Unmet NeedRetinitis pigmentosa (RP) is a genetic disorder with a prevalence of 1 in 4000 people worldwide. RP can be the result of a number of mutations that all result in severe rod photoreceptor degeneration and death, causing excess oxygen in the outer retina and subsequent oxidative damage to cone photoreceptors. Symptom onset begins in childhood and eventually results in significant vision loss and blindness in adulthood. Presently, RP has no cure. While life-style modifications and disability services can improve life with vision loss, there is no effective treatment for RP. A retinal implant is available for those with profound disease but this does not restore one’s normal vision. Consequently, there is a great need for novel therapeutics to treat RP that can be easily delivered to the retina over the life-time of the individual and are well-tolerated in the eye.
Technology OverviewJohns Hopkins researchers have demonstrated repurposing of the approved and well-tolerated drug, Metipranolol, as a novel, noninvasive therapeutic for RP. In the
rd10 mouse model of RP, Metipranolol treatment administered by eye drop or subcutaneous injection, slowed the death of rod photoreceptors compared to vehicular treated controls. Additionally, Metipranolol reduced levels of nitrosative stress in the retina, promoting enhanced cone survival and photoreceptor function.
Stage of DevelopmentThe inventors have identified a novel therapeutic function for Metipranolol as a treatment of retinitis pigmentosa. Further studies will be conducted to determine efficacy in preserving photoreceptor cells and vision in human patients and to also optimize formulation and delivery to the human retina.
