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Breakthroughs in Bioscience


Title: Science Fair Project Author: cwolinetz Last modified by: Lawrence Green Created Date: 6/16/2006 2:42:23 PM Document presentation format: On-screen Show (4:3) – PowerPoint PPT presentation

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Title: Breakthroughs in Bioscience

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

New Jersey
National Institutes of Health (NIH)
  • Nations 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 States

Research Enterprise Is Critical to New Jerseys
  • The state of N.J. received more than 246 million
    overall from NIH in FY 11
  • New Jersey is home to 19 of the 25 largest
    pharmaceutical and medical technology companies
    in the world, including Aventis Pharmaceuticals,
    Bristol-Myers Squibb, Johnson Johnson, Merck
    Co., and Roche Pharmaceuticals
  • N.J. biotech firms contributed 1.3 billion in
    income to state residents in 2003, providing
    nearly 8,000 full-time jobs
  • New Jersey is the first state to invest public
    funds in research using human embryonic stem

NIH Grants Support Many Research Programs in NJ
  • 3.7 million grant awarded to Rutgers professor
    to investigate genetic basis of autism
  • School of Pharmacy professor was awarded 1.7
    million MERIT award to continue research into
    nanotechnology for treatment of HIV
  • A Rutgers-led Consortium was awarded 6.5 million
    for genomic research to help develop technologies
    to analyze molecules coded by the Human Genome
  • NIH funding helps support the Rutgers Center for
    Cancer Prevention Research, which serves as a
    focal point for basic cancer research in the
  • At Princeton, NIH funding created the Center for
    Quantitative Biology, which investigates complex
    biological systems

NIH Saving Lives Through Science
  • Current annual budget (FY2012) of around 30.6B
  • Greater than 80 distributed throughout the
  • 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//
  • Portfolio of basic, translational, and clinical

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
  • Researchers write grant proposals to compete for
  • 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
  • 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
  • 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

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
  • 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 cardiovascular 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

  • 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
  • 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
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

  • 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
  • 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 1970s 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

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 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
  • 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

Urgent Need for Prosthetics Research and
  • 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

Future Directions
  • After amputation, the nerves controlling the
    missing limb remain active
  • Scientists have developed superfine electrode
    arrays to connect these nerves with prosthetic
  • This will allow amputees to control and
    sense their prosthetics intuitively, making them
    feel more like their original limbs

NIH-Funded Discoveries at N.J. Schools
  • Determined the cause of Niemann-Pick type C2
    disease (NP-C2), a fatal hereditary disorder
    characterized by neurodegeneration and death by
    age 3
  • Developed platform technology composed of
    natural, biodegradable materials for delivery of
    drugs, vaccines and gene therapy
  • Research teams substantiated the link between
    autism and EN2, a gene important in central
    nervous system development and also implicated a
    gene called CAPON in schizophrenia
  • Found that herbal remedies and supplements may
    have adverse effects on men undergoing treatment
    for prostate cancer

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
N.J.s Congressmen Need to Advocate for NIH
  • 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 HelpWorking Together for NIH
  • Contact Senators Lautenberg and Menendez, 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
  • 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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