‘Secret’ Ebola Treatment And Other Stories

The outbreak of Ebola virus disease (formerly known as Ebola hermorrhagic fever) in western Africa is no laughing matter.  The way it’s been inserted into fights over immigration reinforces the need to fight misinformation.  (That one of the politicians concerned that Ebola will come into the U.S. via Central American immigrants is a retired doctor reinforces my belief that not all doctors are scientists.)

Here’s what the Centers for Disease Control (CDC) has to say (effective August 6).

“The World Health Organization, in partnership with the Ministries of Health in Guinea, Sierra Leone, Liberia, and Nigeria announced a cumulative total of 1711 suspect and confirmed cases of Ebola virus disease (EVD) and 932 deaths, as of August 4, 2014. Of the 1711 clinical cases, 1070 cases have been laboratory confirmed for Ebola virus infection.”

Contrary to the concerns of several elected officials and media outlets, there is no significant risk of Ebola in the United States.  Two researchers with the disease were evacuated to the United States for treatment at Emory University in Atlanta.  As long as a hospital follows CDC infection control recommendations and can isolate the patient, it can contain the disease.

There is word of a ‘secret serum’ that the U.S. has, but is not currently going to send over to Africa.  This likely refers to the experimental treatment ZMapp, which has not undergone testing on humans.  While it was used in connection with one of the U.S. cases, neither the National Institutes of Health nor the CDC were involved in procuring the experimental treatment or getting it to the infected person in Africa. Continue reading

Google’s Baseline Study – Will It Avoid 23andMe’s Pitfalls?

Last week The Wall Street Journal reported on a new project from Google.org – the Baseline Study (H/T ScienceInsider).  Starting with a pilot group of 175, the study would collect anonymous genetic and molecular information (according to the article) to construct a more comprehensive data-based model of what a human is.  Such a picture would ideally help researchers better identify symptoms and causes of various diseases.  A desired outcome would be the ability to find markers of various diseases much earlier in people than is currently possible.

Of course, such a long-term study is not new.  What would be different here is the potential scope of the project, should it expand beyond the initial population of 175.  The project is lead by Dr. Andrew Cohen, a molecular biologist whose previous major achievement has been in high-volume HIV tests of blood plasma.  Working in volume is something he’s comfortable with.  The project is mindful of the need to preserve anonymity, and has stated information will be used strictly for health and medical purposes and not shared with insurance companies.  Institutional review boards will be involved with the study, and once it grows beyond the initial group of 175, boards at the medical schools at Duke and Stanford Universities will be involved in controlling access and use of the information.

For reasons that escape me, I can find little mention of the project on the Google.org website.  Even the mentions found on Google + don’t connect to the organization.  It would seem to me that the Wall Street Journal article, and the subsequent press on it, could be part of an effort to gauge public interest and concerns with the project.  The lack of details (the articles have little more to go on than the information provided to The Wall Street Journal) are frustrating.

And then there’s 23andMe.  Continue reading

The National Institutes Of Health Has An (Im)Modest Grant Proposal

The National Institute for General Medical Sciences (NIGMS), part of the National Institutes of Health (NIH), issued a Request for Information (RFI) last week on a new kind of research funding program (H/T Science magazine – $ for full version).  The deadline for comments is August 15.

The input NIGMS receives from the comments and other input from stakeholders will inform a funding opportunity announcement for a pilot of this program, which would link funding to a lab/principal investigator more than to a single project. As the RFI describes it (in part) (a link has been removed):

“An NIGMS MIRA would provide support for a lab’s research program, which represents a compilation of the investigator’s NIGMS research projects (research areas supported by NIGMS are outlined at our website). Researchers would have the freedom to explore new avenues of inquiry that arise during the course of their work as long as those avenues are relevant to the mission of the Institute and do not require additional review for regulatory compliance (e.g., new human subjects research).”

Now, I’m not a research scientist, but this program would represent a notable change in how research funding is normally disbursed in the U.S.  Grants are typically considered primarily on the basis of scientific merit and broader impacts and associated with discrete research projects.  By aggregating support to the level of a research lab (and the associated principal investigator), NIGMS will be, if only indirectly, putting more stock into the past work and future promise of the lead researcher than it has before.

Continue reading

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.