Breakthroughs in Bioscience

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Breakthroughs in Bioscience

• From NIH-Funded Basic Research to Improved Health

Maryland

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National Institutes of Health (NIH)

• Nations medical research agency
• Funds the science that leads to medical
  advancement
• Campus in Bethesda, MD but most funding is
  distributed to university researchers throughout
  the United States
• The state of Maryland received more than 1.3B in
  NIH awards for FY2011

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Research Enterprise Is Critical to Marylands
Economy

• NIH directly employs more than 18,000 in Maryland
• Every year, Johns Hopkins (JHU) Institutions
  generate about 10B in economic activity for the
  state
• JHU directly and indirectly supports more than
  100,000 jobs one out of every 29 jobs in the
  state
• In 2007 alone, JHU received 43 U.S. patents and
  licensed 61 technologies for commercial
  development
• More than 50 start-up companies have sprung from
  JHU technologies
• The University of Maryland (UM) Medical System
  contributes nearly 3.5B in economic activity to
  the state, supporting more than 28,000 jobs
• Maryland has the 4th largest cluster of
  biotechnology companies in the U.S.
• BioMaryland 2020, a 10-year 1.3B strategic
  initiative, includes
• Creation of the Maryland Biotechnology Center
• 59M over 3 years for Marylands Stem Cell
  Research Fund
• 18M over 3 years for the Biotechnology Tax
  Credit
• 9M for nanobiotechnology research grants

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NIH Grants Support Many Programs in Maryland

• JHU received an award of more than 100M to
  establish the Institute for Clinical and
  Translational Research, part of a national
  consortium to improve the development of basic
  research into clinical applications
• A 2.5M award to a JHU infectious disease expert
  will support development of a virus surveillance
  system
• NIGMS is funding research at JHU on how to speed
  healing and reduce scarring in burn wounds
• NIAID granted the UM School of Medicine 9.3M to
  evaluate compounds to prevent, reduce, or treat
  symptoms of radiation exposure
• A Pathway to Independence grant was awarded to an
  obesity researcher at JHU to study effects of
  fetal distress and nutrition on behavior and
  development

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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 in Maryland

• JHU scientists identified several unique
  variations in the human genetic code among
  patients with ALS, narrowing the search for the
  gene which accounts for non-inherited forms of
  the disease
• Scientists at JHU discovered mutations in a
  family of genes linked to more than a quarter of
  colon cancers as well as several other common
  cancers such as breast and lung cancer, revealing
  options for creating therapies tailored for
  individual patients
• JHU Childrens Center discovered one of the first
  methods of gene therapy for cystic fibrosis
• Researchers at JHU discovered a potent class of
  compounds that selectively inhibits a protein
  known as MetAP, thereby blocking the growth of
  tuberculosis
• UM School of Medicine co-discovered a common
  genetic variation that could determine whether a
  person with heart failure would benefit from
  beta-blockers
• UM School of Medicine created E. coli vaccine

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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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Marylands 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

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We Need Your HelpWorking Together for NIH

• Contact Senators Mikulski and Cardin, 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?
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