Science Policy

Rock Talk, a National Institutes of Health (NIH) blog on extramural research policy, debuted two years ago as a way to enhance transparency about how the federal agency develops policies and present data that influence such decisions.

In a commentary published yesterday in Nature, Sally Rockey, PhD, deputy director for extramural research at the NIH, discusses her motivation for launching the blog and how it has increased dialogue with constituents about science policy. She writes:

We learned a lot about our constituents’ interests and needs through the blog, and we have been able to highlight behind-the-scenes data, actively engage the community in policy-making and provide insight into our decisions. Without public input, effective and impactful policy cannot be created. My blogging experience has convinced me that using social-media platforms is one effective way for science-funding agencies to successfully support research.

The blog is not the official vehicle for communicating policy changes. We have the NIH Guide for Grants and Contracts for that. And we have official channels for soliciting public feedback — through requests for information in the NIH Guide, for example. But the blog allows me to extend that conversation to people worldwide, many of whom I would not be able to reach in other ways. People are sometimes concerned that offering a dissenting opinion to NIH officials might affect their chances of getting funding. Although that is absolutely not the case, one advantage the blog does have over some other channels is that it allows people to remain anonymous if they wish.

In addition to fostering conversations on policy, Rockey offers examples of how the blog has allowed her team to provide real-time updates during national emergencies, such as Hurricane Sandy, and debunk urban myths about NIH-supported research.

Previously: Veteran blogger offers tips for starting a science blog

When I first interviewed Brian Kobilka, MD, winner of the 2012 Nobel Prize for Chemistry in October, I was struck by an off-hand comment about his motivation for his near-obsessive two-decades long research quest to uncover the workings of GPCRs, or G-protein-coupled receptors, which serve as one of the main methods of molecular communication within the body.

The research, which it’s believed will lead to the creation of new drugs for clinical care, was not originally done for this purpose. It was motivated by simple scientific curiosity - the kind that often leads to amazing discoveries that help cure suffering or save lives. Initially, Kobilka just really wanted to know how it worked.

In a story published in today’s Inside Stanford Medicine, I describe the success of research based on scientific curiosity in leading to clinical care breakthroughs. The story describes the 30-year history of scientific breakthroughs that led to the approval of a new drug called vismodegib that is used to treat inoperable basal cell carcinomas, and how the drug helped save the eyesight of 101-year-old Winnie Bazurto of San Mateo, Calif. It’s a story that begins with a similar motivation - basic scientific interest - and ends with discoveries that help patients in a very practical way. As Jean Tang, MD, PhD, Bazurto’s dermatologist and vismodegib researcher, says in the article:

If a patient only knew the whole story — how the happenstance of science led to their treatment… If they could go back to when this molecular pathway was first discovered in fruit flies, they’d be amazed. It’s not until the dots are connected 30 years later that it begins to make sense.

Stanford’s Matthew Scott, PhD, one of the key players in this basic-science success story, commented to me in an e-mail just how essential it is for future clinical discoveries that basic science continues to be funded. He expressed concern about a current trend toward conservatism in funding that requires much quicker results that lead to treatment options for patients saying, “Current conservatism in funding asks for translational work that gives cures in a few years (which never happens). Far-sighted funding of basic science … pays off big time.”

The vismodegib story illustrates just how essential basic science is to the future of clinical discoveries:

For many of the basic scientists involved in this research, the clinical use of hedgehog-inhibiting drugs to treat patients like Bazurto — while not the original goal of their research — is the ultimate success.

Previously: Why basic research is the venture capital of the biomedical world, Future of medical research is at risk, says Stanford medical school dean and The economic benefits of publicly funded medical research
Photo by Norbert von der Groeben

Yesterday, the Supreme Court declined to hear the case of Shirley vs. Seblius, which challenged the federal government’s right to fund research on human embryonic stem cells. This is good news for stem cell researchers. I asked Stanford law professor and bioethicist Hank Greely, JD, about the verdict, and he told me this morning:

At last, it’s over! To call Sherley v. Sebelius our long national nightmare is a bit of an overstatement, except as to those keenly interested in pluripotent stem cell research. This case, filed in August 2009, should have been thrown out by October 2009. (Ironically, the district court judge did try to throw it out then, but used what the appellate court considered the wrong approach.) But, at long last, after 41 months of uncertainty, leavened with occasional shock and panic, the case is dead. The Supreme Court has, not surprisingly, denied review of the decision by the Court of Appeals for the District of Columbia Circuit, which upheld the district court’s summary judgment in favor of the plaintiffs.

And so it is finally established that, no, Congress did not, in the Dickey-Wicker Amendment, as re-passed every year since 1996, try to outlaw the positions taken by the Clinton, Bush, and Obama Administration. Funding human embryonic stem cell research does not violate the NIH appropriations provisions. It does, however, still rest within the discretion of the sitting President. That seems safe for the next four years; it would be good if Congress could pass legislation expressly supporting such research for the four, and eight, and twelve, years beyond that.

Greely has been outspoken about the ongoing case on the Center for Law and the Biosciences‘ blog. You can read more about the legal background of the case here and in subsequent entries.

Previously: Judge Lamberth dismisses stem cell lawsuit, Stanford law professor on embryonic stem cell ruling and Stem cell funding injunction overturned by federal court

In the wake of last week’s tragic school shooting in Connecticut, three Journal of the American Medical Association publications are running editorials today about gun safety. All three are free online and worth reading. Archives of Internal Medicine‘s piece advocates for tighter restrictions on the sale of guns and ammunition, while the editorial in Archives of Pediatric & Adolescent Medicine describes the legal battle in Florida to defeat a law that would muzzle pediatricians’ first-amendment right to talk with their patients’ parents about gun safety in the home.

The piece that I found most powerful, however, was JAMA‘s Silencing the Science on Gun Research, in which the authors describe how Congress has systematically stripped all Department of Health and Human Services agencies of the ability to fund research into the epidemiology of gun injuries:

In 1996, pro-gun members of Congress mounted an all-out effort to eliminate the National Center for Injury Prevention and Control at the Centers for Disease Control and Prevention (CDC). Although they failed to defund the center, the House of Representatives removed $2.6 million from the CDC’s budget—precisely the amount the agency had spent on firearm injury research the previous year. Funding was restored in joint conference committee, but the money was earmarked for traumatic brain injury. The effect was sharply reduced support for firearm injury research.

To ensure that the CDC and its grantees got the message, the following language was added to the final appropriation: “none of the funds made available for injury prevention and control at the Centers for Disease Control and Prevention may be used to advocate or promote gun control.”

A new study from Stanford molecular and cellular physiologist Axel Brunger, PhD, and colleagues clears up a controversy in the neuroscience community by pinpointing a critical feature of the mechanism by which our nerve cells manage to talk to one another in something approaching real time. If that conversation were stuck in slo-mo, the distinction between brain and blob would vanish.

While the study is noteworthy in itself, the fact that its findings appear in the first issue of eLife, a newly launched open-access journal, rather than Science or Nature is also significant. As a Howard Hughes Medical Institute investigator with close to 250 peer-reviewed publications under his belt, Brunger is hardly hard-up for high-rated scientific outlets. But his experience, he tells me, has made it clear that “our peer-reviewed publication system is in a state of crisis.”

It seems other scientists feel the same. A University of Montreal study published in November concluded that the most prestigious journals were publishing fewer and fewer of the most frequently cited articles.

“For many ‘high-impact factor’ journals, initial triaging and final decisions aren’t made by active scientists,” says Brunger. “That’s not to say that these journals don’t publish excellent work, but the criteria for acceptance seem rather arbitrary and random.”

The brainchild of three heavyweight research-funding entities – HHMI, the Max Planck Society, and Wellcome Trust – eLife is not only open-access, but publication-immediately-upon-acceptance and embargo-free. (Not to mention just plain free, for both authors and readers.) Brunger’s is one of a score or so of research papers selected for eLife’s first issue, which published today.

Photo by mikebaird

Back in 2001, scientist Roger Pedersen, PhD, famously left the United States over concerns about an unfriendly climate for stem cell research in the country. Pedersen has now returned to the Bay Area for a one-year sabbatical at Stanford’s Institute for Stem Cell Biology and Regenerative Medicine, and an Inside Stanford Medicine story today describes the circumstances surrounding his relocation to England’s University of Cambridge and details what he’ll be working on here.

Previously: After the lawsuit, what’s next for stem cell research?, Stem cell ruling throws Stanford researcher’s project into limbo and Stem cell guidelines under fire

As we all head to the polls today, this Scientific American piece on the presidential candidates’ views on science may be of interest. President Barack Obama and Governor Mitt Romney were asked over the summer to share their thoughts on 14 science- and technology-related topics, including food safety and vaccinations, and the publication’s editors rated the candidates’ responses in the piece.

Previously: Hey, president-to-be: What are your views on science?

A U.S. appeals court has affirmed (.pdf) the ruling last year by district court judge Royce Lamberth that federal funding of human embryonic stem cell research is legal. Although the decision effectively puts to rest opponents’ arguments that such funding violates the Dickey-Wicker amendment, at least one judge asked Congress for clarification about the power of the federal government to regulate such research.

Bioethicist Christopher Scott, who directs Stanford’s Program on Stem Cells in Society, has this to say about today’s ruling:

This is a major victory for stem cell researchers. It affirms that the National Institutes of Health’s interpretation of the 1996 Dickey-Wicker amendment is reasonable. Importantly, it removes a barrier of uncertainty for stem cell scientists and the patients who stand to benefit from their discoveries. The Obama administration should be congratulated for its assiduous work to guarantee that the president’s policy makes the difference when the rubber meets the road: funding research that could lead to the next generation of treatments and cures.

Scott has previously published research showing that limiting human embryonic stem cell research is likely to also slow research on induced pluripotent stem cells-an alternate way to create stem cells that does not require the destruction of human embryos-because the two fields are so closely intertwined.

Science writer Maggie Fox, writing for NBC news, has posted a nice summary of the issues leading up to today’s decision. But I found reading the actual text (.pdf) of the ruling to be very enlightening. In particular, Judge Janice Rogers Brown (one of three judges who ruled on the case) commented about the Dickey-Wicker amendment:

The challenging—and constantly evolving—issues presented by bioethics are critical and complex. Striking the right balance is not easy and not, in the first instance, a task for judges. What must be defended is “the integrity of science, the legitimacy of government, and the continuing vitality” of concepts like human dignity. Given the weighty interests at stake in this encounter between science and ethics, relying on an increasingly Delphic, decade-old single paragraph rider on an appropriations bill hardly seems adequate.

Previously After the lawsuit, what’s next for stem cell research, Judge Lamberth dismisses stem cell lawsuit and Embryonic stem cell regulation will affect iPS cell research

Compact fluorescent bulbs (often referred to as CFLs) burn less energy per unit of emitted light. That’s something pretty much everbody can agree on. But that’s about it.

As some readers may know, the U.S. government effectively banned the production of incandescent bulbs going forward by mandating energy-consumption standards no incandescent bulb is likely to meet. At least for the short term, this has tilted the playing field in favor of CFLs.

You either love the temperamental twisted tubes, presumably because they’re energy efficient, or hate them for any of a number of reasons. First, they’re expensive. Worse, because they contain mercury, CFLs have to be disposed of carefully - and should you, heaven forbid, drop one and have it smash into smithereens on the floor, the Environmental Protection Agency says you are supposed to open the windows immediately and clear the room.

I’ve got one in my kitchen. When I turn it on, it takes a while to warm up - and when it finally does, the light it gives off is, well, dirty. It’s depressing. I hate it.

Now, it turns out, there could be a medical reason to hate CFLs too: A recent study in the journal Photochemistry and Photobiology by researchers at the State University of New York, Stony Brook, indicates that, despite advocates’ claims to the contrary, these bulbs give off significant amounts of ultraviolet light, namely in the UVA and UVC range. The SUNY investigators explain that stresses introduced in the bulbs’ X-ray-absorbing coatings during the tubes’ manufacture cause minute cracks or lapses in those coatings:

Closer examination of some of these commercially available bulbs showed multiple defects in their coating, thus allowing UV-light emission. . . . These data are particularly disturbing as the UVC emission is even larger than ambient sunlight on a mountain.

To see if these emissions were physiologically harmful to human skin, the researchers used various biological assays on two kinds of human skin cells: keratinocytes and dermal fibroblasts. For example, the cells were exposed to CFLs so that they got the same UV doses a person’s skin would get from the bulbs after 45 hours at the typical working distance from a desk lamp. The damage to the cells was noticeable and was similar to the kind that occurs in the aging process. (It took only five hours at that distance from a CFL to get UV exposure exceeding recognized safety standards, the scientists noted.)

Ironically, adding either of two forms of titanium oxide (the chief UV-absorbing component of commercial sunscreens) to the skin cells made the damage that CFLs inflicted on them even worse.

Photo by Nioxxe

Peter Aldhous from New Scientist reports today that the U.S. District Court in Washington, DC, has ruled that a person’s own cultured stem cells are drugs subject to regulation by the Food and Drug Administration.

This is a big deal, as it’s the cornerstone of an ongoing argument between the agency and Colorado-based Regenerative Sciences (The FDA Law Blog summarized the legal tussles nicely last October). It’s also germane to the issues surrounding Texas-based Celltex, which I’ve blogged about before.

According to Aldhous:

It’s official: stem cells are drugs. At least, that’s the opinion of the [court]… which has ruled that the Food and Drug Administration (FDA) has the authority to regulate clinics offering controversial stem cell therapies.

Treatments in which stem cells are harvested from bone marrow and injected straight back into the same patient are deemed part of routine medical practice - not regulated by the US government. But if the cells are subjected to more than “minimal manipulation”, the FDA maintains that the therapy becomes a “drug”, which must be specifically approved for use.

Aldhous also quotes Regenerative Sciences’ medical director Christopher Centeno, MD, vowing to appeal the ruling, as well as Stanford’s own Christopher Scott:

“I think it’s a good ruling, and I’m glad to see that that the FDA has exercised its muscle on the case,” says Christopher Scott, who heads the Program on Stem Cells in Society at Stanford University in California.

Scott hopes that the FDA will now step up its efforts to regulate other clinics offering unproven stem cell therapies. These include Celltex of Sugar Land, Texas, which rose to prominence after Texas governor Rick Perry was injected with stem cells supplied by the company to aid his recovery from back surgery.

Previously: FDA audit of Texas stem cell clinic revealed by Houston Chronicle and Stanford’s Irving Weissman on the (lost?) promise of stem cells
Photo by sideonecincy