Bone Marrow Disease Drug Restores Hair Growth in Alopecia

Ruxolitinib, an FDA-approved drug used for treatment of a rare bone marrow disease, could help alopecia areata patients regrow hair, according to a new study.

Four years ago, a Columbia University Medical Center-led team of researchers conducted a study of more than 1,000 patients with alopecia areata, a common autoimmune disorder that results in unpredictable hair loss. The authors of that study recently studied mice with the disease, identifying a certain set of T cells responsible for attacking hair follicles.
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Through their additional research into the cells of mice as well as humans, the investigators pinpointed a number of immune pathways, through which these T cells initiate their attack on hair follicles. According to the authors, these pathways can be targeted by a class of drugs known as JAK inhibitors.

The team tested two FDA-approved JAK inhibitors—ruxolitinib and tofacitinib, which has been approved for the treatment of rheumatoid arthritis—on mice with alopecia areata that had severe hair loss hair from the disease. These drugs fully restored the rodents' hair within 12 weeks, according to the authors, who note that this hair regrowth lasted for months after treatment had ceased.

In the most recent study, the CUMC researchers reported on the findings of a small, open-label clinical trial of ruxolitinib on patients with moderate-to-severe alopecia areata, which is defined as having more than 30 percent hair loss. Early results indicated that hair growth was fully restored within 4 months to 5 months of initiating treatment in 3 participants. Additionally, the T cells that attack the hair follicles were no longer found in the scalps of these participants.

While noting that further testing is necessary to determine whether ruxolitinib should be used for individuals with alopecia areata, the authors described the findings as a potentially significant step toward improving the standard of care for those with the disease.

“Most primary care practitioners will refer patients with alopecia areata to a dermatologist if they wish to pursue steroid injections,” says Angela Christiano, PhD, a professor of dermatology and genetics at CUMC, and a co-author of the study.

“Some primary care physicians may offer topical steroids, but in many difficult cases, these will usually be seen in dermatology practices.”

These findings “offer all practitioners the opportunity to discuss ongoing clinical trials in alopecia areata in a meaningful and exciting way for patients,” continues Christiano.

“For the first time, these findings represent testing of a therapy that was predicted based on understanding the underlying science. In a very short timeframe of 4 years since the discovery of the first genes associated with alopecia areata, the field has moved very rapidly toward testing new drugs selected on the basis of those predictions.”

—Mark McGraw 

Reference

Clynes R, Christiano A, et al. Alopecia-Areata is Driven by Cytotoxic T Lymphocytes and is Reversed by JAK Inhibition. Nature Medicine. 2014.