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By John Gever, Senior Editor, MedPage Today
Published: January 06, 2011

A clinical trial of a human embryonic stem cell (ESC) therapy for age-related macular degeneration has been approved by the FDA, making it the first such study for a common disease.

Advanced Cell Technology, based in Marlborough, Mass., said it had received the agency’s clearance to begin a phase I/II trial in 12 patients with age-related “dry” macular degeneration, which affects about 10 to 15 million people in the U.S.

Patients will receive implants with retinal pigment epithelial (RPE) cells derived from the company’s ESC lines.

Other clinical trials of ESC-based therapies are under way or approved to begin, but they have been for comparatively rare conditions: certain severe spinal cord injuries and Stargardt’s macular dystrophy, a childhood-onset form of blindness that resembles age-related macular degeneration.

Advanced Cell Technology is also conducting the latter trial, using the same ESC-derived RPE cells. The two studies will run simultaneously, the company said.

In a statement, the company’s chief scientific officer, Robert Lanza, MD, said that 50,000 to 200,000 cells would be implanted beneath the retina in each patient in the open-label macular degeneration trial.

The relatively small cell numbers mean “manufacturing and distribution of the therapeutic product is scalable,” Lanza said.

Studies with its lab-grown RPE cells in rodent models of macular degeneration and Stargardt’s disease have demonstrated improvements in visual function, according to the company.

Both clinical trials are designed primarily to assess safety and tolerability.

Clinical sites for the macular degeneration study have not yet been finalized, but Advanced Cell Technology listed centers at UCLA and Stanford as under consideration.

This information is brought to you by Clarin Eye Care, a family Optometry practice in Palmetto Bay, Florida.  Please call or contact our office for more information.

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