Advanced Cell Technology (ACT;OTCBB:ACTC), Inc. has received clearance from the US Food and Drug Administration to initiate a Phase I/II multicenter clinical trial using retinal cells derived from human embryonic stem cells (hESCs) to treat patients with Stargardt’s Macular Dystrophy (SMD). This decision has removed the clinical hold that the FDA had previously placed on the trial, allowing ACT to move forward.
Stargardt’s Macular Dystrophy usually first presents itself in children between the age of 10 and 20. Early on, this disease causes progressive vision loss which can eventually lead to blindness due to photoreceptor loss associated with the degeneration in the pigmented layer of the retina, referred to as the retinal pigment epithelium (RPE).
Unfortunately, there is no treatment currently available for Stargardt’s Macular Dystrophy. ACT Chief Scientific Officer, Dr. Robert Lanza, said, “Using stem cells, we can generate a virtually unlimited supply of healthy RPE cells, which are the first cells to die off in SMD and other forms of macular degeneration. We’ve tested these cells in animal models of eye disease. In rats, we’ve seen 100% improvement in visual performance over untreated animals without any adverse effects. Our studies showed that the cells were capable of extensive rescue of photoreceptors in animals that otherwise would have gone blind. 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.”
The main goal of the Phase I/II trial will be to determine the tolerability of the RPE cells following sub-retinal transplantation. At this time, 12 students will be taking part in the study at multiple sites across the United States. The sites being considered for the study include: University of Massachusetts Memorial Medical Center in Worcester, Massachusetts; UMDNJ – New Jersey Medical School in Newark, New Jersey; Casey Eye Institute in Portland, Oregon; and the Jules Stein Eye Institute at UCLA. Other sites may also be considered.
In early 2010, ACT was also granted Orphan Drug designation by the FDA. This means that the company is eligible to receive benefits including access to grant funding, tax credits, accelerated FDA approval, and allowance for marketing exclusivity for up to seven years.
Did you know that nearly 30 million people in the Europe and United States suffer from macular degeneration? This is one of the most common causes of untreatable blindness in the world.
With Advanced Cell Technology moving onto a Phase I/II multicenter clinical trial there is great hope in better treating age-related macular degeneration in the years to come.
Disclosure: no positions