Breakthroughs in Bioscience From NIH-Funded Basic Research to Improved Health

National Institutes of Health (NIH)  Nation’s medical research agency  Funds the science that leads to medical advancement  Located in Bethesda, MD – but most funding is distributed to university researchers and physicians throughout the United States

NIH: Saving Lives Through Science  Current annual budget of around $28 billion  Greater than 80% distributed throughout the country  More than 50,000 grants  212,000 scientists  2,800 universities  How much money is being spent in your local area?  See:  Portfolio of basic, translational, and clinical research NIH has been involved in nearly all the medical & health related discoveries of the past century

How NIH Makes Science Happen…  Researchers working at local universities, hospitals and research institutions are dependent on federal support to fund their research, hire lab personnel and train young scientists  Write research grant proposals to compete for funding  Must explain why they think it’s a good idea, how they’re going to do the experiments, and what impact it will have on science & medicine  Proposal are reviewed in a two-tier system  Peer-reviewed by scientists to ensure highest quality science  Reviewed again for applicability to scientific or health priorities, by NIH officials and other stakeholders, including public members  NIH review system is the envy of the world!  Very competitive!!!  Before = 1 in 3 proposals funded, now closer to 1 in 6  Lots of high quality research not being done for lack of funding

Basic Research: From Bench to Bedside  Much of NIH funding goes to basic or fundamental research  Basic research is driven by interest in a scientific question  The main motivation is to expand knowledge and understanding, not to create or invent something  However, the insight into how the human body works and understanding of how diseases and disorders operate provides the foundation for medical progress "People cannot foresee the future well enough to predict what's going to develop from basic research. If we only did applied research, we would still be making better spears." Dr. George Smoot, Berkeley National Lab

What about medical breakthroughs?  Medical breakthroughs often come from unrelated areas of science or medicine  Research on cancer biology has led to drugs for: heart disease; viral diseases like influenza, Herpes & AIDS; and osteoporosis  Physicists studying the effects of magnets on atomic particles made the discovery that gave us MRI  Usually based on years or decades of fundamental knowledge  Over time, scientists solve or find different pieces of the puzzle  This makes it difficult to predict where the next breakthrough will come from  Makes it imperative to support a broad range of scientific research  Too risky for the private sector, federal funding is critical for research

Evolution of Research to Healthcare Selected modern examples…

Cardiovascular disease  Information on the biochemical structure & synthesis of cholesterol led to the development of statins  Discoveries in basic kidney biology, and increased understanding of the molecular regulation of blood pressure converged with an unexpected finding involving snake venom to give us ACE inhibitors, one of our most effective hypertension medications  Research into the mechanism of how the blood formed clots, together with the search to find a new cancer treatment and the first commercial use of recombinant technologies, resulted in rt-PA, a clot-busting drug that can prevent death from heart attack or stroke RESULTS?? 63% REDUCTION IN DEATHS FROM HEART DISEASE AND A 70% REDUCTION IN DEATHS DUE TO STROKE; MORE THAN 1 MILLION LIVES SAVED IN 2006 ALONE

Cardiovascular disease Deaths per 100,000 Year ~ 514,000 Actual Deaths in 2000 ~ 1,329,000 Projected Deaths in year Investment per American ~$ Total Economic return of improved treatment & prevention $2.6 TRILLION

HIV / AIDS  Fundamental knowledge of how viruses replicate gave scientists targets for therapy. Researchers looking for a new cancer drug hit one of those targets when they discovered a way to block replication, resulting in the development of AZT.  Increased understanding of how HIV operates at the cellular and molecular level identified more targets, and eventually led to the combination of drugs knows as the ‘triple cocktail.’ RESULTS?? AIDS HAS BEEN TRANSFORMED FROM AN ACUTE FATAL ILLNESS TO A CHRONIC CONDITION; THE PROPHYLACTIC USE OF ANTI-VIRALS PREVENTED ALMOST 350,000 DEATHS WORLDWIDE IN 2005

Deaths from AIDS dropped nearly 70% between HIV / AIDS Survival rates for those infected with HIV has increased by 10 years

Cancer  The discovery that estrogen played a role in some types of breast cancer, together with very basic research into the shape and characteristics of the estrogen receptor, gave us tamoxifen, which can reduce breast cancer incidence among women at risk by over 45%.  The breakthrough finding that human papillomavirus (HPV) could cause cervical cancer has led to a new vaccine that NIH estimated could reduce cervical cancer incidence by as much as 90%.  While investigating the cellular machinery controlling cell growth, scientists found the 26S proteasome, the inhibition of which is the power behind bortezomib- now used to treat patients with multiple myeloma. RESULTS?? FROM , CANCER DEATH RATES DROPPED 1.1% PER YEAR; MORE THAN 2/3 OF PEOPLE DIAGNOSED WITH CANCER CAN EXPECT TO LIVE 5+ YEARS

Cancer Decrease in # of Cancer Deaths Millions of People Increase in # Cancer Survivors 30-year Investment per American ~$ Total

Infant mortality  Studies on the fundamental biology of lung function led to the discovery of a protein called surfactant. It was later discovered that this protein was crucial for survival of premature infants, decreasing the number of infant deaths from respiratory distress from 15,000 per year to less than 1,000 by  The use of anti-virals to prevent mother to child HIV transmission has reduced the rate from 25% to nearly 1% in the U.S.  Studies on a metabolite of progesterone, known as progesterone 17P, have led to the finding that injections of this compound could prevent pre-term delivery by as much as 30%, which is particularly significant in African American women. RESULTS?? IN LESS THAN A CENTURY, INFANT MORTALITY IN THE U.S. HAS BEEN REDUCED BY 90%; TRANSLATING TO ALMOST 500,000 BABIES SAVED PER YEAR

Provide examples from your home institution or laboratory  Information on NIH funding in Congressional districts or geographic areas can be found here:   You can search the CRISP database for grants funded at your local institution here:  Interested in a specific disease or condition? Go here: 

The Bottom Line…  People are living longer, healthier lives because of NIH funded medical research  What were once swiftly fatal illnesses have become treatable or manageable conditions  For those suffering from diseases that have no current treatment or cure, medical research provides hope – which has a major impact on quality of life

The Challenge…  NIH funding is entirely dependent on Congressional support  In recent years, Congressional support has diminished, and the NIH budget is slowly eroding from lack of funding and inflation  Lack of understanding in Congress about the importance of medical research and the treatments and hope it provides Diminished investment in NIH = loss of talented researchers = missed opportunities = delays in medical progress

All Politics is Local  Insert here information about the importance of NIH to your local community: Amount of funding to your institution ( estimates of jobs created, which your university or local economic development council may be able to provide  For information on how your members of Congress have voted or spoken out on NIH related issues, please contact the FASEB Office of Public Affairs: or

Working Together for NIH  Contact your member of Congress, write a letter to the newspaper  Let them know that medical research is important to you  Help educate policymakers and neighbors about the important work NIH is doing  Nothing is more important than our health – the National Institutes of Health (NIH) should be an American priority

Want to know more?? Please visit Federation of American Societies for Experimental Biology (FASEB)