Lessons learnt, and still to learn, in first in human stem cell trials
Roger A. Barker, Melissa Carpenter, Catriona Jamieson, Charles E. Murry, Graziella Pellegrini, Rajesh C. Rao, Jihwan Song
Abstract
Developing cellular therapies is not straightforward. This Perspective summarizes the experience of a group of academic stem cell investigators working in different clinical areas and aims to share insight into what we wished we knew before starting. These include (1) choosing the stem cell line and assessing the genome of both the starting and final product, (2) familiarity with GMP manufacturing, reagent validation, and supply chain management, (3) product delivery issues and the additional regulatory challenges, (4) the relationship between clinical trial design and preclinical studies, and (5) the market approval requirements, pathways, and partnerships needed.