An effective drug in the treatment of ulcerative colitis, an inflammatory bowel disease, soon could be approved for clinical practice, both in the United States and Europe. The validation of the action of tofacitinib was an international study published in the New England Journal of Medicine, which also includes Professor Silvio Danese, coordinator of the Immune Center at Humanitas, head of the Center for Inflammatory Chronic Intestinal Diseases of Humanitas and lecturer at Humanitas University, among the authors.
The team tested the effects of tofacitinib on a group of adults with ulcerative colitis for at least four months, with varying degrees of severity. 598 individuals were given 10 mg of tofacitinib orally daily for eight weeks while 541 patients were given a placebo. 593 patients who responded to the therapy were then randomly assigned to three groups: each was asked to take 5 mg or 10 mg tofacitinib or placebo twice daily for 52 weeks.
At the end of the tests, the drug proved to be more effective than placebo in treating ulcerative colitis. In particular, in the first phase, higher rates of induction of disease remission after eight weeks were found, with healing of damaged intestinal mucous membranes and an improvement in quality of life. Colitis remission was also maintained in the second phase of research after 52 weeks, with both 5 mg tofacitinib and 10 mg taken.
What is tofacitinib?
“The tofacitinib – explains prof. Danish – is a so-called small molecule, a drug of JAK inhibitor synthesis, which can block the action of all enzymes in the Janus Chinasi family, enzymes involved in the activation of an inflammatory process. The therapeutic innovation is that this is the first oral drug that blocks numerous cytokines (inflammation molecules) at the same time, compared to biological drugs with monoclonal antibodies that inhibit one cytokine at a time”.
In recent years, scientific research has tested its use for the treatment of other diseases, from rheumatoid arthritis to psoriasis. The first theory that blocking JAK could stop immunity reactions and out of control inflammation and cause disease, was made by Dr. Anna Villa, CNR researcher and director of Human Genome Lab at Humanitas, in a study on immune-deficiencies published in Cell.
A new hope for patients
Ulcerative rectocolitis is a disease characterized by inflammation of the intestine that always affects the rectum, but can also extend to the rest of the colon. Injuries form on the intestinal mucous membranes, which cause the typical symptoms of the disease, i.e. diarrhea and abdominal pain. “Having found a new effective drug for its treatment – concludes prof. Danish – is important because many patients do not respond to their current therapies or tend not to take them after an initial positive response.
