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ACT gets FDA Nod for Stem Cell Trial Targeting Juvenile Eye Disease

Kathleen Blanchard's picture

Advanced Cell Technology (ACT) has received approval to go forward with stem cell therapy trials for treatment of a retinal eye disease that strikes children and leads to blindness. There is currently no treatment for the disease called Stargardt’s Macular Dystrophy, a type of macular degeneration. ACT spokespersons say stem cell therapy for the eye disease opens up a worldwide umet medical need.

Stem Cells Resulted in 100 Percent Vision Improvement in Mice

The stem therapy uses retinal cells derived from human embryonic stem cells (hESCs) transplanted directly into the retina that resulted in 100 percent vision improvement in mice. Advanced Cell Technology’s Scientific Officer, Robert Lanza says, “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.”

The company will move forward to Phase I/II clinical trials in hopes of producing the same results in humans after receiving an immediate “go-ahead” from the FDA.

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Stargardt’s Macular Dystrophy that strikes young adults age 10 to 20 causes blindness from destruction of photoreceptor cells, eventually leading to blindness. Stem cell treatment of the disease is geared toward saving the retinal pigment epithelium (RPE).

The trials will take place at multiple sites. Under consideration are Jules Stein Eye Institute at UCLA, the University of Massachusetts Memorial Medical Center in Worcester Massachusetts and the UMDNJ – New Jersey Medical School in Newark, New Jersey.

William M. Caldwell IV, ACT’s Chairman and CEO says, "I think generations will look back at this time as one of the most exciting in the history of medicine. With the initiation of this clinical trial, and that of Geron’s earlier this fall, the field of regenerative medicine is poised to take embryonic stem cell therapies from the realm of nebulous potential to that of tangible and real treatments that will make a significant difference in the lives of millions of people worldwide. This is truly a ‘game changer’ for the medical community.”

Treatment for macular degeneration is an “unmet” need that destroys quality of life says Edmund Mickunas, ACT’s Vice President of Regulatory. In the near future Advanced Cell Technology hopes to use stem cell therapy from human embryonic retinal cells to target age-related macular degeneration that affects a significant number of older adults. ACT also plans to file for approval of clinical trials in Europe. The company says targeting retinal eye disease opens a “$25-30 billion worldwide market that has yet to be effectively addressed.”

Advanced Cell Technology