U.S. Government Not Keen On Germ-Line Editing, At Least Right Now

Yesterday John Holdren, Director of the Office of Science and Technology Policy (OSTP), posted to the OSTP blog about recent activities in genome editing.  While the Chinese effort to conduct genome editing in human embryos attracted headlines and an announcement from the National Institutes of Health stating it would not fund similar research, it was not the specific focus of the statement.

That was reserved for the recent announcement that the National Academy of Sciences and the National Academy of Medicine (the new name of the Institute of Medicine) launched an initiative to address these technologies.  The initiative will include an international meeting this fall, along with a committee to study gene-editing technologies and an advisory board to guide the effort.  The project will include, technical, social, legal and ethical issues related to these technologies.

Dr. Holdren’s blog post and the Academies’ announcement noted the historical precedents for this kind of a priori assessment, including the restrictions imposed on recombinant DNA research, gain-of-function research, stem cell research and human cloning while researchers and policymakers discussed the implications of that work.

While not going so far as to echo calls for a moratorium on germline genetic editing, Dr. Holdren’s blog post can be read to imply Administration support for such a move.

“The White House applauds NAS and NAM for convening this dialogue and fully supports a robust review of the ethical issues associated with using gene-editing technology to alter the human germline. The Administration believes that altering the human germline for clinical purposes is a line that should not be crossed at this time.”

Given the nature of how the National Academies typically prepare their study committees, the international meeting this fall will more likely be the start of its process rather than a culmination.  I would also not be surprised to see the Presidential Commission for the Study of Bioethical Issues engage with these technologies, either through its own initiative or a request from the President.

Not All Scientists Are Opposed To GMO Labeling

ScienceInsider reported yesterday that scientists in Germany are calling for labeling of genetically modified organisms (GMO).  The petition (which should be online any time now) goes beyond labeling for GMOs in food, to include such organisms in feed, drugs, textiles, chemicals, and other products that have been produced using genetic engineering.  Should the petition receive enough signatures by a certain time, the German Bundestag would have to consider the proposal.

I remain skeptical that the no-label position regarding GMO’s is the right move, so I welcome this petition effort.  Opposing labeling makes it look like there’s something to hide, which feeds into GMO opponents’ argument that the development and use of GMO’s has been deceptive in some fashion.  It also strikes me as anti-democratic and anti-transparency.  And while those might not be value positions linked to science, they are important values in policy decisions (the current debates over the Trans-Pacific Partnership trade agreement suffer from a similar challenge since the text of the agreement is not widely available).

Arguably the pro-GMO side has won, given the prevalence of these organisms in many items.  But the effort to prevent labeling has the potential to snatch defeat from the jaws of victory.  Maybe the German scientists are onto something.

NIH Reiterates Opposition To Funding Gene Editing For Human Embryos

In light of Chinese researchers reporting their efforts to edit the genes of ‘non-viable’ human embryos, the National Institutes of Health (NIH) Director Francis Collins issued a statement (H/T Carl Zimmer).

(For what it’s worth, the research indicated a very low success rate in editing the gene.)

The statement mentions the various legal and regulatory prohibitions on funding the kind of research the Chinese conducted.  In this case, the editing was of a gene responsible for a particular blood disorder.  But the changes to the gene would be heritable by the descendants (if the embryos in question were viable), and that is the source of concern.

From the Director’s statement (CRISPR/Cas9 is the editing technique in question):

“NIH will not fund any use of gene-editing technologies in human embryos. The concept of altering the human germline in embryos for clinical purposes has been debated over many years from many different perspectives, and has been viewed almost universally as a line that should not be crossed. Advances in technology have given us an elegant new way of carrying out genome editing, but the strong arguments against engaging in this activity remain. These include the serious and unquantifiable safety issues, ethical issues presented by altering the germline in a way that affects the next generation without their consent, and a current lack of compelling medical applications justifying the use of CRISPR/Cas9 in embryos.”

While Collins also notes the federal laws and regulations that restrict funding, I do not expect the statement to be the end of the discussion around the gene-editing research reported on in China (which is probably continuing).  I suspect many would find the use of non-viable embryos in this research acceptable, even if it punts on the questions of consent to changes for future generations and the safety of the techniques on viable embryos.  After all, stem cell research lines have been derived from non-viable embryos.  I think that the need to (eventually) work with these technologies on viable human embryos makes the stem cell comparison problematic, but that won’t likely matter in the policy debates to come.

 

Dr. Varmus To Step Away From National Cancer Institute

Ending what is arguably the science policy equivalent of Presidents Adams or Johnson serving in Congress following their time in the White House, Dr. Harold Varmus announced he will step down as chief of the National Cancer Institute (NCI) effective March 31st.  Varmus was head of the entire National Institutes of Health between 1993 and 1999, meaning he was a predecessor of his current boss, Dr. Francis Collins.  Both of these stints in public service came after Varmus was recognized as a 1989 Nobel Laureate in Physiology or Medicine

Varmus highlighted the National Cancer Institute accomplishments during his tenure in a letter to NCI staff.  They include two new centers, improvements in its clinical trials program, innovations in research funding and significant changes to the NCI’s grant process.

Varmus will now live full time in New York City (he was commuting to the NCI facility in Bethesda, Maryland), conducting cancer research in his own lab at the Meyer Cancer Center at the Weill-Cornell Medical Center.  He is likely to apply for funding for the cancer components of the Precision Medicine Initiative.  He will also advise the newly formed New York Genome Center.

The current NCI Deputy Director, Doug Lowy, will serve as Acting Director once Varmus departs.  With less than two years remaining in the current administration, it is possible that Lowy will remain in the position until at least the next President is sworn in.

Appointment Heading For The Exits

While the confirmation challenges continue, at least one high-level science and technology appointee is stepping down.  Margaret Hamburg, Commissioner of the Food and Drug Administration, will step down at the end of next month (H/T ScienceInsider).  Even with slightly less than two years remaining in the Obama Administration, there is a chance Dr. Hamburg’s successor may not be confirmed before a new President is sworn in.  (I am encouraged by indications that a nominee has already been identified.)

Hamburg’s tenure is one of the longest in the agency’s history.  When she took over, the agency was struggling, with ethical challenges over drug approvals.  The agency is currently in the middle of regulatory changes for food safety oversight and medical device approvals, and it has not been perfect.  There were complaints over the agency’s inaction over compounding pharmacies (which are not presently regulated by the FDA), and Hamburg was overruled by Health and Human Services Secretary Sebelius over Plan B emergency contraception (though the courts eventually sided with the FDA).

One Thread Of Post Myriad Activity Seems To Be Ending

Since the 2013 decision in the Myriad case, which invalidated some patent claims on isolated sequences of two genes relevant in some cases of breast cancer, there have been two major actions taking place.  Following the decision the Patent and Trademark Office (USPTO) has been working on guidance for potential patent applicants working with products of nature.  The latest guidance memo was released in December (H/T ScienceInsider), and the USPTO is taking comments until March 15.  There has been a back and forth with the biomedical research community, which has argued that the initial guidance (from March 2014) went further than the Supreme Court intended.  The USPTO has made changes in response.

The other post-Supreme Court thrust has involved lawsuits by Myriad Genetics against companies developing their own genetic tests for the breast cancer genes where Myriad held patent claims.  The company was protecting its market share and investment through the lawsuits and the settlement terms it has been able to reach..  ScienceInsider is reporting that many of the remaining cases have settled and The New York Times has noted that Myriad is working to address the remaining cases.

So, What Is Precision Medicine, Exactly?

Amidst the laundry list that is the State of the Union was the Precision Medicine Initiative.  In short, it’s a program geared toward expanding treatments crafted to address patients that don’t respond to treatments targeted to an ‘average patient.’ The initiative was announced with Bill Elder in the audience.  Elder benefited from a ‘precision’ treatment for his cystic fibrosis.  The cause of his disease is shared by only 4 percent of cystic fibrosis patients.

Details at the moment are scarce.  The Federal budget may give additional details once it’s released next month.  The program would likely be led by the National Institutes of Health, though the focus of the Initiative would not necessarily be restricted to medicines, wider use of DNA testing to help craft cures or similar clinical treatments.  Refinements in medical imaging can be helpful in this area and health information technology may also be leveraged to develop new cures that can assist those not helped by treatments geared toward the ‘average’ patient.

Setting aside the usual concerns about getting approval for new initiatives in a tight budgetary environment, it remains to be seen if this program can successfully be sold to the general public.  If the focus is on treatments focusing on the margins, it could be hard to sell the benefits to a broader public.  Of course, if there are members of Congress with direct experience benefiting from targeted treatment, then the acceptance of this program might be easier to get.  Unlike the BRAIN Initiative, focused on better understanding of something we all have, the Precision Medicine Initiative runs the risk of being a niche program, not likely to get the large investments it might need.  If it follows the organization of the BRAIN Initiative, including several agencies and a few public-private partnerships, the total investment might be spread around sufficiently to mitigate the political challenges of getting lots of money.