UCSF scientists awarded NIH research awards encouraging innovation

By Jennifer O'Brien

Seven UCSF scientists have received research awards from the National Institutes of Health under an initiative designed to encourage innovative, risk-taking investigations.

The three types of awards—the NIH Director’s New Innovator Awards, Pioneer Awards and Transformative R01 (T-R01) Awards –  are intended to promote “ideas that have the potential to catapult fields forward” and ultimately drive medical advances. The Transformative R01 Awards are being issued for the first time.

A total of 115 2009 NIH Director’s High-Risk Research Awards were granted nationwide:  55 New Innovator Awards for early-stage investigators; 18 Pioneer Awards, and 42 T-R01 Awards.

“These are the kinds of scientific investigations that distinguish UCSF worldwide,” says Keith Yamamoto, PhD, executive vice dean of UCSF School of Medicine. “With these three grant mechanisms, NIH is seeking bold, ground-breaking ideas that can create the scientific paradigms of tomorrow. They depend upon the quality of the investigator and the new concepts proposed, rather than the acquisition of preliminary data.”

The programs are among those that Yamamoto has proposed or championed in the course of his activities in developing national science policy.

New Innovators Awards: UCSF scientists received three of the 55 New Innovator Awards http://nihroadmap.nih.gov/newinnovator/.  The grants provide $1.5 million over five years. They are designed to support promising new investigators, with the goal of advancing exceptionally innovative research ideas that lack the preliminary data to fare well in the traditional NIH peer review system. The UCSF recipients are:

  • Daniel A. Lim, MD, PhD, assistant professor of neurological surgery, director of restorative neurosurgery at UCSF Medical Center and staff physician, surgery service at the San Francisco VA Medical Center. He also is a member of the Eli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research at UCSF
  • Stavros Lomvardas, PhD, assistant professor of anatomy
  • Erik M. Ullian, PhD, assistant professor of ophthalmology and physiology

The Pioneer Awards: UCSF scientists received one of the 18 Pioneer Awards http://nihroadmap.nih.gov/pioneer/. The grants provide $2.5 million over five years. They are intended to support investigators of exceptional creativity who propose pioneering – and possibly transformative—approaches that have the potential to produce an unusually high impact on a broad area of biomedical or behavioral research.  The UCSF recipient is:

  • Nirao M. Shah, MD, PhD, assistant professor of anatomy

The Transformative RO1 (T-RO1) Awards: UCSF scientists received two of the 42 Transformative RO1s http://nihroadmap.nih.gov/T-R01/. The funding awards vary by investigation. They are designed to support unusually innovative, high risk, original and/or unconventional research projects that have the potential to create or overturn fundamental paradigms. These projects tend to be inherently risky, but if successful can profoundly impact a broad area of biomedical research The UCSF recipients are:

  • Long-Cheng Li, MD, assistant professor of urology, and Hao Li, PhD, professor of biochemistry and biophysics (shared) – They will receive a grant of $2.71 million over five years.
  • Wallace Marshall, PhD, associate professor of biochemistry and biophysics. The notice of grant award is being finalized.

The award programs are supported by the NIH Common Fund’s Roadmap for Medical Research. The Common Fund, enacted into law by Congress through the 2006 NIH Reform Act, supports cross-cutting, trans-NIH programs with a particular emphasis on innovation.

“The fact that we continue to receive such strong proposals for funding through the programs reflects the wealth of creative ideas in science today,” says NIH Director Frances S. Collins, MD, PhD.

The NIH expects to make competing awards of approximately $131 million to New Innovators, $13.5 million to Pioneer awardees and $30 million to Transformative-R01 awardees in Fiscal Year 2009. The total funding provided to this competing cohort over a five-year period is estimated to be $348 million. The New Innovator total includes $23 million in funds through the Recovery Act.

UCSF New Innovators Award studies:

  • Daniel A. Lim

    Lim will study the gene expression “switches” in developing neural stem cells. These switches progressively control the specialization of the cells into the multitude of cell types that form the brain. The research has importance for potential cell-based and gene therapies of neurodegenerative disorders such as Parkinson’s and Alzheimer’s disease, as well as the treatment of certain types of brain tumors.

  • Stavros Lomvardas

    Lomvardos has used a novel, high-throughput approach to study an unusual regulatory sequence in the neurons that control the ability to smell, a sequence that is also abundant in genes that are involved with guiding axons.
    His research will investigate brain plasticity processes and could provide potential therapeutic targets for the origins and treatment of learning disabilities and other neuropsychiatric conditions.

  • Erik M. Ullian

    Ullian will attempt to understand why the adult brain is less “plastic” than the developing brain. Plasticity indicates an ability to change the number and strength of synaptic connections. Ullian previously uncovered a role for astrocytes, a common cell type in the brain, in regulating plasticity in the developing brain.  Changes in this regulatory ability may underlie developmental neurological diseases, as well as diseases of the adult brain where circuitry is impaired.  His work has implications for treating a spectrum of neurological diseases ranging from autism and mental retardation to Alzheimer’s disease and neurodegeneration.

UCSF Pioneer Award study:

  • Nirao M. Shah

    Shah will develop genetic techniques to understand the neural circuits that enable animals to form long-lasting social attachments.  His research aims to provide insight into how social ties are regulated in health and how mental disorders such as autism manifest with a profound inability to form interpersonal bonds.

UCSF Transformative R01 Award studies:

Hao Li

According to the NIH, the Transformative R01 award provides an opportunity unmatched by any other NIH funding program. Because no budget cap is imposed and preliminary results are not required, scientists are free to propose new ideas that may require significant resources to pursue. They are also given the flexibility to work in large, complex teams if the complexity of the research problem demands it.

  • Long-Cheng Li

    Li and Li will use a combination of genomic, bioinformatic, and molecular biology approaches to studying small double stranded RNAs. Understanding the molecular mechanism of gene activation by small double stranded RNA and the general rules for promoter targeting is likely to establish a new paradigm in the field of gene regulation, and transform the way gene function is studied and diseases are treated.

  • Wallace Marshall

    Marshall’s research, which offers an alternative to the traditional stem-cell approach to regenerative medicine, will focus on inducing injured cells to repair themselves in human tissues and organs that have been damaged. His team is using modern genomic methods to study the processes of wound healing and regeneration in a single-celled organism called the Stentor, to understand what molecular pathways underlie the animal’s ability to regenerate itself following damage.

Of the Transformative RO1 awards, Collins wrote, they are “intended to support research that has the potential to transform the way we think about and conduct science, so the recipients represent an elite few with truly bold ideas. Competition for the awards was fierce, and standards very high.”

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