mr. Gregory Hines, CEO of ArcticDx. "We think this approval is an important departure from the growing trend of direct to consumer marketing of genetic tests that have only a weak link to science and are often of no clinical value. Macula Risk stands as the best example of a validated test for a multi-genetic common human disease. Achieving FDA approval will position Macula Risk for wide spread adoption."
The Macula Risk test will be marketed to eye care professionals who manage most cases of AMD in North America. These doctors will offer the test to individuals with the dry form of the disease who have not yet lost vision.
In the industrialized world, AMD is the major cause of uncorrectable vision loss in the elderly, affecting over 2.5 million people in Canada and over 25 million people in the USA. Age?related macular degeneration is generally a disease of the elderly with the worldwide incidence of the disease growing from one in ten people over the age of 60 to more than 1 in 4 people over the age of 75. Macular degeneration is more common than Parkinson's disease, Alzheimer's disease, breast cancer and prostate cancer combined.
"Application of genomics technologies is opening the door to an era of personalized medicine in our approach to preventing, detecting and treating human disease," commented Dr. Christian Burks, President and CEO, OGI. "We are particularly pleased to be investing in a company that grew out of applied research funded by Genome Canada through OGI."
The funded work will focus on a cohort of patient samples who had early stage AMD in the Age-Related Eye Disease Study (AREDS), a large eye survey carried out by the American National Eye Institute. These patients were followed over a five-year period to determine progression of the disease. The ArcticDx team will undertake a prospective study on this cohort to evaluate use of Macula Risk in predicting which patients will progress to wet AMD (the late form of AMD) and which will not.
OGI's PBDF program invests in opportunities ? based in genomics, proteomics or associated technologies ? that fall in the proof-of-principle (validation) phase of research and that have the short-term potential to secure a significant next step towards the marketplace. Previous recipients have included Ontario universities, research institutes and companies.
Established in 2007 and based in Toronto, ArcticDx has developed a test, Macula Risk®, the first of its kind and specifically designed to determine one's inherited risk for age-related macular degeneration (AMD), the most common form of acquired blindness in the developed world, affecting over 10% of individuals. ArcticDx will use the PBDF investment to undertake studies in support of a planned filing for Food and Drug Administration (FDA) approval for Macula Risk.
Macula Risk detects variations in genetic markers known to predict the progression of early asymptomatic AMD to blindness using a cheek swab sample. The eyesight of individuals who are genetically predisposed to blindness can be saved through enhanced surveillance and early treatment. Macula Risk helps target effective care to those who need it most and relieves others who would otherwise live with uncertainty.
"The investment from OGI will support our filing for FDA approval for Macula Risk," commented Mr. Gregory Hines, CEO of ArcticDx. "We think this approval is an important departure from the growing trend of direct to consumer marketing of genetic tests that have only a weak link to science and are often of no clinical value. Macula Risk stands as the best example of a validated test for a multi-genetic common human disease. Achieving FDA approval will position Macula Risk for wide spread adoption."
The Macula Risk test will be marketed to eye care professionals who manage most cases of AMD in North America. These doctors will offer the test to individuals with the dry form of the disease who have not yet lost vision.
In the industrialized world, AMD is the major cause of uncorrectable vision loss in the elderly, affecting over 2.5 million people in Canada and over 25 million people in the USA. Age?related macular degeneration is generally a disease of the elderly with the worldwide incidence of the disease growing from one in ten people over the age of 60 to more than 1 in 4 people over the age of 75. Macular degeneration is more common than Parkinson's disease, Alzheimer's disease, breast cancer and prostate cancer combined.
"Application of genomics technologies is opening the door to an era of personalized medicine in our approach to preventing, detecting and treating human disease," commented Dr. Christian Burks, President and CEO, OGI. "We are particularly pleased to be investing in a company that grew out of applied research funded by Genome Canada through OGI."
The funded work will focus on a cohort of patient samples who had early stage AMD in the Age-Related Eye Disease Study (AREDS), a large eye survey carried out by the American National Eye Institute. These patients were followed over a five-year period to determine progression of the disease. The ArcticDx team will undertake a prospective study on this cohort to evaluate use of Macula Risk in predicting which patients will progress to wet AMD (the late form of AMD) and which will not.
OGI's PBDF program invests in opportunities ? based in genomics, proteomics or associated technologies ? that fall in the proof-of-principle (validation) phase of research and that have the short-term potential to secure a significant next step towards the marketplace. Previous recipients have included Ontario universities, research institutes and companies.
Saturday, October 23, 2010
CIRM to dole Out $72M to Advance Research and Recruit Stem Cell Scientist
The California Institute for Regenerative Medicine (CIRM) approved funding for 19 awards worth $67 million under the Early Translation II Awards program. The 29-member governing board also voted to approve the second Research Leadership Award of $4.8 million, given to aid in recruiting Peter Coffey, D.Phil., from the University College London to the University of California, Santa Barbara.
The Early Translation II Awards are the second of what CIRM expects to be a 12- to 18-month award cycle for translational research grants. The funded projects are expected to either result in a candidate drug or cell therapy or make significant strides toward such a candidate.
“We are looking for ways to complement our leading edge of stem cell-based treatments for patients, and these projects will load our frontline portfolio with promising studies on autism, muscular dystrophy, Canavan disease, and liver disease,” says Alan Trounson, CIRM president.
The awards went to one for-profit and 11 not-for-profit institutions. The for-profit company iPierian will take its award in the form of a loan. Three of the awards include collaborators in Germany. The portion of the projects carried out by these collaborators will be supported by the Federal Ministry of Education and Research, the science financing agency in Germany, which will fund up to $15 million for this round of awards.
The $4.8 million grant under the Research Leadership Award program will be spread over six years and will back Dr. Coffey’s research on maturing embryonic stem cells into retinal pigment epithelial cells to treat macular degeneration and other forms of vision loss such as diabetic retinopathy and retinitis pigmentosa. Dr. Coffey is part of a team working toward a therapy for macular degeneration led by Mark Humayun, M.D., Ph.D., at the University of Southern California.
“Recruiting internationally renowned stem cell experts such as Dr. Coffey builds a critical mass of stem cell leadership in California to drive the creation of innovative therapies for patients suffering from chronic disease or injury,” notes Robert Klein, chair of the CIRM governing board.
The Early Translation II Awards are the second of what CIRM expects to be a 12- to 18-month award cycle for translational research grants. The funded projects are expected to either result in a candidate drug or cell therapy or make significant strides toward such a candidate.
“We are looking for ways to complement our leading edge of stem cell-based treatments for patients, and these projects will load our frontline portfolio with promising studies on autism, muscular dystrophy, Canavan disease, and liver disease,” says Alan Trounson, CIRM president.
The awards went to one for-profit and 11 not-for-profit institutions. The for-profit company iPierian will take its award in the form of a loan. Three of the awards include collaborators in Germany. The portion of the projects carried out by these collaborators will be supported by the Federal Ministry of Education and Research, the science financing agency in Germany, which will fund up to $15 million for this round of awards.
The $4.8 million grant under the Research Leadership Award program will be spread over six years and will back Dr. Coffey’s research on maturing embryonic stem cells into retinal pigment epithelial cells to treat macular degeneration and other forms of vision loss such as diabetic retinopathy and retinitis pigmentosa. Dr. Coffey is part of a team working toward a therapy for macular degeneration led by Mark Humayun, M.D., Ph.D., at the University of Southern California.
“Recruiting internationally renowned stem cell experts such as Dr. Coffey builds a critical mass of stem cell leadership in California to drive the creation of innovative therapies for patients suffering from chronic disease or injury,” notes Robert Klein, chair of the CIRM governing board.
Monday, October 18, 2010
Vitamin A pill could save sight
By Fiona Macrae
A drug based on vitamin A could prevent millions from going blind as they get older, doctors believe.
The treatment was able to stop the most common cause of blindness in old age during trials.
Researchers behind the drug, fenretinide, found it halted the advance of age-related macular degeneration, for which there is currently no cure.
They targeted the most prevalent form of the condition, known as ‘dry’ AMD, which is caused by the deterioration and death of cells in the macula – the part of the retina used to see straight ahead.
The disease robs sufferers of their sight by creating a blackspot in the centre of their vision.
It can make it impossible to carry out everyday tasks such as reading, driving and watching television.
While the less common ‘wet’ form can be treated, nothing can be done to help the bulk of patients.
The U.S. research studied fenretinide, which is derived from vitamin A, the vitamin found in carrots, and which was originally designed to tackle arthritis.
Almost 250 men and women with dry AMD took a fenretinide pill a day or a placebo.
In the highest dose, the drug halted visual deterioration after a year. This suggests that while it was unable to do anything to stop cells that were already damaged from dying, it protected healthy cells. Although the research is still preliminary, it offers promise of a treatment for the disease.
More...
* The age of the pensionista: Why most women are totally unprepared for retirement
* The rise of the silver surfer: How half of women over 55 log on to Facebook
It affects millions across the world and 300,000 Britons. The number of UK sufferers could more than treble to one million within 25 years as the population ages.
Dr Jason Slakter, of New York University School of Medicine, said: ‘There are currently no effective treatments for dry AMD and the need for finding one is grave.
‘Our study wasn’t designed to give a final answer.
‘It was designed to see if there was a biological effect and if the drug was working in the way we’d expect and to find out if it was well tolerated by patents.
‘I think we answered all of these points favourably. The bottom line is that I am excited about doing more studies.’
Further, larger trials are planned for the end of next year.
If the drug lives up to its initial promise, it could be in widespread use for dry AMD by 2015.
The treatment works because in normal circumstances the eye needs vitamin A to help it see. The retina naturally uses the vitamin and is helped to do so by a compound called retinol binding protein, or RBP.
However in some patients, the vitamin can produce poisons that kill the delicate cells, leading to loss of vision.
Fenretinide acts as a decoy, attaching itself to the RBP and stopping vitamin A from causing harm, the American Academy of Ophthalmology’s annual conference heard.
Wet AMD, in which tiny blood vessels bleed into the retina, is less common, but progresses more rapidly, with central vision being lost within months of diagnosis.
Caught early enough, wet AMD can be stopped in its tracks by a technique called photodynamic therapy, which uses a light-activated dye to destroy abnormal blood vessels. Drug treatments are also available.
Fenretinide also halved the odds of the patients, who already had dry AMD, going on to develop wet AMD.
A spokesman for the research team said: ‘Years of use of fenretinide to treat cancers, rheumatoid arthritis have shown it to be safe and well-tolerated.’
A drug based on vitamin A could prevent millions from going blind as they get older, doctors believe.
The treatment was able to stop the most common cause of blindness in old age during trials.
Researchers behind the drug, fenretinide, found it halted the advance of age-related macular degeneration, for which there is currently no cure.
They targeted the most prevalent form of the condition, known as ‘dry’ AMD, which is caused by the deterioration and death of cells in the macula – the part of the retina used to see straight ahead.
The disease robs sufferers of their sight by creating a blackspot in the centre of their vision.
It can make it impossible to carry out everyday tasks such as reading, driving and watching television.
While the less common ‘wet’ form can be treated, nothing can be done to help the bulk of patients.
The U.S. research studied fenretinide, which is derived from vitamin A, the vitamin found in carrots, and which was originally designed to tackle arthritis.
Almost 250 men and women with dry AMD took a fenretinide pill a day or a placebo.
In the highest dose, the drug halted visual deterioration after a year. This suggests that while it was unable to do anything to stop cells that were already damaged from dying, it protected healthy cells. Although the research is still preliminary, it offers promise of a treatment for the disease.
More...
* The age of the pensionista: Why most women are totally unprepared for retirement
* The rise of the silver surfer: How half of women over 55 log on to Facebook
It affects millions across the world and 300,000 Britons. The number of UK sufferers could more than treble to one million within 25 years as the population ages.
Dr Jason Slakter, of New York University School of Medicine, said: ‘There are currently no effective treatments for dry AMD and the need for finding one is grave.
‘Our study wasn’t designed to give a final answer.
‘It was designed to see if there was a biological effect and if the drug was working in the way we’d expect and to find out if it was well tolerated by patents.
‘I think we answered all of these points favourably. The bottom line is that I am excited about doing more studies.’
Further, larger trials are planned for the end of next year.
If the drug lives up to its initial promise, it could be in widespread use for dry AMD by 2015.
The treatment works because in normal circumstances the eye needs vitamin A to help it see. The retina naturally uses the vitamin and is helped to do so by a compound called retinol binding protein, or RBP.
However in some patients, the vitamin can produce poisons that kill the delicate cells, leading to loss of vision.
Fenretinide acts as a decoy, attaching itself to the RBP and stopping vitamin A from causing harm, the American Academy of Ophthalmology’s annual conference heard.
Wet AMD, in which tiny blood vessels bleed into the retina, is less common, but progresses more rapidly, with central vision being lost within months of diagnosis.
Caught early enough, wet AMD can be stopped in its tracks by a technique called photodynamic therapy, which uses a light-activated dye to destroy abnormal blood vessels. Drug treatments are also available.
Fenretinide also halved the odds of the patients, who already had dry AMD, going on to develop wet AMD.
A spokesman for the research team said: ‘Years of use of fenretinide to treat cancers, rheumatoid arthritis have shown it to be safe and well-tolerated.’
Sunday, October 10, 2010
Prizes Honor Studies in Vision Loss
Posted by admin
NEW YORK – Three scientists have won prestigious medical prizes — one for devising a treatment for a major cause of vision loss and two for laying the groundwork for an explosion in obesity research.
The Lasker Awards, worth $250,000 apiece, will be presented Oct. 1 by the Albert and Mary Lasker Foundation. A fourth scientist is being honored for decades of statesmanship in biomedical sciences.
The clinical research award goes to Dr. Napoleone Ferrara, 54, of the biotech company Genentech in South San Francisco, Calif. He is honored for discovering a protein called VEGF in 1989 and using it to develop a treatment that significantly improves sight for people with a devastating type of age-related macular degeneration.
More than a million people worldwide have been treated based on Ferrara’s research, the Lasker foundation says. The type of age-related macular degeneration his research addressed — “wet” as opposed to the more common “dry” form — accounts for a tenth or more of the 25 million to 30 million cases of AMD worldwide.
Two drugs based on Ferrara’s VEGF research, Lucentis and the cancer medicine Avastin, are used for wet AMD, attacking it by discouraging the formation of an abnormal growth of blood vessels behind the retina.
The Lasker prize for basic research is shared by Douglas Coleman, 78, of the Jackson Laboratory in Bar Harbor, Maine, and Jeffrey Friedman, 56, of Rockefeller University in New York. They are honored for the discovery of the hormone leptin, which helps regulate appetite and body weight.
In the 1970s, Coleman showed that mice have some sort of appetite-suppressing substance in the blood. Friedman identified the substance in 1994 and named it leptin. People have leptin too, and the research opened new avenues for exploring the biological basis of human obesity, the foundation said.
A Lasker award for special achievement in medical research goes to Dr. David Weatherall, 77, of Oxford University. He is honored for 50 years of “international statesmanship in biomedical science,” including his research on an inherited anemia called thalassemia.
The Lasker foundation was established in 1942. Albert Lasker was an advertising executive who died in 1952. His wife Mary was a longtime champion of medical research before her death in 1994.
NEW YORK – Three scientists have won prestigious medical prizes — one for devising a treatment for a major cause of vision loss and two for laying the groundwork for an explosion in obesity research.
The Lasker Awards, worth $250,000 apiece, will be presented Oct. 1 by the Albert and Mary Lasker Foundation. A fourth scientist is being honored for decades of statesmanship in biomedical sciences.
The clinical research award goes to Dr. Napoleone Ferrara, 54, of the biotech company Genentech in South San Francisco, Calif. He is honored for discovering a protein called VEGF in 1989 and using it to develop a treatment that significantly improves sight for people with a devastating type of age-related macular degeneration.
More than a million people worldwide have been treated based on Ferrara’s research, the Lasker foundation says. The type of age-related macular degeneration his research addressed — “wet” as opposed to the more common “dry” form — accounts for a tenth or more of the 25 million to 30 million cases of AMD worldwide.
Two drugs based on Ferrara’s VEGF research, Lucentis and the cancer medicine Avastin, are used for wet AMD, attacking it by discouraging the formation of an abnormal growth of blood vessels behind the retina.
The Lasker prize for basic research is shared by Douglas Coleman, 78, of the Jackson Laboratory in Bar Harbor, Maine, and Jeffrey Friedman, 56, of Rockefeller University in New York. They are honored for the discovery of the hormone leptin, which helps regulate appetite and body weight.
In the 1970s, Coleman showed that mice have some sort of appetite-suppressing substance in the blood. Friedman identified the substance in 1994 and named it leptin. People have leptin too, and the research opened new avenues for exploring the biological basis of human obesity, the foundation said.
A Lasker award for special achievement in medical research goes to Dr. David Weatherall, 77, of Oxford University. He is honored for 50 years of “international statesmanship in biomedical science,” including his research on an inherited anemia called thalassemia.
The Lasker foundation was established in 1942. Albert Lasker was an advertising executive who died in 1952. His wife Mary was a longtime champion of medical research before her death in 1994.
Monday, October 4, 2010
Portable Macular Degeneration (AMD) Early Detection & Screening Device
Health Research Sciences has developed a new portable device allowing early detection of the leading cause of blindness in the US; Age-Related Macular Degeneration (AMD) and Diabetic Maculopathy. This inexpensive and portable device will be an integral part during the next few years in the fight to reduce AMD in the US.
Lighthouse Point, Florida September 30, 2010. Health Research Sciences introduces a new approach for testing macular function with the purpose of early detection of Age-Related Macular Degeneration (AMD), Diabetic Maculopathy and other retinal pathologies. The MDD-2 Macular Degeneration Detection & Screening Device has a unique, hand-held design that measures photostress recovery and solves the problem of inconsistent macular function testing.
The MDD-2 provides reproducible measurements of macular function (precise photostress recovery times) which are documented in a concise format for health professional interpretation. The MDD-2 also permits monitoring of central retinal health over time and can warn of deterioration in function at an early stage. Over 10 million Americans suffer from vision loss due to Macular Degeneration and approximately 4 million Americans are at risk for vision loss from Diabetes and further that these vision disorders cost all Americans over $1 billion annually.
The MDD-2 enables Ophthalmologists, Optometrists, Primary Care Physicians and Endocrinologists to easily and efficiently measure macular function for the purpose of early detection of AMD, Diabetic Retinopathy and other central retinal diseases. The test may be administered by a trained technician/assistant and takes approximately 4 minutes to complete. Results are available immediately and easily interpreted by the physician.
“As Professor of Ophthalmology and Pathology, Johns Hopkins University, School of Medicine, I am very concerned about the need for early detection of Age Related Macular Degeneration (AMD) and Diabetic Retinopathy in the United States. I believe that dark adaptation and photostress recovery measurement are both effective tests for evaluating the function of the macula, detecting macular degeneration and diabetic retinopathy at an early stage.”
Mark O.M. Tso, M.D., D. Sc.
Lighthouse Point, Florida September 30, 2010. Health Research Sciences introduces a new approach for testing macular function with the purpose of early detection of Age-Related Macular Degeneration (AMD), Diabetic Maculopathy and other retinal pathologies. The MDD-2 Macular Degeneration Detection & Screening Device has a unique, hand-held design that measures photostress recovery and solves the problem of inconsistent macular function testing.
The MDD-2 provides reproducible measurements of macular function (precise photostress recovery times) which are documented in a concise format for health professional interpretation. The MDD-2 also permits monitoring of central retinal health over time and can warn of deterioration in function at an early stage. Over 10 million Americans suffer from vision loss due to Macular Degeneration and approximately 4 million Americans are at risk for vision loss from Diabetes and further that these vision disorders cost all Americans over $1 billion annually.
The MDD-2 enables Ophthalmologists, Optometrists, Primary Care Physicians and Endocrinologists to easily and efficiently measure macular function for the purpose of early detection of AMD, Diabetic Retinopathy and other central retinal diseases. The test may be administered by a trained technician/assistant and takes approximately 4 minutes to complete. Results are available immediately and easily interpreted by the physician.
“As Professor of Ophthalmology and Pathology, Johns Hopkins University, School of Medicine, I am very concerned about the need for early detection of Age Related Macular Degeneration (AMD) and Diabetic Retinopathy in the United States. I believe that dark adaptation and photostress recovery measurement are both effective tests for evaluating the function of the macula, detecting macular degeneration and diabetic retinopathy at an early stage.”
Mark O.M. Tso, M.D., D. Sc.
Saturday, September 18, 2010
Shark Liver Oil and Cartilage could provide Rescue
By Debbie Nicholson
Sharks are labeled with a bad persona. Mainly due to movies like "Jaws" where they go around and chomp down for a nightly feast on any human or animal they can find. There are one million slaughters going on each year on this creature. However, these creatures of the sea have the ability to aide in certain medical conditions such as anemia, brain bleeding and cancer.
Scientists for decades have been studying the shark, well at least his cartilage. They believe this creature has something that can offer the medical community especially in the treatment of cancer. It is a known fact that sharks hardly ever get cancer. There must be something in their bodies that aide in protection from this disease.
The theory of the shark cartilage and cancer among most of the scientific community is having to do specifically with the composition of the cartilage. For one thing cartilage cannot grow cancer due to the fact it has no blood vessels. The theory is that the cartilage manufactures elements that barricade the growing of new blood vessels that would feed on the tumor. So by using the cartilage to treat the tumor the blood vessels then should be stopped from developing on the tumor.
A study was performed to scientifically prove or disprove the theory of shark cartilage. Nine patients had participated in this study. When the study had been completed the evidence indicated that seven of the nine patients who had advanced cancer there was an 87% positive response from the treatment. It is important to note that no other treatment was applied during this study.
The evidence laid out had shown the following results (in brief form here after seven weeks of treatment and all patients were diagnosed as terminal)
A few results:
Female patient stage three uterine cancer going into bladder. Radiation had no effects on the tumor. After seven weeks of the cartilage the tumor reduced in size by 80% and after eleven weeks the tumor had gone and so did her pain. The only remaining factor was scar tissue.
Female with vaginal hemangioma in size of 5"x 5" after hysterectomy and partial vaginectomy along with radiation. Heavy bleeding was life threatening complication. At seven weeks tumor reduced by 60% and bleeding had stopped. At eleven weeks tumor size was 3"x 3" with further treatment it was stated tumor would appear to decrease.
Male stage three cancer on back of right thigh. After nine weeks of cartilage therapy surgery was done. It was noticed that 60% of the tumor was dead and surgically removed.
The summary of this research had indicated that further research was still required but shark cartilage treatments had displayed promise.
Studies on shark cartilage are still being conducted however, these studies are extremely expensive. At the same time environmentalists are advocating for the shark and making the fact known that the shark population has decreased significantly in the prior fifteen years due to over fishing and the ever on going demand for shark products.
The compounds of the shark cartilage are being tested by a few pharmaceutical companies. If developed it would only be able to be available by prescription and it is stated the cartilage is coming from those sharks who have been slaughtered for meat.
Shark cartilage in research has shown it can help alleviate arthritis symptoms. In all reality scientists had determined it is one of the main components in the shark cartilage which is chondroitin sulfate that is the magic behind the alleviation. To make a note the supplements which are sold in health food stores usually come from cows and that of sharks.
Shark cartilage has demonstrated benefits in patients with arthritis. It decrease joint pain and inflammation which enabled patients to use less pain relievers. Good news is a lot of persons who use it in small doses have no major problems. Some can have side effects which include nausea, vomiting, cramping, can lower blood pressure and raise sugar levels. Pregnant women, children and those recovering from surgery are advised against using the product. It has been validated for use in osteoporosis.
Shark cartilage has also been noted to be used for psoriasis, healing wounds, retina damage due to diabetes and inflammation of the intestines. The FDA has given the cartilage "Orphan Drug Status"for renal cell carcinoma. This law allows drug pharmaceuticals special incentives to study drugs for rare conditions.
Currently there has been no reported cases for drug interactions of this product. Doses are dependent on a variety of factors such as age, health and other conditions. There is no current information for proper dosage of this product. Follow directions on label and consult health practitioner before using this product.
Shark liver oil is also being used to treat health conditions. The liver oil is either brown or yellow in appearance. Fisherman for a very long time have used the oil for health treatments. The oil is an abundant source alkyiglycerols which just possibly contain anti-cancer elements. There are currently two other chemicals in the oil which are being studied for cancer they are squalene and squalamine.
Shark liver oil is vastly used in northern Europe as one of the conventional cancer treatments. However, in the United States it is sold as a dietary supplement. Current research is aiming at the components of the oil alkyiglycerols, squalene and squalamone. Past scientific studies have demonstrated the oil possibly has anti-cancer reactions on tumors in animals. Clinical trails based on if it works in humans are currently being studied.
We do know that shark liver oil aides in heightening the immune system, fighting infections, in cancer treatments (northern Europe) and decreases side effects of conventional cancer treatments.
The theory of the alkyiglycerols provide benefits in several different ways. It has been proposed that when fighting cancer they kill off the tumor cells indirectly. Advocates allege they stimulate the immune system by activation of immune cells (macrophages) that eat germs that are trying to penetrate along with damaged cells. The other way is that they have the ability to lessen side effects from chemotherapy and radiation therapy. This is due to the fact they can guard the cell membrane.
Due to the ability to heighten the immune system, there are claims that they provide protection against colds, flu, chronic infections, asthma, arthritis and AIDS.
Previous studies have demonstrated that squalamine can slow down the growing of tumor blood vessels. Advocates lay claim that it just possibly could treat cancer either independently or with chemotherapy. It is currently in studies for macular degeneration, eye condition in which ends in vision loss. Squalene is advocated to having cell protection ability which in turn could possibly lessen side effects that occur from chemotherapy.
All claims to shark liver oil are currently being researched.
Based on how the oil is prepared for commercial use it possibly can be loaded with omega 3 fatty acids and Vitamin A. The oil has been used in already in some moisturizing skin creams and lotions. Just to note some cosmetic companies have removed the ingredient over concern for the decline in the shark population.
The shark liver oil is being sold in capsules and liquid forms. You can purchase it at many of the health food stores and over the internet.
To clarify a few points in the shark liver oil. There is no current evidence that the supplement are effective to treat cancer. There have been a couple of studies that did present some benefit to women who were having radiation therapy for cervical cancer. There has been no other confirmed research in this area since 1980.
Recent studies have demonstrated squalamine has lessened the amounts of lung metastases, tumors in which spread to lungs from a primary cancer which is elsewhere in the body and this was found in animals. Studies conducted early in humans did show it can be used safely with chemotherapy. It is still not determined if it does shrink the tumor or prolongs survival of patients. It is undergoing studies with other treatments for lung and prostate cancers.
Some side effects include nausea and upset stomachs have been noted. In animal studies it had shown it could raise blood pressure. If you are allergic to seafood you could have a reaction.
The dosage for shark liver oil is also undetermined at this time so it is advisable to talk with your practitioner first.
Please do not use this product on its own current merit solely. It is used to aide in other treatments and best used with the advice of your health care practitioner.
Sharks are labeled with a bad persona. Mainly due to movies like "Jaws" where they go around and chomp down for a nightly feast on any human or animal they can find. There are one million slaughters going on each year on this creature. However, these creatures of the sea have the ability to aide in certain medical conditions such as anemia, brain bleeding and cancer.
Scientists for decades have been studying the shark, well at least his cartilage. They believe this creature has something that can offer the medical community especially in the treatment of cancer. It is a known fact that sharks hardly ever get cancer. There must be something in their bodies that aide in protection from this disease.
The theory of the shark cartilage and cancer among most of the scientific community is having to do specifically with the composition of the cartilage. For one thing cartilage cannot grow cancer due to the fact it has no blood vessels. The theory is that the cartilage manufactures elements that barricade the growing of new blood vessels that would feed on the tumor. So by using the cartilage to treat the tumor the blood vessels then should be stopped from developing on the tumor.
A study was performed to scientifically prove or disprove the theory of shark cartilage. Nine patients had participated in this study. When the study had been completed the evidence indicated that seven of the nine patients who had advanced cancer there was an 87% positive response from the treatment. It is important to note that no other treatment was applied during this study.
The evidence laid out had shown the following results (in brief form here after seven weeks of treatment and all patients were diagnosed as terminal)
A few results:
Female patient stage three uterine cancer going into bladder. Radiation had no effects on the tumor. After seven weeks of the cartilage the tumor reduced in size by 80% and after eleven weeks the tumor had gone and so did her pain. The only remaining factor was scar tissue.
Female with vaginal hemangioma in size of 5"x 5" after hysterectomy and partial vaginectomy along with radiation. Heavy bleeding was life threatening complication. At seven weeks tumor reduced by 60% and bleeding had stopped. At eleven weeks tumor size was 3"x 3" with further treatment it was stated tumor would appear to decrease.
Male stage three cancer on back of right thigh. After nine weeks of cartilage therapy surgery was done. It was noticed that 60% of the tumor was dead and surgically removed.
The summary of this research had indicated that further research was still required but shark cartilage treatments had displayed promise.
Studies on shark cartilage are still being conducted however, these studies are extremely expensive. At the same time environmentalists are advocating for the shark and making the fact known that the shark population has decreased significantly in the prior fifteen years due to over fishing and the ever on going demand for shark products.
The compounds of the shark cartilage are being tested by a few pharmaceutical companies. If developed it would only be able to be available by prescription and it is stated the cartilage is coming from those sharks who have been slaughtered for meat.
Shark cartilage in research has shown it can help alleviate arthritis symptoms. In all reality scientists had determined it is one of the main components in the shark cartilage which is chondroitin sulfate that is the magic behind the alleviation. To make a note the supplements which are sold in health food stores usually come from cows and that of sharks.
Shark cartilage has demonstrated benefits in patients with arthritis. It decrease joint pain and inflammation which enabled patients to use less pain relievers. Good news is a lot of persons who use it in small doses have no major problems. Some can have side effects which include nausea, vomiting, cramping, can lower blood pressure and raise sugar levels. Pregnant women, children and those recovering from surgery are advised against using the product. It has been validated for use in osteoporosis.
Shark cartilage has also been noted to be used for psoriasis, healing wounds, retina damage due to diabetes and inflammation of the intestines. The FDA has given the cartilage "Orphan Drug Status"for renal cell carcinoma. This law allows drug pharmaceuticals special incentives to study drugs for rare conditions.
Currently there has been no reported cases for drug interactions of this product. Doses are dependent on a variety of factors such as age, health and other conditions. There is no current information for proper dosage of this product. Follow directions on label and consult health practitioner before using this product.
Shark liver oil is also being used to treat health conditions. The liver oil is either brown or yellow in appearance. Fisherman for a very long time have used the oil for health treatments. The oil is an abundant source alkyiglycerols which just possibly contain anti-cancer elements. There are currently two other chemicals in the oil which are being studied for cancer they are squalene and squalamine.
Shark liver oil is vastly used in northern Europe as one of the conventional cancer treatments. However, in the United States it is sold as a dietary supplement. Current research is aiming at the components of the oil alkyiglycerols, squalene and squalamone. Past scientific studies have demonstrated the oil possibly has anti-cancer reactions on tumors in animals. Clinical trails based on if it works in humans are currently being studied.
We do know that shark liver oil aides in heightening the immune system, fighting infections, in cancer treatments (northern Europe) and decreases side effects of conventional cancer treatments.
The theory of the alkyiglycerols provide benefits in several different ways. It has been proposed that when fighting cancer they kill off the tumor cells indirectly. Advocates allege they stimulate the immune system by activation of immune cells (macrophages) that eat germs that are trying to penetrate along with damaged cells. The other way is that they have the ability to lessen side effects from chemotherapy and radiation therapy. This is due to the fact they can guard the cell membrane.
Due to the ability to heighten the immune system, there are claims that they provide protection against colds, flu, chronic infections, asthma, arthritis and AIDS.
Previous studies have demonstrated that squalamine can slow down the growing of tumor blood vessels. Advocates lay claim that it just possibly could treat cancer either independently or with chemotherapy. It is currently in studies for macular degeneration, eye condition in which ends in vision loss. Squalene is advocated to having cell protection ability which in turn could possibly lessen side effects that occur from chemotherapy.
All claims to shark liver oil are currently being researched.
Based on how the oil is prepared for commercial use it possibly can be loaded with omega 3 fatty acids and Vitamin A. The oil has been used in already in some moisturizing skin creams and lotions. Just to note some cosmetic companies have removed the ingredient over concern for the decline in the shark population.
The shark liver oil is being sold in capsules and liquid forms. You can purchase it at many of the health food stores and over the internet.
To clarify a few points in the shark liver oil. There is no current evidence that the supplement are effective to treat cancer. There have been a couple of studies that did present some benefit to women who were having radiation therapy for cervical cancer. There has been no other confirmed research in this area since 1980.
Recent studies have demonstrated squalamine has lessened the amounts of lung metastases, tumors in which spread to lungs from a primary cancer which is elsewhere in the body and this was found in animals. Studies conducted early in humans did show it can be used safely with chemotherapy. It is still not determined if it does shrink the tumor or prolongs survival of patients. It is undergoing studies with other treatments for lung and prostate cancers.
Some side effects include nausea and upset stomachs have been noted. In animal studies it had shown it could raise blood pressure. If you are allergic to seafood you could have a reaction.
The dosage for shark liver oil is also undetermined at this time so it is advisable to talk with your practitioner first.
Please do not use this product on its own current merit solely. It is used to aide in other treatments and best used with the advice of your health care practitioner.
Saturday, September 4, 2010
Federal stem cell ruling blocks Yale scientists
By Rachel Gilmore
For two decades, Lawrence Rizzolo, the director of medical studies at the Yale School of Medicine, has been working toward a project that aims to transplant young, healthy retinal cells to replace diseased tissues in the eyes of patients who are going blind.
But now Rizzolo fears he may have to delay, or even stop, his research because of last week’s ruling by a federal judge that prevents federal funding for studies involving embryonic stem cells, the building blocks for human organs and tissues that Rizzolo needs for his project.
Rizzolo had applied for a grant from the National Institutes of Health to replace his funding from the nonprofit International Retinal Research Foundation, which ends in December. Rizzolo also has a three-year state grant from Rocky Hill, Conn.-based state holding company Connecticut Innovations, but he says he cannot continue his research without the federal funding.
Rizzolo’s laboratory is one of about a dozen facilities on campus that use stem cells. Haifan Lin, director of the Yale Stem Cell Center, said he did not yet know how the moratorium will affect Yale researchers.
“We’re all waiting for clarification on the implications of the judge’s ruling,” University President Richard Levin said.
But frozen funding could lead scientists to lose their jobs. Rizzolo said that although a fourth researcher will join his laboratory in October, he may soon have to fire his workers because the grants and not the University pay for his researchers’ salaries.
Although the U.S. Department of Justice has filed a motion seeking to delay the federal judge’s ban on funding, the relief would only be temporary, NIH spokesman Don Ralbovsky said. The Justice Department declined to comment Thursday.
Opponents of embryonic stem cell research have hailed the ruling.
“The American people should not be forced to pay for experiments — prohibited by federal law — that destroy human life,” said Steven Aden, the legal counsel for the Alliance Defense Fund, which advised the party that brought the lawsuit to court.
Most labs at Yale’s Stem Cell Center are supported primarily by state funds, which are not affected by the ruling, said Diane Krause, the center’s assistant director. But, she explained, those state grants were meant to be a springboard for acquiring federal funding.
“Discontinuation of NIH funding of work with human embryonic stem cells could be a disaster,” she said.
School of Medicine Dean Robert Alpern agreed, saying that halting the stem cell research would be a “loss to society.”
Krause and other Yale researchers met Aug. 26 with Connecticut attorney general and U.S. Senate candidate Richard Blumenthal LAW ’73, who told the News this week that he plans to file a brief in support of the appeal.
“It is incredibly important for our scientific and legislative leaders to come together and solve this issue quickly,” Blumenthal said in a statement. “We have already lost so many years of work to backwards-looking and bureaucratic federal policy.”
Linda McMahon, his Republican challenger for Chris Dodd’s Senate seat, also supports stem cell research, according to her website. The McMahon campaign did not respond to multiple phone requests for comment this week.
Even if the Justice Department successfully delays the ruling, NIH has not said whether it would accept new applications. That would be a problem for Yibing Qyang, an assistant professor of cardiology at the School of Medicine who currently has a $95,499 federal grant and had planned on applying for another in October.
“I cannot live without federal funding,” said Qyang, who is priming stem cells to become healthy heart cells in patients with heart disease.
Yale has received at least $17 million in state funding from a $100 million stem cell research pot funded in part by tobacco taxes. It also received $4.9 million in NIH stem cell research grants in 2009 and 2010. NIH gave $123 million to researchers in fiscal year 2010 for embryonic stem cell research, Ralbovsky said.
Rizzolo studies eye tissues called retinal pigment epithelia. Many eye diseases — including macular degeneration, from which more than 10 million Americans suffer — affect these tissues, which function as a support for the retina, the part of the eye responsible for sight. Doctors have tried to transplant retinal and epithelial cells to combat these diseases. But most of the transplants fail because they are risky and the patients who choose to undergo them are usually almost blind. Patients in the early stages of macular degeneration are unlikely to attempt a transplant; Rizzolo said he hopes that his research with stem cells, which he started to use about a year ago, would lead to other, less risky, treatments for the disease.
Rizzolo added that, in the past year, two Yale colleagues had entirely shut down their stem-cell research labs because of funding shortages.
“It’s pretty frustrating when you work so hard to see something go unfunded for political reasons,” he said.
The Yale Stem Cell Center, established in 2006, brings together more than 30 faculty members across the University.
For two decades, Lawrence Rizzolo, the director of medical studies at the Yale School of Medicine, has been working toward a project that aims to transplant young, healthy retinal cells to replace diseased tissues in the eyes of patients who are going blind.
But now Rizzolo fears he may have to delay, or even stop, his research because of last week’s ruling by a federal judge that prevents federal funding for studies involving embryonic stem cells, the building blocks for human organs and tissues that Rizzolo needs for his project.
Rizzolo had applied for a grant from the National Institutes of Health to replace his funding from the nonprofit International Retinal Research Foundation, which ends in December. Rizzolo also has a three-year state grant from Rocky Hill, Conn.-based state holding company Connecticut Innovations, but he says he cannot continue his research without the federal funding.
Rizzolo’s laboratory is one of about a dozen facilities on campus that use stem cells. Haifan Lin, director of the Yale Stem Cell Center, said he did not yet know how the moratorium will affect Yale researchers.
“We’re all waiting for clarification on the implications of the judge’s ruling,” University President Richard Levin said.
But frozen funding could lead scientists to lose their jobs. Rizzolo said that although a fourth researcher will join his laboratory in October, he may soon have to fire his workers because the grants and not the University pay for his researchers’ salaries.
Although the U.S. Department of Justice has filed a motion seeking to delay the federal judge’s ban on funding, the relief would only be temporary, NIH spokesman Don Ralbovsky said. The Justice Department declined to comment Thursday.
Opponents of embryonic stem cell research have hailed the ruling.
“The American people should not be forced to pay for experiments — prohibited by federal law — that destroy human life,” said Steven Aden, the legal counsel for the Alliance Defense Fund, which advised the party that brought the lawsuit to court.
Most labs at Yale’s Stem Cell Center are supported primarily by state funds, which are not affected by the ruling, said Diane Krause, the center’s assistant director. But, she explained, those state grants were meant to be a springboard for acquiring federal funding.
“Discontinuation of NIH funding of work with human embryonic stem cells could be a disaster,” she said.
School of Medicine Dean Robert Alpern agreed, saying that halting the stem cell research would be a “loss to society.”
Krause and other Yale researchers met Aug. 26 with Connecticut attorney general and U.S. Senate candidate Richard Blumenthal LAW ’73, who told the News this week that he plans to file a brief in support of the appeal.
“It is incredibly important for our scientific and legislative leaders to come together and solve this issue quickly,” Blumenthal said in a statement. “We have already lost so many years of work to backwards-looking and bureaucratic federal policy.”
Linda McMahon, his Republican challenger for Chris Dodd’s Senate seat, also supports stem cell research, according to her website. The McMahon campaign did not respond to multiple phone requests for comment this week.
Even if the Justice Department successfully delays the ruling, NIH has not said whether it would accept new applications. That would be a problem for Yibing Qyang, an assistant professor of cardiology at the School of Medicine who currently has a $95,499 federal grant and had planned on applying for another in October.
“I cannot live without federal funding,” said Qyang, who is priming stem cells to become healthy heart cells in patients with heart disease.
Yale has received at least $17 million in state funding from a $100 million stem cell research pot funded in part by tobacco taxes. It also received $4.9 million in NIH stem cell research grants in 2009 and 2010. NIH gave $123 million to researchers in fiscal year 2010 for embryonic stem cell research, Ralbovsky said.
Rizzolo studies eye tissues called retinal pigment epithelia. Many eye diseases — including macular degeneration, from which more than 10 million Americans suffer — affect these tissues, which function as a support for the retina, the part of the eye responsible for sight. Doctors have tried to transplant retinal and epithelial cells to combat these diseases. But most of the transplants fail because they are risky and the patients who choose to undergo them are usually almost blind. Patients in the early stages of macular degeneration are unlikely to attempt a transplant; Rizzolo said he hopes that his research with stem cells, which he started to use about a year ago, would lead to other, less risky, treatments for the disease.
Rizzolo added that, in the past year, two Yale colleagues had entirely shut down their stem-cell research labs because of funding shortages.
“It’s pretty frustrating when you work so hard to see something go unfunded for political reasons,” he said.
The Yale Stem Cell Center, established in 2006, brings together more than 30 faculty members across the University.
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