Stem Cells Get FDA Nod For Stargardt's Disease

by Randall V. Wong, M.D.

Advanced Cell Technology, Inc., received FDA approval for starting Phase I/II clinical trial to use stem cells for the treatment of Stargardt’s disease. Stargardt’s disease is an inherited type of macular degeneration affecting individuals at age 10-20. There is no cure.

Similar to other macular diseases, the photoreceptors, and the layer of cells just beneath, call the retinal pigment epithelium (RPE), become destroyed due to a genetic defect. Due to the macular involvement, central vision is lost.

Stargardt’s disease is the a common form of “macular degeneration” that afflicts the young. It is estimated to affect about 30,000 people worldwide. It shares in common with ARMD (age related macular degeneration) the loss of photoreceptors secondary to RPE damage and degeneration.

Advanced Cell Technology (ACT) claims the ability to generate healthy RPE cells from human embryonic stem cells. The idea is to replace the genetically diseased RPE cells with healthy replacements. In theory, the healthy RPE cells should prevent loss of the photoreceptors, thereby preserving vision.

The phase I/II study will involve 12 patients enrolled into several centers across the United States. The initial experiments will determine if the RPE cells are indeed safe and if they can be tolerated by the human recipients (i.e., does the body reject the new RPE cells?).

What Does This Mean? Stargardt’s disease has no cure, and therefore, are great subjects to consider for this possible treatment. In a way, there is nothing to lose. Stargardt’s patients also have or had vision, that is, they have experience with vision. We know that these patients have fully developed visual pathways.

While this group is very small in comparison to patients with macular degeneration, the significance this holds for a potential, effective treatment can only be left to our imagination. Even a small success in this trial is exciting.

The news of this trial is exciting, but remember further testing (clinical trials III and IV) need to be completed. Also, the techniques for introducing the cells safely underneath the retina need to be accomplished, too.