OVERVIEW OF A QUALITY ASSURANCE QUALITY-CONTROL COMPLIANCE PROGRAM CONSISTENT WITH FDA REGULATIONS AND POLICIES FOR SOMATIC-CELL AND GENE THERAPIES - A 4-YEAR EXPERIENCE

Somatic eel and gene therapy involve the application of biological tec hnologies to an individual patient through the use of living cells whi ch provide a therapeutic benefit (Aliski, 1998). Various forms of cell ular and gene therapies are being developed and evaluated in an increa sing number of clinical trials for congenital and acquired disorders. The potential and progress of these therapeutic applications have resu lted in an increasing effort by the Food and Drug Administration (FDA) to develop the regulatory framework under which these therapeutic app roaches would insure safety and efficacy, the primary mandate of the F DA. Over five years ago Cellcor began to define the parameters, specif ications, and conditions relevant to a Quality Assurance / Quality Con trol (QA/QC) program that has evolved to insure safety and maximize th e efficacy of applications of the company's ex vivo technology, autoly mphocyte therapy. Autolymphocyte therapy is an outpatient form of soma tic cell immunotherapy based upon the infusion of T cells that have be en activated ex vivo using a combination of previously generated autol ogous cytokines and an anti-CD3 monoclonal antibody. We have been able to demonstrate the feasibility for the safe, controlled, and consiste nt preparation and delivery of a cellular therapy by application of re levant GMP regulations. This presentation reviews aspects of this prog ram and chronicles our experience which at present amounts to over 440 0 infusions for over 700 patients. This program provides a high degree of assurance that a cellular therapy program can be carried out in a multisite mode involving hundreds of patients through the strict adher ence to cGMP as set forth in existing regulations. It would be prudent that developers of cellular and ex vivo gene therapies establish a si milar cell processing and QA/QC infrastructure at an early development al stage to optimize safety and reproducibility and facilitate regulat ory review.