Lessons learnt, and still to learn, in first in human stem cell trials

Roger A Barker; Melissa Carpenter; Catriona H M Jamieson; Charles E Murry; Graziella Pellegrini; Rajesh C Rao; Jihwan Song

doi:10.1016/j.stemcr.2022.11.019

Lessons learnt, and still to learn, in first in human stem cell trials

Stem Cell Reports. 2023 Aug 8;18(8):1599-1609. doi: 10.1016/j.stemcr.2022.11.019. Epub 2022 Dec 22.

Authors

Roger A Barker¹, Melissa Carpenter², Catriona H M Jamieson³, Charles E Murry⁴, Graziella Pellegrini⁵, Rajesh C Rao⁶, Jihwan Song⁷

Affiliations

¹ Department of Clinical Neuroscience and Wellcome-MRC Cambridge Stem Institute, John van Geest Centre for Brain Repair, Forvie Site, Robinson Way, Cambridge CB2 0QQ, UK. Electronic address: rab46@cam.ac.uk.
² ElevateBio, Cambridge, MA 02142, USA.
³ Division of Regenerative Medicine, Department of Medicine, Sanford Stem Cell Clinical Center, University of California San Diego, Sanford Consortium for Regenerative Medicine, 2880 Torrey Pines Scenic Drive #0695, La Jolla, CA 92037-0695, USA.
⁴ Institute for Stem Cell and Regenerative Medicine, Center for Cardiovascular Biology; Departments of Laboratory Medicine & Pathology, Bioengineering, and Medicine/Cardiology, University of Washington, Seattle, WA 98109, USA; Sana Biotechnology, Seattle, WA 98102, USA.
⁵ Centre for Regenerative Medicine, University of Modena and Reggio Emilia, 41125 Modena, Italy.
⁶ Departments of Ophthalmology & Visual Sciences, Pathology, and Human Genetics, University of Michigan, Surgery Service, VA Ann Arbor Health System, Ann Arbor, MI 48105, USA.
⁷ Jihwan Song, Department of Biomedical Science, CHA University, 335 Pangyo-ro, Bundang-gu, Seongnam-si, Gyeonggi-do 13488, Republic of Korea; iPS Bio, Inc., 16 Daewangpangyo-ro 712 Beon-gil, Bundang-gu, Seongnam-si, Gyeonggi-do 13488, Republic of Korea.

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.

Keywords: stem cell therapy; stem cells; translation.

Publication types

Review
Research Support, N.I.H., Extramural
Research Support, Non-U.S. Gov't

MeSH terms

Cell Line
Cell- and Tissue-Based Therapy*
Humans
Stem Cells*

Abstract

Publication types

MeSH terms

Grants and funding