In a June 13 announcement, the U.S. Food and Drug Administration approved the use of the Janus kinase (JAK) inhibitor baricitinib as a treatment for severe alopecia areata, a disfiguring skin disease.
It is the first approved treatment for alopecia areata, an autoimmune disorder that affects about 7 million people in the United States. The often-disfiguring disease, in which the body’s immune system attacks hair follicles, is marked by patchy or complete loss of scalp hair and sometimes eyebrows, eyelashes, facial hair, and body hair.
Dr. Brett King, an associate professor of dermatology at Yale Medical School, worked with the pharmaceutical company Eli Lilly and Company to conduct a series of clinical trials with the new medicine, a once-daily pill which goes by the product name Olumiant.
In the trials, Olumiant helped one in three patients with severe alopecia areata regrow their hair — almost half of the patients had no scalp hair at the start of the trials — resulting in 80% or more scalp coverage. Improvements were also achieved for patients with significant eyebrow or eyelash hair loss.
Over the past decade, King has conducted innovative research using JAK inhibitors — which were originally designed to treat rheumatoid arthritis and certain blood disorders — to treat a range of intractable skin diseases, including eczema, vitiligo, granuloma annulare, sarcoidosis, and erosive lichen planus. Earlier this year, the American Academy of Dermatology named King a “Patient Care Hero.”
King spoke with Yale News about the new treatment, how it fits into his overall research efforts, and what it means for patients.
In practical terms for patients, what does this FDA approval mean for treating alopecia areata?
Brett King: Until now, there have been no FDA-approved treatments for alopecia areata, and the medicines that have been used in the past to treat severe cases of alopecia areata are largely ineffective. There is, however, lots of data to show that a relatively new class of medicines called JAK inhibitors work for the treatment of severe alopecia areata. Patient access to these medicines is extremely limited, though, because JAK inhibitors were not FDA-approved for this purpose. FDA approval will bring greater access, via insurance coverage, to patients.
FDA approval has an additional benefit, as well. When a medicine is approved for treatment of a disease, doctors feel more comfortable prescribing the medicine for that purpose. Therefore, FDA approval will empower and enable health care providers to treat patients with severe alopecia areata.
How did you get involved in the baricitinib trials?
King: I had been very actively exploring the use of JAK inhibitors, mostly one called tofacitinib, for the treatment of patients with alopecia areata when Eli Lilly became interested in alopecia areata treatment with baricitinib, and so I became a key consultant to them in the development of these clinical trials.
Do you remember the first patient, or group of patients, you treated with JAK inhibitors?
King: I vividly remember the first patient I treated. He had almost no scalp hair, his eyebrows and eyelashes and facial hair were missing, and, in addition, he had red, scaly psoriasis plaques all over his body. It was in 2013. The JAK inhibitor tofacitinib was a new medicine, not in dermatology but in rheumatology, and wasn’t a year old. There was data to suggest that JAK inhibitors might interrupt alopecia areata in mice with the condition, and so I thought to use tofacitinib in a human with the disease. This had never been done before, and there was no guidance for how to do it. I explained to the patient that use of tofacitinib in him would be exploratory, and he agreed to try it. I had to fight with his insurance to get him the medicine, but I got it. Not long after he started taking tofacitinib, his hair started to grow. I published the results of his treatment not long after that and history was made, forever changing this disease.
What was the early response from colleagues as you explored this treatment approach?
King: Many were very intrigued. A few wanted to be involved. Some thought that I was crazy to be using a new medicine that did not really exist in dermatology in so many people. Others were intrigued and thought I was crazy. But I was compelled to do what I did because there were so many people asking for help — literally I received hundreds and hundreds of emails in the days and weeks after my paper was published. I was amazed by the stories and how much suffering there was from this disease. I had to try to help.
What advice would you give to other physicians and researchers with novel treatment ideas?
King: Try hard. Whether or not we succeed, the process is unbelievably rewarding and worth the painful challenges.
How meaningful is this development for you, personally?
King: I am elated and deeply grateful to have been able to shepherd this discovery from the very first patient to now, when treatment will become available for countless people. I celebrate my mom, who worked tirelessly, cleaning toilets among other thankless jobs, to provide opportunities for me so that I could do what I’ve done. It’s an incredible joy to watch a hypothesis unfold and become a life-changing treatment for people. My mother, too, would be elated.