Yale experts treat severe, disfiguring sarcoidosis with novel therapy

December 26, 2018

New Haven, Conn. -- An all-Yale team of researchers successfully treated a patient with disfiguring sarcoidosis, a chronic disease that can affect multiple organs, with a drug approved for rheumatoid arthritis. Successful treatment of two other patients with similarly severe disease suggests an effective treatment for an incurable, sometimes life-threatening illness is within reach, the scientists said.

The research was published in the New England Journal of Medicine.

Sarcoidosis is an inflammatory disease that affects multiple organs in the body. While some sarcoidosis patients recover without treatment, others suffer damage to the lungs, heart, lymph nodes, skin, and other organs. Current treatments, including steroids, are not reliably effective for the skin and can cause serious side effects.

Based on clues gleaned from prior studies, the Yale team decided to try the arthritis medication tofacitinib. The drug, a Jak inhibitor, blocks a pathway known as Jak-STAT. The lead author, Brett King, M.D., has pioneered the use of Jak inhibitors to treat other intractable skin diseases, including vitiligo, alopecia areata, and eczema.

For several months, a 48-year-old female patient was treated with the drug, a twice-daily pill. The researchers observed that her skin lesions nearly disappeared. They also performed RNA sequencing on biopsied skin from the patient before and during treatment. "Before treatment, we were able to show that the Jak-STAT pathway is activated," King said. "During treatment, not only does her skin disease go away, but there is no activation of the pathway."

"We plan to evaluate the activation of the Jak-STAT pathway in the lung fluid and blood of over 200 patients with pulmonary and multiorgan sarcoidosis," said co-author Nkiruka Emeagwali. These are big steps toward understanding a disease that has been a mystery for years, the researchers said.

The findings are being tested further by the Yale team in a clinical trial. If confirmed, they could represent a breakthrough for sarcoidosis patients, King noted.

"A frequently awful disease, which to date has no reliably effective therapy, may now be targeted with Jak inhibitors," he said. "We have a relatively safe medicine that works."

Other Yale authors are William Damsky, Durga Thakral, and Anjela Galan.
-end-
This work was funded in part by the Ranjini and Ajay Poddar Resource Fund for Dermatologic Diseases Research, the National Institutes of Health, and The Dermatology Foundation.

King is a consultant to and a clinical trials investigator for Pfizer, the maker of tofacitinib.

Citation: New England Journal of Medicine

Reporters can contact the author at brett.king@yale.edu.

Yale University

Related Rheumatoid Arthritis Articles from Brightsurf:

Reducing dementia in patients with rheumatoid arthritis
The incidence of dementia in patients with rheumatoid arthritis is lower in patients receiving biologic or targeted synthetic disease modifying antirheumatic drugs (DMARDs) than in patients who receive conventional synthetic DMARDs, according to a new study.

Is rheumatoid arthritis two different diseases?
While disease activity improves over time for most rheumatoid arthritis (RA) patients, long-term outcomes only improve in RA patients with autoantibodies, according to a new study published this week in PLOS Medicine by Xanthe Matthijssen of Leiden University Medical Center, Netherlands, and colleagues.

Does the Mediterranean diet protect against rheumatoid arthritis?
Previous research has demonstrated a variety of health benefits associated with the Mediterranean diet, which is rich in olive oil, cereals, fruit and vegetables, fish, and a moderate amount of dairy, meat, and wine.

Reducing corticosteroid use in rheumatoid arthritis
Is the long-term use of glucocorticoids essential in people with chronic inflammatory diseases such as rheumatoid arthritis, or can early discontinuation prevent characteristic side effects?

Rheumatoid arthritis patients under treatment with methotrexate
Patients with rheumatoid arthritis (RA) often suffer from what is referred to as interstitial lung disease (ILD).

Rheumatoid arthritis -- can its onset be delayed or prevented?
Rheumatoid arthritis (RA) is a chronic inflammatory autoimmune disorder that leads to significant health issues as well as high treatment costs.

Disease burden in osteoarthritis is similar to rheumatoid arthritis
Osteoarthritis (OA) has traditionally been viewed as a highly prevalent but milder condition when compared with rheumatoid arthritis (RA), and some may believe that it is part of a normal aging process requiring acceptance, not treatment.

Prospect of a new treatment for rheumatoid arthritis
An international research group led by Charité -- Universitätsmedizin Berlin has completed testing a new drug to treat rheumatoid arthritis.

Can rare lymphocytes combat rheumatoid arthritis?
Immunologists at Friedrich-Alexander-Universität Erlangen-Nürnberg have demonstrated that ILC2, a group of rare lymphoid cells, play a key role in the development of inflammatory arthritis.

Rheumatoid arthritis meets precision medicine
Scientists are bringing precision medicine to rheumatoid arthritis for the first time by using genetic profiling of joint tissue to see which drugs will work for which patients, reports a new multi-site study.

Read More: Rheumatoid Arthritis News and Rheumatoid Arthritis Current Events
Brightsurf.com is a participant in the Amazon Services LLC Associates Program, an affiliate advertising program designed to provide a means for sites to earn advertising fees by advertising and linking to Amazon.com.