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

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  California ranks 1 st overall in NIH funding ($3.2B)  California successfully competes for 14% of the total NIH budget

Research Is Critical to California’s Economy  Los Angeles has a large base of biomedical research activity, including its universities (UCLA,UCSB, USC, UC Irvine, and the California Institute of Technology) as well as private research institutions (such as Cedars Sinai Medical Center, the City of Hope National Medical Center, and Huntington Medical Research Institute).  Three research institutions in the Los Angeles area are among the top 100 institutions that receive funding from the National Institutes of Health  The leading research institutions in the San Diego area include the Salk Institute, Scripps Research Institute, and the University of California San Diego  Almost 7,000 life scientists work in California  One in four U.S. biotech companies is located with in 35 miles of a UC campus  One in three CA biotech firms (one in six nationwide) was founded by UC scientists

CA Leverages the State’s Investment with Extramural Research Dollars  The area around Los Angeles has attracted more than $180 million in venture capital investment in biopharmaceutical firms since 1995 and investments have been made in 26 new firms.  UCSF generates more than 23,000 full-time jobs and $1.8B in sales and income in the area  UCSF researchers have contributed to the start-up of 60 life science and pharmaceutical companies, including Genentech and Chiron  For every state dollar invested in UC Davis, the state of California got at least $5 in return  UC Davis has generated 45,000 jobs for California  For each dollar of taxpayer investment, UCLA generates almost $9 in economic activity, resulting in $6B impact on the region

The NIH Top 20 Universities: An Elite Group 1.Johns Hopkins 2.Pennsylvania 3.Univ. Washington 4.UC San Francisco 5.Washington Univ 6.Duke 7.Michigan 8.UCLA 9.Pitt 10.Yale 11. Columbia 12. Harvard 13.UC San Diego 14.Stanford 15.North Carolina 16.Case Western Reserve 17.Vanderbilt 18.Wisconsin 19.Baylor 20.UAB

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

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

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

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 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

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 1995 and 2000 HIV / AIDS Survival rates for those infected with HIV has increased by 10 years

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 , CANCER DEATH RATES DROPPED 1.1% PER YEAR; MORE THAN 2/3 OF PEOPLE DIAGNOSED WITH CANCER CAN EXPECT TO LIVE 5+ YEARS

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

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

NIH-Funded Discoveries in California  Identified the protein that more reliably signals warning signs for cervical cancer, leading to an improved standardized screening test (UC – Irvine, College of Medicine)  Bioengineered corneal tissue to restore or improve vision in blind or visually impaired patients (UC – Davis, School of Medicine)  Discovered a new, progressive neurological disorder affecting men over age 50 that causes tremors, balance problems and memory deficits (UC –Davis, School of Medicine)  Developed first new breast cancer CT scanner to reach clinical testing in a generation (UC – San Diego, School of Medicine)  Isolated the gene for basal cell carcinoma, a form of skin cancer that affects about 750,000 Americans each year (Stanford University School of Medicine)  Researchers reported that memory loss from minor strokes deep in the brain could now be distinguished from that caused by mild Alzheimer’s disease  Discovered how and where Listeria bacteria invade cells in the intestine (Stanford University School of Medicine)

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  As the NIH budget falls, success rates also fall Diminished investment in NIH = loss of talented researchers = missed opportunities = delays in medical progress

California’s Members of Congress 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

We Need your Help: Working Together for NIH  Contact Senators Boxer and Feinstein, or the Congressional Representative for your district  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

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