UCSF researchers receive five NIH awards for high-risk scientific research projects

UC San Francisco researchers received five awards announced this week by the National Institutes of Health (NIH) for high-risk, high-reward scientific research projects. Their work will focus on novel approaches for diagnosing and treating diseases ranging from autoimmune and chronic inflammatory diseases, to cancer, diabetes and neurological disorders.

The High-Risk, High-Reward Program, created to support the work of exceptionally creative scientists, is supported by the NIH's Common Fund. A total of 85 grants were awarded.

"Supporting innovative investigators with the potential to transform scientific fields is a critical element of our mission," said NIH director Francis S. Collins, MD, PhD. "This program allows researchers to propose highly creative research projects across a broad range of biomedical and behavioral research areas that involve inherent risk but have the potential to lead to dramatic breakthroughs."

Awardees from previous years have made scientific leaps, established new scientific paradigms and in some cases, revolutionized entire fields, according to Collins.

UCSF researchers were recognized in three out of four award categories:

NIH Director's Transformative Research Award

The Transformative Research Award, established in 2009, promotes crosscutting interdisciplinary approaches and is open to individuals and teams of investigators who propose research that could potentially create or challenge existing paradigms.

Wendell Lim, PhD, a professor of cellular and molecular pharmacology, investigator of the Howard Hughes Medical Institute and director of the UCSF Center for Systems and Synthetic Biology, was awarded $430,000 per year for five years to investigate techniques for engineering living immune cells in a predictable and controllable way in order to use them as therapeutic agents against complex diseases such as cancer.

"This grant provides an amazing opportunity for us to push our work in new directions," said Lim. "We have been pursuing fundamental basic research on understanding how cellular decision-making circuits work and have evolved. We now have the opportunity to apply the knowledge that has emerged to a tremendously exciting new problem — trying to reprogram the immune system to specifically recognize and kill tumor cells."

Michael McManus, PhD, an associate professor of microbiology and immunology, the Vincent and Stella Coates Endowed Chair in the UCSF Diabetes Center and the founder and director of the UCSF W.M. Keck Center for Noncoding RNAs, was awarded $370,000 per year for five years to support the development of a transformative technology for ancestral tracking of individual cells in an organism back to the single fertilized egg.

"This award will help us fulfill our greater scientific mission to solve important biological problems using highly creative approaches," said McManus. "In essence, it will help us transform science." If successful, the technology could provide new insights into fundamental developmental processes and diseases such as diabetes, cancer and neurological diseases, McManus said.

NIH Director's New Innovator Award

The New Innovator Award initiative, established in 2007, supports unusually innovative research by investigators who have completed their academic and clinical training within the past 10 years, but who have not previously received a research project grant (R01) or an equivalent NIH grant. The awards emphasize the creativity of individuals early in their careers, the innovativeness of the research approaches, and the potential of the projects, if successful, to have significant impacts on important biomedical or behavioral research problems.

Adam R. Abate, PhD, an assistant professor of bioengineering and therapeutic sciences, was awarded $300,000 per year for five years to support the development of a new approach for profiling the immune systems of patients with rheumatoid arthritis by identifying exactly which B cells - a type of immune cell - are mistakenly recognizing the body's own tissues as foreign. The new technology will analyze thousands of cells per second using microfluidics, a miniaturized fluid-handling system that employs pipes the diameter of a human hair or smaller. "Our aim," said Abate, "is to develop an approach that will allow us to detect autoimmune disorders and potentially develop therapeutics that can be used to treat them."

Michael Rosenblum, MD, PhD, an assistant professor of dermatology, was awarded $300,000 per year for five years to support research to better understand how the immune system functions in the skin. Rosenblum called the award "a great opportunity for my lab to perform cutting-edge research that will also give us the flexibility to pursue new scientific questions that may arise in the process." The ultimate goal of his research, he said, is to find ways to enhance the skin's immune system to treat chronic inflammatory diseases and skin cancer with minimal side effects.

NIH Director's Early Independence Award

The Early Independence Award, established in 2011, provides an opportunity for exceptional junior scientists who have recently received a doctoral degree or finished medical residency to skip traditional post-doctoral training and move immediately into independent research positions.

Robert Judson, PhD, a postdoctoral research fellow at the UCSF Helen Diller Family Comprehensive Cancer Center, was awarded $250,000 per year for five years to discover more accurate ways of predicting which of a patient's moles are at greater risk of eventually becoming an invasive melanoma, and which are likely to remain benign. Incidence of melanoma is on the rise, and melanoma is frequently lethal in less than a year if given the opportunity to metastasize.

"Receiving this award is a true game changer for me," Judson said. "This funding, plus the support I've received from the UCSF Sandler Fellows program and the Department of Dermatology, enables me to launch my independent lab and to apply my expertise in stem-cell biology to novel research directions in the study of melanoma. Without the Early Independence Award, I would not have the opportunity to pursue these novel research directions with potentially high clinical impact."