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.”

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

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)

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.

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."

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.

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.

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.

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.

Alcon Terminates the Development of Anecortave Acetate in Age-Related Macular Degeneration

HUENENBERG, Switzerland -- Alcon, Inc. (NYSE:ACL) announced today it has terminated the development program designed to evaluate the benefit of anecortave acetate treatment on the risk for developing sight-threatening choroidal neovascularization secondary to age-related macular degeneration. The decision followed a planned interim analysis of studies C-02-60 A and B that was performed after 2,546 patients had completed the 24 month time point. In this analysis, anecortave acetate showed no effect on the primary or secondary endpoints. In addition to terminating studies C-02-60 A and B, the company also terminated two smaller studies with an identical design that were being conducted in Asia, C-04-30 and C-05-34.

The company continues to study anecortave acetate administered as an anterior juxtascleral depot to reduce intraocular pressure in patients with open-angle glaucoma.

About Alcon

Alcon, Inc. is the world's leading eye care company, with sales of approximately $5.6 billion in 2007. Alcon, which has been dedicated to the ophthalmic industry for more than 60 years, researches, develops, manufactures and markets pharmaceuticals, surgical equipment and devices, contact lens care solutions and other vision care products that treat diseases, disorders and other conditions of the eye. Alcon's majority shareholder is Nestle, S.A., the world's largest food company. For more information on Alcon Inc., visit the company's Web site at www.alcon.com.

The Aging Eye: Researchers Aim To Stop the Clock

By JANE E. BRODY

AGING Americans expect more from their eyes these days than ever before. People in their 70's and 80's want to be able to drive, play cards, recognize people on the street, travel with their grandchildren, take advantage of senior discounts in the movies and read the books they missed while working full time.

But eyes have a way of aging that can render such expectations unrealistic. Far worse than the loss of visual acuity that prompts most middle-aged people to resort to magnifying lenses are sight-robbing diseases like glaucoma, cataracts, age-related macular degeneration, diabetic retinopathy and other retinal disorders that afflict tens of millions of Americans, usually after age 50.

The incidence of such potentially blinding disorders is increasing rapidly as the number of older people grows. Experts predict, for example, that by the year 2030, 6.3 million older Americans will develop macular degeneration, up from 1.7 million in 1995. It is a still-irreversible disorder that robs people of the central vision needed to drive, read, watch television, recognize faces, play cards or do any fine work. If Grandma Moses had had macular degeneration, her artistic talents would never have been noticed.

Fortunately, research is progressing on a number of promising new treatments, including low doses of radiation, and a combination of lasers and light-activated chemicals, both of which are used for some particularly hard-to-treat forms of macular degeneration. Other research is concentrating on how to stop toxins that damage the eye in glaucoma, and the genetics of several different eye diseases.

''Older Americans today expect to enjoy their retirement with the same visual capacity that they had in their younger years,'' said Dr. Harold Spalter, professor of ophthalmology at Columbia-Presbyterian Medical Center in New York. But, alas, as was apparent at a four-day seminar that Dr. Spalter chaired here last month, researchers are still a long way from knowing how to reverse most blinding eye disorders.

Still, major progress in understanding and treating these conditions -- and perhaps detecting them early enough to blunt their effects -- was evident at the seminar, organized by Research to Prevent Blindness, a New York-based voluntary organization. Unfortunately, though, many elderly Americans cannot afford the early detection procedures described at the seminar because Medicare and many other insurance programs do not cover such preventive measures.

For example, while insurance companies would routinely cover a visual field examination for a patient who already has glaucoma, most would not pay for this test for a person who has not yet experienced vision loss, when the disease process might be stopped without lasting vision damage.

Furthermore, an ongoing study of 2,520 men and women aged 65 to 84 in Salisbury, Md., has revealed that the usual eye chart test for visual acuity is inadequate to assess vision losses that interfere with the ability of elderly people to get around on their own, perform tasks of daily living and avoid accidents that can result in serious or fatal injuries. Rather, Dr. Sheila West, professor of ophthalmology at Johns Hopkins Medical Institutions, reported that tests for contrast sensitivity -- the ability to distinguish, say, a step from the one below it -- are more revealing of functional disability in older people.

''We have found that loss of contrast sensitivity is as important as arthritis and heart failure in determining loss of mobility in the aged,'' Dr. West said. She traced this loss to ''nonspecific retinal changes'' and the beginnings of cataracts, a gradual clouding of the lens of the eye that eventually obscures vision. However, early-stage cataracts are rarely recognized by those who have them and are often dismissed as inconsequential by eye doctors.

Sunlight, Dr. West said, is a major factor in the formation of cataracts, and the damage is cumulative. By assessing the exposure of study participants to sunlight, Dr. West and colleagues determined that for every 1 percent increase in exposure to ultraviolet-B light, the risk of developing cataracts rose by 10 percent.

''There is no threshold for sun-related damage, the dosage is cumulative and no group is immune to it,'' Dr. West said the study showed. She recommended that when out of doors, everyone -- starting in childhood -- should wear lenses that block ultraviolet light and a cap with a brim that shades the eyes.

Dr. M. Cristina Leske, head of preventive medicine at University Medical Center in Stony Brook, N.Y., and associates, identified other risk factors for cataracts. Through a five-year study of 764 patients, they found that Caucasians are three times as likely as blacks to develop cataracts. Those who take the gout medicine allopurinol face more than a two-fold increase in risk, and smokers have a 60 percent increase.

On the other hand, certain nutrients appear protective. The risk was 30 percent lower among those who took multivitamin-mineral supplements and nearly 60 percent lower among those who took a vitamin E supplement, a finding that is now being tested in a clinical trial sponsored by the National Eye Institute. Still another study of 247 women aged 56 to 71 conducted at Tufts University in Boston found that taking vitamin C supplements for more than 10 years reduced the risk of early cataracts by 77 percent and the risk of moderately advanced cataracts by 83 percent.

But while cataracts can usually be treated very successfully by surgically removing the damaged lens and replacing it with a synthetic lens implant, age-related macular degeneration, the leading cause of legal blindness in the elderly, has yet to yield to an effective treatment. Macular degeneration involves progressive damage to the cells in the center of the retina that are responsible for straight-ahead vision.

Early cases are often treated with lasers, which have the unfortunate side effect of destroying normal retinal cells as well as the damaged areas beneath them. Furthermore, after laser treatment, the vision-damaging tissue often grows back.

Dr. Dennis M. Marcus, an ophthalmologist at the Medical College of Georgia in Augusta, said that laser therapy usually cannot be used for the most severe form of the disease -- so-called wet macular degeneration, which involves the growth of leaky blood vessels beneath the central retina. Instead, he and his colleagues are testing low-dose radiation to destroy the blood vessels but spare the normal retinal cells. Thus far, 100 patients have been treated in a clinical trial that will eventually involve 500 people with wet macular degeneration. While it is too soon to evaluate the effectiveness of the treatment, Dr. Marcus said that he has seen no radiation-induced complications.

Another clinical study is testing a technique called photodynamic therapy. It starts with the intravenous administration of a photosensitive dye that collects in the damaging blood vessels that are growing beneath the retina. The eye is then exposed to laser light that activates the dye, destroying those vessels only. Dr. Joan W. Miller, an ophthalmologist at the Massachusetts Eye and Ear Infirmary, said that preliminary studies showed that the technique effectively closes off the leaky vessels in the majority of patients. And while these vessels reopen and leak in some patients, the treatment can be repeated, if needed, without harm to the eye.

Some seminar participants said the best hope for conquering blinding eye diseases was unraveling the sometimes complex genetics underlying many if not all of these conditions. Just last month, for example, a team of scientists announced the discovery of the first genetic link to age-related macular degeneration, which strikes 25 percent of Americans over the age of 65 and is the major cause of vision loss in the elderly. The researchers hope that by studying mutations in this gene they will gain an understanding of how the disease damages the eye, a means of identifying those at risk and methods of prevention and treatment.

Glaucoma, for example, usually involves elevated pressure inside the eye, leading eventually to the death of ganglion cells, the nerve cells that transmit information from the eye to the brain. This disease afflicts perhaps eight million Americans and causes blindness in 5,500 each year. Currently the only available treatment involves continual use of eye drops that reduce intraocular pressure. This only works if treatment is begun early.

Dr. Robert W. Nickells, an eye researcher at the University of Wisconsin in Madison, said, however, that ''new advances suggest that glaucoma could be treated during the second or even the third stage of the disease.'' The second stage involves the release of high concentrations of ''excitotoxins,'' amino acids that are toxic to nerve cells. Dr. Nickells said that several compounds that intervene in the formation of excitotoxins have been developed to treat other neurodegenerative disorders and may also prove useful in treating glaucoma.

As for the third stage, he and his colleagues have found in monkeys and rodents that ganglion cells succumb to a form of programmed cell death that appears to be controlled by three genes that act as a molecular switch. One of the genes, called bcl-x, prevents the fatal blow and might be harnessed therapeutically to override the cell death mechanism, Dr. Nickells said.

But discoveries about the genetics of eye disorders can sometimes raise more questions than they answer. For example, Dr. Fulton Wong of Duke University Medical Center reported that as many as 50 genes are believed to be involved in the progressive disease retinitis pigmentosa, which begins as night blindness and loss of peripheral vision and eventually destroys central vision, leaving people blind. Thus far, four genes have been identified, each with multiple mutations that may result in different aberrations of the condition. One of the genes that codes for the production of the visual pigment rhodopsin can exist in 92 different mutated forms, Dr. Wong reported.

This year alone, three genes for various forms of glaucoma have been identified. But Dr. Janey Wiggs, an ophthalmologist and geneticist at Tufts University School of Medicine in Boston, said: ''This is a very complicated disease, with maybe 20 or 30 genes involved. And finding genes is only the first step. Where and when is the gene required and how does it produce disease? Does it result in too much or not enough of a gene product or make a toxic product?''

Still, she and others expressed guarded enthusiasm for the prospects of gene therapy to treat various devastating eye diseases. ''The eye is accessible,'' she said. ''It can be given selective treatment, using the other untreated eye as a control to see how well the treatment is working.''

Dr. J. Timothy Stout, head of the division of ophthalmology at Children's Hospital in Los Angeles, said, ''The potential use of gene therapy is nearly limitless for the ophthalmologist.'' He and his colleagues are exploring in animals the potential of a so-called suicide gene to treat eye diseases that involve excessive cell division, such as intraocular proliferative disease, macular degeneration and diabetic retinopathy. Normally, most cells in an adult eye are not dividing. Using as a gene carrier a virus that infects only actively dividing cells, he introduces into the eye a gene that by itself is not toxic but that results in cell death when combined with the drug ganciclovir, which also does not harm normal cells. Thus, the treatment is specific for the proliferating cells involved in the eye disease.