Imagine that you are 13 years old and discover you are losing eyesight in the central portion of the visual field. There is hardly a part of the great adventure ahead, from learning to sports to dating, that is not changed forever by Stargardt’s Disease, an inherited disorder that resembles adult macular degeneration. A familiar trope of conservative critics of embryonic stem cell research was that regarding health problems as a crisis could provide ethical justification for just about anything. But how exactly is a young teenager’s degenerative eye disease not a crisis, not only for that child but for that family?

Medical intervention should not be the only response; parental support, counseling, classroom arrangements, and low vision devices are all important. Patients can and do live active and happy lives with a wide range of opportunities. There is no denying that we all live with disabilities or acquire them, nor that we cannot profit from life’s challenges.

And if regenerative medicine can offer a promising new therapy with reasonable risks to subjects, well, that’s not bad either. On Tuesday, the Food and Drug Administration granted orphan drug status to cells derived from human embryonic stem cells by Advanced Cell Technology that will treat Stargardt’s. This bodes well for approval for a clinical trial.

This is not only an important step in normalizing the field as a regulated scientific activity. It also speaks to the sometimes-unpredictable ways that experimentation can address sources of human suffering. And it is a clear example of the way a smart regulatory system in a liberal democracy can respond to both a medical need and a market opportunity with no moral inconsistency.

Thus what is in some respects even more interesting than the fact that the company’s approach to a retinal disorder involves cells from embryos is that the FDA approval took place under the auspices of the Orphan Drug Act. The Act encourages innovative approaches to serious diseases that affect fewer than 200,000 Americans, conditions unlikely to attract private investment without additional incentives. The rare kind of macular degeneration that the therapy will attempt to alleviate afflicts mostly children. The idea—for which there is already support in laboratory and animal studies—is that embryonic stem cells can be pushed to become the retinal cells destroyed by Stargardt’s.

It was just early last year that biotech company Geron obtained permission from the FDA for the first embryonic stem cell trial. It will never be known how much of a chilling effect Bush administration policy had on the willingness of private companies to invest in the field, but conservatives and progressives alike understand that targeted, private-sector innovation is as critical for a healthy economy as it is for improving human life.

Someday therapeutic modifications in the gene that causes Stargardt’s Disease might be possible, but there might be too many harmful mutations to make direct gene therapy practical. That question, too, will surely be answered someday. Meanwhile, we may hope that the recent designation succeeds in moving the therapy forward and throws more light on this form of macular degeneration and on the lives of the kids who suffer from it.

Jonathan D. Moreno, Ph.D., is the David and Lyn Silfen University Professor of Ethics and Professor of Medical Ethics and of the History and Sociology of Science at the University of Pennsylvania, and the Editor-in-Chief of Science Progress.

Predictably, Obama has run into some political pushback. The complaints have arisen primarily over two issues, neither of which is substantial and both of which deserve to be countered.

For one, some opponents of the research have inferred that because the president himself did not announce in his executive order any preordained limits on the research field, there is no aspect of human embryonic stem cell research that he is not willing to endorse. But anyone who paid attention to Obama’s words during the signing ceremony last Monday would know better than that. He spoke repeatedly about the need for such research to be “legal”—a clear reference to the Dickey-Wicker amendment that Congress has renewed annually for 13 years running, which precludes the use of federal funds for any work that could cause harm to a human embryo—and “responsible,” an even more demanding level of ethical care than that of mere legality. Obama also spoke forcefully against the prospect of human cloning in words that could leave no question in any listener’s mind that the president is not going to let this field of science run amok.

Second, some research opponents are upset that the president turned to the National Institutes of Health to create guidelines that will set limits on embryonic stem cell research. In their eyes, these critics saw that approach as one that puts scientists unilaterally in charge of overseeing science—arguably no better than putting theologians in charge.

But that view ignores the expertise within the NIH in areas such as research ethics, biomedical distributive justice, and other fields of social science that focus on the fair integration of pluralistic American values with the intellectual and humanistic imperative to explore science and reduce suffering. The executive order also charges the NIH with reviewing “existing NIH guidance and other widely recognized guidelines.” This refers to the guidelines put out by organizations such as the National Academies and the International Society for Stem Cell Research, which both include ethical safeguards that ensure responsible conduct of embryonic stem cell research. As the president noted, the point is not to assume that science and ethics are opposed, but to view ethics as inherent in the pursuit of scientific knowledge.

Finally, Obama observed that the stem cell policy issue is only one example of the need for policymakers to have access to the best evidence, an important realization after the last eight years. Reasonable people may disagree, but reason cannot flourish without the facts.

Earlier this year, Science Progress and the Center for American Progress released a report, “A Life Sciences Crucible: Stem Cell Research and Innovation Done Responsibly and Ethically,” in which Michael Rugnetta of CAP and Michael Peroski spell out a basic formula that would lead to a new openness in this research field without overstepping ethical lines. We review some of the key recommendations below in the hope that it will help the NIH and assorted experts find a path to bringing this important field to maturity while addressing the concerns of those who have good questions with this new and promising frontier.

Key recommendations from the report

Currently, federal funding is only available for research on the 21 lines of embryonic stem cells that were derived before August 9, 2001. Once this arbitrary limit is lifted, the National Institutes of Health will be able to issue grants to scientists who wish to research embryonic stem cells in accordance with guidelines for ethically derived cells.

CAP believes that the quickest and best way to open up the stem cell field is to focus first on those areas where there is common ground among a wide array of Americans, by allowing funding for research in which:

• The stem cells come from embryos that were originally created at in vitro fertilization clinics for the purpose of fertility treatment and are now stored at these IVF clinics because more were created than required to fulfill the patient’s clinical need.
• Proper written informed consent is obtained from the donors.
• As part of the informed consent process, the embryo donors determine along with the physician that the embryos will never be implanted in a womb and would otherwise be destroyed.
• There are no financial inducements and the donors understand the purpose of the research is not to eventually confer therapeutic benefits upon the donors.

CAP also recommends that embryonic stem cell research requirements along these lines be codified in legislation by the 111th Congress and become law so that future presidents can not obstruct this research.

Over a period of time the NIH must also address the more contentious issue of deploying federal funds for research on stem cells that have been derived with private funds from embryos created for research.

To enforce ethical guidelines and to ensure that all stem cell research (embryonic or otherwise) is conducted cautiously and responsibly so as not to threaten the safety or autonomy of research subjects or the donors of research materials, the following administrative oversight requirements should be included either in the NIH guidelines that respond to the president’s executive order or in legislation that should be passed in the first session of the 111th Congress:

• The National Institutes of Health should require that all research be conducted under the review of a stem cell research oversight committee that adheres to the standards put forth in the regulations issued by the NIH and the Department of Health and Human Services as informed by the National Academies or the International Society for Stem Cell Research guidelines. Any embryonic stem cells that are not in compliance with these rules, or are derived from embryos that are not in compliance with these rules, will not be eligible for federal funding.
• The one caveat to this requirement is that the 21 cell lines that were approved by the Bush administration should be grandfathered into the new policy because federal funding has already been provided for research that is now well underway.

These policy guidelines will ensure that human embryonic stem cell research is carried out with the highest ethical standards. It will also ensure that U.S. public and private biomedical research laboratories live up to the highest scientific standards.

Jonathan Moreno is a Senior Fellow at the Center for American Progress. Michael Rugnetta is a Research Assistant with the Progressive Bioethics Initiative at the Center for American Progress.

See also:

• Report: A Life Sciences Crucible: Stem Cell Research and Innovation Done Responsibly and Ethically
• Column: Eight Reasons to Applaud Action on Stem Cells
• News: Stem Cell Science Takes an Ambitious Step Forward
• Timeline: A Brief History of Stem Cell Research

This is an idea that I and others-especially the Union of Concerned Scientists, or UCS-have explicitly pushed for in the past. The basic notion is to be able to conduct an intellectually sound version of what Bush science adviser John Marburger himself purported to do back in 2004, after leading scientists, organized by UCS, brought scathing charges against the administration on scientific integrity grounds. The UCS claimed that scientific information had been undermined across the government, at agencies ranging from the National Cancer Institute to the Environmental Protection Agency to the Fish and Wildlife Service. It was among the earliest-and by far the most prominent-airings of what would become the Bush “war on science” allegation.

In his response, Marburger took the UCS head-on. He claimed that his office conducted a “thorough investigation into all the allegations”-which necessarily involved getting information about what had happened from each federal agency involved in a science scandal. And yet Marburger summarily dismissed the charges in a way that few observers found remotely credible. One of those unsatisfied observers? John Holdren, who told me of Marburger’s effort for my book The Republican War on Science: “One supposes he was ordered to produce a rebuttal, but they could have produced a more nuanced rebuttal than that crass, heavy-handed, and grossly wrong one that they issued.” Indeed, in my book I compared many of the original charges with Marburger’s attempted rebuttal, and found the latter largely wanting on points of substance.

Now Holdren will get to try his own hand at this scientific integrity business. Thanks to the president’s memorandum, there are many reasons to expect he’ll do a better job of it. The memorandum makes official that this is not to be a rearguard, wagon circling action, but rather, forward-looking and comprehensive. Moreover, the federal agencies and their leaders have to cooperate with the science adviser to make sure it’s done properly.

Officially, Holdren is to take no more than 120 days to come up with a plan for how the White House can ensure, across the government, that scientific information is used properly in decision-making; that such decision-making is transparent; that scientific whistleblowers are heeded and protected; that scientific advisory committees are properly staffed with experts rather than ideological hacks, and so on. Most important, there will be rules that agencies must follow to ensure scientific integrity; and procedures in place to investigate, should anyone allege that they haven’t done so.

Provided the executive branch does indeed set up a system set up like this, it would be a huge step forward. The whole problem with the Bush administration’s responses to many allegations of political interference with science is that the answer was always the same: Nothing to see here folks, move along. Repeatedly, Bush spokespeople-Marburger, and also various press secretaries-simply asserted that all the whistleblowers were wrong, all the journalists were wrong, heck, anybody was wrong who suggested anything untoward had happened. They didn’t seriously investigate the problems; they dismissed the idea that there were any problems. Needless to say, it wasn’t a very credible approach.

Now, not only can we hope for a more transparent method of dealing with any potential new politics and science allegations; we can also hope for a much stronger presidential science adviser with the power to investigate them. For that’s perhaps the most significant aspect of the President’s scientific integrity memorandum. It puts John Holdren on a par with the heads of the federal agencies-with the cabinet-who need to report to him to show that their houses are in order. In other words, he’ll serve as a central science czar whose role is to provide good advice and preserve informational integrity, and who will actually be listened to and heeded.

Now, if we could only get Holdren through the Senate and into his job.

Chris Mooney is contributing editor to Science Progress and author of several books, including The Republican War on Science and the forthcoming Unscientific America: How Scientific Illiteracy Threatens Our Future, co-authored by Sheril Kirshenbaum. He and Kirshenbaum blog at “The Intersection.”