“Long-term Efficacy and Safety of Stem Cell Therapy (Cx601) for Complex Perianal Fistulas in Patients With Crohn’s Disease”: this is the title of the study, just published in Gastroenterology, of which Professor Silvio Danese, Head of the Centre for Inflammatory Chronic Intestinal Diseases in Humanitas and Professor at Humanitas University, is a senior author.
The study involves 212 patients with Crohn’s disease with refractory perianal fistulae who are enrolled in 49 hospitals (Europe, Canada, USA, Israel), and investigates the efficacy of stem cell therapy: mesenchymal cells from adipose tissue represent a valid therapeutic option compared to surgery, treatments with systemic immunosuppressants, antibiotics or inhibitors of tumor necrosis factor.
The study had already yielded its first results at 24 weeks, published in The Lancet in July 2016, and its results at 52 weeks have now been published.
How does the study work?
“Therapies for perianal fistulae in patients with Crohn’s disease often do not guarantee long-term healing. We conducted a double-blind, randomized, placebo-controlled study to determine the long-term efficacy and safety of a single local administration of stem cells derived from allogeneic expanded adipose (Cx601),” explained professor Danese.
The patients were divided into two groups: the first, composed of 107 patients, received treatment with mesenchymal cells from adipose tissue (Cx601); the 105 patients of the second group were instead treated with placebo. 24 weeks after the injection of 120 million Cx601 cells, in 50% of patients in the first group, compared to 34% in the second, the fistulae were completely healed and the treatment was well tolerated by the patients themselves.
Results at 52 weeks
The beneficial effect observed at week 24 was confirmed at week 52: a significantly higher percentage of patients treated with Cx601 in fact obtained a radiological and clinical remission (56.3%) compared to the control group (38.6%).
Safety was maintained over the long term with overlapping side effects between placebo and treatment group.
“In conclusion, the efficacy and safety of Cx601 compared to placebo in treating perianal fistulae in Crohn patients was maintained even after one year. For these reasons, the European Commission of Evaluation (CHMP) has given a favorable opinion on this new treatment”, concluded the specialist.