Production Assistance for Cellular Therapies (PACT): Four-Year Experience from the United States National Heart, Lung, and Blood Institute (NHLBI) Contract Research Program in Cell and Tissue Therapies
Year of Publication
Reed, W; Noga, S; Gee, A; Rooney, C; Wagner, J; McCullough, J; McKenna, D; Whiteside, T; Donnenberg, A; Baker, A; Lindblad, R; Wagner, E; Mondoro, T
Algorithms; Biological Specimen Banks; Contracts; Education-Medical-Continuing; Laboratory Techniques and Procedures; National Heart Lung and Blood Institute (US); Research NIH Extramural; Tissue Therapy
BACKGROUND: In 2002, the US National Heart, Lung, and Blood Institute (NHLBI) conducted a workshop to determine needs of the cell therapy community. A consensus emerged that improved access to cGMP facilities, regulatory assistance, and training would foster the advancement of cellular therapy. STUDY DESIGN AND METHODS: A 2003 NHLBI request for proposals resulted in four contracts being awarded to three cell-manufacturing facilities (Baylor College of Medicine, University of Minnesota, and University of Pittsburgh) and one administrative center (The EMMES Corporation). As a result, Production Assistance for Cellular Therapies (PACT) was formed. RESULTS: As of October 1, 2008, PACT has received 65 preliminary applications of which 45 have been approved for product manufacture. A variety of cell therapies are represented including T-regulatory cells, natural killer cells, adipose-derived stem cells, cardiac progenitor cells for cardiac disease, hematopoietic progenitor cells (HPCs) for central nervous system applications, cytotoxic T lymphocytes, and dendritic cells. A total of 169 products have been administered under 12 applications and 2 reagents were manufactured and delivered. Fourteen peer-reviewed publications and 15 abstracts have resulted from the PACT project to date. A cell therapy textbook is nearly complete. PACT technical projects have addressed assay development, rapid endotoxin testing, shipping of cell products, and CD34+ HPC isolation from low-volume marrow. Educational Web seminars and on-site training through workshops have been conducted. CONCLUSIONS: PACT is an active and successful cell therapy manufacturing resource in the United States, addressing research and training while forging relationships among academia, industry, and participating institutions.