Title: Breakthroughs in Bioscience
1Breakthroughs in Bioscience
- From NIH-Funded Basic Research to Improved Health
Kentucky
2National Institutes of Health (NIH)
- Nations 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
3 Research Enterprise Is Critical to Kentuckys
Economy
- In 2011, Kentucky received more than 154 million
in NIH awards - The states 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
4Facing the Challenges Kentucky Health from a
National Perspective
- 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
5NIH Saving Lives Through Science
- Current annual budget (FY2012) of around 30.6B
- Greater than 80 distributed throughout the
country - Almost 50,000 grants
- More than 325,000 scientists at over 3,000
research sites - 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
6How 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
funding - Must explain why they think its a good idea, how
theyre going to do the experiments, and what
impact it will have on science and medicine - Proposals 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!
- Only 1 in 4 proposals funded in the 2008 fiscal
year - Lots of high quality research not being done for
lack of funding
7Basic 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
8What 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, osteoporosis, and viral diseases
like influenza, herpes AIDS - 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 from - Makes it imperative to support a broad range of
scientific research - Too risky for the private sector, federal funding
is critical for research
9Evolution of Research to Healthcare
Some recent examples
10Cardiovascular 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
11Results of Cardiovascular Disease Research
CVD disease death rates(United States 1900-2006)
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.
12Future 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
13HIV / 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
14Results 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
15Future 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
16Cancer
- The discovery that estrogens 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
17Results of Cancer Research
Overall cancer survival rates have improved
significantly, from about 50 in the 1970s to
66 in recent years. This is due, in part, to
both earlier detection and advances in
treatment.
18Future 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
19Infant 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
20Results 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
21Future 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)
22Neural Prosthetics
- The groundwork for neural prosthetics was laid by
more than a centurys 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
23Urgent Need for Prosthetics Research and
Development
- Body armor saves lives, but provides little to no
protection for a soldiers limbs - One of the major impairments seen in veterans
returning from Iraq and Afghanistan is amputations
24Future 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
25NIH-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 Huntingtons,
Alzheimers and Parkinsons - The UK College of Medicines molecular/cellular
biochemistry department is pursuing a therapeutic
approach using bone marrow stem cells to prevent
and treat Alzheimers disease
26The 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
27The 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 essential
Diminished investment in NIH loss of talented
researchers missed opportunities delays in
medical progress
28Kentuckys 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
29We Need Your HelpWorking 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
30Want to Know More?
Please visit opa.faseb.org