PCAST Meets To Take A Deep Dive

When I first posted about tomorrow’s meeting of the President’s Council of Advisors on Science and Technology (PCAST), it was without benefit of an agenda.  Now that I have seen it, my mildly informed speculation has been confirmed.

The meeting will start at 9:15 Eastern time tomorrow in Washington.  A webcast will be available, as usual.  Simply visit the PCAST meetings page tomorrow.  The morning starts with progress updates (and perhaps final approvals) on PCAST reports on the National Nanotechnology Initiative (NNI) and antibiotic resistance.  The NNI report is required every other year by law, so PCAST will be returning to somewhat familiar territory.

The presentation part of the meeting concludes with a panel on oceans policy.  As I guessed, Beth Kertulla, Director of the National Ocean Council, will be part of the panel.  She will be joined by other leaders in the ocean science research community: Robert Gagosian, President of the Consortium for Ocean Leadership and Anthony Knap, head of the Geochemical and Environmental Research Group at Texas A&M University.

As usual, there is time set aside for public comment.  The public session is scheduled to end by lunchtime.

NIH Translational Center Scores Drug Development Deal

One of the goals of the National Institutes of Health’s (NIH) National Center for the Advancement of Translational Sciences (NCATS) has been to facilitate the conversion of research output into clinical inputs (treatments, medicines, and other tools to help patients).  Today it announced its first success in this area where drug development is concerned (H/T ScienceInsider).

The development comes through the center’s Therapeutics for Rare and Neglected Diseases (TRND) program.  Resources in this program are to encourage collaborations between NIH researchers and outside researchers working on conditions that due to rarity or other circumstances don’t receive my attention through traditional drug development channels.  The drug at the heart of today’s announcement addresses the underlying molecular mechanism of sickle cell disease.  The company that collaborated with NCATS staff, AesRx, has been acquired by Baxter International.  Before working with TRND resources, AesRx was having difficulty obtaining private investment in early-stage development.

This is, of course, not the only translational research program NCATS supports.  It’s not even the first TRND program to be completed.  But it does appear to be the first to lead to commercial acquisition.  In an era where economic impacts of scientific research are given greater scrutiny (not necessarily with additional understanding), this is certainly a positive development.  It’s also a validation of the need to look at all aspects of the research process to facilitate innovation.  The ‘valley of death’ (the gap between initial development and commercialization) is not just a challenge in technology.

NIH Expands Its Mystery Diseases Operations

While Princeton-Plainsboro is a fictitious teaching hospital that focused on diagnosing rare diseases on House, M.D., there are people suffering from such ailments.  The National Institutes of Health (NIH) has been growing its Undiagnosed Diseases Program (UDP) since 2008, and it just named six centers that will do the same kind of work currently available only at the NIH Clinical Center in Bethesda.  The centers were decided after   The program has been effective, and the expansion should be able to expand both the number of patients served and the breadth of diseases studied.

The UDP is supported by the National Human Genome Research Institute (NHGRI), and has seen approximately 750 patients in the last six years.  (This 2012 journal article provides a more detailed understanding of how the program functions.)  Genetics are not the exclusive focus of the program, which also analyzes clinical and cellular mechanisms behind these rare diseases.  Beyond discovery of cures and better understanding of the underlying disease mechanisms, study of abnormal conditions like these diseases can help inform how affected systems function normally.

Joining the Clinical Center in what is now the Undiagnosed Diseases Network are the Harvard teaching hospitals, Duke, Vanderbilt, Baylor, Stanford and the University of California, Los Angeles.  Harvard Medical School will serve as coordinating center. The Network will share information from the UDP with several public repositories.

California Jumps On The BRAIN Wagon

Many of the scientists who proposed the national BRAIN Initiative (Brain Research Through Advancement of Innovative Neurotechnologies), and several members of the Advisory Committee to the Director of the National Institutes of Health on the BRAIN program are at California institutions.  California has also established, in part due to a voter initiative, a state institute to support research in stem cells and other regenerative therapies.

So it’s news, but perhaps not surprising, that California has officially started Cal-BRAIN (California Blueprint for Research to Advance Innovations in Neuroscience) (H/T Nature News).  Required under a law passed earlier this year, Cal-BRAIN is a project of the University of California system, with the University of California, San Diego in charge.  The state set aside $2 million for the effort, which is to establish a blueprint for university and industry participation in the program.  The law specifically states that a goal is to have an industrial investment matching the state investment in Cal-BRAIN.  It also requires a technology transfer program and web presence for Cal-BRAIN.  The University of California has yet to determine how much it will invest in the project, but that should be announced soon.

Court Takes The Procedural Way Out Of Ruling On Stem Cell Patent

Consumer Watchdog will apparently need to get into stem cell research in order to get Courts to act on the validity of stem cell patents.  The U.S. Court of Appeals for the Federal Circuit has dismissed Consumer Watchdog’s challenge to a stem cell patent granted in 2007 to the Wisconsin Alumni Research Foundation (WARF).  The court did not rule on the merits of the challenge, but dismissed it based on Consumer Watchdog not being involved in the work on human embryonic stem cells.  As they weren’t directly involved or affected by stem cell research, the court decided that Consumer Watchdog lacked standing to challenge the patent.  The U.S. Patent and Trademark Office (USPTO) made that argument in filings earlier this year.

Consumer Watchdog filed the case because it felt that aggressive defense of the patent by WARF could effectively pre-empt research on stem cells in the State of California.  Perhaps the challenge would have met the court’s requirements for standing if a California research university, or the California Institute for Regenerative Medicine (which supports stem cell research in the state) had filed it.  At least for now, the patent stands.  As it expires in 2015, the window of opportunity to successfully challenge it, and change related patent law, is small.  The decision also suggests, as one of the legal experts quoted by ScienceInsider implies, that challenges by consumer groups like California Watchdog (or the ACLU) may not get much further than the USPTO.

NIH Advisory Group Outlines Long-Term Plan For BRAIN Initiative

While budget pressures may turn it into a wish list, the Advisory Committee to the Director of the National Institutes of Health (NIH) has outlined a long-term plan for the NIH portion of the BRAIN Initiative (Brain Research through Advancing Innovative Neurotechnologies).  NIH is one of three federal agencies involved, along with several private sector entities and foundations.  The NIH intends, per its press release, to “map the circuits of the brain, measure the fluctuating patterns of electrical and chemical activity flowing within those circuits, and understand how their interplay creates our unique cognitive and behavioral capabilities.”

The estimated necessary investment is significantly larger than the $140 million expected between the current fiscal year and fiscal year 2015.  The Advisory Committee sees a 12-year investment of $4.5 billion as important toward achieving its vision, which would include doing the following (again, from the press release):

  • Pursu[ing] human studies and non-human models in parallel
  • Cross[ing] boundaries in interdisciplinary collaborations
  • Integrat[ing] spatial and temporal scales
  • Establish[ing] platforms for preserving and sharing data
  • Validat[ing] and disseminat[ing] technology
  • Consider[ing] ethical implications of neuroscience research
  • Creat[ing] mechanisms to ensure accountability to the NIH, the taxpayer, and the community of basic, translational, and clinical neuroscientists

The Advisory Committee describes its plan in a report released last week.  BRAIN 2025 is pretty thorough, certainly for a policy document (rather than a research paper).  It covers why the Initiative is needed, a scientific review intended to justify the choices for high-priority research areas, and a detailed implementation plan that includes deliverables, milestones, and cost estimates.  It’s worth taking the time to review and digest.

As ambitious, and arguably as valuable, as the BRAIN Initiative is, the recent budget fights suggest to me that there is no stomach in Congress for major scientific investments.  There’s barely enough interest in maintaining a status quo that doesn’t consider inflation.  I strongly suspect that a large chunk of the $4.5 billion (again, this would be spread out over 12 years) will come – if it comes at all – from non-governmental sources.  That may not be a problem, depending on what expectations come with the additional outside funding.

The White House intends to hold an event later this year to discuss further efforts supporting the BRAIN Initiative.  No date has been announced as yet.

Bioethics Commission Continues Assessment Of Neuroscience Research

The Presidential Commission for the Study of Bioethical Issues will hold its next meeting on Monday and Tuesday, June 9 and 10, in Atlanta.  Per the draft agenda, it will continue the work it started last summer on the ethics of neuroscience research and its applications.  As is customary, the meeting will be webcast, simply visit the Commission website during the meeting.

On the first day the Commission will hear from experts on data sharing in neuroscience research, the potential in the field, and the differences in neuroscience research for various stages of life.  On the second day the focus shifts slightly to the ethical and social implications of neuroscience research.  The material on the second day will likely inform the second volume of the Commission’s report, Gray Matters.  The first volume was released in May and focuses more on how to better integrate ethical principles into neuroscience research.  During this meeting, the Commission may give some idea of when that report might be available.

PCAST Releases Second Report Of May On Health Care Delivery

Last Thursday the President’s Council of Advisers on Science and Technology (PCAST) released a report on health care.  The title, Better Health Care and Lower Costs: Accelerating Improvement Through Systems Engineering, implies the thrust of the report.  The report group suggests that the use of systems engineering panels can enhance health care in several ways, including:

  1. reforming payment systems
  2. building the Nation’s health-data
  3. providing technical assistance to providers
  4. increasing community
  5. sharing best practices, and
  6. training health professionals in systems-engineering approaches

The recommendations, according to the report, should be implemented in that order, because the current payment model (which encourages volume of treatment rather than value of treatment) and the ability to measure and analyze health care activity are significant impediments to using systems engineering in this area.

For those not familiar with systems engineering, I would jump to the appendices first before diving into the body of the report.

Making Research Subjects Better Reflect The Population At Large

Recently the National Institutes of Health (NIH) announced that starting in October, populations of lab animals and research cells will need to follow the same requirements for gender equity as required for research on human populations.  Put in other words, the lab mice (and cell samples) used in an experiment need to have as many males as females.  NIH Director Francis Collins, along with Janine Clayton, the Director of the NIH Office of Research on Women’s Health, outlined the reasoning behind the new policy in Nature.

While the tools and processes are in place to increase the diversity of research subjects, there are other areas where research could do better.  A recent opinion column in The New York Times (H/T ScienceInsider) argues that older people are not adequately represented in clinical trials.  While there are issues related to informed consent for research on younger subjects, such barriers – to the extent they exist – are much smaller for older test subjects.  I agree with the column that there is no reason to exclude subjects on the basis of age, but I suspect properly recruiting older people for research trials and controlling for the factors that might influence experimental results will make it harder to address this gap.

Gene Therapy May Have Revised Oversight

The National Institutes of Health (NIH) will stop requiring special review of gene therapy trials (H/T ScienceInsider) currently conducted by the Recombinant DNA Advisory Committee (RAC).  NIH Director Francis Collins justified the decision, which is based on recommendations from a study it requested of the Institute of Medicine, by noting the progress in the field since the formation of the RAC over 40 years ago, as well as the additional regulatory reviews in place for this kind of research.  The RAC would remain to review special kinds of gene therapy trials, provided they meet the following requirements:

  1. The protocol review could not be adequately performed by other regulatory and oversight processes (for example, the institutional review boards, institutional biosafety committees, and the FDA).
  2. One or more of the following criteria are satisfied:
    • Protocol uses a new vector, genetic material, or delivery method that represents a first-in-human experience, thus representing unknown risk.
    • Protocol relies on preclinical safety data that were obtained using a new preclinical model system of unknown and unconfirmed value.
    • Proposed vector, gene construct, or method of delivery is associated with possible toxicities that are not widely known and that may render it difficult for local and federal regulatory bodies to evaluate the protocol rigorously.

I understand the idea that the RAC likely conducts a certain amount of review that is redundant.  Given the challenges facing other bodies with NIH-relevant ethics responsibilities, I would certainly understand if anyone took pause in response to the decision.  Especially since the NIH has yet to decide whether to follow another IOM recommendation – to replace the RAC with another body focused on gene therapy and other kinds of risky research.