Seventy-five percent of patients with moderate to severe alopecia areata—an autoimmune disease that causes patchy and, less frequently, total hair loss—had significant hair regrowth after treatment with ruxolitinib.
This is reported by Columbia University Medical Center (CUMC).
By the end of treatment, average hair regrowth among the patients was 92 percent.
Findings from an open-label clinical trial of 12 patients with alopecia areata are published in the Journal of Clinical Investigation/Insight, alongside a paper reporting results of a separate study from Stanford University and Yale University that tested a similar drug.
“Although our study was small, it provides crucial evidence that JAK inhibitors may constitute the first effective treatment for people with alopecia areata,” said Julian Mackay-Wiggan, MD, MS.
“This is encouraging news for patients who are coping with the physical and emotional effects of this disfiguring autoimmune disease.”
Alopecia areata, the second most common form of hair loss, can occur at any age and affects men and women equally.
The disease usually causes hair loss on the scalp, but some patients also experience facial and body hair loss with devastating consequences, particularly in children. Currently, there are no known treatments that can completely restore hair.
Previously, the Columbia researchers identified the specific immune cells and the dominant inflammatory signaling pathways responsible for attacking the hair follicle in alopecia areata, putting them into a dormant state.
Subsequent experiments with mouse and human hair follicles showed that topical and oral drugs that inhibit the Janus kinase (JAK) family of enzymes, known as JAK inhibitors, reawaken these dormant follicles by blocking inflammatory signaling.
Two such JAK inhibitors already approved by the U.S. FDA are ruxolitinib, a medication that is used to treat bone marrow malignancies, and tofacitinib, a treatment for rheumatoid arthritis.
In the study, the researchers initiated a small, open-label clinical trial of 12 patients with moderate to severe alopecia areata (more than 30 percent hair loss).
All patients were given 20 mg of oral ruxolitinib, twice a day, for three to six months. Participants were followed for an additional three months to assess the durability of treatment response.
Nine of the patients had hair regrowth of 50 percent or greater. By the end of the treatment period, 77 percent of those who responded to the therapy achieved hair regrowth of more than 95 percent.
A third of the responders had significant hair loss in the follow-up period after the medication was stopped, although hair loss did not reach pre-treatment levels.
Skin biopsies performed before, during, and after treatment also revealed that responders had less inflammatory response—and higher levels of hair growth.
These levels were similar to those in people without alopecia areata.
The drug was well-tolerated in all participants, with no serious adverse events. Those that did occur were infrequent and included bacterial skin infections, skin allergy symptoms, and lower hemoglobin levels, which resolved with dose adjustment.
In the Stanford/Yale study, a series of patients with moderate to severe alopecia areata responded to another JAK inhibitor called tofacitinib.
“Together, the two studies show that we’re on the right track,” said Dr. Christiano, who is a co-author of the tofacitinib paper.