American Regulators approve Trial for Stargardt’s
November 22, 2010 - The Food and Drug Adminstration in the United States will permit Advanced Cell Technology, Inc. to test a new treatment for Stargardt’s Macular Dystrophy (MD). The treatment uses embryonic stem cells to restore a layer of eye cells known as the retinal pigment epithelium (RPE). These cells are rapidly lost in Stargardt’s MD resulting in a loss of vision. Read our stem cell therapies fact sheet to learn more about how stem cell therapies work.
This is a very early stage trial that will enrol only 12 people from across the United States. It will test the safety of the treatment and how well patients can tolerate the symptoms. Although Stargardt’s MD often begins showing symptoms in the early teens, only people over 18 years of age will be permitted to enter the trial. This is only the second trial of a treatment using embryonic stem cells to be allowed in the United States. The first trial, which explored the use of embryonic cells to restore the spinal cord of injured patients, was halted for more than a year over safety concerns.
Dr. Robert Lanza, Chief Scientific Officer for Advanced Cell Technology Inc. says this trial is much less likely to encounter problems. “The advantage, of course, is that we’re talking about a very small number of cells going into a very local area,” he told the science journal Nature.
The company has tested the treatment in various animal models. “In rats,” says Dr. Lanza, “we’ve seen 100% improvement in visual performance over untreated animals without any adverse effects."
Although this trial does not aim to directly replace lost photoreceptors, if a person has already lost their vision, the company's pre-clinical animal studies suggest that replenishing the retinal pigment epithelium may help damaged photoreceptors repair themselves and restore some vision.
"Our studies showed that the cells were capable of extensive rescue of photoreceptors in animals that otherwise would have gone blind," says Lanza. "Near-normal function was also achieved in a mouse model of Stargardt’s disease. We hope to see a similar benefit in patients with various forms of macular degeneration.” (See the scientific paper describing these findings, if desired.)
While testing will begin on Stargardt’s MD, the company is hopeful that this therapy may also be useful for other forms of macular degeneration – including age related macular degeneration (AMD). AMD is the leading cause of blindness in Canadians over the age of sixty.
Update: A trial of this stem cell treatment to treat dry AMD (age-related macular degeneration) has now also been approved in the USA. Both the AMD and Stargardt Disease trials are very early Stage 1 trials, designed only to test the safety of the treatment and how well patients can tolerate any side-effects. Both trials will be conducted at the Jules Stein Eye Institute at the UCLA School of Medicine in California under the direction of Dr. Steven Schwartz. A second site at the Oregon Health and Sciences University in Portland is also recruiting patients for the Stargardt trial. Use this link for more details about the criteria for enrolling in the Stargardt disease trial.
In Sept 2011, investigators at Moorfields Eye Hospital in London, England also received approval to begin testing this therapy.
Learn more about this trial on the Advanced Cell Technology Inc. web site.