Mesenchymal Stromal Cell Therapy Yields Improvements in Colitis

Remestemcel-L, Mesoblast’s mesenchymal stromal cell therapy, demonstrated rapid mucosal healing and disease remission in patients with refractory ulcerative (NCT04543994) or Crohn colitis (NCT04548583), according to results from a phase 1b/2a study published in the Journal of Crohn’s and Colitis.¹

These results, from the first cohort of patients treated, were also presented at the 17th Congress of European Crohn’s and Colitis Organisation (ECCO), February 16-19, by principal investigator Amy L. Lightner, MD, associate professor of surgery, department of colon and rectal surgery, and associate chief, surgical research, Cleveland Clinic.

“Mesenchymal stromal cells (remestemcel-L) offer a safe therapeutic for the treatment of medically refractory Ulcerative Colitis and Crohn colitis,” Lightner said in a statement.² “Early data suggests improved clinical and endoscopic scores as early as two weeks following remestemcel-L delivery.”

Cleveland Clinic is assessing remestemcel-L in up to 48 patients with refractory Crohn or ulcerative colitis at high risk of progressing to surgery. Participants receive a single dose of 150 to 300 million cells ofremestemcel-L or placebo delivered via direct injection using a 23 G sclerotherapy needle during colonoscopy.

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“We are delighted to be involved with Dr. Lightner and her team at Cleveland Clinic in this latest cutting-edge study. This randomized controlled trial is the first to evaluate local delivery of remestemcel-L directly into the inflamed colon, using objective endoscopic measures of mucosal healing, in patients with colitis who are at high risk of surgical resection of their colon,” Eric Rose, MD, chief medical officer, Mesoblast, added to the statement.²

Investigators observed improvements in clinical and endoscopy scores as assessed by Mayo clinical score and Mayo endoscopic severity (MES) score within the first 12 enrolled patients. All patients had excellent or good responses on inflammatory bowel disease patient reported treatment impact (IBD-PRTI) scale. All patients exhibited reductions in fecal calprotectin from a mean of 231 at baseline to 67 at 3 months.

All patients with ulcerative colitis achieved clinical and endoscopic remission by 6 weeks. These patients were either satisfied or extremely satisfied with their treatment at 3months as according to IBD-PRTI scale. Patients with Crohn exhibited remissions or responses as measured by the Simple Endoscopy Score for Crohn’s Disease (SES-CD), with mean score decreasing from 17 at baseline to 5 at 3 months.

Comparatively, patients in the control cohort had increases in endoscopy scores and fecal calprotectin levels. Correspondingly, clinical responses were poor or unchanged. Remestemcel-L delivered during colonoscopy was not associated with any treatment-related adverse events (AEs).

REFERENCES

1. Lightner A, Ream J, Nachand D, Fulmer C, Regueiro M, Steele S. P428 A Phase IB/IIA study of remestemcel-L, an allogeneic bone marrow derived mesenchymal stem cell product, for the treatment of medically refractory Crohn’s colitis: A preliminary analysis. J. Crohn's Colitis.2022. 16(S1); i412–i413. doi:10.1093/ecco-jcc/jjab232.555
2. Rapid improvement and remission in patients with refractory ulcerative colitis and Crohn’s Colitis after direct injection of remestemcel-L by colonoscopy. News release. Mesoblast Limited. February 20, 2022. https://www.biospace.com/article/releases/rapid-improvement-and-remission-in-patients-with-refractory-ulcerative-colitis-and-crohn-s-colitis-after-direct-injection-of-remestemcel-l-by-colonoscopy/?s=110