Breakthroughs in Bioscience

1. Breakthroughs in Bioscience From NIH-Funded Basic Research to Improved Health North Carolina

2. National Institutes of Health (NIH) • Nation’s medical research agency • Funds the science that leads to medical advancement • Campus in Bethesda, MD – but most funding is distributed to university researchers throughout the United States • Duke ranks 9th overall in NIH funding; UNC ranks 11th& the state ranks 7th overall, with more than $918 million.

3. Research Enterprise Is Critical to North Carolina’s Economy • The state ranksin the top 10 for NIH funding, with more than $918 million awarded. • Duke University received more than $358 million in NIH awards in FY08. UNC-Chapel Hill topped $337 million. Wake Forest received about $93 million. • Faculty discoveries have created 25 spin-off companies that are attracting new investments and creating jobs for North Carolinians. • Duke is the largest employer in Durham County. NIEHS, a division of NIH, employs more than 850. • The state has approx. 150 biotech companies that generate $3 billion in revenue a year and employ about 20,000 residents.

4. Leveraging the State’s Investment with Extramural Research Dollars • NIH funding for research conducted jumped almost 7 percent in fiscal year 2004 • The Southeast Regional Centers of Excellence for Biodefense and Emerging Infectious Disease aims to develop vaccines, drugs and diagnostic tests against emerging infections and organisms such as smallpox and Severe Acute Respiratory Syndrome

5. The NIH Top 20 Universities: An Elite Group • Johns Hopkins • UC San Francisco • Pennsylvania • Michigan – Ann Arbor • Washington • Washington U • Pitt • Yale • Duke • UCLA • UNC – Chapel Hill • UC San Diego • Stanford • Vanderbilt • Columbia • Minnesota • Wisconsin • Emory • Baylor • MIT

6. NIH: Saving Lives Through Science • Current annual budget of around $29.3 billion • Greater than 80% distributed throughout the country • More than 50,000 grants • 212,000 scientists • 2,800 universities • Portfolio of basic, translational, and clinical research NIH has been involved in nearly all the medical & health related discoveries of the past century

7. 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 • They 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 • Proposals are reviewed in a two-tiered 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 • High quality research is not being done for lack of funding

8. 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

9. 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 • Much of this research is too basic for the private sector • The federal investment often lays the foundation for advances in healthcare

10. Evolution of Research to Healthcare Selected modern examples…

11. Cardiovascular disease • Information on the biochemical structure & synthesis of cholesterol led to the development of statins • Discoveries in basic kidney biology and blood pressure regulation converged with an unexpected finding involving snake venom to yield ACE inhibitors, one of our most effective hypertension medications • Understanding how the blood clots, together with a new cancer treatment and the first commercial use of recombinant technologies, resulted in rtPA, 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

12. 500 ~ 1,329,000 Projected Deaths in 2000 400 300 Deaths per 100,000 200 ~ 514,000 Actual Deaths in 2000 100 50 55 65 75 80 85 90 95 00 Year Cardiovascular disease 60 70 Economic return of improved treatment & prevention $2.6 TRILLION 30-year Investment per American ~$110.00 Total

13. 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

14. Deaths from AIDS dropped nearly 70% between 1995 and 2000 HIV / AIDS Survival rates for those infected with HIV has increased by 10 years

15. Cancer • 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 developed bortezomib - now used to treat patients with multiple myeloma. RESULTS??FROM 1993-2002, CANCER DEATH RATES DROPPED 1.1% PER YEAR; MORE THAN 2/3 OF PEOPLE DIAGNOSED WITH CANCER CAN EXPECT TO LIVE 5+ YEARS

16. Cancer Increase in Cancer Survivors 9 6 Millions of People 3 1971 1986 1990 2003 30-year Investment per American ~$260.00 Total

17. Infant mortality • Studies on lung function led to the discovery of surfactant. This protein-lipid mixture is crucial for the survival of premature infants, decreasing the number of infant deaths from respiratory distress from 15,000 per year to less than 1,000. • The use of anti-virals to prevent mother to child HIV transmission has reduced the rate from 25% to about 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 can reduce pre-term deliveries by as much as 30%, a particularly important result for 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

18. NIH-Funded Discoveries in N.C. • First to discover ApoE, a major genetic risk factor for Alzheimer’s (Duke) • Discovered a novel genetic trait that protects carriers from deadly malaria (Duke) • Dukeresearchers were the first to identify a gene that causestesticular cancer in three out of every five men who have it • Wake Forest researchers first used MRI to view the heart during vigorous beating, pioneering use of MRI as screening and diagnostic tool for heart disease • UNC researchers discovered that rheumatoid arthritis patients treated with methotrexate have higher risk of reactivating cancer • New digital mammography techniques helped detect more cancers than previous technique (UNC) • Duke is working with AIDS patients in Tanzania to study low-cost methods for diagnosing TB & meningitis, and treating infectious diseases affecting people with HIV/AIDS

19. 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

20. 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 • As the NIH budget falls, success rate also falls Diminished investment in NIH = loss of talented researchers = missed opportunities = delays in medical progress

21. North Carolina’s Congressmen Need to Advocate for NIH Funding • Nothing should surpass improving our health as a national priority • Opportunities for discoveries that translate to improved health for our citizens have never been greater • Every increase in the NIH budget means additional funding for research in the state and new jobs

22. We Need your Help:Working Together for NIH • Contact Senators Hagan and Burr, and your Congressional Representative • Let them know that medical research is important to you and what a bargain it is • Write a letter to the newspaper and talk to your friends • 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

23. Want to know more?? Please visit http://opa.faseb.org Federation of American Societies for Experimental Biology (FASEB)