Breakthroughs in Bioscience
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Transcript of Breakthroughs in Bioscience
Breakthroughs in Bioscience
From NIH-Funded Basic Research to Improved Health
KentuckyKentucky
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
Research Enterprise Is Critical to Kentucky’s Economy
In 2011, Kentucky received more than $154 million in NIH awards
The state’s $400 million Bucks for Brains program leverages donor money to attract top-notch faculty to endowed chairs at University of Louisville
The UK Behavioral Science Department ranks #1 in NIH funding nationwide
A single $6.6 million award to the UK College of Agriculture directly created over 50 jobs at the institution
Kentucky is home to the biotech company Alltech, which plans to build the first Center for Animal Nutrigenomics and Applied Animal Nutrition
• In Kentucky, more adults smoke than any other state
• Kentucky ranks sixth in prevalence of obesity nationwide
• Cardiovascular disease is the leading cause of death in Kentucky
• Kentucky is ranked 3rd highest in cancer mortality rates
• Kentucky is 5th in the nation for deaths from coronary heart disease
• Nationally, Kentucky ranks 13th in premature deaths
Facing the Challenges: Kentucky Health from a National Perspective
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 Research in KY Shows Promise
UK investigators in physical medicine and rehabilitation have found a promising compound that targets mitochondrial function and has potential for acute treatment of patients sustaining head injury or spinal cord injury
A neurology professor at UK is engaged in a multi-institutional study which looks at methods of nutritional and respiratory support for amyotrophic lateral sclerosis (ALS) patients
Univ. of Louisville has created interdisciplinary centers to identify mechanisms to decrease heart damage after a heart attack, support cardiovascular function with internal artificial hearts, and stimulate spinal cord regeneration with neural stem cells
The UK department of neurobiology is also exploring RNA therapy to treat Huntington’s, Alzheimer’s and Parkinson’s
The UK College of Medicine’s molecular/cellular biochemistry department is pursuing a therapeutic approach using bone marrow stem cells to prevent and treat Alzheimer’s disease
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
Kentucky’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
We Need Your Help:Working Together for NIH
Contact Senators McConnell and Bunning and your 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