Tofacitinib effective against vitiligo, the “Michael Jackson disease,” in first patient treated

by Bruce Sylvester: Treatment with tofacitinib, a drug for rheumatoid arthritis, has restored skin color in a patient suffering from vitiligo, researchers reported on June 24 in JAMA Dermatology.

Vitiligo causes skin to lose its pigmentation and is best known for afflicting the singer Michael Jackson.

“It’s a first, and it could revolutionize treatment of an awful disease,” said investigator Brett King, M.D., assistant professor of dermatology at Yale University School of Medicine in New Haven, Connecticut. “This may be a huge step forward in the treatment of patients with this condition.”

There are no reliably effective treatments for vitiligo. However, some research has suggested that an existing class of FDA-approved medications, Janus kinase (JAK) inhibitors, might hold some treatment efficacy.

Last year, King published a study showing the efficacy of the JAK inhibitor tofacitinib citrate in treating hair loss caused by alopecia areata. King and co-author Brittany Craiglow, M.D., also an assistant professor of dermatology at Yale, hypothesized that tofacitinib might also be effective for vitiligo.

They investigative team administered tofacitinib to a 53-year-old patient with white spots covering her body. During the year prior to tofacitinib therapy, the spread of the spots had been increasing.

Within two months of treatment, the patient achieved partial repigmentation on her face, arms, and hands. After five months, the white spots on her face and hands had largely disappeared, with only a few remaining spots in other less obvious regions of her body.

There were no adverse side effects associated with tofacitinib treatment.

King hopes to follow-up this research with a full-scale clinical trial using tofacitinib, or a similar medicine, ruxolitinib, for the treatment of vitiligo.