March 2005
The Business of Stem Cells: Re-Examining Federal, State, and Private Funding and Regulatory Initiatives
As scientists around the world race to explore the potential of stem cells, the regulatory and research funding picture has become increasingly complex. Critics believe the limitations posed by the U.S. federal stem cell policy have put a damper on public and private research. Other countries are setting up research facilities and have begun luring bio-researchers, many from the United States, to grow these new ventures. In the United States, stem cell institutes that combine private financing with state funding have been established at a number of institutions. A number of states are also spearheading initiatives, such as Proposition 71 in California. Some observers are concerned that efforts by states to supersede federally supported stem cell research will open a regulatory free-for-all. A March 9 AEI conference brought to light the various issues that have arisen in the United States and abroad in response to federal stem cell policy and the slew of recent state initiatives encouraging private investments and public/private partnerships.
Human Embryonic Stem Cells: Current Challenge and Future Promise
Dr. James Battey
Director NIDCD, NIH Task Force on Stem Cell Research
Human embryonic stem cells represent one of the most promising platforms for entering the era of regenerative medicine. The scientific challenges of human stem cells include building scientific capacity, proving the long term stability of the cells, understanding cell specialization and cell cycle control, and evaluating cell-host interactions. Given these issues, the immediate challenges for the NIH and human embryonic stem cell (HES cell) research are to generate quality HES cell lines within the NIH registry for distribution, stimulate more research on the necessary biology, and train investigators on the culture and use of stem cells. With adequate funding, training, and research, we can better define and characterize these HES cells, and we can better determine and control the necessary conditions for the successful transplantation of the cells.
Panel I: State Models/Initiatives for Stem Cell Research
David Gollaher
California Health Care Institute
When Californians approved a $3 billion bond to fund stem cell research, they embarked on a bold civic experiment, unprecedented in the annals of science. Its sheer magnitude and support base in California makes Proposition 71 the greatest application of direct democracy to science policy in history. And for both science and politics, the implications are far-reaching and profound. Having passed Proposition 71, the immediate issues at hand include building a leadership organization, handling litigation and intellectual property issues, administering the technology transfer, and sorting out conflicts of interest among supporters. As California’s stem cell initiative progresses, critical issues will continue to include addressing moral and ethical issues and educating the public. California will also be faced with the protection of state bioscience funding and meeting expectations as it strives to best utilize this elaborate funding mechanism to produce breakthrough science.
John Gearhart
Institute for Cell Engineering, Johns Hopkins School of Medicine
Legislation is pending in Maryland both for and against stem cell research. Two bills are currently under consideration. In the Maryland Stem Cell Research Act of 2005, $25 million a year has been proposed for all forms of stem cell research, excepting controversial somatic cell nuclear transfer. The second bill is the Human Cloning Prohibition Act of 2005, which bans all cloning as a felony, including techniques to extract HES cells. Governor Bob Ehrlich has taken no position on stem cell research as of yet, but has indicated that he finds it to be a federal government issue and may prefer to invest funds elsewhere. Opinions on the matter are certainly still heated, as a recent editorial in the Baltimore Sun by the William Brody, the president of Johns Hopkins University, refers to the state initiatives for scientific research as unsupportable scientific tourism. Another recent editorial in the Washington Post voices disapproval of states taking individual positions on a small area of medical research activity.
Carl E. Gulbrandsen
Wisconsin Alumni Research Foundation (WARF)
Founded in 1925 and supported with a $1.4 billion endowment, WARF is a tax-exempt, not-for-profit charity and patent management organization for the University of Wisconsin-Madison. WARF aims to pass its inventions into the scientific marketplace and invest licensing proceeds into further research. The WiCell Research Institute was founded by WARF in 1999 to conduct research on HES cells, distribute the cells to researchers worldwide, and train researchers in the culture, maintenance, and licensing of HES cells. Stem cell research at the University of Wisconsin-Madison now involves over fifty researchers dedicated to studying HES cell biology and lineage. The recent passing of Proposition 71 in California enhances the value of HES cell technology and has spurned interest in licensing. Despite an unfriendly climate surrounding stem cell research in the state of Wisconsin, the success of Proposition 71 helped push along a recent initiative by Wisconsin’s Governor Jim Doyle facilitating the funding of $0.75 billion to Integrated and Systems Biology--which includes HES cell research--over the next ten years. However, the recruiting and retaining of faculty, as well as the ability to stay competitive, will pose future challenges.
Charles Jennings
Harvard Stem Cell Institute
The Harvard Stem Cell Institute (HSCI) has broad interests ranging from the basic biology of stem cells to its implications in neurological and blood diseases, cancer, and diabetes. The HSCI is currently seeking regulatory approval to derive new embryonic stem cell lines by somatic cell nuclear transfer (sometimes known as “therapeutic cloning”). Financial, ethical, and legal regulatory hang-ups continue to act as obstacles in stem cell research. In Massachusetts, the political climate remains complex, as Governor Mitt Romney has voiced his opposition to stem cell research, and yet pro-HES cell legislation has been proposed. Current sources of funding for stem cell research in academic settings include the NIH, individual states, the biotech community, pharmaceutical companies, and philanthropic outlets. Nonetheless, all of the above are either severely restricted to such funding or not yet fully committed to the studies.
Wise Young
Rutgers University’s Keck Center for Collaborative Neuroscience
Most scientists believe that stem cells will revolutionize treatment of many incurable diseases. Congress, however, has long prohibited federal funding of research that harms human embryos, a major source of stem cells. In 2001, President George W. Bush allowed NIH to fund HES cell research but only on cells derived before August 2001. Since 2001, NIH has spent only $50 million on HESC research, while numerous other countries have spent hundreds of millions. Not surprisingly, the major advances in HESC research have occurred overseas, and clinical trials are imminent. Although human umbilical cord blood and adult tissues are non-controversial and promising sources of stem cells, NIH spent less than $250 million in 2004 on human stem cell research.
In response to growing public demand, California and New Jersey recently committed $3 billion and $380 million to stem cell research. Other states have announced their intentions to fund stem cell research. Congress ought to significantly increase NIH funding for human neonatal and adult stem cell research for the following reasons. If umbilical cord blood or bone marrow stem cells turn out to be effective and safe, they will be the therapies of choice, thereby resolving the stem cell debate. NIH funding will allow scientists in all states to contribute to the therapeutic revolution.
Finally, we must develop diverse and scaleable sources of stem cells. World supplies of even umbilical cord blood or bone marrow stem cells are insufficient to treat millions of people, should stem cells prove to be beneficial for even one of the following conditions: diabetes, heart failure, neurodegenerative diseases, multiple sclerosis, brain or spinal cord injury. We must learn how to grow and program large populations of stem cells to treat many conditions and people.
Luncheon Keynote
Beyond Bioethics: New Approaches to the Governance of Human Biotechnology
Francis Fukuyama
Bernard L. Schwartz Professor of International Political Economy
Paul H. Nitze School of Advanced International Studies, Johns Hopkins University
The Washington, D.C.-based Human Biotechnology Governance Project has examined the framework of biotech regulation, weighing alternatives, international considerations, and constitutional constraints. The study group found the Human Fertilisation and Embryology Authority (HFEA) in Britain and the Assisted Human Reproduction Agency (AHRA) of Canada to be positive models for regulating reproductive biomedicine in the United States.
Ultimately, possible means of handling the stem cell dilemma include 1) using existing statutory powers, 2) direct legislative intervention, 3) self-regulation, and 4) creating a new regulatory authority. To facilitate stem cell research, it would be most effective to pursue the latter (in the model of the HFEA and AHRA) to create new independent, representative agencies. Despite expense and the potential implications of setting a regulatory precedent, new authority and a fresh approach are necessary in overcoming interest group deadlock, although continued measures should be taken to prohibit reproductive cloning and embryo patenting. Appointment terms, voting rules, independent commissioners, and mechanisms for public consultation will be necessary. Practical questions and constitutional constraints will need to be addressed. Furthermore, international considerations will come into play, although at this time there is no need to harmonize domestic legislation with the international realm.
Panel II: Private versus Public Financing of Stem Cell Research
Opportunity and Concern
Lori Knowles
University of Alberta
Nations around the globe continue to create policy that will allow stem cell science to proceed while respecting the values each society holds dear. A survey of selected regulatory responses around the world, with particular emphasis on the United Kingdom and Canada, reveals that international stem cell initiatives result in a continually evolving regulatory patchwork. While there are a number of policy alternatives for regulating HES cell research, all require political willingness to engage in transparent and controversial political debate and a commitment of both time and money. It is essential that the United States review licensing schemes and regulate specific, informed consent of human biological material donors. Legislative review must be regulated, as well as ancillary development of infrastructure and the facilitation of public discussion and consultation.
Ken Giacin
StemCyte
The demand for umbilical cord blood is rising, as it has major advantages over bone marrow and continues to gain ground and may even overtake bone marrow as a viable alternative in regenerative medicine and therapeutic products. StemCyte, incorporated in 1997, is one of the largest and most trusted umbilical cord blood stem cell banks in the world and a leading developer and provider of stem cell therapy products. StemCyte partners with world class researchers and is currently expanding its New Jersey operations in collaboration with a New Jersey research and development initiative and a California cell biology company, while also exploring collaborations with other institutions in California and Taiwan, and a cell biology company on the east coast. Prospects for business growth and performance look promising because of increasing use of umbilical cord blood in transplants, Proposition 71 and New Jersey stem cell research programs, international interest, and potential NIH funding. Umbilical cord blood cell therapy works, and its success hinges on public and private support for this lifesaving potential.
Robert Lanza
Advanced Cell Technology and Wake Forest University School of Medicine
While the number of organ transplants has remained steady over recent years, waiting lists for transplants are rising rapidly. This need not be the case. Stem cell progeny may be able to reconstitute more complex tissues and organs, including myocardial patches, kidneys, and even entire hearts, in conjunction with the generation of functional replacement cells such as cardiomyocytes, neurons, or insulin-producing cells. Body parts can be replaceable, and conditions such as Parkinson’s disease, cardiovascular disease, diabetes mellitus, and retinal degenerative diseases could all be treated with stem cell technology. Nevertheless, federal funds currently cannot be used for any such HES cell research, the clinical applications of which are currently considered safe. It is essential to generate funding to advance this research and ultimately help the millions of Americans afflicted with diseases potentially treatable by HES cell technologies.
AEI program assistant Elizabeth White prepared this summary.
