Inherited Retinal Diseases

Stem cell therapy shows promise in inherited retinal diseases

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Stem cell therapy appears to be a potentially effective and safe treatment for patients with retinitis pigmentosa or Stargardt disease, according to a study, although long-term vision improvement appears more limited for patients with retinitis pigmentosa.

The study encompassed 21 prospective trials, involving 496 eyes (404 with retinitis pigmentosa and 92 with Stargardt disease) of 382 patients (306 with retinitis pigmentosa and 76 with Stargardt disease).

For those with retinitis pigmentosa, there was a 49% improvement rate in BCVA at 6 months and 30% at 12 months. Furthermore, BCVA was notably enhanced in the treated eyes at 6 months post-therapy, with a mean difference of -0.12 logMAR. However, no significant difference was observed at the 12-month mark post-treatment.

For those with Stargardt disease, there was a 60% improvement in BCVA at 6 months, followed by 55% at 12 months. The operative eyes exhibited substantial improvement at both time points, with mean differences of -0.14 logMAR and -0.17 logMAR  at 6 and 12 months, respectively.

Subgroup analyses indicated that the suprachoroidal space injection of stem cells might hold promise for retinitis pigmentosa patients. The study reported 11 treatment-related ocular adverse events from 3 studies, but no related systemic adverse events were recorded.

Chen X, Xu N, Li J, et al. Stem cell therapy for inherited retinal diseases: a systematic review and meta-analysis. Stem Cell Res Ther. 2023;14(1):286. doi: 10.1186/s13287-023-03526-x. PMID: 37798796.