UCSF Tops California Universities with Four NIH Director's New Innovator Awards

By Jeffrey Norris

The National Institutes of Health (NIH) on September 30 announced 52 highly competitive awards for high-risk, high-payoff research for young biomedical scientists, and UCSF tops California institutions with four recipients. The four recipients of the NIH Director’s New Innovator Awards will use their new funds to further understanding of cancer, autism, stem cells and the potential for microbes to yield new drugs. Each individual award covers up to $1.5 million in research costs over five years. The recipients of the new innovators awards are:

  • Lauren Weiss, PhD, assistant professor of psychiatry;
  • Diana Laird, PhD, an assistant professor in residence in the UCSF Department of Obstetrics, Gynecology and Reproductive Sciences and Eli and Edythe Broad Center for Regeneration Medicine;
  • Michael Fischbach, PhD, an assistant professor in the Department of Bioengineering and Therapeutic Sciences; and
  • Ophir Klein MD, PhD, faculty member in the Schools of Dentistry and Medicine and director of the Craniofacial and Mesenchymal Biology (CMB) Program.

According to NIH Director Francis S. Collins, MD, PhD, "The NIH is pleased to be supporting early-stage investigators from across the country who are taking considered risks in a wide range of areas in order to accelerate research. We look forward to the results of their work." As the agency’s website explains, the NIH Director's New Innovator Award program is “designed specifically to support unusually creative new investigators with highly innovative research ideas at an early stage of their career. “The procedure for evaluating applicants' qualifications is distinct from the traditional NIH peer review ‘study section’ process and will emphasize the individual’s creativity, the innovativeness of the research approaches, and the potential of the project, if successful, to have a significant impact on an important biomedical or behavioral research problem.”

Lauren Weiss – Elusive Autism Genes

About 90 percent of autism risk is thought to be due to inherited genetic variants. However the genes responsible are for the most part unidentified. Implicating genes has proved difficult because autism is a complex disease in which multiple genes are likely to contribute to the risk. Although model organisms suggest that interactions between genes are important determinants of complex traits, genetic interactions have been difficult to identify for human disease. To eliminate one of the unknowns, with her new innovator award Lauren Weiss will conduct a genome-wide association study of families that include individuals with an already identified genetic mutation known to cause a rare inherited syndrome --- one in which many of those afflicted also have autism. She will look for additional genetic variants that influence whether an individual with the syndrome has autism.

Diana Laird – Competing Cells and a Healthy Germ Line

Competition between cells in the body contributes to the health of tissues, regulates the size of organs, and underlies the growth of tumors. In the case of the germ cells —- egg and sperm — or their precursors, the outcome of a competition not only affects the quality and quantity of germ cells, but also is inherited by the offspring, making it relevant to evolution. With her new innovator award developmental biologist Diana Laird will study the mechanisms of competition between the precursors of egg and sperm in the embryo to shed light on cancer, where similar mechanisms are likely exploited by tumor cells. This competition among cells will need to be considered in stem cell therapy and other treatments that introduce cells into the body, Laird says.

Michael Fischbach – Will Microbes Inside Us Yield New Drugs?

Bacteria and fungi that grow inside us or on our skin are the focus of Michael Fischbach, another UCSF new innovator award recipient. He aims to identify potentially therapeutic, drug-like molecules. Fischbach is an expert on the chemistry of small molecules formed during metabolic processes. He also is a bioinformatics expert. Although microbial cells outnumber the cells of their human host by about a factor of 10, these microbes have rarely been investigated in drug screening studies. A recent and ongoing NIH-funded initiative to map the genomes of human-associated microbes and to have information about the microbes deposited in extensive databases will make it easier for Fischbach to apply his expertise to the task of identifying potential drugs.

Ophir Klein – Stem Cells for Teeth Shed Light on Organ Development

New innovator Ophir Klein wants to use stem cells that give rise to teeth as a convenient starting point and proving ground for regenerative medicine. Klein will identify the genetic changes that led stem cells to arise in teeth during the evolution of mammals. He will take advantage of the fact that teeth in a number of different mammals grow continuously, providing a system for comparing those animals that have stem cells in their teeth with those that don't. Klein hopes that by exploring the mechanisms that underlie the evolution of stem cells, he will be able to understand which genes are important regulators of these cells. Because teeth are relatively simple in comparison to the large, vital organs, they provide an excellent prototype for learning how to grow these larger, more complex organs.

Supporting Bold Research Ideas

Keith Yamamoto, PhD, executive vice dean for the UCSF School of Medicine, earlier co-chaired a working group for the NIH that advised the agency on its peer-review grant-funding process. “We wanted to know how we could get the scientific community to know that the NIH will recognize the potential impact of really bold research ideas,” Yamamoto says. “I think it’s important that the federal funding system for biomedical research is welcoming of bold initiatives, especially from young investigators.” The new innovator program is one initiative that addresses issues raised in those earlier workshop discussions. As for what might account for the success of young UCSF scientists in the new innovators award competition, Yamamoto points to UCSF’s own Program for Breakthrough Biomedical Research. The program has the same standards as the NIH Director’s New Innovator Awards and has contributed to a culture in which individuals can hope to be supported for taking on high-risk, high-payoff research, he says. Work performed with funds awarded through the program, which are provided by donors, have been leveraged into NIH grants, and many discoveries stemming from the research have led to new patents, according to Yamamoto.

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