Breakthroughs in Bioscience

From NIH-Funded Basic Research to Improved Health

ArkansasArkansas

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

University of Arkansas ranks in the top 100 schools for NIH funding

Research Enterprise Is Critical to Arkansas’s

Economy The state of Arkansas received more than

$62M in NIH awards for FY2011 The University of Arkansas for Medical

Sciences (UAMS) and its affiliates generate more than $1 billion in direct impact statewide

UAMS is the state’s largest public employer, with over 10,000 employees

UAMS Arkansas BioVentures and its Technology Licensing Office were established to help translate research into products for improving health

Researchers have obtained 150 patents for inventions or applications, with an equal number of pending patents

NIH Grants Support Many Programs in Arkansas

UAMS, the University of Arkansas-Fayetteville, the University of Arkansas at Little Rock, and seven partner institutions previously received a five-year, $16.7 million grant to expand and improve biomedical research in Arkansas

In 2009 NIH awarded UAMS its largest ever research grant (almost $20 million) to fund their Center for Clinical and Translational Research

The Myeloma Institute for Research and Therapy received a $19.5 million five-year grant to continue developing new cancer treatments

NIH: Saving Lives Through Science Current annual budget (FY2012) of around $30.6B

Greater than 80% distributed throughout the country

o Almost 50,000 grants

o More than 325,000 scientists at over 3,000 research sites

o How much money is being spent in your local area?

• http://report.nih.gov/award/organizations.cfm

Portfolio of basic, translational, and clinical research

NIH has been involved in nearly all the major medical & health related discoveries of the past

fifty years

How NIH Makes Science Happen… Most 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

Researchers write grant proposals to compete for fundingo 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 and medicine

Proposals are reviewed in a two-tier systemo Peer-reviewed by scientists to ensure highest quality scienceo Reviewed again for applicability to scientific or health priorities,

by NIH officials and other stakeholders, including public memberso NIH review system is the envy of the world!

Very competitive! o Only 1 in 4 proposals funded in the 2008 fiscal yearo Lots of high quality research not being done for lack of funding

Basic Research: From Bench to Bedside

A portion 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

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 medicineo Research on cancer biology has led to drugs for heart

disease, osteoporosis, and viral diseases like influenza, herpes & AIDS

o Physicists studying the effects of magnets on atomic particles made the discovery that gave us magnetic resonance imagining (MRI)

It often takes years or decades of fundamental knowledge to solve or find different pieces of the puzzle

This makes it difficult to predict where the next breakthrough will come fromo Makes it imperative to support a broad range of scientific

researcho Too risky for the private sector, federal funding is critical for

research

Evolution of Research to Healthcare

Some recent examples…

Cardiovascular Disease Information on the biochemical structure and

synthesis of cholesterol led to the development of statins, a class of drug used to lower cholesterol

Discoveries in basic kidney biology and an 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 blood forms 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 of Cardiovascular Disease

Research“Americans can expect to live an average of four years longer due to the reductions in deaths due to cardiovascular disease, largely as a result of NIH research.”

CVD disease death rates(United States: 1900-2006)

Future Directions… Genome-wide associations studies (GWAS) are

providing unprecedented insight into the intricate role genetics plays in the development of heart disease and identifying possible targets for novel drug therapies

Research on the effect of air pollution on blood vessel constriction is helping scientists understand how environmental factors effect cardiovascular health

Innovative imaging systems are being developed to allow for simultaneous evaluation of electrical activity and metabolic properties in the heart, allowing for the study of the complex mechanisms which lead to sudden cardiac arrest

HIV / AIDS Fundamental knowledge of how viruses replicate

gave scientists targets for therapy that led to the discovery of a way to block replication, resulting in the development of azidothymidine (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 of HIV / AIDS Research

The number of cases has remained relatively stable while the number of deaths has decreased

AIDS has been transformed from an acute, fatal illness to a chronic, manageable condition

Future Directions… Topical antimicrobial products, or microbicides,

offer one of the most promising avenues to primary prevention of HIV transmission

A number of HIV vaccine clinical trials have begun, which depend on fundamental research of the human immune response and on understanding of the way in which HIV infects cells

Investigators have identified the existence of HIV reservoirs that persist despite antiretroviral therapy, and efforts are now being focused on understanding and eliminating these reservoirs

Cancer The discovery that estrogen’s role in breast cancer,

together with 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 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 of Cancer Research

“Overall cancer survival rates have improved significantly, from about 50% in the 1970’s to 66% in recent years. This is due, in part, to both earlier detection and advances in treatment.”

Future Directions… Medical researchers have found certain antibodies

that are present only in tumors and may enable early detection and diagnosis of certain cancers

The Cancer Genome Atlas (TCGA), a project dedicated to accelerating our understanding of cancer genetics, has enabled deeper understanding of the most common form of adult brain cancer, glioblastoma multiforme

By suppressing the action of a certain cellular receptor, CD47, researchers have developed a method to protect healthy tissue from radiation therapy while making cancerous cells more vulnerable

Infant Mortality Research on the fundamental biology of lung

function enabled the discovery of surfactant, a protein crucial for survival of premature infants, and enabled a decrease in the number of infant deaths from respiratory distress from 15,000 per year to less than 1,000 by 2002

The use of anti-virals to prevent mother-to-child HIV transmission reduced the rate from 25% to nearly 1%

Studies on a metabolite of progesterone led to the finding that injections of this compound, progesterone 17P, could prevent pre-term delivery by as much as 30%, which is particularly significant in African American women

Results of Research on Infant Mortality

In less than a century, infant mortality in the United States has been reduced by 90%

This translates to almost 500,000 babies saved per year

Future Directions… In order to better diagnose and treat congenital

heart defects, a leading cause of infant mortality, scientists are developing new non-invasive imaging technologies for prenatal heart studies

Novel diagnostic techniques for amniotic fluid infection, a major risk factor for preterm birth, are being developed based on a recent finding that bacteria in the amniotic cavity can form biofilms (which make infections harder to detect)

Neural Prosthetics The groundwork for neural prosthetics was laid by

more than a century’s worth of basic research by anatomists, biochemists, and electrophysiologists

The first cochlear implant was introduced in the 1970s; today, more than 23,000 adults and 15,000 children in the U.S. owe their hearing to this device

The artificial retina is delicate enough not to damage the eye yet complex enough to provide visual input to the human brain; by 2011, the research team expects to start clinical testing on a version that enables reading and facial recognition

Urgent Need for Prosthetics Research and Development Body armor saves lives, but provides little to no

protection for a soldier’s limbs One of the major impairments seen in veterans

returning from Iraq and Afghanistan is amputations

Future Directions…

After amputation, the nerves controlling the missing limb remain active

Scientists have developed superfine electrode arrays to connect these nerves with prosthetic limbs

This will allow amputees to control and sense their prosthetics intuitively, making them feel more like their original limbs

NIH-Funded Discoveries in Arkansas

At the Food Allergy Research Program at Arkansas Children’s Hospital Research Institute (ACHRI), an oral immunotherapy is proving effective to protect children from severe allergic reactions to peanuts

Researchers at ACHRI found that developmental exposure to the environmental toxin trichloroethelene (TCE) causes neuroinflammation, which is linked to neurological disorders ranging from Autism to Parkinson’s disease

Discoveries from the Myeloma institute at UAMS have increased patient survival rates to approximately eight years, as compared to the national median of three years

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

The Challenge…

NIH funding requires congressional support

Sustainable budget growth is needed to achieve the full promise of medical research

Strong, outspoken champions for NIH in Congress and within the Administration are essentialDiminished investment in NIH = loss of

talented researchers = missed opportunities = delays in medical

progress

Arkansas’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

Working Together for NIH

Contact Senators Lincoln and Pryor, 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

Want to Know More?

Please visit opa.faseb.org