Phase 1 Study Finds Bone Marrow Stem Cells Safe as Potential Treatment for Retinitis Pigmentosa

Wednesday, February 12 2025 | 08 h 49 min | Vision Science

A team of researchers at University of California Davis has published the results of a Phase 1 clinical trial testing stem cells therapy for retinitis pigmentosa (RP). The study which involved seven participants found that intravitreal injection of the cells was safe and all, but one participant had either stable or improved best corrected visual acuity (BCVA) six months after treatment.

There are two main types of stem cell therapy currently being developed for treatment of retinal degeneration, including for inherited retinal diseases like RP, Stargardts and Usher syndrome as well as age-related macular degeneration.

Restorative cell therapy tries to replace damaged cells (like photoreceptors) with new cells grown from stem cells. This type of cell therapy could be useful for individuals with advanced vision loss.

The UC David team is developing a second type of cell therapy is called neuroprotective therapy which takes advantage of the fact that stem cells secrete growth and survival factors that can help other cells survive. This study was promising but needs to be tested
in larger Phase 2/3 clinical trials to assure safety and efficacy.

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