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

Abstract

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.

Fig. 1

Organization of the NHLBI program Production Assistance for Cellular Therapies (PACT).

Fig. 2

(A) Preliminary applications by origin, on a quarterly basis, from January 2004 through September 2008. (B) Preliminary application by review status and application origin on a semiannual basis. (Period 10 for this figure only contains data from July 2008 through September 2008.) (C) Data shown are full applications by application type and origin on a semiannual basis. (Period 10 for this figure only contains data from July 2008 through September 2008.)

Fig. 3

Workflow, product manufacturing, and product administration associated with the current 45 approved full applications to NHLBI PACT.