Even after the approval of multiple new drugs for ulcerative colitis (UC), up to 30% of UC patients still require major abdominopelvic surgery to remove the colon and rectum for medically refractory disease.
That is why the development of new and better treatment strategies has been under investigation. The use of mesenchymal stem cells (MSCs) is one of them. They were introduced as a safe and effective therapy for the treatment of Crohn’s disease in 2003 following an initial report of complete healing of a refractory rectovaginal fistula following MSCs delivery.
There have been many trials showing the safety of local application of MSCs for perianal Crohn’s disease. In the case of UC, there is one study that showed a good clinical response and increased remission in the treatment group when using a systemic application (intravenously) of MSCs.
Stem Cells Applied Directly into the Colon
Recently, a group of researchers evaluated the use of MSCs delivered locally via endoscopic delivery. The results of the study appear in the journal Colorectal Disease.
For the study, the team use an allogenic bone marrow-derived MSC product called remestemcel-L at a dose of 150 million MSCs and compared it to a placebo. They delivered the MSCs using sclerotherapy during colonoscopy.
The team then evaluated the Mayo score and Mayo endoscopic severity scores at 2 and 6 weeks, and at 3 months after the procedure.
The study included a total of 6 patients with UC diagnose. Four of them received the MSCs treatment. The patients were previously treated with anti-tumor necrosis factor or anti-integrin therapy.
There were no adverse or serious adverse events related to the administration of the MSCs product.
All Patients Achieved Endoscopic Remission
The Mayo score and Mayo endoscopic severity score decreased in all patients 2 weeks after the procedure, and by week 6 post-treatment all treated patients in the treatment group had achieved clinical and endoscopic remission. Also, the patient-reported number of daily bowel movements decreased as did urgency and presence of blood in the stool. All patients were extremely satisfied with their treatment.
On the other hand, the control group had no Mayo score improvement, the daily bowel movements increased, as did the number reporting blood in their stool, and urgency also remained unchanged.
Although the study was performed in a pretty small sample of patients, the early findings suggest that the treatment with MSCs administered via endoscopy could be an alternative therapy for patients with refractory ulcerative colitis.
Lightner AL, Dadgar N, Matyas C, Elliott K, Fulmer C, Khaitan N, Ream J, Nachand D, Steele SR. A phase IB/IIA study of remestemcel-L, an allogeneic bone marrow-derived mesenchymal stem cell product, for the treatment of medically refractory ulcerative colitis: an interim analysis. Colorectal Dis. 2022 Jun 29. doi: 10.1111/codi.16239. Epub ahead of print. PMID: 35767384.
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