Maneesh Dave1, Palashkumar Jaiswal, Fabio Cominelli. 1. aDivision of Gastroenterology and Liver Disease, University Hospitals of ClevelandbDigestive Health Research Institute, Case Western Reserve University, Cleveland, OhiocDivision of Internal Medicine, John H Stroger Jr. Hospital of Cook County, Chicago, Illinois, USA.
Abstract
PURPOSE OF REVIEW: There is a need for novel therapies for inflammatory bowel diseases (IBDs) that are well tolerated and effective. Currently, mesenchymal stem/stromal cells (MSCs) are being investigated in clinical trials for treatment of IBD. In this review, we update the recently published studies with an emphasis on the long-term efficacy of MSC therapy for IBD. RECENT FINDINGS: A cumulative body of data, including a recent phase III randomized controlled trial demonstrated excellent fistula healing in patients with refractory Crohn's perianal fistulae treated via local injections of MSCs and with a good safety profile. Follow-up studies suggest long-term efficacy of MSC therapy for complex perianal Crohn's disease fistulae; however, the efficacy decreases over time and may necessitate repeat treatment. Systemic (intravenous) therapy for luminal IBD offers a relatively well tolerated alternative but its efficacy remains unclear. SUMMARY: Recent studies demonstrate that MSCs are well tolerated and have an excellent short-term efficacy for the management of refractory fistulizing perianal Crohn's disease. The current data suggest that its influence may 'wear off' over time. More data on larger number of patients with longer duration of follow-up in the setting of a randomized placebo controlled trial are needed to confirm these promising results. For luminal IBD, there is a need for more mechanistic studies in representative preclinical murine models, and the results of an ongoing phase III randomized controlled trial are eagerly awaited.
PURPOSE OF REVIEW: There is a need for novel therapies for inflammatory bowel diseases (IBDs) that are well tolerated and effective. Currently, mesenchymal stem/stromal cells (MSCs) are being investigated in clinical trials for treatment of IBD. In this review, we update the recently published studies with an emphasis on the long-term efficacy of MSC therapy for IBD. RECENT FINDINGS: A cumulative body of data, including a recent phase III randomized controlled trial demonstrated excellent fistula healing in patients with refractory Crohn's perianal fistulae treated via local injections of MSCs and with a good safety profile. Follow-up studies suggest long-term efficacy of MSC therapy for complex perianal Crohn's disease fistulae; however, the efficacy decreases over time and may necessitate repeat treatment. Systemic (intravenous) therapy for luminal IBD offers a relatively well tolerated alternative but its efficacy remains unclear. SUMMARY: Recent studies demonstrate that MSCs are well tolerated and have an excellent short-term efficacy for the management of refractory fistulizing perianal Crohn's disease. The current data suggest that its influence may 'wear off' over time. More data on larger number of patients with longer duration of follow-up in the setting of a randomized placebo controlled trial are needed to confirm these promising results. For luminal IBD, there is a need for more mechanistic studies in representative preclinical murine models, and the results of an ongoing phase III randomized controlled trial are eagerly awaited.