The ASGCT in 2012: Charting a Path to Bring Novel Biologic Therapies to the Clinic
Authors: Xandra O Breakefield
Journal: Molecular Therapy (2012)

Many of us in the American Society of Gene and Cell Therapy (ASGCT) have dedicated our careers to developing novel biologic therapies for incurable diseases. For me, this path opened when I was 10 years old. My mother came home and told the family that my baby sister had a hereditary disease of unpredictable outcome, for which the doctors could offer neither insight into cause nor hope for therapy. There continue to be thousands of hereditary and nonhereditary diseases with such a prognosis. Although the underlying science is both fascinating and essential, the main goal of our Society must be to facilitate the development of new therapies and to bring promising ones into the clinic to help patients. In recent years, we have had exciting evidence that the vision we set out to follow so many years ago can in fact bring benefit to patients. We are beholden to the brilliant basic research efforts that have contributed to these successes. We now face a problem engendered by this success; how to bring these biologic therapies to patients both safely and expeditiously. This is new, uncharted territory, and it will take a multifaceted force of scientists, clinicians, insurers, business people, and patient advocacy groups to generate an operational map forward.1Wilson JM It's time for gene therapy to get disruptive.Hum Gene Ther. 2012; 23: 1-3Crossref PubMed Scopus (8) Google Scholar,2Philippidis A Developing a balanced business model for gene therapy.Hum Gene Ther. 2011; 22: 645-646Crossref PubMed Scopus (2) Google Scholar,3Ettinger WH Flotte RR The role of gene and cell therapy in the era of health care reform.Hum Gene Ther. 2011; 22: 1307-1309Crossref PubMed Scopus (4) Google Scholar As President of the ASGCT, I will place a major focus on fostering the delivery of gene and cell therapy to the patient, including education, advocacy, and public awareness of the issues. I will start by organizing a commercialization workshop to be scheduled for 2013. My presidential predecessors, Barrie Carter and Jude Samulski, launched several important initiatives that have added new dynamic aspects to the ASGCT. I will keep those efforts moving forward. With the launch of the strategic plan for the Society, initiated by Dr. Carter, several important new committees have been formed. These will expand our educational and leadership role; they will promote partnerships with related societies, the media, Congress, bio-pharma, foundations, and the public at large; and they will nurture young investigators in the field through mentorship–leadership programs. These committees are off to a running start under the energetic leadership of Carter (Development Committee), Savio Woo (Outreach Committee), and Matthew Weitzman (New Investigator Committee). Although American in name, ours is a truly international society with about 30% of attendees at the 2012 ASGCT Annual Meeting traveling from outside the United States. I will continue the mission launched by Dr. Samulski to strengthen international collaborations with gene therapy societies so that we function as a synergistic network. Efforts on this front include presidential trips to China and Japan as well as a Global Gene Therapy Symposium and Latin American reception at the 2012 Annual Meeting. In addition, we will be hosting a joint clinical trials training course with our sister society, the European Society of Gene and Cell Therapy (ESGCT), to be held in the fall of 2012. There has been much discussion—even commotion—following the recent recommendation of the ASGCT Board of Directors, under the guidance of Dr. Samulski, to the Office of Biotechnology Affairs that “the NIH guidelines be amended such that the Recombinant DNA Advisory Committee (RAC) would terminate review of individual protocols and would instead identify new areas of research that required a public forum for discussion and review.” This does not mean that we do not appreciate the historical contributions of the RAC in advising the field as it has evolved, nor that we request the abolition of the RAC. We simply feel that the regulatory agencies responsible for review of these protocols—including the US Food and Drug Administration, the Office for Human Research Protections, and institutional studies review boards and biosafety committees—are fully competent to carry out these reviews. Furthermore, it is our consensus that the RAC could function more effectively in its role in advising the National Institutes of Health director and maintaining a public forum by focusing on novel issues of concern through targeted workshops and symposia. For investigators, the additional paperwork required by the RAC, as well as presentations at RAC meetings regarding these protocols, results in a significant loss of time and energy that would be better devoted to ensuring that promising therapies are efficiently translated into clinical trials.4McGarrity GJ It's time to end RAC review of gene therapy protocols.Mol Ther. 2012; 20: 1079-1080Abstract Full Text Full Text PDF PubMed Scopus (1) Google Scholar To realize our vision of successful biologic therapies for patients, our Society should capitalize on our strengths in all areas, including vector-, cell-, and oligonucleotide-mediated therapies, as well as engage all critical partners, including basic research scientists, physicians, regulatory agencies, ethicists, foundations, and the bioindustry community. We can succeed only by working together and by appreciating the critical contributions of all components of the enterprise. I look to all members of the ASGCT and our international partners to keep me informed over the coming year of critical issues and ways to improve our success in this mission. We are a Society on a quest to improve health care in diseases that defy current medical practice. Together we can chart a straight, swift, and safe course toward that goal.