Reserpine: A New Hope for Inherited Blinding Diseases

A recent NIH animal study has shown promising results for repurposing reserpine, an old antihypertensive drug, in preventing vision loss in retinitis pigmentosa (RP) — a rare, inherited blinding disease. No definitive treatment currently exists for RP, which progressively damages retinal photoreceptors starting from childhood.

Key Findings:

• In a rat model of autosomal dominant RP (rhodopsin mutation), reserpine preserved rod photoreceptor function and structure, particularly in female rats.
• The drug’s neuroprotective effects appear independent of specific gene mutations, making it potentially useful for various inherited retinal dystrophies.
• Interestingly, females showed greater preservation of both rod and cone photoreceptors, suggesting possible sex-specific responses worth further investigation.

Why This Matters:

• Reserpine is a small molecule drug — easier and cheaper to deliver than gene therapies, with potential for low-dose, intraocular use.
• Could act as a bridge therapy to slow vision loss in RP and related conditions until more definitive, gene-targeted treatments become widely available.

The research team is now developing more potent reserpine-related compounds for future studies.

Reference:

Song HB et al. eLife. 2025. DOI: 10.7554/eLife.103888.1