Congratulations to our medical advisory committee members, Drs. Michail Lionakis and Maureen Su, on their recent publications in the prestigious New England Journal of Medicine, entitled The Role of Interferon-γ in Autoimmune Polyendocrine Syndrome Type 1 and JAK Inhibition Immunotherapy for APS-1, respectively. In his article, Dr. Lionakis, whose work led to the first-ever clinical trial for APS Type 1, reports on the results of treating APS Type 1 patients with Ruxolitinib. These articles are behind a paywall, but if you sign up with the New England Journal of Medicine you can access several articles each month without charge.
Dr. Lionakis’ article discusses the development of a possible new therapy to treat individuals with APS Type 1. The article traces the development of this treatment beginning with his natural history study of more than 100 adult and pediatric patients with APS Type 1. Based on conclusions drawn from the natural history study (in which so many of us participate), responses to a variety of treatments were evaluated using APS Type 1 animal models. Based on these findings, Ruxolitinib was used to treat five patients with severe autoimmunity, which led to remission of alopecia, oral candidiasis, nail dystrophy, gastritis, enteritis, arthritis, Sjögren’s-like syndrome, urticaria, and thyroiditis. Dr. Lionakis is currently enrolling patients in his Stage 2 Clinical Trial for Ruxolitinib. If you have alopecia and APS Type 1, you may be eligible.