Cellular Therapies Clinical Research Roadmap: lessons learned on how to move a cellular therapy into a clinical trial

Stacy Ouseph; Darah Tappitake; Myriam Armant; Robin Wesselschmidt; Ivy Derecho; Rebecca Draxler; Deborah Wood; John M Centanni

doi:10.1016/j.jcyt.2014.10.008

Cellular Therapies Clinical Research Roadmap: lessons learned on how to move a cellular therapy into a clinical trial

Cytotherapy. 2015 Apr;17(4):339-43. doi: 10.1016/j.jcyt.2014.10.008. Epub 2014 Dec 4.

Authors

Stacy Ouseph¹, Darah Tappitake², Myriam Armant², Robin Wesselschmidt³, Ivy Derecho³, Rebecca Draxler⁴, Deborah Wood⁵, John M Centanni⁶

Affiliations

¹ Baylor College of Medicine, Houston, Texas, USA. Electronic address: ouseph@bcm.edu.
² Center for Human Cell Therapy, Boston, Massachusetts, USA.
³ City of Hope, Duarte, California, USA.
⁴ University of Minnesota, Minneapolis, Minnesota, USA.
⁵ The EMMES Corporation, Rockville, Maryland, USA.
⁶ University of Wisconsin, Madison, Wisconsin, USA.

Abstract

Background aims: A clinical research roadmap has been developed as a resource for researchers to identify critical areas and potential pitfalls when transitioning a cellular therapy product from the research laboratory, by means of an Investigational New Drug (IND) application, into early-phase clinical trials. The roadmap describes four key areas: basic and preclinical research, resource development, translational research and Good Manufacturing Practice (GMP) and IND assembly and submission.

Methods: Basic and preclinical research identifies a new therapeutic concept and demonstrates its potential value with the use of a model of the relevant disease. During resource development, the appropriate specialists and the required expertise to bring this product into the clinic are identified (eg, researchers, regulatory specialists, GMP manufacturing staff, clinicians and clinical trials staff, etc). Additionally, the funds required to achieve this goal (or a plan to procure them) are identified. In the next phase, the plan to translate the research product into a clinical-grade therapeutic is developed. Finally regulatory approval to start the trial must be obtained. In the United States, this is done by filing an IND application with the Food and Drug Administration.

Results: The National Heart, Lung and Blood Institute-funded Production Assistance for Cellular Therapies program has facilitated the transition of a variety of cellular therapy products from the laboratory into Phase1/2 trials.

Conclusions: The five Production Assistance for Cellular Therapies facilities have assisted investigators by performing translational studies and GMP manufacturing to ensure that cellular products met release specifications and were manufactured safely, reproducibly and at the appropriate scale. The roadmap resulting from this experience is the focus of this article.

Keywords: FDA; GMP; IND filing; NHLBI; PACT; cellular therapy.

Publication types

Research Support, N.I.H., Extramural

MeSH terms

Biomedical Research
Cell- and Tissue-Based Therapy / trends*
Clinical Trials as Topic / methods*
Humans
United States
United States Food and Drug Administration

Abstract

Publication types

MeSH terms

Grants and funding