Over the past decade regenerative medicine has become a highly active area of investigation with the potential to address many unmet medical needs. Hematologists have played a prominent role in advancing this emerging field because of prior expertise in stem cell and transplantation biology as well as extensive experience in related areas of translational research. Despite the impetus for growth this field has experienced, investigators continue to face numerous challenges in conducting translational and clinical research in regenerative medicine and cell-based therapy.
In response to these concerns, and at the urging of the ASH Committee on Government Affairs, we were asked by the ASH Executive Committee to co-chair an expert working group in October 2009 and develop recommendations to the Society and to the National Institutes of Health (NIH) on enhancing research in regenerative medicine. The conclusions, recommendations, and composition of the working group are outlined in an editorial in Blood [116: 866-7, 2010].
Workshop participants identified a number of challenges that can be broadly classified into two areas: funding and implementation. Funding research in this area is a challenge for a number of reasons. For example, pre-clinical research is difficult to fund within the usual NIH study section format because the proposals seek to validate initial findings, establish safety, and determine efficacy rather than address a hypothesis-driven aim; hence, these proposals are often noncompetitive in study section reviews. Nonetheless, the work is essential to validate clinical protocols in support of Investigational New Drug (IND) applications. Other challenges include the considerable expense of conducting phase I trials, a lack of participation by industry, and the possibility that the applications may cross the jurisdictions of individual Institutes within NIH.
To address these and other challenges that were identified during the meeting, workshop participants recommended that a new grants panel/study section be established with broad expertise in translational and clinical research, including matters related to cell manufacturing under Good Manufacturing Practice (GMP) conditions. It was suggested that an NIH Center for Regenerative Medicine be established to champion and coordinate NIH activities in this area.
Recommendations to the general scientific community focused on the need to improve communications between basic and clinical scientists in regenerative medicine and to obtain consensus on the most appropriate design of clinical trials, including not only trials methodology but also cell characterization, cell and tissue banking, long-term followup of the recipients of the cell therapy (potentially utilizing a national database), and identification of the most relevant pre-clinical animal models for safety and efficacy testing.
The workshop recommendations to ASH included the need to meet with the leadership of other learned societies interested in regenerative medicine to promote education and research across disciplines. There was agreement that ASH was particularly well placed to foster such interactions. ASH members, as experts in stem cell biology and blood and marrow transplantation, were in an excellent position to assume a leadership role in the field of cell therapy and regenerative medicine. This leadership could extend to developing educational material on a range of topics relevant to regenerative medicine, including guidance on the risks of traveling to other countries for unapproved clinical interventions utilizing stem cells (stem cell tourism) and recent developments with reprogramming of somatic cells to stem cells, termed induced pluripotent stem (iPS) cells. It should also be extended to leadership in developing a consensus on efficacy, safety, and cell product release criteria. Specific short-term recommendations to ASH included forming an ad hoc scientific committee on regenerative medicine and including a session on regenerative medicine at its annual meeting.
ASH has already made progress on two fronts in response to the workshop recommendations. First, a scientific session on regenerative medicine is being planned for inclusion at the 2011 annual meeting. Second, the hard work of the Committee on Government Affairs, armed with the workshop recommendations, has led to incorporation of language recommended by ASH in the U.S. Senate Appropriations Committee report to the full Senate. The Senate Committee language reads in part:
The field of regenerative medicine represents a unique approach to treating diseases and disorders by enabling the body to repair, replace, restore and regenerate damaged or diseased cells, tissues and organs. The Committee believes that the NIH should carefully and deliberatively consider how best to organize and undertake research in this promising field, with input from experts in multiple disciplines. The Committee urges the Director to develop a plan that would: assess current research; identify research gaps including research methodologies; develop a mechanism to allow for the coordination of research between Institutes; consider the development of a separate study section; and in coordination with FDA, develop clinical trial methodologies and measures to assure the safety and efficacy of therapies, including data and sample registries. The Committee requests a response in the fiscal year 2012 congressional budget justification.
It is highly gratifying that, in a remarkably short time, the ASH workshop recommendations have led to the same recommendations by a Senate committee to the NIH. It is now up to the ASH membership to take a leadership position on regenerative medicine in the scientific community at large.
