StemCells, Inc. Launches Clinical Trial for Dry AMD Treatment

June 27, 2012

A potential stem cell treatment for preserving vision in people with dry age-related macular degeneration (AMD) is moving into a Phase I/II clinical trial at the Retina Foundation for the Southwest (RFSW) in Dallas. Developed by StemCells, Inc. (SCI) of Newark, California, the therapy is designed to integrate into the patient’s retina and release proteins that keep his or her existing retinal cells healthy and functional.

In January 2012, SCI reported that, in a rodent study, its treatment “significantly protected photoreceptors from degeneration.” The company noted that the number of cone cells, the photoreceptors responsible for central and color vision, remained constant for an extended period of time. In untreated animals, the cones were lost.

SCI’s treatment is now the second stem-cell-based therapy to move into human studies for retinal disease. Advanced Cell Technology (ACT) launched Phase I/II clinical trials of its treatment in 2011 for people with dry AMD and Stargardt disease. Early results of ACT’s studies were encouraging.

“We are very pleased to see another stem cell treatment move into in a clinical trial for retinal degenerations,” says Dr. Stephen Rose, chief research officer, Foundation Fighting Blindness. “Stem cells hold great therapeutic promise, and these human studies are a critical step toward saving vision for people affected by AMD and potentially inherited retinal diseases such as retinitis pigmentosa.”

While both approaches are stem-cell-based, SCI’s treatment differs from ACT’s therapy in that it involves transplantation of human, neural stem cells. ACT’s therapy involves the transplantation of mature retinal pigment epithelial (RPE) cells, which were derived from embryonic stem cells. RPE cells provide a critical supportive role in the retina and are affected in diseases such as AMD,Stargardt disease and some forms of retinitis pigmentosa.

“Both approaches have shown promise for preserving vision,” says Dr. Rose. “It will be interesting to see how well they perform over the long term in clinical trials. Ideally, we will see benefits from both.”

In May 2012, StemCells, Inc. reported positive interim results of their Phase I/II clinical trial of a stem cell treatment for spinal cord injury.

The company issued a press release on June 21, 2012, announcing its clinical trial for AMD. Additional information on the study will be posted soon to and the Foundation’s Web site. The trial will be led by Dr. David Birch, chief scientific and executive officer of the RFSW, and co-director of the Foundation’s Southwest Regional Research Center for the Study of Retinal Degenerative Diseases.