Wednesday, March 3, 2010

A Human Embryonic Stem Cell Therapy Moves Forward

Tuesday, March 2, 2010
By: Meredith Wadman, Nature

Advanced Cell Technology of Worcester, Massachusetts announced on Tuesday that a human embryonic stem cell therapy it is developing for a rare form of juvenile blindness has been granted orphan drug status by the US Food and Drug Administration (FDA).

The special status gives companies tax breaks, access to grant funding for clinical trials, and up to seven years of market exclusivity under the 1983 Orphan Drug Act . The Act is aimed at speeding therapies for diseases afflicting fewer than 200,000 Americans.

ACT applied last November for permission to begin human trials of its stem-cell-based therapy for Stargardt’s disease. Formally known as Stargardt’s Macular Dystrophy, it is a degenerative disease of the retina that affects roughly 1 in 10,000 US youngsters. A closely related cause of blindness, Age-related Macular Degeneration (AMD), affects millions of Americans.

Edmund Mickunas, ACT’s vice president for regulatory matters, said that the company plans to use the orphan drug designation to “accelerate” clinical development. (Along with providing tax breaks, the designation lessens some of the statistical burdens in proving safety and efficacy to FDA.)

The company’s therapy uses human embryonic stem cells to recreate retinal pigment epithelium cells, which support the photoreceptors needed for vision. It has shown promise in rat and mouse models of macular degenerative disease. Abstracts of the relevant papers are here and here.

More details are available at this FDA webpage, which links to a searchable database of orphan-designated products. Search for ACT’s therapy under its generic name, MA09-hRPE.

Significantly, the cells used in the ACT therapy are derived from pre-blastocyst human embryos, meaning that work on them should become eligible for federal funding under a change to regulations proposed last month by the National Institutes of Health.

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