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.
Saturday, January 15, 2011
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.
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