Alopecia alert: Drugs for arthritis and bone marrow disease restore hair growth

By John Murphy, MDLinx
Published September 23, 2016

Key Takeaways

Two new studies have shown that two different Janus kinase (JAK) inhibitors regrew hair in patients with alopecia areata. Researchers at Yale and Stanford demonstrated hair regrowth with tofacitinib, a rheumatoid arthritis drug, and researchers at Columbia University showed similar results with ruxolitinib, used for myelofibrosis. The two studies were published September 22, 2016 in JCI Insight.

No effective treatments are available for alopecia areata, an autoimmune disease characterized by partial or complete loss of scalp or body hair. However, preliminary studies have indicated that JAK inhibitors can reverse the type I inflammatory response that underlies alopecia areata, even in patients with longstanding or more severe forms of disease.

These studies aimed to further investigate this effect.

Tofacitinib trial

Tofacitinib is a JAK inhibitor in pill form that is marketed as Xeljanz® by Pfizer Inc. for the treatment of moderate to severe rheumatoid arthritis. For this clinical trial, researchers treated 66 alopecia patients with 5 mg of tofacitinib twice daily for 3 months. By the end of the trial, nearly two-thirds of the subjects had at least some hair regrowth. Of these, almost one-third recovered 50% or more of the scalp hair that was lost.

However, after the treatment stopped, so did the hair growth. All patients who responded to tofacitinib began losing hair again about 2 months after discontinuing the drug. Additional research is needed to determine a dosing duration that can provide a longer-lasting response, the researchers concluded.

“The only missing pieces are, one, the results of treatment over longer periods of time and, two, looking at treatment of the pediatric age group, where the disease frequently shows up first and can be particularly devastating,” said co-senior author Brett A. King, MD, PhD, Assistant Professor of Dermatology at Yale University School of Medicine, in New Haven, CT. “We’ll have those results soon.”

Ruxolitinib trial

A smaller, open-label pilot study of ruxolitinib—which is marketed as Jakafi® by Incyte Corp. for treating intermediate or high-risk myelofibrosis—involved 12 patients with moderate to severe alopecia, all of whom were given a 20 mg oral tablet of ruxolitinib twice a day for 3 to 6 months. The researchers followed the subjects for an additional three months after the treatment period.

Nine out of 12 patients responded to treatment, with hair regrowth of 50% or greater. Regrowth began within 1 month in responders and progressed at a rapid rate, the researchers observed. By the end of the treatment period, 7 of the 9 responders to the therapy achieved hair regrowth of more than 95%. Six months of therapy was enough to achieve the maximum amount of hair regrowth in the majority of responders.

“Although our study was small, it provides crucial evidence that JAK inhibitors may constitute the first effective treatment for people with alopecia areata,” said study co-author Julian Mackay-Wiggan, MD, Associate Professor and Director of the Clinical Research Unit in Dermatology at Columbia University Medical Center, in New York, NY. “This is encouraging news for patients who are coping with the physical and emotional effects of this disfiguring autoimmune disease.”

As in the tofacitinib study, subjects in this trial began to shed hair within weeks of discontinuing ruxolitinib, with one-third of respondents experiencing significant hair loss. However, hair loss did not reach pre-treatment levels in any patient.

“Our findings suggest that initial treatment induces a high rate of disease remissions in patients with moderate to severe alopecia areata, but maintenance therapy may be needed,” Dr. Mackay-Wiggan said. “While larger, randomized trials are needed to confirm the safety and efficacy of ruxolitinib in people with moderate to severe alopecia areata, our initial results are very encouraging.”

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