2010 Gene Therapy Year in Review

As the Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) drew to a close, I had the opportunity to reflect on the Society and on our mission of moving gene and cell technologies into clinical practice. While significant challenges remain, participants at the thirteenth Annual Meeting exhibited a renewed optimism and excitement owing to the significant advances occurring in our field. Although each year I am impressed by the increasing sophistication of vector design, an important change this year was the advance of clinical gene and cell therapies toward the clinic. While the experience with clinical trials is still limited, the field is moving beyond feasibility studies to develop trials that assess clinical efficacy. My time as an officer of the ASGCT has also allowed me to see the evolution of our Journal. As most in the Society know, Molecular Therapy is the leading gene therapy journal, with an impact factor of 6.239. Our bylaws mandate that a new Editor-in-Chief be selected every five years. Inder Verma served as the first Editor-in-Chief and fostered a remarkable rise in the journal's impact factor. Dave Williams, who just completed his five-year term, also provided outstanding stewardship of Molecular Therapy. In addition to continued improvement in impact factor, Dave oversaw the transition of the journal to Nature Publishing Group (NPG). The business arrangement negotiated with NPG has markedly increased the value of the Journal, and Molecular Therapy is now the Society's major source of revenue. This past January, Malcolm Brenner became the third Editorin- Chief. Selection of the Editor-in-Chief is a highly competitive process overseen by the ASGCT Publication Committee and the Board of Directors. From among many highly qualified candidates, Malcolm was chosen for his scientific expertise, his past editorial experience with Blood and other journals, and his ability to articulate a vision for continuing the high standards set by the previous editors. While our Annual Meeting and our Journal are the most visible efforts of our Society, I have had the opportunity to participate in other important activities that foster our research and education missions. One such activity is career development of students, postdoctoral fellows, and junior investigators. Each year the Society awards 60 $500 travel awards to trainees at the pre- and postdoctoral levels. Selection is based on the scientific merit of the abstracts submitted by the trainees. Approximately half of the awards this year were facilitated by a grant from the National Heart, Lung, and Blood Institute. The remainder were supported by meeting sponsors, revenue from Molecular Therapy, and membership dues. Each year, the Society selects four promising individuals just beginning their independent research careers to receive the Outstanding New Investigator Award. An additional outreach to junior investigators was begun this year through the efforts of Matthew Weitzman and the New Investigator Center for Educational Resources committee. This group has developed a dedicated area of the ASGCT website to provide information to junior investigators about gene and cell therapy funding, regulatory guidance, and job opportunities. As the site develops, it will seek to promote networking and expand the existing career-development resources. Members should also expect to see a changing footprint for ASGCT in cyberspace. A major effort is under way to improve the educational content on the ASGCT website. The Society has contracted a science writer to work with our membership to expand the content so that our website can serve as a resource for our members, the public, and the media. Savio Woo and the Membership Committee have also inspired our foray into social media through the development of the ASGCT Facebook page. Members who use social media can now obtain news about gene and cell therapy through this outlet, which also offers the opportunity to discuss and exchange ideas in real time. It will be interesting to see how our members use the Facebook site. As part of the Society's educational mission, Barrie Carter and I spent two days meeting with 13 directors of National Institutes of Health (NIH) institutes and centers. Prior to the call, we contacted our membership to better understand the challenges faced by our investigators. Those concerns were summarized and shared with the NIH leadership. Overall, the trip was very encouraging. Clearly, the NIH leaders are aware of the advances in our field. Barrie and I also used the visit to discuss the difficulty in obtaining funds for clinical trial development. We pointed out that many of the recent clinical successes were achieved in Europe, which reflects, in part, on the manner in which translational research is funded in the United States. Rather than viewing preclinical development, vector manufacture, toxicology studies, and early-phase clinical trials as a continuum, most institutes require investigators to submit multiple, independent grant applications for each element of translational research. This system often leads to a catch-22 scenario and challenges the five-year limit on many grants. While these challenges have been discussed at a variety of NIH workshops, most institutes have not implemented the recommendations developed at these forums. Another area about which our members have expressed significant concern is regulatory oversight. Historically, review processes were put into place at a time when investigators had focused research projects, so there was little overlap of review by animal-care, biosafety, and human subject protection committees. Now, with the evolution of translational research, investigators routinely perform work that includes animal studies, recombinant DNA, and human research. If the NIH wishes to significantly foster translational research, they need to help develop a review process that reflects the current research environment. By consolidating review committees, the quality of a review can be improved by allowing reviewers with diverse expertise to discuss the merits of a proposal in a collaborative fashion. This will also result in a considerable cost savings to the research enterprise, given the time invested by institutions in staffing multiple separate review committees. At the NIH meeting, we also discussed a concern by our members that current local and federal regulations do not appear to be evidence-based. I believe that the Society will be fully supportive of any new requirement that promotes safety, but current requirements should be assessed for effectiveness, and those that do not add value should be eliminated. Finally, we addressed the challenges our members have in navigating the NIH. Translational research often spans the mission of multiple institutes, and funding mechanisms for translational research vary widely among the different institutes. The silos within the NIH make it difficult for investigators to identify the most relevant and efficient pathway to move translational research forward. Barrie and I summarized our visit in a letter to the NIH Director. He provided an encouraging response, which is available on the ASGCT website. Although changes will take time, I was encouraged to hear that the concerns expressed by our members were also of concern to NIH leaders and that many of them had already been considering ways to address these challenges. While the Society had a very active year, there are many opportunities ahead. One of the most significant changes was the addition of “Cell” to the name of our society. Even prior to this change, about one-third of our meeting abstracts reflected cell therapy, but stem cell science and cell therapies are rapidly growing. Many workers in this area are using genetic modifications developed by ASGCT members. It will be critical for the Society's leadership to engage those working in cell therapies as well as to foster scientific exchange with our members developing novel gene transfer technologies. Finally, a major strength of our Society is the varied interests and background of our membership. We have had a shared goal of using genetic technologies to develop novel therapies. As our efforts move into clinical practice, the Society will need to adapt from its current role in fostering the development of theoretical treatments to interacting with individuals who are overseeing the use and development of licensed products. Ensuring that ASGCT continues to be of value to academic, nonprofit, and industrial members will take considerable thought and engagement with our membership. Nevertheless, adapting to licensed gene and cell therapy products is a challenge that we all welcome. Although it will be some time before these therapies are in the mainstream of medical care, the positive events of the past few years suggest that the progress is steady and we will see continued positive clinical outcomes.

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