Daily Topical Rapamycin for Vitiligo

While rapamycin is not approved for the treatment of vitiligo, it has been found to be efficacious in stopping disease progression in animal models. Another recent study in humans showed that doses of 0.5cc daily of 0.001% of the topical formulation are able to achieve improvement in hypopigmented lesions of the skin at both the clinical and histological level. At this dose, improvement in cell function was observed regarding maintenance of proliferative potential and prevention of senescence, with avoidance of total inhibition of cell growth as has been seen at higher potencies used for immunosuppression.

Rapamycin is an immunosuppressant that works by inhibiting the mammalian target of rapamycin (mTOR) leading to inhibition of the cell cycle and antibody production. It has also been shown to promote expansion of Treg populations. Although studies evaluating alternative dosing for vitiligo are limited, topical rapamycin has previously been used to treat angiofibromas related to Tuberous Sclerosis, vascular malformations such as port-wine stains, and inflammatory lesions such as plaque psoriasis. In a 2019 meta-analysis, 38 out of 40 reports included used topical formulations of 1% or lower potency rapamycin; the majority of publications were focused on the treatment of Tuberous Sclerosis, where the median concentration of mTOR inhibition was 0.1% dosed twice daily.

All patients will be assigned to received topical placebo cream to the lesion not being treated with the active study drug.