Polyamine-Based Therapeutic Approach for Alopecia Areata

Executive Summary:

Oral administration of DFMO (Eflornithine), a polyamine inhibitor, offers a novel treatment to reduce elevated polyamine levels and stimulate hair regrowth in alopecia areata patients.

Description:

Alopecia areata is an autoimmune hair loss disorder that affects approximately 5 million patients in the United States. Laboratory research in alopecia mouse models by Dr. André Bachmann (Michigan State University) and clinical insights by Dr. Caleb Bupp (Corewell Health) and their teams, as well as independently published literature, found that elevated ODC protein and high polyamines correlate with hair loss in alopecia areata. Therefore, they proposed that the FDA-approved drug DFMO (Eflornithine) can reduce these polyamine levels. Evidence from alopecia mouse models demonstrates that oral DFMO effectively promotes hair regrowth by normalizing polyamine concentrations, providing a promising therapeutic option for people with alopecia areata.

Of note, Dr. André Bachmann (Michigan State University), Dr. Caleb Bupp (Corewell Health), and colleagues discovered Bachmann–Bupp syndrome (BABS), a neurodevelopmental condition caused by mutations in the ODC1 gene that includes alopecia. Their research has focused on elucidating the molecular basis of the disorder, measuring polyamines, and evaluating the use of DFMO as a potential therapeutic intervention which has shown hair regrowth in treated patients. This work laid the foundation for their research into DFMO-based treatment of alopecia areata.

Bupp et al., 2025. Repurposing With Purpose: Treatment of Bachmann-Bupp Syndrome With Eflornithine and Implications for Other Polyaminopathies. Am J Med Genet C Semin Med Genet. Sep;199:183-188.

Bachmann et al., 2023. Bachmann-Bupp syndrome and treatment. Dev Med Child Neurol. Apr;66:445-455.

Key Advantages:

• Utilizes an already FDA-approved drug with established safety profile for continuous, long-term use
• Targets a novel biochemical pathway for hair regrowth, supported by human genetic and mouse model evidence
• Potential for lifelong treatment with well-known side effect profile (compared to JAK inhibitors)
• Overcomes limitations of or supplements existing hair regrowth treatments for alopecia areata
• Addresses large untreated worldwide patient population need

Applications: Hair regrowth therapy

Patent Status: PCT patent application filed

Licensing Rights: Full licensing rights available