An Important Science Update from the Foundation Fighting Blindness

FDA Authorizes Stem Cell Clinical Trial for RP in Los Angeles

Phase 1/2a human study will evaluate neural progenitors for preserving vision
Cedars-Sinai, a non-profit healthcare organization in Los Angeles, has received authorization from the US Food & Drug Administration (FDA) to launch a 16-person, Phase 1/2a clinical trial of human neural progenitor cells—stem cells that have almost developed into neural cells—for slowing retinal degeneration and preserving vision in people with retinitis pigmentosa (RP). Investigators will be able to launch the trial after their study protocol receives final institutional review. The trial is being funded by a $10.5 million grant from the California Institute for Regenerative Medicine, a state-based stem-cell research institute.

The study's principal investigator is Clive Svendsen, PhD, professor of Biomedical Sciences and Medicine and director of the Cedars-Sinai Board of Governors Regenerative Medicine Institute. David Lao, MD, from Retina-Vitreous Associates Medical Group in Beverly Hills, will be injecting the cells subretinally in patients. Shaomei Wang, MD, PhD, a professor of Biomedical Sciences and a research scientist in the Eye Program at the Board of Governors Regenerative Medicine Institute, conducted the initial lab research that showed these cells offer promise for treating RP.

When launched, the Cedars-Sinai clinical trial will be the third stem-cell study for people with RP. Two companies, ReNeuron and jCyte, are conducting Phase 2 trials for retinal progenitors–stem cells that have almost matured into photoreceptors–for RP patients. The emerging ReNeuron treatment is designed to restore vision by replacing photoreceptors. The emerging jCyte therapy is designed to preserve and rescue the patient's existing photoreceptors. Thus far, both the ReNeuron and jCyte therapies have had good safety profiles and shown early signs of efficacy.

"We are glad to see more cell-based therapies move into clinical trials for RP. These trials are all at an early stage, so we have a lot to learn. But results so far are encouraging," says Brian Mansfield, PhD, executive vice president of research and interim chief scientific officer at the Foundation Fighting Blindness.