$17.3 Million Invested in Stem Cell Treatment for RP

Sept. 10, 2012 – Stem cell research focused on saving vision is getting a big boost thanks to a $17.3 million award from the California Institute of Regenerative Medicine (CIRM) to Dr. Henry Klassen, an associate professor of ophthalmology at the University of California, Irvine and a former Foundation Fighting Blindness Career Development Awardee. His ultimate goal is to seek FDA authorization to launch a clinical trial of a stem cell treatment for people with retinitis pigmentosa, or RP, a condition that causes severe vision loss and blindness in 100,000 people in the United States and thousands more around the world.

Established in November 2004 with the passage of Proposition 71, CIRM is authorized to provide $3 billion in funding over 10 years for stem cell research in facilities throughout California.

Dr. Klassen will use eye-derived “progenitor” stem cells — cells that have partially developed into retinal cells — as the basis for his emerging treatment. He is optimistic that transplanted progenitors can “rescue and reactivate” inactive cones in the retinas of people with even advanced cases of RP.

Cones are a key target for therapies, because they provide central vision and the ability to read, drive and recognize faces. His work will include growing pharmaceutical-grade progenitors, testing them for safety and efficacy in animal models, and then launching a human study.

“We are delighted by this major investment in stem cell research for an inherited retinal disease,” says Dr. Stephen Rose, chief research officer, Foundation Fighting Blindness. “Foundation-funded research has shown that stem cells hold extraordinary promise for saving and restoring vision in people with RP and a variety of retinal degenerations. This funding will help get the treatment out to those who need it.”