A new open-access paper in nature communications, published August 1, 2026, reports on two people with APECED who were treated with ruxolitinib as autoimmune endocrine dysfunction was developing.
Dr. Lionakis and his team of investigators reported that JAK1/2 inhibition with ruxolitinib halted progression and reversed biochemical and clinical abnormalities associated with evolving hypoparathyroidism in one patient and hypergonadotropic hypogonadism in another, preserving endocrine function during the reported follow-up periods. The authors describe the findings as proof-of-concept for a “disease-interception” approach—identifying autoimmune injury early enough that targeted treatment may preserve organ function before irreversible damage occurs.
These are encouraging observations, but the study involved only two patients. The authors emphasize the need for larger prospective studies and longer follow-up to determine generalizability, durability, optimal dosing, and long-term safety. Patients should discuss any treatment questions with their own physicians.