In the absence of broader federal support, alternate funding sources are taking the lead in promoting embryonic stem (ES) cell research in the United States. During the last year, Stanford and Harvard universities have launched multimillion-dollar ES cell programs with private funds, while California taxpayers authorized Proposition 71, a $3 billion, 10-year effort to advance the field's medical goals. States including Wisconsin, Illinois, Florida, New Jersey, and Massachusetts are now considering their own stem cell programs, hoping to secure a foothold in this growing field of research.
As the main backers of stem cell science, these entities complement the National Institutes of Health, whose $28 billion annual budget supports most of the basic biomedical research in this country today. The NIH is a minor player in the field, however, committing just $24 million to ES cell research in 2004. Constrained by Bush administration policy, NIH-funded scientists are limited to studying federally approved ES cell lines that are useful for studies of early-stage events in differentiation, says James Battey, M.D., Ph.D., who chairs the NIH's Stem Cell Task Force.
But many researchers consider these lines poor in quality relative to those being generated in the private sector, which is where much of the research is now unfolding, far removed from NIH research protocols. The long-term consequences for U.S. leadership in the field are unknown and debated among scientists and health policy analysts.
Varying Attitudes
Some take a dim view, suggesting that without traditional NIH support, stem cell science in this country may lag behind progress elsewhere, for instance in the United Kingdom, Israel, Singapore, and Australia, all countries where the field has broad government backing.
"Federal funding has been key to the success of U.S. science for 30 years," said Massachusetts Institute of Technology professor Rudolph Jaenisch, M.D. "It's good that private organizations are stepping up to the plate, but they aren't available to all scientists and there's no way they can fill the hole left by the NIH." Jaenisch says a troubling parallel can be drawn between ES cell science now and human in vitro fertilization (IVF), which was also denied NIH funding in 1980 by federal officials who cited ethical concerns. More than 25 years after the technique was first demonstrated in humans, IVF in this country has emerged wholly in the private sector. "But everything we learned about IVF and how to make it better came from European academic investigations," Jaenisch said.
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Dan Perry, the executive director of the Alliance for Aging Research, says the projected $300 million in annual spending under Proposition 71in addition to the "half again or more" expected from other statesis enough to keep stem cell scientists engaged. "It's not ideal," he said. "But it offers hope the science will go forward, although probably not as fast as it would under a unified policy."
Short-Term Implications?
Most stakeholders do agree, however, that the lack of federal support poses some difficult challenges. A chief concern is that states will have to replicate a research infrastructure that has already been in place at NIH for decades. From grant writing to publishing, the NIH provides a uniform approach to the business of science. Without this national protocol, each state will have to reinvent the wheel, so to speak, and decide how ES cell research funds will be disseminated and findings evaluated. Some worry that a checkerboard of varying institutional policies might emerge state by state, forcing scientists to contend with a bewildering array of procedures.
Moreover, the financial resources devoted to stem cells will also differ by state, conceivably pulling scientists in different directions according to the local funding climate. "One of the things that makes the NIH work so well is that you have all the scientists in the country competing for the same money," said Sean Tipton, a spokesperson with the Coalition for the Advancement of Medical Research, in Washington, D.C. "What we're going to have in stem cells is smaller groups of scientists competing for smaller amounts of funding."
Funding Challenges
The obvious roots of a national funding disparity are now emerging with Proposition 71. Carl Gulbrandsen, Ph.D., president of WiCell Research Institute, a nonprofit organization formed to promote stem cell research, based in Madison, Wis., says that plentiful dollars in California are a potential magnet for scientists who would migrate westward for greater opportunity, "which means that we have to spend more money in Wisconsin to recruit and retain faculty," he said.
The University of Wisconsin has a stem cell program funded with both private and public funds that employs roughly 50 researchers, making it one of the largest in the country. So far, researchers there have not needed to derive human ES cells beyond those made available by the Bush administration. But Gulbrandsen says that the university is on the verge of introducing a more optimal media that will allow scientists to culture ES cells for human therapy. (Culture media for the current NIH-approved cell lines are prepared with the use of mouse feeder cells, making them unattractive for use in humans because of possible contamination by pathogens such as retroviruses.) At that point, new ES cell lines will be required, forcing the university to seek alternative funding sources. The state's governor, Jim Doyle, recently announced a plan to invest nearly $750 million from a combination of public and private sources to support human ES cell research.
Scientists suggest that in this alternative funding environment, money won't necessarily flow to the most talented scientistswho could be located anywherebut will instead go to institutions and organizations set up for ES cell research.
Robert Lanza, M.D., is the medical director at Advanced Cell Technology (ACT), a stem cell research firm based in Worcester, Mass. ACT scientists recently showed that ES cell technology might be able to generate retinal cells to treat macular degeneration, a common cause of blindness, he said.
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Another potentially more challenging issue could be the expectations of those who pay for the research. NIH-funded scientists are to some extent shielded from economic and political concerns. From their basic researchwhich is often time-consuming and expensivecomes the fundamental knowledge in cell and molecular biology that fuels therapeutic discovery in the private sector.
Alternative funding sources, on the other hand, might apply unique pressures that could influence the course of the science. In some instances, the research could be targeted toward the aims of donor foundations representing specific diseases, such diabetes or Alzheimer's disease. This might discourage researchers from pursuing more open-ended studies that often lead to serendipitous findings. "Quite often, the most important cures are made by accident," Tipton said. "With a tight focus to achieve therapeutic benefits you can miss out on these opportunities."
Additional pressures could arise from demands to deliver therapies quickly. Political advertisements mounted during the campaign for Proposition 71 featured celebrities such as Christopher Reeve and pleas from advocates who claimed that ES cells could supply cures within a decade, in time to help some people who are suffering today. Taxpayers who agreed to fund the program are pinning their hopes on the technology, perhaps in vain.
Moreover, millions of dollars in capital came from corporate donors and venture capitalists who could be expected to seek a timely return on their investment. But the reality is that viable treatments are unlikely to arise from ES cells for many years, if at all.
In this charged environment, privately funded scientists might be overly selective about how they release research findings, thus undermining a dialogue that benefits the entire community. Unlike NIH-funded researchwhich has strict requirements for openness and transparencyprivately generated results can be withheld if they don't favor a sponsoring company's interests or if intellectual property concerns are at stake. It is unclear whether this will be an issue and if it will negatively affect the field.
Moving Forward
And so there are many important questions about the future of ES cell research. Will the technology succeed? How will the states structure their own programs and will these programs mirror each other or diverge in inconsistent ways? How will taxpayers and private sponsors respond to the realities of basic research; that painstaking process from which they are more often shielded by the federal government?
What is perhaps most clear is that much of the country has rallied around ES cell technology even in the context of the Bush administration policy. Should the technology yield even one reasonably achievable cure, elected officials may face some hard choices and pressures from the public. "I'm fond of saying that if you cure diabetes in dogs, the pressure to move with more federal funding could be overwhelming and you won't be able to stop it," Lanza said. "Right now, scientists are very conservative. We need answers. But without the research, we'll never know if this will work."
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