By Rita Rubin
WebMD Health News
Reviewed by Michael W. Smith, MD
Aug. 19, 2014-- A drug used to treat a rare bone marrow cancer caused hair to grow back in some people with alopecia areata, an autoimmune disease that attacks the hair follicles, scientists have found.
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A small study shows that the drug, ruxolitinib, was effective in mice and in three people with the disease. Alopecia areata is thought to affect between 4 million and 5 million Americans, can strike at any age, and affects men and women equally. They are generally otherwise healthy. But they lose patches of hair and, in rare cases, all of their body hair.
The two scientists who led the new study are Raphael Clynes, MD, PhD, who recently left Columbia University Medical Center to work for Bristol-Myers Squibb, and Angela Christiano, PhD, a professor in the departments of dermatology and of genetics and development at Columbia. Here, they discuss their research, which appears online in Nature Medicine.
Q. Why would you think to use a cancer drug to treat alopecia?
A. Christiano's past research suggested that in people with alopecia, hair follicles send out a false "danger signal" that triggers the immune system to attack them. Further research, which she and Clynes describe in their new report, identifies one way to stop the attack -- a new class of drugs that includes ruxolitinib, approved in 2011 to treat the bone marrow cancer myelofibrosis.
Q. How many people with alopecia have been treated with ruxolitinib?
A. Clynes and Christiano have treated a total of a dozen, the first three of whom are included in the new study. After 5 months of treatment, all three regrew more than half of the hair they had lost.
"They were very pleased and happy," Clyne says. Of the next six patients, though, only three regrew that much hair, he says. The last three haven't been treated long enough to reach any conclusions.
Q. If ruxolitinib promotes hair growth in people with alopecia, could it be used to treat run-of-the mill male-pattern baldness?
A. "It's not obvious at this point," Christiano says. She's looking into that question, though.
Q. What are the side effects?
A. The labeling for the drug lists a number of serious potential side effects, such as low blood counts and infection. More common and less serious side effects include headache and dizziness, according to the labeling. Clynes says one person in his study has had "a touch of anemia. Nothing significant."
Because people with alopecia are generally healthy, he says, "we didn't expect much change in the blood counts." The side effects may be worse in people with underlying and chronic illnesses, he says. And ruxolitinib probably does suppress the immune system, putting people at risk for infections.
Q. Why would anyone take a potentially risky medication for months just to regrow hair?
A. "This is a group of patients who've really had nothing," says Christiano, who was diagnosed with alopecia at age 30 in 1996. "There's no FDA-approved treatment. They're desperate for something that works."
Christiano, who'd lost 20% to 25% of her hair when she was diagnosed, tried steroid injections into the affected areas, a common treatment. "Some people spontaneously recover with no treatment," she says, while in others the disease progresses to the point that they don't have hair anywhere on their body, a condition called alopecia universalis. As a result of some publicity about her study, she says, her inbox has been flooded with emails from people eager to test ruxolitinib for alopecia.
Q. When the people in the study stopped taking ruxolitinib, did their hair fall out again?
A. Scientists are continuing to check their hair growth every couple of months. It's plausible that the drug could have a permanent effect, Clynes says. On the other hand, the drug might have only put the disease into remission, so it could eventually come back and cause hair loss.
Q. Wouldn't it make more sense to rub the drug on the skin where you'd like to grow hair, rather than swallowing it in a pill?
A. "It would be wonderful to have both an oral and a topical" version of ruxolitinib, Clynes says. But topical ruxolitinib has not been approved for human use, so he and Christiano mixed up their own with ingredients from China and tested it on the mice, with excellent results.
Q. Since ruxolitinib is already on the market to treat that bone marrow cancer, couldn't people with alopecia ask their doctor for a prescription?
A. Once the FDA approves a drug to treat one condition, doctors are free to prescribe for others, a practice called "off-label" use. But insurers generally cover medications only for their approved uses.
Jakafi, the brand name for ruxolitinib, costs $8,753 for a month's supply, which amounts to more than $100,000 a year, says Pamela Murphy. She's vice president for investor relations and corporate communications at Incyte, the Wilmington, DE, company that markets the drug.
"I think that is a pretty high price point," Clynes says. "Our hope is a year of therapy wouldn't be required." If a wealthy person with alopecia wanted to try the drug, he says, "I would not personally be uncomfortable prescribing the medicine if the patient was otherwise healthy and young and had an expected low-level risk of complications."
Q. What's the next step?
A. In a partnership with drug companies Novartis and Eli Lilly, Incyte does not have any rights to develop Jakafi tablets for any use other than treating cancer, so it won't ever get approved to treat alopecia, Murphy says. "We don't want to raise expectations of patients inappropriately," she says.
But, she says, Incyte can do what it likes with the topical form of the drug. While the company has tested the topical form on people with psoriasis (a long-term skin disorder that, like alopecia, is an autoimmune disease), it has no plans to pursue the topical form for that purpose.
"Right now we're not doing anything with the topical, but we're interested in working with Columbia [University Medical Center]," she says.
SOURCES: Raphael Clynes, MD, PhD, Bristol-Myers Squibb. Angela Christiano, PhD, professor, department of dermatology, department of genetics and development, Columbia University Medical Center. Pamela Murphy, vice president for investor relations and corporate communications, Incyte. Xing, L. Nature Medicine, published online Aug. 17, 2014.
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