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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:

Clinical Breakthroughs

Prevention, Diagnosis, and Treatment of Human Disease

Mosquito feeding on a person.

Malaria Vaccine Found Safe and Protective

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.
Protection against malaria by intravenous immunization with a nonreplicating sporozoite vaccine.

Happy woman with a wicker basket full of fresh vegetables.

Gene Variants Predict Response to Breast Cancer Drugs

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.
Selective estrogen receptor modulators and pharmacogenomic variation in ZNF423 regulation of BRCA1 expression: individualized breast cancer prevention.

Woman listening to doctor.

Urine Test Detects Kidney Transplant Rejection

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 kidneys.
Urinary-cell mRNA profile and acute cellular rejection in kidney allografts.

Transmission electron micrograph of a lymphoblast. All rights reserved by Wellcome Images.

Technique Directs Immune Cells to Target Leukemia

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.
CD19-targeted T cells rapidly induce molecular remissions in adults with chemotherapy-refractory acute lymphoblastic leukemia.

Happy mature couple looking at view.

Medical Management Best to Prevent Second Stroke

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.
Aggressive medical treatment with or without stenting in high-risk patients with intracranial artery stenosis (SAMMPRIS): the final results of a randomised trial.

Doctor showing patient prescription bottle.

Genetic Testing Doesn’t Improve Warfarin Dosing

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.
A pharmacogenetic versus a clinical algorithm for warfarin dosing.

Infant watching someone while drinking from a bottle.

Eye Contact Declines in Young Infants with Autism

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.
Attention to eyes is present but in decline in 2-6-month-old infants later diagnosed with autism.

Doctor using a stethoscope to examine an overweight African-American woman

Duration of Obesity May Affect Heart Disease

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.
Association between duration of overall and abdominal obesity beginning in young adulthood and coronary artery calcification in middle age.

Promising Medical Advances

Findings with Potential for Enhancing Human Health

Image showing effects of concussion on the human brain.

Insights into Brain Injury

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.
Transcranial amelioration of inflammation and cell death after brain injury.

Image of tiny blood vessels.

Stem Cells Coaxed To Create Working Blood Vessels

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.
Self-organized vascular networks from human pluripotent stem cells in a synthetic matrix.

Two smiling women.

Genomic Analysis of Endometrial Tumors

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.
Integrated genomic characterization of endometrial carcinoma.

Rear view photo of a family walking together in a park.

Common Genetic Factors Found in 5 Mental Disorders

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.
Identification of risk loci with shared effects on five major psychiatric disorders: a genome-wide analysis.

Transmission electron micrograph of AIDS virus particles budding from an immune cell.

Vaccine Clears Away Monkey AIDS Virus

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 AIDS vaccine.
Immune clearance of highly pathogenic SIV infection.

Malassezia fungi.

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 and rheumatoid 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 bypass surgery.
Topographic diversity of fungal and bacterial communities in human skin. Expansion of intestinal Prevotella copri correlates with enhanced susceptibility to arthritis.
Intestinal microbiota metabolism of L-carnitine, a nutrient in red meat, promotes atherosclerosis. Gut microbiota from twins discordant for obesity modulate metabolism in mice. Commensal bacteria control cancer response to therapy by modulating the tumor microenvironment. The intestinal microbiota modulates the anticancer immune effects of cyclophosphamide. Conserved shifts in the gut microbiota due to gastric bypass reduce host weight and adiposity.

Bacterial cells.

Method Quickly Assesses Antibiotics

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.
Bacterial cytological profiling rapidly identifies the cellular pathways targeted by antibacterial molecules.

Photo of a medical team rushing a sick patient to the emergency ward.

Strategy May Improve Survival after Shock

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 outcomes.
Pancreatic digestive enzyme blockade in the intestine increases survival after experimental shock.

Insights from the lab

Noteworthy Advances in Basic Research

A mouse hippocampus.

Seeing Into the Brain

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.
Structural and molecular interrogation of intact biological systems.

Human glucagon receptor.

Structure of a Potential Diabetes Drug Target

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.
Structure of the human glucagon class B G-protein-coupled receptor.

Cerebrospinal fluid flowing through the brain.

How Sleep Clears the Brain

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.
Sleep drives metabolite clearance from the adult brain.

Stem-cell-derived sensory hair cells.

Technique Forms Working Inner Ear Cells

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 ear.
Generation of inner ear sensory epithelia from pluripotent stem cells in 3D culture.

Green cells with red clusters.

Study Reveals New Targets for Parkinson’s Disease

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 involve mitochondria.
High-content genome-wide RNAi screens identify regulators of parkin upstream of mitophagy.

Grapefruits.

Therapeutic Nanoparticles from Grapefruit Juice

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.
Delivery of therapeutic agents by nanoparticles made of grapefruit-derived lipids.

Close-up photo of a woman speaking.

Understanding How We Speak

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 disorders.
Functional organization of human sensorimotor cortex for speech articulation.

Escherichia coli.

Scientists Recode Organism's Genome

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.
Genomically recoded organisms expand biological functions.

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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.

ISSN 2375-9593

This page last reviewed on December 23, 2013

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