Repurposing Bone Marrow Stem Cell Therapy for Vision Loss Caused by Retinitis Pigmentosa

Principal Investigator: Dr. Susanna Park

​

Disease: Retinitis Pigmentosa

Research Description: Retinitis pigmentosa (RP) is a rare hereditary form of retinal degeneration that can lead to total blindness. Although gene therapy has been developed recently for the rarest, severest form of RP, there is no effective treatment to date for most patients with RP. This clinical trial will investigate whether a patient’s own bone marrow stem cells can be used to treat vision loss from RP. Stem cells transplants have been for years used to treat cancer and also have been investigated to treat heart disease. In addition, previous research has shown that injecting stem cells into the eye can slow retinal degeneration in animals. Eight patients with RP will be treated with their own stem cells to determine the safety and efficacy of this treatment. The team will also evaluate any possible beneficial effects of the stem cell therapy on patient’s vision. If successful, this research could become the basis for larger clinical trials and could lead to improved vision and quality of life for RP patients.

Funding Partners: Goldman Philanthropic Partnerships

CWR funding role: Participating funder