A PHASE IB/IIA STUDY OF REMESTEMCE-L, AN ALLOGENEIC BONE MARROW DERIVED MESENCHYMAL STEM CELL PRODUCT, FOR THE TREATMENT OF MEDICALLY REFRACTORY ULCERATIVE COLITIS: AN INTERIM ANALYSIS
Amy L. Lightner*, Neda Dadgar, Clifton G. Fulmer, Justin Ream, Douglas Nachand, Scott Steele
Cleveland Clinic, Cleveland, OH
Background: No studies have looked at the direct injection of mesenchymal stem cells (MSCs) for luminal ulcerative colitis (UC). We designed a phase IB/IIA randomized control clinical trial in a 2:1 fashion to receive remestemcel-L, an ex vivo expanded allogeneic bone marrow derived MSC product, at a dose of 150 million MSCs versus placebo in order to study the safety and efficacy of endoscopic delivery of MSCs for UC.
Methods: Patients with medically refractory UC who have lost response to at least one monoclonal antibody were randomzied to MSCs versus placebo delivered via direct injection using a 23 G sclerotherapy needle at the time of colonoscopy. Data collected included adverse events; validated clinical (Mayo score), endoscopic (Mayo endoscopic severity score), and histologic (Simplified Geboes score) scoring systems at 2 weeks, 6 weeks, and 3 months post MSC delivery; and patient reported outcomes.
Results: Six patients were enrolled and treated; 4 received MSCs and 2 placebo. All had been on prior anti-TNF or anti integrin-therapy. There were no adverse events related to investigational product. In the treatment group (n=4), the Mayo endoscopic severity score decreased in all patients by two weeks after MSC delivery. At three months, based on the inflammatory bowel disease patient reported treatment impact (IBD-PRTI), all patients were extremely satisfied or satisfied with their MSC treatment. Treatment response was described as excellent or good in all patients. In the control group (n=2), the Mayo endoscopic severity score did not increase by being off alternative therapy. At three months on IBD-PRTI, one patient was neutral and one patient was dissatisfied. Treatment response was described as poor or unchanged in control patients.
Conclusions: MSCs may offer a safe therapeutic for the treatment of medically refractory UC. Early data suggests improved clinical and endoscopic scores by 2 weeks post MSC delivery.
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