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Gain Without Pain: New Clues for Analgesic Design

Posted on by Dr. Francis Collins

A mouse and a scorpion sharing a space and facing nose-to-nose.

Photo Credit: Matthew Rowe, Michigan State University

If you’re a southern grasshopper mouse, nothing beats a delicious snack of scorpion. But what, you might ask, prevents that from being a painful or even fatal event?  Well, this native of the Arizona desert has evolved an amazing resistance to the stings of the bark scorpion—stings so painful and toxic they kill house mice and other rodents of similar size.

Why am I sharing this bit of natural history? Well, it turns out that by studying the grasshopper mouse and its unusual diet, NIH-funded researchers at the Indiana University School of Medicine and collaborators at the University of Texas, Austin, have identified a new target on nerve fibers that could lead to more effective and less addictive pain medications for humans.


Yeast Reveals New Drug Target for Parkinson’s

Posted on by Dr. Francis Collins

Untreated yeast shows clumps of brightly colored spots, while treated yeast are more even in their color.
Caption: Left, yeast sick with too much α-synuclein, a protein that is implicated in Parkinson’s disease. Right, the same yeast cells after a dose of NAB, which seems to reverse the toxic effects of α-synuclein.
Credit: Daniel Tardiff, Whitehead Institute

Many progressive neurodegenerative disorders like Alzheimer’s, Huntington’s, and Parkinson’s disease, are characterized by abnormal clumps of proteins that clog up the cell and disrupt normal cellular functions. But it’s difficult to study these complex disease processes directly in the brain—so NIH-funded researchers, led by a team at the Whitehead Institute for Biomedical Research, Cambridge, MA, have turned to yeast for help.

Now, it may sound odd to study a brain disease in yeast, a microorganism long used in baking and brewing. After all, the brain is made up of billions of cells of many different types, while yeast grows as a single cell. But because the processes of protein production are generally conserved from yeast to humans, we can use this infinitely simpler organism to figure out what the proteins clumps are doing and test various drug candidates to halt the damage.


A White Halloween Costume That’s Not a Ghost

Posted on by Dr. Francis Collins

Photo of a tall man in glasses wearing a tie looking down at a young boy wearing play glasses, a tie, a white coat, and a stethoscope.

Caption: Dr. Jay Rubinstein and his mini-me, Landon Browne
Credit: Courtesy of Mary Guiden, Seattle Children’s Hospital

What costume to wear for Halloween? For many kids, it’s a difficult choice, but not so for 7-year-old Landon Browne. This year, he’s not going as a zombie or an action hero—he’s going as an NIH-funded researcher!

Landon, who was born almost completely deaf, has decided to dress up as his real-life superhero: Jay Rubinstein, M.D., Ph.D., a physician-scientist at Seattle Children’s Hospital who performed the surgeries that have enabled the boy to hear.


Basic Science Finds New Clue to Bipolar Disorder

Posted on by Dr. Francis Collins

Greek comedy tragedy play masks -- altered with ATCGs to create the shadows and double helix to create the ties.We know that heredity, along with environment, plays an important role in many mental illnesses. For example, studies have revealed that if one identical twin has bipolar disorder, the chance of the other being affected is about 60%. There are similar observations for autism, schizophrenia, and major depression. But finding the genes that predispose to these conditions has proven very tricky.

Now, an NIH-funded team at Baylor College of Medicine has demonstrated for the first time that extra copies of a gene that codes for a protein called Shank3 can cause manic episodes similar to those seen in some types of bipolar disorder [1]. The researchers initially tested their hypothesis in mice and then, building upon those findings, went on to find extra copies of the SHANK3 gene in two human patients—one with seizures and attention deficit hyperactivity disorder and another with seizures and bipolar disorder.


Snapshots of Life: Sore Throat as Art

Posted on by Dr. Francis Collins

Scanning electron micrograph of Strep A bacteria

Credit: Vincent A. Fischetti, The Rockefeller University

Most parents and kids wouldn’t consider strep throat the subject of high art. But the judges of the Federation of American Societies for Experimental Biology’s 2013 BioArt competition think it is. In this silver-toned scanning electron micrograph, you can see hundreds of tiny spheres—bacteria called Group A streptococci—attached to a human pharyngeal (throat) cells grown in a lab dish. These bacteria are responsible for a very nasty type of pharyngeal inflammation commonly known as strep throat. Strep infections are usually treated with antibiotics; left untreated, they can lead to rheumatic fever, rheumatic heart disease, and even kidney disease.


Different Cancers Can Share Genetic Signatures

Posted on by Dr. Francis Collins

Cancer types floating over a cell with unraveling DNA

NIH-funded researchers analyzed the DNA of these cancers.

Cancer is a disease of the genome. It arises when genes involved in promoting or suppressing cell growth sustain mutations that disturb the normal stop and go signals.  There are more than 100 different types of cancer, most of which derive their names and current treatment based on their tissue of origin—breast, colon, or brain, for example. But because of advances in DNA sequencing and analysis, that soon may be about to change.

Using data generated through The Cancer Genome Atlas, NIH-funded researchers recently compared the genomic fingerprints of tumor samples from nearly 3,300 patients with 12 types of cancer: acute myeloid leukemia, bladder, brain (glioblastoma multiforme), breast, colon, endometrial, head and neck, kidney, lung (adenocarcinoma and squamous cell carcinoma), ovarian, and rectal. Confirming but greatly extending what smaller studies have shown, the researchers discovered that even when cancers originate from vastly different tissues, they can show similar features at the DNA level


Driving Innovation and Creativity with High Risk Research

Posted on by Dr. Francis Collins

Girl in a lab

Caption: One of the many faces of NIH-supported innovation, Stanford’s Christina Smolke is exploring how synthetic biology and microbes can be used to produce new drugs. She is a 2012 Pioneer Award winner.
Credit: Linda Cicero/Stanford News Service

High-risk research isn’t for the faint of heart. It’s for fearless researchers who envision and develop innovative projects with unconventional approaches that, if successful, may yield great leaps in our understanding of health problems and/or biological mechanisms. It takes nerve and creativity to conceive such projects—and, often, special support to bring them to fruition.  And, as the name implies, there is a significant chance of failure.


One Nation in Support of Biomedical Research?

Posted on by Dr. Sally Rockey and Dr. Francis Collins

Graph of % change in scientific R&D spending from 2012 to 2013 in China (15%), Germany (5%), S. Korea (5%), Canada (-3%), and U.S. (15%)“It was the best of times, it was the worst of times.” Until recently, we’d never have dreamed of mentioning the famous opening line of Charles Dickens’ Tale of Two Cities in the context of U.S. biomedical research. But now those words ring all too true.

The “best of times” reflects the amazing technological advances and unprecedented scientific opportunities that exist right now. We’ve never had a better chance to make rapid progress in preventing, diagnosing, and curing human disease. But the “worst of times” is the other reality: NIH’s ability to support vital research at more than 2,500 universities and organizations across the nation is reeling from a decline in funding that threatens our health, our economy, and our standing as the world leader in biomedical innovation.


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