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Breakthroughs in Bioscience
From NIH-Funded Basic Research
to Improved Health
Massachusetts
National Institutes of Health (NIH)
 Nation’s 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
 Massachusetts received over
$2.4B in funds from NIH in
FY2011
Research Is Critical to the
Massachusetts Economy
 Boston has a dense concentration of institutions –
MIT, Harvard, Massachusetts General Hospital,
Beth-Israel Deaconess Medical Center and
New England Medical Center
 The region has more than 4,900 life scientists
 Harvard is the region’s largest private employer
 provides/generates more than 48,500 jobs
 has a statewide economic impact of more
than $5.3B
 Boston is a center for venture capital investment in biotechnology –
the area has attracted over $2B of venture capital investment in
biopharmaceutical firms
 Many biotechnology firms in the area rely on local research, and more
than 20 companies were founded or co-founded by MIT graduates,
faculty or have licensed MIT biotechnology patents
Harvard Leverages the State’s Investment
with Extramural Research Dollars
 FY2008, Harvard spent a total of $660 million
on research funded from external sources
 an increase of 22.5% during the past five years
 more than 80% from federal grants
 The primary source of research funding is the
National Institutes of Health
 Harvard Medical School received over $200M in
FY2011
 Harvard School of Public Health received over
$120M in FY2011
 In the past few years, 24 companies with roots at
Harvard have collectively secured more than $280M
in financing
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?
• 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
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
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
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
(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
o Makes it imperative to support a broad range of scientific research
o 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 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
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
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
Cancer
 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
improved
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
treatment.”
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
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 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 at
Harvard
 Discovered the gene for Huntington’s Disease, allowing the
development of a diagnostic test for individuals at risk
 Developed the first vaccine for cholera
 Created the “OncoMouse,” a transgenic, highly cancer-prone mouse
that offers important insights into human cancers
 First successful leukemia remission (with the Children’s Hospital)
 New drug for bone marrow cancer was based on research by scientists
at Harvard Medical School
 Synthesized a cancer drug more powerful than any other in use
 A simple device was invented to quickly determine if a person is
having a heart attack, as well as the invention of the defibrillator,
cardioverter, and heart pacemaker
 Determined the value of estrogen replacement therapy for reducing
the risk of heart disease in women
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
Massachusetts’s 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
 The region has produced a number of research
breakthroughs that have driven the biotech industry,
including five discoveries that have earned Nobel prizes
We Need your Help:
Working Together for NIH
 Contact Senators Brown and Kerry, or the
Congressional Representative for your
district
 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
Want to Know More?
Please visit opa.faseb.org