In the near future, a new effective medication for ulcerative colitis, an intestinal chronic inflammatory disease, may be available for clinical purposes both in the US and Europe. Tofacitinib was validated by an international study, published on the New England Journal of Medicine, authored (among others) by Prof. Silvio Danese, coordinator of the Immuno Center at Humanitas, supervisor of the Intestinal Chronic Inflammatory Diseases Center at Humanitas and professor at Humanitas University.

The team tested the effects of tofacitinib on a group of adults suffering from ulcerative colitis, at different stages, for at least four months. 598 individuals were orally administered 10 mg tofacitinib per day for eight weeks, while 541 patients only got a placebo. 593 patients who responded to the therapy, were then randomly divided into three groups. Each one had to take twice a day for 52 weeks 5 mg or 10 mg of tofacitinib or placebo.

At the end of the tests, the medication proved to be more effective in the treatment of ulcerative colitis, than the placebo. In particular, in the first step there were higher remission induction rates after the first eight weeks. There was a healing in the damaged intestinal mucous membranes and improvement in the life quality. The remission of the colitis was also maintained in the second step of the study, after 52 weeks, both with 5 mg and with 10 mg tofacitinib.

What is Tofacitinib?

As Prof. Danese explains, “Tofacitinib is a so-called small molecule, a JAK-inhibiting synthetic medicine. It is able to block every enzyme in the Janus Kinase family, that play a part in the activation of an inflammatory process. This is a therapeutic novelty. It is the first oral medication to block many cytokines (inflammatory molecules) at the same time, while biological medications with monoclonal antibodies only inhibit a cytokine at a time”.

In the last years, scientific research has sperimented its use also for the treatment of other diseases, such as rheumatoid arthritis or psoriasis. Doctor Anna Villa, CNR researcher and director of the Human Genome Lab at Humanitas, was the first to suggest that inhibiting JAK, could stop out-of-control disease-causing immune responses and inflammations, in a study about immunodeficiencies published on Cell.

A new hope for Patients

Ulcerative colitis is an intestinal inflammation that typically affects the rectum. However, it can also spread to the rest of the colon. Lesions form on the intestinal mucous membranes and provoke the typical symptoms of the disease: diarrhea and abdominal pains. “The creation of a new medication to treat it, is important because many patients do not respond to current therapies or stop doing so after an initial positive response”, Prof. Danese concludes.