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

National Institutes of Health (NIH)
                 Nation’s medical
                  research agency
                 Funds the science that
                  leads to medical
                 Located in Bethesda,
                  Md. – but most funding
                  is distributed to
                  university researchers
                  and physicians
                  throughout the United
    Research Enterprise Is Critical to
           Maine’s Economy
   The state of Maine received more than $74 million in
    NIH awards for FY11
   The Jackson Laboratory received over $49 million from
    NIH in FY2011 and is one of 21 NCI designated Cancer
   The Jackson Lab employs more than 1,270 people
   Between November 2000-2004, Maine’s biomedical
    research sector grew by 44%, twice the state average
   Maine Biomedical Research Coalition members have
    spent more than $30 million at Maine businesses
   Maine Medical Center is the largest hospital in northern
    New England and received more than $12 million from
    NIH in 2011
NIH Grants Support Many Programs in
   Mount Desert Island Biological Lab received a grant of $17.8
    million to strengthen research and training programs at 9 Maine
   Jackson Lab researchers were awarded $15.1 million over 5 years
    for a systems approach to genetic research using a mouse model
   Maine Medical Center Research Institute is the recipient of an
    $11.2 million award to establish a COBRE in stem cell biology
    and regenerative medicine, generating 40 new jobs by 2008;
    MMCRI also received funding to purchase an animal MRI
    machine for statewide use
   Foundation for Blood Research (FBR) was awarded $1.3 million
    for pre-natal research into a woman’s thyroid function and its
    effects on a developing fetus
   FBR also recently received $1.24 million to develop a program
    that introduces Maine teachers and students to the importance of
    clinical research trials in medicine
     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?

 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 funding
    o 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 system
    o Peer-reviewed by scientists to ensure highest quality science
    o Reviewed again for applicability to scientific or health priorities, by NIH
      officials and other stakeholders, including public members
    o NIH review system is the envy of the world!
 Very competitive!
    o Only 1 in 4 proposals funded in the 2008 fiscal year
    o 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
 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
   o 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
 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
   o Makes it imperative to support a broad range of scientific research
   o Too risky for the private sector, federal funding is critical for
Evolution of Research to

   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
      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
                                  disease, largely
                                  as a result of
                                  NIH research.”
           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
 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
                  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
              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

This translates
to almost
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 Maine

   Researchers at Maine Medical Center Research Institute
    discovered that re-narrowing of arteries following balloon
    angioplasty can be halted by copper chelation therapy
   Maine Medical Center was first in the state to use a computed
    tomography imaging system to diagnose cardiovascular disease
   Jackson Lab researchers discovered new information about the
    mechanisms of Philadelphia-positive (Ph+) leukemias that will
    affect how they are treated clinically
   Scientists at Jackson Lab found a gene mutation that leads to
    cerebral hemorrhaging and a condition known as porencephaly in
    newborn mice, which could have implications for humans
           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 essential

   Diminished investment in NIH = loss of
talented researchers = missed opportunities
        = delays in medical progress
       Maine’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

 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 Snowe and Collins, 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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