Millions of people in North America live with varying degrees of irreversible vision loss. Some good news is that a novel stem cell therapy for retinitis pigmentosa (RP), an inherited condition that slowly damages the retina and can result in blindness, has been cleared by the U.S. Food and Drug Administration (FDA) to start a Phase I clinical trial.
The therapy for RP, which affects 1 in 3500 Canadians, was developed by Dr. Henry Klassen, Associate Professor at the Gavin Herbert Eye Institute, UC Irvine School of Medicine, and will consist of injecting patients with stem cells to help replace the cells destroyed by the disease and to save those cells that are not damaged yet.
The goal of the trial, which will involve up to 16 patients, is to evaluate the safety and identify side effects of the treatment. Although it is early to speak about a cure for RP, the researchers are hopeful.
“This milestone is a very important one for our project. It signals a turning point, marking the beginning of the clinical phase of development, and we are all very excited about this project.” said Dr. Klassen in the CIRM press release.
The trial has received almost $20 million in funds from the California Institute for Regenerative Medicine (CIRM).
“One of the goals of the agency is to provide the support that promising therapies need to progress and ultimately to get into clinical trials in patients. RP affects about 1.5 million people worldwide and is the leading cause of inherited blindness in the developed world. Having an effective treatment for it would transform people’s lives in extraordinary ways.” Jonathan Thomas, Ph.D., J.D., Chair of the Governing Board of the CIRM, said in the CIRM press release.
Click here to discover more about how stem cells are being used to understand and treat eye diseases on our Toward Treatments page.