The first problem is that people who get Crohn’s disease are getting younger and younger: we are talking about twenty and thirty year olds. The second that, in about a third of cases, have complications, such as perianal fistulas, which limit their physical activity, have a heavy impact on social and working life, interfere with sexual activity and give rise to disorders of anxiety and mood.

A new drug

But, for these people, there is good news that comes to us from the Sacco Hospital in Milan: in the last four months, despite the difficulties encountered from the logistical point of view and with part of the staff involved in the Covid emergency, the first patients, suffering from perianal disease, with an innovative drug: it is called darvadstrocel, approved by EMA, the European Medicines Agency, as an Advanced Therapy Medicinal Product. The drug was authorized and funded by Aifa (the Italian drug agency) for three patients with Crohn’s disease, complicated by refractory perianal complication, at the Gastroenterology and Digestive Endoscopy of the Sacco Hospital in Milan.


Sant’Orsola and Gemelli Polyclinic

Comment Sandro Ardizzone who heads this Division at the Milanese Hospital, University of Milan: Our hospital is among the first in Italy to use this preparation of expanded allogeneic mesenchymal stem cells, extracted from adipose tissue, together with the Gemelli Polyclinic of Rome and the Policlinico Sant ‘ Orsola of Bologna. Respectively, in each of the other two centers, three patients were treated and, there, the cost of the therapy was covered by the companies themselves (each treatment costs € 60,000 and has not yet been reimbursed by the National Health Service).

Complications of Crohn’s

Before explaining what this complication of Crohn’s disease consists of and how this new therapy works, it is worth giving some more information about the disease and its complications. Crohn’s disease is a chronic inflammatory bowel disease that recognizes an immune origin explains Ardizzone. We add: it causes a series of symptoms ranging from chronic diarrhea to abdominal pain and cramps, sometimes accompanied by low-grade fever or joint pain. And it affects younger and younger people, even if at the moment it is not clear why. To give an idea: today in Italy at least 150 thousand people are affected by this disease (but still, unfortunately, there is no register). The disease occurs preferably in two age groups: between the ages of twenty and thirty (and cases in young people have been increasing in recent years), and between sixty and seventy years – explains Ardizzone -. The causes of the disease are not yet known and the reasons for the increase among young people are not known. There seems to be a genetic predisposition that abnormally modulates the body’s immune response to certain antigens (i.e. molecules of various substances, ed) present in the environment.

Perianal fistulas

The final result is this: uncontrolled inflammation is triggered in the intestine, which causes the symptoms mentioned above and which, in about a third of cases, affects the rectum and leads to the formation of fistulas. Perianal fistulas are formed precisely due to inflammation of the rectum – continues Ardizzone – and affect the rectal muscles. In other words: “fistulous tracts” are created, a sort of channels that invade the muscle, open outwards, can become infected and give rise to abscesses (and in some cases to sepsis: that is to generalized infection of the organism) . With all the symptoms we talked about above. Currently, the treatment of perianal fistulas can count on several options (but not entirely satisfactory): antibiotics work in 21-48% of cases; thiopurines (drugs that interfere with the immune response) in 20-40 percent; anti Tnf (the so-called anti Tumor necrosis factor, which once again interfere with inflammation) in 23-50 percent.

Proctectomia

But at least 40 percent of fistula patients require a proctectomy. That is: an removal of the rectum and consequent “ostomy”, the one which, in common jargon, means “having the bag”. This is why new therapeutic solutions are sought and, in the end, this is what the new therapeutic opportunity with darvadstrocel consists of. Al Sacco, Ardizzone works with the surgeons Piergiorgio Danelli and Francesco Colombo who also intervened in implementing the new stem cell therapy.

Stomia avoided

Darvadstrocel, recently approved by the EMA, after the registration studies published in Lancet and Gastroenterology – comments Ardizzone – consists of human MSCs (Mesenchimal Stem Cells) whose efficacy in the treatment of the disease is attributable to two main biological mechanisms: the anti-inflammatory properties and the secretion of soluble molecules that promote tissue repair and growth. ”The treatment consists of administering 120 million cells into the fistulous lesion and has a safety profile comparable to placebo. The results? Stem cells rebuild the tissues inside the fistula – Ardizzone specifies – because these mesenchymal cells have an inflammatory activity and extinguish inflammation. Then, they secrete molecules that control immune reactions – in other words: at the moment these patients are spared the ostomy, that is, the function (and channeling) of their intestines is preserved. We are at the beginning and the results are encouraging. Now it is a question of verifying, in the so-called “real life”, the real life, the safety of these therapies and the medium-long term results.

April 10, 2021 (change April 10, 2021 | 11:31)

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