Breakthroughs in Bioscience

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Breakthroughs in Bioscience

• From NIH-Funded Basic Research to Improved Health

Alabama
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National 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 U.S.
• Alabama received over 255M in NIH funding in
  FY2011

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UABs Research Enterprise Is Critical to
Alabamas Economy

• In FY2011, UAB received over 209M in NIH
  funding
• For every state dollar invested in UAB, about 11
  is returned to Birminghams economy
• UAB is the states largest employer, with more
  than 18,000 employees and about 56,000 jobs
  statewide related to the university and its
  health system
• Several Alabama-based companies started from UAB
  technologies, including
• Biocryst
• Vaxin
• Discovery BioMed
• Atherotech

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NIH 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
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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
  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

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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
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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, 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

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Evolution of Research to Healthcare
Some recent examples
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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

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Results 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.
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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

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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

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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
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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

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Cancer

• 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

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Results 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.
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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

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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

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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
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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)

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Neural 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

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Urgent 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

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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

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NIH-Funded Discoveries at UAB and Southern
Research Institute

• Studies on nerve cells using a toxin from the
  Israeli yellow scorpion led to the discovery of a
  new treatment for a brain tumor called
  glioblastoma
• Work with transgenic mice engineered to express
  the gene defective in sickle cell disease has led
  to a potential cure that is likely only a few
  years off
• A new type of white blood cell that is important
  in autoimmune diseases was recently described
• Drugs that cure viral infections such as those
  caused by West Nile virus or SARS have been
  discovered and tested
• A new understanding of the molecular basis for
  learning and memory may lead to better treatments
  for Alzheimers Disease and dementia
• Researchers discovered the first bio-marker for
  multiple sclerosis (MS) that might predict how
  patients will respond to treatment

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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

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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 essential

Diminished investment in NIH loss of talented
researchers missed opportunities delays in
medical progress
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Alabamas 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

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Working Together for NIH

• Contact Senators Shelby and Sessions, 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

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Want to Know More?
Please visit opa.faseb.org