Testing a Generic Malarial Drug in a Rare Ophthalmic Condition, Retinitis Pigmentosa

Principal Investigator: Dr. David Zacks

Disease: Retinitis Pigmentosa

Research Description: Certain mutations are a common cause of retinitis pigmentosa (RP), a rare ophthalmic condition resulting in night blindness, loss of peripheral vision and ultimately, profound vision loss. Currently, there are no approved therapies for patients with RP. Previous animal work by Dr. Zacks and his team have shown that drugs blocking a process called autophagy, or the breaking down and recycling of cell structures, may have potential for treating patients with RP caused by a specific mutation. Dr. Zacks and his team are testing a drug originally used to treat malaria that has already been repurposed to treat rheumatologic diseases. This proof of concept clinical trial will test the safety and tolerability of this drug in RP patients with a specific mutation, and will evaluate its ability to improve patients’ symptoms.

Funding Partners: Goldman Philanthropic Partnerships

CWR funding role: Primary funder