Science Policy

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

Last week, the European Commission pledged to offer free access to publicly funded scientific research and set a goal of making 60 percent of studies and papers produced with taxpayer dollars available by 2016.

In this recently posted video, Neelie Kroes, European Commission Vice-President for the Digital Agenda, talks with three prominent European scientists about how granting open access to research benefits not only science and innovation but also public health and the economy. Kroes shares her full perspective on the issue on her blog.

The European Commission’s announcement follows news from the United Kingdom that the British government will grant free public access to government-funded scientific research by 2014. A campaign to adopt an open access science policy has also been gaining momentum in the United States; a recent petition urging President Obama to implement open access policies for federal agencies that fund scientific research exceeded the number of required signatures. Signees are now awaiting a response from the Obama Administration.

Previously: How open access publishing benefits patients, PeerJ open access publishing platform launches today and A guide to transitioning scholarly journals to an open access model
Via PLoS blog

This is very cool: In an effort to ensure continued dialogue on important science issues this election year, Scientific American will be asking the two presidential candidates their views on 14 science- and technology-related topics. The answers will be published in the November issue of the magazine.

Christine Gorman talks about the plan and provides the questions over on the Observations blog today; several jumped out as me as being particularly relevant to Scope readers:

• Pandemics and Biosecurity. Recent experiments show how Avian flu may become transmissible among mammals. In an era of constant and rapid international travel, what steps should the United States take to protect our population from emerging diseases, global pandemics and/or deliberate biological attacks?
• Food. Thanks to science and technology, the United States has the world’s most productive and diverse agricultural sector, yet many Americans are increasingly concerned about the health and safety of our food.  The use of hormones, antibiotics and pesticides, as well as animal diseases and even terrorism pose risks.  What steps would you take to ensure the health, safety and productivity of America’s food supply?
• Science in Public Policy. We live in an era when science and technology affect every aspect of life and society, and so must be included in well-informed public policy decisions.  How will you ensure that policy and regulatory decisions are fully informed by the best available scientific and technical information, and that the public is able to evaluate the basis of these policy decisions?
• Vaccination and Public Health. Vaccination campaigns against preventable diseases such as measles, polio and whooping cough depend on widespread participation to be effective, but in some communities vaccination rates have fallen off sharply. What actions would you support to enforce vaccinations in the interest of public health, and in what circumstances should exemptions be allowed?

Following my colleague Marissa Fessenden’s entry on the difficulty – and importance – of publishing results from duplicate studies that contradict previous findings, there’s some more news on biases in published research. The current issue of the NIH Record newsletter reports on a recent NIH-sponsored talk that John Ioannidis, MD, DSc, gave on the topic. Among his concerns:

Ioannidis said… editors want to publish research that is novel and will have a large impact on the field, which generally means papers that report very large, statistically significant effects.

At the seminar, Ioannidis outlined other instances of bias and made nine recommendations to improve biomedical research by overcoming these. Registering clinical trials, even if the results are never published, is “one of the best ideas ever to appear in clinical research,” Ioannidis said, and it will ensure the important data won’t get “lost.”

The article concludes:

…Under the weight of the great promises they make, investigators often abandon studies whose results seem uninteresting or selectively report only statistically significant portions of their results.

“We need to move away from the requirement to make big promises,” [Ioannidis] said. “Very little of what we do will be so lucky as to break new ground.” He suggested that instead of funding specific research projects, NIH and other funding bodies should support individual researchers with a track record of excellence. “Maybe we should promise instead just to do our best,” he said.

Previously: A critical look at the difficulty of publishing “negative” results, Testing medical ‘truths’ and “Omics” studies need validation, says Stanford’s Ioannidis
Via HealthNewsReview.org

In case you missed it, the U.K. government has announced its plans to make publicly funded research freely available online. A portal called Gateway To Research, which will provide links to published studies and related data, is currently being developed.

paidContent’s Robert Andrews reports today:

The news will prove unpopular with academic publishers, which license and peer-review researchers’ work and charge libraries to make it available.

“As taxpayers put their money towards intellectual enquiry, they cannot be barred from then accessing it,” science minister David Willetts said in a speech to the Publishers Association on Wednesday (transcript).

“They should not be kept outside with their noses pressed to the window – whilst, inside, the academic community produces research in an exclusive space.”

The Guardian also ran an interesting piece earlier this week on Wikipedia co-founder Jimmy Wales’ role in the government’s endeavor.

This (moving) video, featuring edited narrative elements from Neil deGrasse Tyson, isn’t explicitly about medicine. But the point it makes is as true for biomedicine as it is for space: Public support for the sciences dares us to dream about the future and empowers us to invent it.

Take five minutes and watch the video. It’s worth your time.

Via Devour

Over at the Nature News blog, there’s a great summary of the ways that last night’s State of the Union address by President Obama tackled issues near to scientists’ hearts. Several of these points are particularly relevant for medical researchers. For instance, the post says:

- Obama urged lawmakers not to “gut” US investments in basic research, arguing among other things that “the discoveries taking place in our federally-financed labs and universities could lead to new treatments that kill cancer cells but leave healthy ones untouched” and create lightweight vests that protect police and soldiers from “any bullet.”

- Calling on Congress to “stop expelling responsible young people who want to staff our labs,” the president argued that a path to citizenship should be created for foreign students who come here to study subjects like business, science and engineering. “Send me a law that gives them the chance to earn their citizenship. I will sign it right away.”

The entire post – which also addresses such areas of science as green technology and high-tech manufacturing – is a worthwhile read. A video of the president’s speech is available here in case you missed it.

Previously: Obama reaffirms commitment to health reform – but does Congress care?
Photo by The White House

I guess you could say that the Stanford Biodesign Program “wrote the book” on how to teach medical technology innovation to multidisciplinary teams. The U.S. Food and Drug Administration recently acknowledged the effectiveness of this training program by signing a “memorandum of understanding” with Stanford, which I discuss in today’s Inside Stanford Medicine.

The agreement lays the groundwork for the FDA and Stanford to collaborate on a number of initiatives, including educational outreach, cross-training of scientific personnel, and the development of new biostatistical methods for more accurately evaluating the safety of emerging medical technologies.

The biodesign program, which is in its 11th year, trains teams of doctors, engineers and business students — in an intensive one-year program — to identify a medical need, develop an invention to fill it, create a business plan, navigate the regulatory process, then present their inventions to venture capitalists. Since its inception, the program has led to more than 200 patents and 24 start-up companies.

Previously: Stanford Biodesign Program releases video series on the FDA system, FDA walks line between innovation and safety, Stanford physician-entrepreneur discusses need to change FDA approval process, and New biomedical device textbook gets early praise

A few years ago, Tom Rando, MD, PhD, found that if the circulatory systems of a young and an old mouse were connected, something in the blood of the young mouse seemed to rejuvenate the old mouse’s liver and muscle. And something in the old mouse’s blood seemed to age the young mouse’s equivalent organs and tissues. Rando taught Tony Wyss-Coray, PhD, the mouse-hookup technique, and last year the latter showed that factors in blood can similarly influence the robustness of stem cells in the brain, too. (This was the subject of a recent Stanford Medicine article.)

Meanwhile, Howard Chang, MD, PhD, and one or two other investigators in the research-osphere were showing the world that a whole lot of the DNA in each of our cells codes for not proteins but RNA molecules whose job it is to fire up or shut down the genes that do code for protein production. (For more, see this.)

In a just-out review article in the journal Cell, Rando and Chang raise the possibility of getting old cells to act younger. And not just any old cells, but the “adult” stem cells that reside in most if not all of our various tissues.

Most of us are more familiar with these cells’ more publicized relatives, embryonic stem cells (eSCs). These superstars are famous for being able to perform two tricks. They can multiply indefinitely in a dish, and they can differentiate into any of the 200-odd cell types in our body. That’s good and it’s bad. Good, because they offer the promise of regenerative medicine: essentially, differentiating eSCs into the needed cell types and infusing them to rejuvenate worn-out or defective tissue. Bad, because on their way to becoming the right kind of cells, they could go wrong instead, and become tumors.

Unlike eSCs, tissue-resident cells already know what they want to be when they grow up: They want to be whatever type of cell within that particular tissue that happens to be in short supply. In other words, these adult stem cells are already committed to a given lineage. A nerve stem cell’s not going to surprise you by turning into a fat stem cell.

Rando and Chang’s review, and this Q-and-A session I conducted with Rando the other day, explore the prospects for restoring aging tissues’ tiring stem cells to more-youthful activity levels. It might be possible to kickstart them with bloodborne factors isolated through experiments of the type I mentioned above – or, better, with pharmaceutical compounds that mimic the actions of those factors.

I feel younger already.

Previously:Old blood + young brain = old brain, Old blood makes young brains act older, and vice versa, New job description for RNA, oldest professional biomolecule
Photo by jikatu

In a fascinating Science Progress blog entry, Jonathan Moreno takes a stock of how America is performing in terms of its investment in the life sciences. He writes:

So how is the country doing in biotech investment? A fascinating and richly detailed new industry report from Jones Lang Lasalle allows us to reach two salient conclusions: first, the United States is holding its own as the global leader; and second, since the 2007 downturn, industry clusters in China, India, and Singapore have displaced traditional powers Ireland, Italy, Germany, and Spain in direct pharmaceutical investment.

This result confirms the narrative we hear in so many fields these days about the turn toward Asia, and well justifies the Obama administration’s reassertion of American interests as a Pacific power.

As you saw in the block quote, Moreno’s entry summarizes a report (.pdf) from Jones Lang Lasalle, a financial and investment services firm. It also identifies several “clusters” in which, with the right funding and policies, biotechnology might begin to flourish.