Congressional Biomedical Research Caucus Archives - Friends of the National Institute on Aging

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Fragile X Syndrome: Can Drugs be Used to Reverse Autism?

Time: September 30, 2009 from 12pm to 1pm
Location: B369 Rayburn House Office Building
City/Town: Washington, DC
Event Type: congressional, biomedical, research,caucus, briefing
Organized By: Congressional Biomedical Research Caucus

Dr. Stephen T. Warren
Emory University

Dr. Warren led the research that discovered how the gene mutation responsible for Fragile X Syndrome (FXS) alters the way brain cells communicate. FXS is the most commonly inherited form of mental retardation, with nearly a third of FXS patients also having autism, making FXS the single best understood cause of autism and a model for autism research.

FXS is caused by a mutation in the FMR1 gene on the X chromosome. Dr. Warren and his colleagues led an international team that discovered the FMR1 gene in 1991.They found that in patients with FXS, the expanded CGG triplet repeats can be repeated from 55 to over 200 times—whereas in healthy individuals the repeats range from 40 to fewer than 10. As a result of the hyper-CGG repeats, the expression of the FMR1 gene is repressed, which leads to the absence of FMR1 protein and subsequent mental retardation.

Dr. Warren and his team have since developed diagnostic tests for FXS. Clinical trials are now under way for FXS, taking advantage of the fundamental basic science research on FXS carried out over the past two decades. FXS is now used as a model of how fundamental research on autism could lead the way for future therapeutic interventions in autistic disorders.

Does Aging Bring Wisdom?

Time: September 16, 2009 from 12pm to 1pm
Location: B340 Rayburn House Office Building
City/Town: Washington, DC
Event Type: congressional, biomedical, research,caucus, briefing
Organized By: Congressional Biomedical Research Caucus

Dr. Shelley Carson
Harvard University

In her briefing for the Congressional Biomedical Research Caucus, Dr. Carson examines the deterioration of memory in older adults in a new light — not that of a mind being ravaged by dementia, but instead a mind widening its ability to process more details than that of a younger brain. For decades, cognitive research on the older brain has focused on the decline of thinking abilities. Newer research, however, suggests that much of this observed decline may actually result from lifestyle changes or illness rather than from inevitable brain atrophy.

Dr. Carson highlights how scientists are now beginning to focus on ways that cognition can actually improve with age. Research suggests that brainpower is not declining but that more information is being processed. Dr. Carson reviews the research on age-related brain changes and brain plasticity, and discusses how these changes affect wisdom and creativity. She also discusses initiatives to maintain and even improve cognition in later years.

FINDING AND FUNDING THE BEST SCIENCE: PEER REVIEW AT NIH

Time: July 22, 2009 from 12pm to 1pm
Location: 121 Cannon House Office Building
City/Town: Washington, DC
Event Type: congressional, biomedical, research,caucus, briefing
Organized By: Congressional Biomedical Research Caucus

Dr. Keith Yamamoto
University of California, San Francisco

Each year Congress appropriates billions of dollars to fund the National Institutes of Health (NIH). Did you ever consider how the money is distributed? With a budget of roughly $30 billion per year, the decisions that most strongly influence allocation of NIH funds are made by peer review by groups of professional scientists who typically are themselves funded by NIH. Is peer review really the best way to fund biomedical research? Are there intrinsic problems that compromise it? Could changes in peer review improve the quality of research?

These and other issues are discussed by Dr. Keith R. Yamamoto, an active scientist who has been involved in NIH peer review for almost 25 years, most recently leading an overall evaluation that produced key changes in the process.

Improving the Quality and Efficiency of Health Care for Older Americans

Time: June 17, 2009 from 12pm to 1pm
Location: 122 Cannon House Office Building
City/Town: Washington, DC
Event Type: congressional, biomedical, research,caucus, briefing
Organized By: Congressional Biomedical Research Caucus

Dr. Chad Boult
Johns Hopkins School of Public Health

Dr. Chad Boult and his team have created a new model of comprehensive health care, called Guided Care, which targets people living with multiple chronic conditions, the typical high-cost Medicare beneficiaries. His plan is based on the simple premise that a patient’s “care plan” is well coordinated, and patients and families are involved in and educated about the care plan. Guided Care is based on the simple notion that one trained professional should guide all aspects of care, uniting the patient, the family, and the medical team.

Using Genes to Redefine Disease

Dr. Atul Butte of Stanford University is at the forefront of the nascent field of translational bioinformatics—a field that seeks to create new diagnostics and therapeutics from genome-era information and data. Here he highlights how new uses for publicly available data have enabled us to ask new questions, including rethinking the nature of disease. Dr. Butte gathers this data on gene activity for scores of diseases. He is looking not at the symptoms or physiological measurements of disease, but at their genetic underpinnings. He performs statistical analyses to map disease based on similarities in their patterns of gene activity. Dr. Butte is able to show how using genes to redefine disease enables the discovery of new causes for disease, suggests novel roles for drugs in the treatment of disease, and, for the first time, allows us to probe the inner commonality across diseases that previously seemed dissimilar.