Intraspinal stem cell transplantation for amyotrophic lateral sclerosis: Ready for efficacy clinical trials?

Cytotherapy. 2016 Dec;18(12):1471-1475. doi: 10.1016/j.jcyt.2016.08.005. Epub 2016 Oct 6.


Intraspinal stem cell (SC) transplantation represents a new therapeutic approach for amyotrophic lateral sclerosis (ALS) clinical trials. There are considerable difficulties in designing future efficacy trials, some related to the field of ALS and some that are specific to SCs or the mode of delivery. In October 2015, the most controversial points on SC transplantation were addressed during an international workshop intended to bring together international SC and ALS researchers in a public discussion on a topic for which expertise is limited. During the meeting, a discussion was started on the basic structure of the ideal clinical trial testing the efficacy and safety of SC transplantation. The current document includes a number of consensus points reflecting the design of phase II/III clinical trials.

Keywords: ALS; clinical trials; stem cells; transplantation.

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Amyotrophic Lateral Sclerosis / therapy*
  • Cell- and Tissue-Based Therapy / methods*
  • Clinical Trials, Phase II as Topic / methods*
  • Clinical Trials, Phase III as Topic / methods*
  • Consensus
  • Humans
  • Middle Aged
  • Neural Stem Cells / transplantation
  • Safety
  • Stem Cell Transplantation*
  • Young Adult