The U.S. Food & Drug Administration approved for clinical trial a novel stem cell?based treatment for retinitis pigmentosa (RP) developed by DEF-funded researcher Dr. Henry Klassen and colleagues at the University of California, Irvine (UCI). The start of this clinical trial will be the culmination of a project that began in 2006, when DEF brought Klassen to UCI to bring this research from the laboratory to human patients.
Retinitis pigmentosa (RP) is a group of hereditary blinding disorders that begins with the loss of rod photoreceptors in the retina. The new treatment is intended to preserve vision by intervening when degenerating photoreceptors (rods and cones) can be protected and potentially reactivated.
?This milestone is a very important one for our project,? says Klassen, an associate professor of ophthalmology with UCI?s Gavin Herbert Eye Institute. ?It signals a turning point, marking the beginning of the clinical phase of development, and we are all very excited about this progress.?
The primary purpose of the trial is to determine the safety of a single injection of retinal progenitor cells into the eyes of patients with advanced RP. The trial will begin enrolling patients at UCI in the next few months.
?We are very pleased that a project our donors have been helping to fund is entering clinical trials,? says DEF Medical Director Dr. Anthony Nesburn. ?This treatment is moving toward its approval for patients with RP; eventually we hope it will be able to help people with AMD, as well. The process can take a long time, but investing in this type of research is the only way we will find treatments and cures for blinding retinal diseases.?
May 2015