NIH Research Matters
December 23, 2013
2013 Research Highlights
With NIH support, scientists across the country and the world conduct wide-ranging research to improve the health of the nation. Groundbreaking NIH-funded research often receives top scientific honors. In 2013, all 3 recipients of the Nobel Prize in Physiology or Medicine, and all 3 awardees of the Nobel Prize in Chemistry, received NIH funding at different times in their careers. Four NIH-funded scientists also won awards from the Lasker Foundation in 2013. Here's just a small sampling of the research accomplishments made by NIH-supported scientists in 2013:
Prevention, Diagnosis, and Treatment of Human Disease
Malaria kills hundreds of thousands of people each year, most of
them young children in Sub-Saharan Africa. While scientists have
made significant gains in understanding, treating, and preventing
the disease, a vaccine has remained elusive. NIH researchers reported
that a candidate malaria vaccine is safe and protected against infection
in an early-stage clinical trial.
Women at risk for breast cancer may take certain types of medications
that reduce the chance of developing the cancer. But in rare cases,
the drugs can cause dangerous side effects. Many women decide that
the chance of success doesn’t outweigh the risks. An international
research team, with NIH support, found genetic variations that can
be used to identify women who are most likely to benefit from this
potentially life-saving strategy—and who should avoid it.
After a kidney transplant, patients must take medications with toxic
side effects to keep their immune system from attacking the new organ.
If doctors could track rejection status over time, they could adjust
drug doses for more effective treatment. NIH-funded researchers found
that certain molecules in urine can provide an early sign of transplant
rejection. The test could allow doctors to act early to protect transplanted
When adult patients with B-cell acute lymphoblastic leukemia have
remission followed by relapse, the prognosis is poor. An NIH-funded
team used a type of targeted immunotherapy to induce remission in
5 patients with this aggressive form of leukemia. The early results
of the ongoing trial highlight the potential of this approach.
After a stroke, treatment for patients at high risk for a second
stroke typically involves a medical program that includes blood-thinning
medications and control of blood pressure and cholesterol. In hopes
of improving the odds, doctors over the past decade began to also
use an intracranial artery stent. An NIH-funded clinical trial confirmed
earlier findings that stenting adds no benefits over aggressive medical
treatment alone for most of these patients.
Warfarin is often prescribed to prevent blood clots in people with
certain conditions. But determining the best dose can be tricky.
Too much can cause excess bleeding; too little can lead to dangerous
clots. Past research suggested that adding genetic data to clinical
information would improve initial dosing. But an NIH-funded study
contradicted that result, highlighting the importance of using clinical
trials to assess the role of genetics in optimizing treatments.
Autism symptoms first appear during early childhood, and a definitive
diagnosis can often be made by 2 years of age. Scientists have long
been searching for ways to identify the condition at even younger
ages, since outcomes tend to be better with earlier intervention.
NIH-funded researchers found evidence that infants later diagnosed
with autism show a steady decline in eye contact beginning as young
as 2 months of age.
Past research has linked obesity to heart disease risk. But few
studies have examined how the duration of obesity affects heart disease.
NIH researchers found that how long a young adult is obese may affect
that person’s heart disease risk in middle age. The finding suggests
that not only preventing but also delaying the onset of obesity can
help reduce heart disease later in life.
Promising Medical Advances
Findings with Potential for Enhancing Human Health
Concussions can have serious and lasting effects. However, the specific
damage that occurs in affected brain tissue hasn’t been well understood.
A study by NIH researchers provided insight into the damage caused
by mild traumatic brain injury and suggested approaches for reducing
its harmful effects.
In an NIH-funded study, scientists were able to direct human stem
cells to form networks of tiny blood vessels that can connect to
the existing circulation in mice. The finding might assist future
efforts to repair and regenerate tissues and organs, which need an
adequate blood supply to grow and survive.
Pathologists currently classify endometrial tumors by examining
tissue under a microscope. A comprehensive genomic analysis of nearly
400 endometrial tumors revealed 4 novel endometrial tumor subtypes
and also found similarities to other cancers. The findings, by an
NIH-funded research network, suggest that genomic classification
of endometrial tumors could help guide treatment strategies.
Autism, attention deficit hyperactivity disorder (ADHD), bipolar
disorder, major depression, and schizophrenia were traditionally
thought of as distinct mental disorders. However, their symptoms
can overlap, making it difficult to distinguish between them. An
international research consortium funded by NIH discovered that these
disorders share certain genetic glitches. The finding may point to
better ways to diagnose and treat these conditions.
HIV, which causes AIDS in people, and the similar monkey virus SIV
are thought to cause permanent infections in the body. Current therapies
can control but not eliminate the virus. In an NIH-funded study,
an experimental vaccine triggered a lasting immune attack in monkeys
that eliminated all traces of SIV infection after a year or more.
The finding points to a new strategy in the search for an effective
The Human Microbiome
The human body hosts trillions of microbes. We’re now gaining
a better understanding of the many roles that microbial communities
and their genes—collectively known as the microbiome—play
in human health and disease. NIH-funded scientists
surveyed all the fungi
living on human skin. They uncovered links between gut microbes
arthritis; discovered interactions among diet, gut microbes,
and both heart
disease and obesity;
and found that microbes may also influence the effectiveness of cancer
therapy and gastric
Decades of widespread antibiotic use have encouraged the spread
of bacteria with resistance to multiple antibiotics. To combat these
multidrug-resistant bacteria, researchers have been searching for
new classes of antibiotics that work by different mechanisms than
current drugs. NIH-funded scientists developed an innovative method
to quickly identify antibiotics that can treat multidrug-resistant
bacteria—and reveal how these bacteria-killing medications work.
Shock is a life-threatening condition in which blood pressure drops
and not enough blood and oxygen can get to organs. Inflammation has
been strongly linked with shock, and past research suggests that
this inflammation involves the digestive system. An NIH-funded study
of rats found that blocking digestive enzymes in intestines
increases survival, reduces organ damage, and improves recovery after
shock. The approach may lead to new therapies to improve patient
Insights from the lab
Noteworthy Advances in Basic Research
Scientists seeking to understand the brain’s fine structure and
connections have been faced with tradeoffs. To examine deeply buried
structures, they had to cut brain tissue into extremely thin sections.
This deforms the tissue and makes it difficult to study brain wiring
and circuitry. NIH-funded scientists developed a new technique to
preserve the brain’s 3-D structure down to the molecular level with
a hydrogel. It allows for study of the brain’s inner workings at
a scale never before possible.
People with diabetes have difficulty maintaining blood glucose levels.
The hormones insulin and glucagon are used by the body—and also used
as medications—to help keep blood glucose in a safe range. An international
team of researchers, funded in part by NIH, determined and analyzed
the structure of the human glucagon receptor. The results may aid
in the development of drugs for diabetes and other metabolic disorders.
Sleep is important for storing memories, and also has a restorative
function. Sleep helps reasoning, problem-solving, and other functions.
However, the mechanisms behind these benefits have been unknown.
An NIH-funded study in mice suggests that sleep helps restore the
brain by flushing out toxins that build up during waking hours though
a special series of channels in the brain.
Specialized cells in the inner ear detect head movements, gravity,
and sound. Researchers know the general scheme of inner ear development,
but deeper knowledge will be critical for developing novel therapies
for hearing loss and balance disorders. Using an innovative 3-D culture
system, NIH-funded researchers were able to coax mouse embryonic
stem cells to form complex cells and structures seen in the inner
Defects in mitochondria, our cells’ biological power plants, have
been associated with certain neurological disorders, including Parkinson’s
disease, Charcot-Marie-Tooth syndrome, and the ataxias. NIH scientists
used a novel approach, involving a protein tied to Parkinson’s disease,
to identify dozens of genes that may contribute to disorders that
Nanoparticles are emerging as an efficient tool for drug delivery.
Microscopic pouches of synthetic lipid can protect drug molecules
within the body and deliver them to specific cells. However, these
nanoparticles pose obstacles, including potential toxicity, environmental
hazards, and large-scale production costs. NIH-funded researchers
made nanoparticles from grapefruits and used them to deliver targeted
drugs to treat cancer in mice. The technique may prove to be a safe
and inexpensive alternative.
Speech disorders, such as stuttering, affect roughly 5% of children
by the first grade. The underlying causes of most speech disorders,
however, aren’t well understood. The process of speaking is one of
the most complex actions humans perform. Scientists funded by NIH
revealed the patterns of brain activity that produce human speech.
The research may one day lead to new methods for treating speech
Living microbes can quickly and reliably produce proteins, the building
blocks of the cell. This ability has long been harnessed to produce
conventional proteins, such as insulin, for medical use. Synthetic
biology seeks to redesign natural biological systems for new purposes.
NIH-funded researchers developed a method to recode a bacterium’s
genome to incorporate synthetic non-standard amino acids into its
proteins. The technique can potentially turn microbes into efficient
living factories that make novel compounds.
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About NIH Research Matters
Editor: Harrison Wein, Ph.D.
Assistant Editors: Vicki Contie, Carol Torgan, Ph.D.
NIH Research Matters is a weekly update of NIH research highlights from the Office of Communications and Public Liaison, Office of the Director, National Institutes of Health.