Mesenchymal stromal cell-based therapy: Regulatory and translational aspects in gastroenterology

World J Gastroenterol. 2016 Nov 7;22(41):9057-9068. doi: 10.3748/wjg.v22.i41.9057.


The past decade has witnessed an outstanding scientific production focused towards the possible clinical applications of mesenchymal stromal cells (MSCs) in autoimmune and chronic inflammatory diseases. This raised the need of novel standards to adequately address quality, efficacy and safety issues of this advanced therapy. The development of a streamlined regulation is currently hampered by the complexity of analyzing dynamic biological entities rather than chemicals. Although numerous pieces of evidence show efficacy in reducing intestinal inflammation, some inconsistencies between the mechanisms of action of rodent vs human MSCs suggest caution before assigning translational value to preclinical studies. Preliminary evidence from clinical trials showed efficacy of MSCs in the treatment of fistulizing Crohn's disease (CD), and preparations of heterologous MSCs for CD treatment are currently tested in ongoing clinical trials. However, safety issues, especially in long-term treatment, still require solid clinical data. In this regard, standardized guidelines for appropriate dosing and methods of infusion could enhance the likelihood to predict more accurately the number of responders and the duration of remission periods. In addition, elucidating MSC mechanisms of action could lead to novel and more reliable formulations such as those derived from the MSCs themselves (e.g., supernatants).

Keywords: Inflammatory bowel diseases; Intestinal disorders; Mesenchymal stem cells; Mesenchymal stromal cells; Translational medicine.

Publication types

  • Review

MeSH terms

  • Animals
  • Biomarkers / metabolism
  • Gastroenterology / legislation & jurisprudence
  • Gastroenterology / methods*
  • Gastrointestinal Diseases / diagnosis
  • Gastrointestinal Diseases / metabolism
  • Gastrointestinal Diseases / physiopathology
  • Gastrointestinal Diseases / surgery*
  • Government Regulation*
  • Health Policy*
  • Humans
  • Mesenchymal Stem Cell Transplantation* / adverse effects
  • Mesenchymal Stem Cell Transplantation* / legislation & jurisprudence
  • Mesenchymal Stem Cells / metabolism
  • Mesenchymal Stem Cells / physiology*
  • Patient Safety
  • Phenotype
  • Regeneration
  • Regenerative Medicine / legislation & jurisprudence
  • Regenerative Medicine / methods*
  • Risk Factors
  • Translational Research, Biomedical / legislation & jurisprudence
  • Translational Research, Biomedical / methods*
  • Treatment Outcome


  • Biomarkers