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Senator Mikulski during her tour of NCATS

Caption: Here I am with Senator Barbara Mikulski (center) and NCATS Director Chris Austin (right). Credit: NIH

Alzheimer’s disease research is among the many areas of biomedical science that Senator Barbara Mikulski has championed during her nearly 40 years on Capitol Hill. And it’s easy to understand why the Senator is concerned: an estimated 5 million Americans age 65 and older have Alzheimer’s disease, and those numbers are expected to rise exponentially as the U.S. population continues to age.

So, I was thrilled to have some encouraging progress to report last week when Senator Mikulski (D-MD) paid a visit to NIH’s National Center for Advancing Translational Sciences (NCATS) in Gaithersburg, MD. After a whirlwind tour of the cutting-edge robotics facility for high throughput screening of small molecules, she joined me and NCATS Director Dr. Chris Austin in announcing that, thanks to an innovative public-private partnership, an experimental drug originally developed to fight cancer is now showing promise against Alzheimer’s disease.


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Photo of parkin protein (green) that tags damaged mitochondria (red)

Caption: NIH scientists used RNA interference to find genes that interact with the parkin protein (green), which tags damaged mitochondria (red). Mutations in the parkin gene are linked to Parkinson’s disease and other mitochondrial disorders.
Credit: Richard J. Youle Laboratory, NINDS, NIH

It would be terrific if we could turn off human genes in the laboratory, one at a time, to figure out their exact functions and learn more about how our health is affected when those functions are disrupted. Today, I’m excited to announce the availability of new data that will empower researchers to do just that on a genome-wide scale. As part of a public-private collaboration between the NIH’s National Center for Advancing Translational Sciences (NCATS) and Life Technologies Corporation, researchers now have access to a wealth of information about small interfering RNAs (siRNAs), which are snippets of ribonucleic acid (RNA) with the power to turn off a gene, or reduce its activity—in much the same way that we use a dimmer switch to modulate a light.


Posted In: Science

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At NIH, our job—in a nutshell—is to turn discoveries into health. Even if you’ve only read this blog a few times, you know we conduct and support basic science advances, clinical breakthroughs—and everything between. We call that “between” process, “translation.” And it takes sustained creativity, innovation, attention—and collaboration—to do it well.

Which brings me to today’s Institute of Medicine (IOM) Report on NIH’s Clinical and Translational Science Awards (CTSA) program [1]. (more…)

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Photo of Woody Guthrie and Led Belly walking down a dirt road

Photo from liner notes of the Folkways CD

Today is International Rare Disease Day. In honor of the occasion, I’d like to pay tribute to a few real-life heroes whose struggles have forever changed the landscape of rare disease research.

Folk singer Woody Guthrie is best known for his song, “This Land Is Your Land.” Written more than 70 years ago, “This Land” has taken its place among our nation’s great anthems, setting forth a vision of inclusiveness that has inspired generations of Americans to “sing along.” But the last couple of verses are often omitted. Here’s a version of one of them:

As I was walkin’I saw a sign there
And that sign said
no trespassin’
But on the other side … it didn’t say nothin’!
Now that side was made for you and me!

These verses brought into the foreground those whom society had marginalized. “This Land” reminded us of their existence, challenged us to live up to our ideals—and include all people in our best vision of ourselves.

Woody performing one version of “This Land”:

Even as he was singing about inclusiveness, Woody Guthrie was starting a long battle against a disease that increasingly cast him outside mainstream society: Huntington’s disease. In most cases—and as was indeed the case for Woody—symptoms of Huntington’s disease do not appear until adulthood. Gradually, this rare, inherited neurological disorder seizes control of its sufferer’s body, mind—and even voice. In 1965, 13 years after he was diagnosed, Woody fell mute. He had long since lost his ability to play guitar. Two years later, he died at the age of 55.


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Can you believe the average length of time from target discovery to approval of a new drug currently averages about 14 years? That is WAY too long. Even more shocking is that the failure rate exceeds 95 percent, and the cost per successful drug surpasses $2 billion, after adjusting for all of the failures. The National Center for Advancing Translational Sciences was specifically established one year ago to apply innovative scientific approaches to the bottlenecks in the pipeline. An example of game-changing innovation is the NCATS collaboration with the Defense Advanced Research Projects Agency (DARPA) to develop a biochip for testing drug safety. Devices like this and other tissue chips may someday reduce the amount of animal and human clinical trials necessary to determine if a drug works. That could be a huge step toward making drug development faster and cheaper—which is better for all of us.

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