Appropriations Subcommittee Statement on the Fiscal Year 2016 Budget

April 30, 2015

Mr. Chairman and Members of the Committee:

I am pleased to present the President’s Fiscal Year (FY) 2016 budget request for the National Institute of General Medical Sciences (NIGMS) of the National Institutes of Health (NIH). The FY 2016 NIGMS budget of $2,433,780,000 includes an increase of $61,479,000 over the comparable FY 2015 level of $2,372,301,000.

NIGMS is the NIH home for basic biomedical research—the wellspring that feeds advances in medicine and technology. By defining the mechanisms of disease and providing targets for drug development, NIGMS-funded basic research is a critical driver of the U.S. economy. NIGMS supports more than 3,000 investigators and 4,500 research grants, helping to maintain a healthy research enterprise in every state in the nation as well as in U.S. territories. NIGMS is dedicated in particular to boosting opportunities for new investigators to establish independent biomedical research programs.

Creativity to Cures: Basic Research Discoveries With Potential Health Impact

Drug design and manufacturing is a multi-billion dollar enterprise with substantial impact on American health and the economy. This enterprise has been helped significantly by NIGMS-supported researchers who have used clever biological techniques to produce molecules that are more useful and safer than their natural counterparts. Take the medicine amphotericin B, the last line of defense in treating fungal infections like valley fever. So notorious for its severe and potentially lethal side effects, the drug is referred to by physicians as “ampho-terrible.” Recent NIGMS-supported research pinpointed the cause of this drug’s toxic effects: unintended removal of cholesterol—an integral component of every human cell. The team went on to develop an alternative molecule that kills fungi but spares human cells, potentially leading to a safer, better tolerated anti-fungal drug.

Other progress arising from NIGMS’ basic research investment is the development and refinement of extremely powerful biotechnology tools. An example of this type of technology emerged from studies of a bacterial antiviral defense system called CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats). After years of careful characterization of CRISPR, scientists recently adapted this bacterial “immune system” to efficiently change gene sequences inside cells with extreme precision and speed. This past year, two new investigators—each using money from their first NIH R01 grant, and one also using a Centers of Biomedical Research Excellence (COBRE) grant from NIGMS’ Institutional Development Award (IDeA) program in Montana—determined the three-dimensional structures of CRISPR’s cellular DNA-repair machines. These new insights will accelerate efforts to engineer even more accurate and efficient gene-therapy and other biotechnology-related applications.

Yet another recent discovery may lead to new treatments for sleep disorders, jet lag and other health problems tied to circadian rhythms. NIGMS-funded scientists at Dartmouth elucidated a key principle determining how our biological clock is set. Circadian function is conserved across species and can be studied with model organisms such as the bread mold Neurospora crassa, which was used in this work.

Maximizing Return on Investment for Taxpayer-Funded Research

To ensure the greatest yield from its investments, NIGMS seeks to increase the efficiency of the biomedical research enterprise. For example, with lower funding success rates over the past decade, scientists have had to devote an increasing proportion of their time to writing research proposals to support their work, leaving less time and energy to perform the important research they propose to do. This past year, NIGMS tackled this and other problems by conceiving a new way of doing business. Currently in pilot testing, the Maximizing Investigators’ Research Award (MIRA) provides support for all NIGMS-relevant research in an investigator’s laboratory. By focusing support on individual investigators rather than on individual projects, extending grant “shelf-life” and broadening grant scope, MIRA aims to promote more ambitious and creative research, enhance flexibility for trying new ideas and maximize the scientific returns on taxpayers’ investment.

Modern biomedical instrumentation is extremely powerful yet very expensive, and NIGMS looks for avenues to create and sustain economies of scale that increase efficiency in the practice of science. The Institute continues to use a variety of approaches to advance research capabilities of faculty and institutions and to ensure broad access to critical, shared research resources and technologies.

Funding Excellence Through Diversity in People, Projects and Places

A core principle of both financial investment and biology is that diversity leads to strength. Diversity at all levels—from the kinds of science supported to the regions in which it is conducted to the backgrounds of the people conducting it—strengthens the Institute’s research portfolio.

The NIGMS IDeA program, for example, increases the geographic distribution of NIH funding for biomedical research. Through the IDeA program, NIGMS stimulates research in states that have traditionally received lower rates of NIH funding. IDeA’s COBRE programs enhance access to cutting-edge technologies and promote career development for new investigators through mentorship and strengthening of research infrastructure. One example is the Oklahoma COBRE, which is determining the three-dimensional structures of important biological molecules. This center, which is led and staffed by an all-female scientific team, provides significant resources for investigators throughout Oklahoma: supporting annual symposia, workshops, a seed-grant program and core research facilities.

COBRE-funded researchers at the University of Kansas Medical Center are pursuing an exciting new lead for treating ovarian cancer, which is usually diagnosed in patients at a late stage of the disease and is prone to drug resistance in which chemotherapy stops working. This group discovered that one component in an herbal preparation of a tropical shrub used in traditional folk medicine in Africa shrank ovarian tumors in experimental mice, either alone or in combination with the standard chemotherapy drug carboplatin. These promising results await testing in humans.

In this statement, I have shared just a few examples of the remarkable types of returns received from NIGMS’ investment in basic biomedical research. The Institute looks forward to the many more advances that will emerge from laboratories across America.

Thank you, Mr. Chairman. I would be pleased to answer any questions that the Committee might have.