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New NIH Leader Backs Personalized Medicine

WASHINGTON - MARCH 09:  U.S. President Barack ...
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No one was surprised last month when the White House nominated world-famous geneticist Francis Collins for the top job at the National Institutes of Health (NIH). The driving force behind NIH’s Human Genome Project for 15 years, Collins is experienced in managing large research projects and big budgets and in making friends on Capitol Hill.

In the year since he left NIH, he actively supported President Barack Obama’s election campaign and wrote a book on personalized medicine that reflects his belief in the power of molecular biology to shape medicine and the world. Collins has expressed disappointment that important genomic discoveries have not led to new life-saving therapies, and he will want to use his new position to change that.

For pharma, Collins’ offers prospects of increased emphasis at NIH on translational research and more collaboration in developing drugs for orphan and neglected diseases. Comparative effectiveness research, he says, should identify subpopulation responses to treatment and support personalized medicine.

Collins has been highly praised for bringing in the human genome project ahead of schedule and below budget; as NIH director he will need all his administrative skills to manage the vast NIH complex and shape how its disparate institutes dole out more than $25 billion in grants to research organizations and scientists. NIH staffers see him as likely to favor big, high-profile projects over the needs of individual researchers. An evangelical Christian, he also will face pressure to clarify how his personal faith can co-exist with support for evolution and science.

Collins takes over NIH at an enviable time. The agency gained some $10 billion in extra funds through the economic recovery legislation enacted earlier this year, providing a nice addition to the agency’s $30 billion budget. Although Congress is not likely to provide such largesse in the future, some increases are likely.

Spurring Stem Cell Research

A high-profile task for Collins is to implement recently finalized guidelines on human embryonic stem cell research (hESC), which NIH issued just as Collins’ nomination was announced. The new program calls for an NIH panel of scientists, ethicists and advocates to assess that all candidate stem cell lines meet ethical standards for government-funded research:  that they are derived from leftover embryos created by in vitro fertilization, that donors understand their options and consent to research uses, and that there is no compensation to or pressure on donors to do so. NIH will establish a registry of all cell lines deemed eligible for federal funding, with an eye to reducing uncertainty and confusion in the research community.

The new rules were proposed last April after President Obama issued an executive order rescinding Bush administration restrictions on government-funded hESC studies. The new rule promises to vastly expand opportunities for research in this area, despite continued curbs on funding studies that use stem cells created solely for research purposes or cell lines derived through somatic cell nuclear transfer, a technique that many researchers consider highly promising. While life-saving medicines ultimately may emerge, a near-term benefit may be the development of new cellular assays for screening drug candidates and other methods useful in drug development.

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2 Comments

  1. Posted July 22, 2009 at 1:24 pm | Permalink

    Excellent choice. There is no denying that we are on the verge of unprecedented discovery and innovation in science and technology that in my opinion will completely change how illnesses and diseases are diagnosed and treated. There will be tremendous opportunity for emerging companies that lead the way in the development of diagnostics and therapeutics based on patients’ individual genetic profiles.

  2. Posted July 29, 2009 at 9:54 am | Permalink

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