First RP Patient Receives Stem-Cell Therapy in ReNeuron’s Clinical Trial

March 17, 2016

ReNeuron, a stem-cell development company in the United Kingdom, has reported that the first patient with retinitis pigmentosa (RP) has been treated with its stem-cell therapy in a Phase I/II clinical trial taking place at Massachusetts Eye and Ear Infirmary (MEEI). While the early-stage trial is focused on safety, ReNeuron plans to report preliminary efficacy results in 2017.

The treatment involves the injection of human retinal progenitor cells (hRPCs)—stem cells that have partially developed into photoreceptors—underneath the patient’s retina. Based on results from preclinical studies, researchers believe the injected hRPCs will integrate into the retina and fully develop into photoreceptors, replacing those lost to disease and, thereby, restoring vision. They also believe the treatment will protect the patient’s retina from further photoreceptor loss. Photoreceptors are the cells that make vision possible, but are lost to retinal diseases such as RP.

“This is a major milestone in the effort to save and restore vision in people who are suffering from RP,” says Patricia Zilliox, Ph.D., chief drug development officer, at the Foundation’s Clinical Research Institute (CRI). “While the primary goal of the initial phase of the trial is to evaluate safety, we are hopeful that the treatment will ultimately have a major impact on patients’ vision.”

The Foundation has funded stem-cell research for decades, including laboratory projects led by Michael Young, Ph.D., and Ray Lund, Ph.D., which made ReNeuron’s clinical trial possible. Dr. Young conducted the final preparatory study in a large-animal model, also funded by the Foundation, before ReNeuron sought authorization from the U.S. Food and Drug Administration to initiate its clinical trial. ReNeuron consulted with the Foundation’s CRI in designing and organizing the groundbreaking human study.

“The launch of the ReNeuron clinical trial is a great moment in the fight against blinding retinal diseases,” says William T. Schmidt, chief executive officer at the Foundation. “It took many years of challenging, painstaking research to get to this promising juncture. But the commitment and tenacity of the retinal-research community has paid off in a huge way.”

Eric Pierce, M.D., Ph.D., director of the Berman-Gund Laboratory at MEEI and former chairman of the Foundation’s Scientific Advisory Board, is the study’s lead investigator.

“We are very pleased to be part of the team effort and working with the Foundation and MEEI to bring this exciting technology to patients,” says Julian Howell, M.D., chief medical officer at ReNeuron. “Although we are just at the start of the clinical development of hRPC cells, our goal is to make safe and successful treatments available as soon as possible to people living with RP.”

RP is an inherited retinal disease affecting approximately 100,000 people in the United States and more than two million people worldwide. Though dozens of genes, when mutated, can cause the condition, ReNeuron believes its hRPCs can benefit most people with RP, regardless of their genetic profile.