Vitiligo causes the development of patches of discolored skin through the loss of pigmentation.
The study, published in JAMA Dermatology, details the case of one patient whose progressive vitiligo was successfully treated by dermatologists from Yale School of Medicine in New Haven, CN.
"While it's one case, we anticipated the successful treatment of this patient based on our current understanding of the disease and how the drug works," says principal investigator Dr. Brett King, an assistant professor of dermatology at Yale.
The drug in question, tofacitinib, is typically used to treat moderate to severe rheumatoid arthritis, a chronic inflammatory disorder affecting the lining of the joints. It belongs to a family of drugs known as Janus kinase (JAK) inhibitors that have been approved for use by the US Food and Drug Administration (FDA).
Vitiligo is also considered to be an autoimmune disorder, with the immune system targeting skin cells called melanocytes that produce the skin's pigmentation. People with vitiligo develop patches on their skin that are devoid of pigmentation and color. In severe cases, the discoloration can cause significant psychological distress.
There are several treatments available for treating vitiligo, such as steroid creams and light therapy, but none of these are reliably effective in reversing the symptoms of the condition.
Researchers believe discovery could revolutionize vitiligo treatment
The subject of the study was a 53-year-old patient with prominent white patches covering the skin of her face, hands and body that had been increasing over the previous year. She had received treatment in the form of ointments and light therapy, but these did not halt the condition's progression.
Due to tofacitinib's success in treating alopecia areata, Dr. King and study co-author Dr. Brittany Craiglow believed that it might also prove effective at treating vitiligo. For their trial, the patient received an oral dose of the drug of 5 mg every other day, which increased to 5 mg a day after 3 weeks.
The researchers found that after 2 months of treatment, the patient had experienced partial repigmentation on her arms, face and hands. After 5 months, the white patches she had been most concerned with on her face and hands were nearly all gone. Only a few white spots remained on other parts of her body.
The dosage administered during the trial was half the approved dosage for treating rheumatoid arthritis. Crucially, the patient did not experience any adverse effects upon taking tofacitinib.
"It's a first, and it could revolutionize treatment of an awful disease," says Dr. King. "This may be a huge step forward in the treatment of patients with this condition."
As the study only involved one patient, Dr. King acknowledges that further research will be needed to confirm that the drug is both safe and effective in treating vitiligo. He now hopes to conduct a clinical trial using tofacitinib or ruxolitinib - another JAK inhibitor that may be able to treat alopecia areata - to treat the disorder.
"Alopecia areata and vitiligo share genetic risk factors and can co-occur within families and individual patients, suggesting a common pathogenesis," write the authors. "As such, it is not surprising that a medication that has been shown to be effective in treating alopecia areata may also be effective in treating vitiligo."