The first-in-human phase I trial of stem cell transplantation for amyotrophic lateral sclerosis was approved by the FDA in 2009 and is ongoing. In their Perspectives article, Eva Feldman and colleagues, who are conducting the trial, discuss the preclinical research supporting this therapeutic approach, key aspects of the trial design to overcome translational issues, and ongoing challenges to address in future studies.
- Nicholas M. Boulis
- Thais Federici
- Eva L. Feldman
