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FDA approves study of embryonic stem cells to treat juvenile macular degeneration

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MARLBOROUGH, Mass. — The U.S. Food and Drug Administration has approved Advanced Cell Technology's investigational new drug application to launch the first clinical trial using retinal cells derived from human embryonic stem cells for treating Stargardt's macular dystrophy, the company announced in a press release.

A form of juvenile macular degeneration, Stargardt's macular dystrophy spurs progressive vision loss in patients between 10 years and 20 years of age. The associated photoreceptor loss stemming from degeneration of the retinal pigment epithelium can eventually result in blindness.

"There is currently no treatment for Stargardt's disease," Robert Lanza, MD, chief scientific officer of Advanced Cell Technology, said in the release. "Using stem cells, we can generate a virtually unlimited supply of healthy [retinal pigment epithelium] cells, which are the first cells to die off in [Stargardt's macular dystrophy] and other forms of macular degeneration."

Preclinical animal studies have shown complete recovery of visual function with no negative side effects, according to the release.

The prospective, multicenter phase 1/2 clinical trial is designed to assess the safety and tolerability of retinal pigment epithelium cells following subretinal implantation in patients with advanced Stargardt's macular dystrophy. To date, the trial has enrolled 12 patients. Proposed trial sites include Jules Stein Eye Institute (Los Angeles); Casey Eye Institute (Portland, Ore.); University of Massachusetts Memorial Medical Center (Worcester, Mass.); and UMDNJ-New Jersey Medical School (Newark, N.J.).

Advanced Cell Technology's retinal pigment epithelium cell technology was granted orphan drug designation by the FDA earlier this year.

"With the momentum of this Stargardt's trial, and the unique experience we have gained as one of the few companies in the world having succeeded in taking a [human embryonic stem cell] program into the clinic, we are preparing to extend our lead with an [investigational new drug application] in the use for treating age-related macular degeneration, as well as filing to begin clinical trials in Europe, in the very near future," Edmund Mickunas, vice president of Advanced Cell Technology's regulatory division, said in the release.