Nominations Corner – Health And Human Services

The recent resignation of Secretary of Health and Human Services Kathleen Sebelius prompts a revisit of the slow march from nominee to confirmation.  While the President acted quickly to nominate Sylvia Burwell (current Director of the Office of Management and Budget) to replace Sebelius, the continuing paralysis of the Senate may mean it will be several months before she takes the job.

Normally the Secretary is, oddly enough, distanced from many of the science and technology functions the Department deals with.  But with the National Institutes of Health, Food and Drug Administration (FDA), Public Health Service (headed by the Surgeon General), the Presidential Commission for the Study of Biomedical Issues, and various research programs connected to Medicare and similar health programs, it would be tough for a Secretary to be completely detached from such actions.  In the case of Secretary Sebelius, perhaps her most controversial science and technology action is her decision to overrule the FDA in connection with the availability of emergency contraception.  While I doubt it will come up during the confirmation hearings, I think there will be an attempt to revisit the decision with a new person in charge of the Department.

(While we’re on the subject of Health and Human Services nominations, it’s worth noting that the latest attempt to nominate a Surgeon General has been blocked due – at least in part – on the refusal of some Senators to accept the nominee’s opinion that gun violence is a public health issue.)

About Those Three-Parent Headlines

A Food and Drug Administration advisory panel provided some filler for the 24-hour news channels this week.  The reason: discussion of mitochondrial replacement as a means of in-vitro fertilization (IVF).  It involves a ‘donor’ cell which has its nucleus removed to receive the nucleus of the mother (it bears some resemblance to somatic-cell nuclear transfer, except there is an additional DNA source involved).  This method could be used for situations where the mother has mitochondrial defects that could be passed on to the offspring.

The method gets the superficial cable news attention because the resulting offspring would have DNA from all three donors.  While the Presidential Commission for the study of Bioethical Issues has not weighed in (it doesn’t meet again until June), the FDA panel discussed the state of science and research on the technique, as well as the design requirements for early-phase clinical trials.  Researchers have produced monkeys via this IVF technique, but panel members were reluctant to recommend human trials at this time.

Over on the other side of the Atlantic, the U.K. is further along in regulating the technique.  The appropriate advisory bodies started assessing mitochondrial replacement in 2011, and the government announced last year that it was working on regulations.  It issued a consultation on Thursday for the draft regulations (questions of interest are on pages 27-28).  Responses will be accepted until May 21.

Antidepressants and Brewers Yeast – Evolutionary Pharmacology

Baba Brinkman is back with another science rap.  The new one is titled Evolutionary Pharmacology.

As you might have gathered, this was done in connection with the lab of Ethan Perlstein.  In fact, the video is taken from on this earlier production that Perlstein put together to promote his independent lab (crowdfunded via Angelist).  Perlstein and his colleagues are working on orphan drug discovery and research related to those diseases.  The evolutionary pharmacology comes in because they are studying drug effects on yeast cells in order to better understand how well the drugs do (and/or do not) affect humans.

Either way, like the man says, don’t give Zoloft to infants.

Whether Stem Cells Can Be Patentable May Be The Least Of Commercial Challenges

Two recent actions involving the Food and Drug Administration (FDA) suggest to me that the matter of whether stem cell patents are valid may not be so critical.

The D.C. Circuit Court of Appeals ruled last week that the FDA has jurisdiction over stem cells cultured for therapeutic use (H/T The Scientist).  This decision upheld a lower court ruling that considered the act of culturing the cells more than ‘minimal manipulation,’ and therefore subject to FDA drug oversight regulations.

(IANAL, but I think this decision could be used to strengthen the case that stem cell patents – at least for cultures of said cells – would be valid.  After all, if there was more than minimal manipulation, wouldn’t that be sufficiently transformative to make the cultures no longer products of nature?  Again – I Am Not A Lawyer.)

Aside from the legal matters, there appears to be a big regulatory mismatch that will hinder commercialization of stem cell treatments.  In the February 6 edition of Cell Stem Cell researchers note (H/T The Scientist) that differing regulations between the National Institutes of Health (NIH) and the FDA may reduce the number of stem cell lines that could be used in clinical practice.

FDA regulations require that stem cell donors be screened for various diseases (so that treatments derived from those cells cannot infect others).  NIH regulations – not focused on commercial applications of stem cell research – do not have this requirement.  Now it is possible, as the article from The Scientist notes, for the FDA to allow some treatments to be approved without such a screening, but some alternative measure will likely be needed to mitigate the risk of infection.

Lawsuits ‘R’ Us: 23andMe & Goldieblox

Two technology-oriented lawsuits of note for your weekend reading:

Besides the issues the Food and Drug Administration has with genetic sequencing firm 23andMe, the company has a lawsuit on its hands.  It’s a class action lawsuit filed in federal court.  Much like the FDA’s concerns, the class action focuses on how 23andMe represents the health screening tests it provide(d).  What I missed during the initial reporting is that there are two aspects to how the company markets its health screenings, both of which are suspect in the absence of rigorous proof of the validity of its tests.  Not only would consumers rely on the results for assessing their genetic health, but researchers using the company’s health databases depend on the reliability of that data.  If the company could not satisfy the FDA on the reliability of its tests, how can its customers depend on its data?

Apparently a letter of apology wasn’t enough to get GoldieBlox off the hook for how it used a Beastie Boys tune in a promotional video.  That GoldieBlox pre-emptively sued to establish a right to fair use certainly didn’t help.  The band has countersued the company, alleging systematic infringement of intellectual property – and not just their own.  In their Answer, the band asserts the following (page 7): Continue reading

New Survey Provides Snapshot On DIY Biology

The Woodrow Wilson International Center for Scholars recently conducted a survey as part of a report on the growing do-it-yourself (DIY) Biology movement.  The newly released report (H/T ScienceInsider) from the Synthetic Biology Project is apparently the first of its kind to track what activities the community is involved with.

The report authors are interested in countering existing stories about the DIYBio community that don’t match with what their research (and survey data) have demonstrated.  The brief takeaway, from their perspective – the threat posed by this research (and these researchers) has been overstated in the press.

While the authors are careful to note that their work is a current snapshot of the field, I am concerned that the press connected to this report may oversimplify what’s going on.  In other words, the new stories will be about how the old stories oversold the magnitude of what is going on in the field and the possible threats of what could be taking place.

I think the most productive recommendations from the report will be those focused on how to grow, support and manage research in this area moving forward.  As a DIY community is not necessarily connected to existing institutions, having the capacity to educate interested researchers and provide them spaces to work is not guaranteed.  Such resources could also ease the burden of monitoring and guiding the research moving forward.  A previous report from the Synthetic Biology Project suggests to me that at least some community self-regulation would be useful in the future, as federal action is coming slowly.

Either way, this survey needs to be the first of several, and not the end of a discussion.

Another Year, Another Genetics Test Patent Challenged In Court

Not that it was really expected, but recent action in the courts suggests that the Myriad Genetics case has not settled the legal standing of patents on testing of genetic sequences.

The new matter involves two companies in a lawsuit over the patenting of a genetic test for Down Syndrome (H/T ScienceInsider).  On the 30th, a Federal judge in California invalidated the patent licensed by Sequenom since 2011.  The test Sequenom developed and patented is new and different, utilizing a different technique and demonstrating marked increases in efficiency compared to other methods.  They were sued by Ariosa Diagnostics for a declaration that a test Ariosa developed did not infringe on Sequenom’s patent.

The judge’s order, which granted Ariosa’s motion for summary judgment (and rejected Sequenom’s motion), focuses on whether various claims of the underlying patent address eligible subject matter.  Specifically, whether or not patenting the uses of paternally-derived cell-free fetal DNA in the test constitutes the patenting of something novel and therefore patent-worthy.  The judge was persuaded that the test constituted the application of conventional techniques to a new natural phenomenon, and did not pass the patent-worthy threshold.  Relying on the decisions in both the Myriad case and the other recent medical test patenting case, Mayo v. Prometheus, the court found that the only inventive part of the patent was the use of cell-free fetal DNA, and thus not patentable.

This case is only at the District Court level, and Sequenom has stated it will appeal the decision.  Ariosa is pleased with the decision, and given the tenor of its press release, would likely meet Sequenom’s ‘vigorous’ defense with vigor of its own.  A visit to the Supreme Court in 2016 or so is plausible.

FDA Takes Additional Steps To Improve Drug Supply Monitoring

I’ve been posting irregularly about shortages in the production of drugs and other critical elements.  But I’ve barely scratched the surface of the drug shortages.  The Food and Drug Administration (FDA) is starting to take steps required by a recent federal law to try and get a better handling on drug shortages as they appear (H/T Scientific American).

In Monday’s Federal Register there is a notice of a proposed rulemaking and request for comments from the FDA on changes in reporting on drug shortages.  Prompted by the 2012 Food and Drug Administration Safety and Innovation Act, companies will now be required to report six months in advance of discontinuing production or a shortage of production of certain drugs.  The FDA will also maintain a list of drugs under shortage conditions.

Much of these actions are putting into law and regulation guidance advanced in a 2011 Executive Order.  The rule would require a minimum of 6 months notice, with rare exception.  Failure to provide notice would prompt a public noncompliance letter, and the rule would give specifics both to the letter and the notice that companies must provide to the FDA.

The agency is persuaded that better information exchange between manufacturers and the FDA could further mitigate shortages.  From the notice:

“Working closely with manufacturers and other stakeholders, FDA was able to help prevent just under 200 drug and biological product shortages in 2011 and more than 280 such shortages in 2012, using tools such as:

“Working with manufacturers to resolve manufacturing and quality issues contributing to short supply.

  • Expediting FDA inspections and reviews of submissions from manufacturers to prevent and/or alleviate shortages.
  • Identifying and working with manufacturers willing to initiate or increase production to cover expected gaps in supply.
  • Exercising enforcement discretion in appropriate circumstances, if this would not cause undue risk to patients.”

An important change is that the proposed rule would require notice for marketers of certain compounds regardless of whether they hold an approved application from the FDA.  IANAL, but I wonder if this might be a means of exerting a bit more control over compounding pharmacies, which are not presently under FDA regulatory control.  (It is arguably a big stretch to suggest it might allow control of these pharmacies over drugs made for execution purposes.)

Comments will be open for roughly 60 days, closing on January 4, 2014.

Do You, For One, Welcome These New Cyborg Cockroach Minions?

Apologies to The Simpsons for the tortured paraphrasing.

This drew my attention in a list of most clicked stories over at ScienceInsider:

Cyborg Cockroach Sparks Ethics Debate

The article mentions the appearance of a RobotRoach at a recent TEDx conference in Detroit.  Here’s the video from a previous TED talk:

If you would like a cyborg cockroach of your very own, you can order a kit from Backyard Brains, co-founded by Greg Gage and Tim Marzullo.  You can purchase many experiments from the company, which focus on neuroscience and engineering.

And that’s great.  But converting the cockroaches into cyborgs gives me pause.  The company is mindful of this, providing some explanation of how it handles these concerns in the experiments it supports in schools that use live animals (crickets, roaches and worms).

However, since that page was posted Backyard Brains has introduced a home kit for the RoboRoach, starting next month you can control a cockroach with your phone.  But not without surgery.

Continue reading

Agricultural Innovation Prize New Drop In Mostly Empty Bucket

The University of Wisconsin, the U.S. Department of Agriculture, and 40 Chances are working together on the 2014 Agricultural Innovation Prize (H/T ScienceInsider).  The prize was prompted by the recommendations from the 2012 report on the agricultural research enterprise from the President’s Council of Advisers on Science and Technology.  Those recommendations included encouraging further collaborations between students and agricultural businesses across disciplines.

The competition is geared around student teams, and their universities (or businesses they create) could receive the prizes, which total over $200,000.  Entries are due February 28.  They start with a business plan for a process, product, service or technology that address one or more challenges in the food system.  The ultimate goal of the entries would be to “address social and agricultural challenges within food systems to improve the standard of living and quality of life for the world’s population.”

While a first read of the prize website comes across to me as a bit too business-oriented, I don’t think entries that are oriented more towards the public sector or are arranged around alternative business models (such as open source) would be dismissed out of hand.  I think some of this uncertainty may be resolved as judges are announced.

The prize organizers are also offering funds to help support interested student teams in learning more about agricultural innovation to help them develop their entries.

As Science magazine noted in a rare exploration of agricultural science policy, the nation’s agricultural research capacity needs rebuilding.  This prize can help, but it’s one small measure that should be joined with several other efforts to grow and nurture practices and institutions that support agricultural research.