Novel therapies for hair loss

Hair loss affects hundreds of millions worldwide and can be androgenetic, stress-associated, or age-related. Current treatments are limited to oral dihydrotestosterone (DHT) inhibitors and topical minoxidil. DHT inhibitors require continuous use, carry systemic side effects, and are not approved for use in women. Minoxidil promotes vasodilation but has limited efficacy and does not restore stem cell function. There is a clear unmet need for therapies that safely and effectively reactivate hair follicle stem cells (HFSCs) to enable hair growth across a wide range of conditions.

Our studies have identified a class of small-molecule compounds that potently promote HFSC activation and hair regeneration. In mouse models, a single treatment induces rapid and robust stem cell activation and hair growth, with efficacy exceeding all HFSC-targeting approaches evaluated to date. These compounds have favorable drug-like properties and represent a promising therapeutic strategy for directly promoting endogenous hair regeneration.

In this proposal, we will (1) rigorously assess the efficacy and safety profile of these compounds on hair growth, including local versus systemic effects, and (2) test their ability to promote human hair follicle regeneration in culture and in xenograft models using human scalp skin transplanted onto immunodeficient mice. These studies will establish therapeutic potential and evaluate relevance to human biology. Together, this work will advance a new class of regenerative therapeutics toward more effective, localized, and broadly applicable treatments for hair loss.