Sharing a Story of Hope

Whether by snail mail, email, or social media, it’s the time of year for catching up with family and friends. As NIH Director, I’m also fortunate to hear from some of the amazing people who’ve been helped by NIH research. Among the greetings to arrive in my inbox this holiday season is this incredible video from a 15-year-old named Aaron, who is fortunate enough to count two states—Alabama and Colorado—as his home.

As a young boy, Aaron was naturally athletic, speeding around the baseball diamond and competing on the ski slopes in freestyle mogul. But around the age of 10, Aaron noticed something strange. He couldn’t move as fast as usual. Aaron pushed himself to get back up to speed, but his muscles grew progressively weaker.

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Creative Minds: Designing Personalized Clinical Trials

Karina Davidson

Karina Davidson/Jörg Meyer

It might have been 25 years ago, but Karina Davidson remembers that day like yesterday. She was an intern in clinical psychology, and two concerned parents walked into the hospital with their troubled, seven-year-old son. The boy was severely underweight at just 37 pounds and had been acting out violently toward himself and others. It seemed as though Ritalin, a drug commonly prescribed for Attention Deficit Disorder, might help. But would it?

To find out, the clinical team did something unconventional: they designed for the boy a clinical trial to test the benefit of Ritalin versus a placebo. The boy was randomly assigned to take either the drug or placebo each day for four weeks. As a controlled study, neither clinical staff nor the family knew whether he was taking the drug or placebo at any given time. The result: Ritalin wasn’t the answer. The boy was spared any side effects from long term administration of a medication that wouldn’t help him, and his doctors could turn to other potentially more beneficial approaches to his treatment.

Davidson, now an established clinical psychologist at the Columbia University Irving Medical Center, New York, wants to take the unconventional approach that helped this boy and make it more of the norm in medicine. With support from a 2017 NIH Director’s Transformative Research Award, she and her colleagues will develop three pilot computer applications—or digital platforms—to help doctors conduct one-person studies in their offices.

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How Can You Take Part in Clinical Research? Looking Beyond “First in Human”


For a remarkable journey through the front lines of clinical research, I’d like to invite you to join me in viewing First in Human, which premieres tonight at 9 p.m. ET on the Discovery Channel. This three-part docuseries, to be aired August 10, 17, and 24, provides an unprecedented look inside the NIH Clinical Center here in Bethesda, MD, following four of the many brave patients who’ve volunteered to take part in the clinical trials that are so essential to medical breakthroughs.

You’ll learn about what it’s like to take part in an experimental trial of a new treatment, when all standard options have failed. You’ll see that the NIH Clinical Center and its staff are simply amazing. But keep in mind that you don’t have to travel all the way to Bethesda to be part of outstanding, NIH-funded clinical research. In fact, we support clinical trials all across the country, and it’s often possible to find one at a medical institution near your home. To search for a clinical trial that might be right for you or a loved one with a serious medical problem, try going to ClinicalTrials.gov, a web site run by NIH.

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You’ll Want to See This! “First in Human” Debuts August 10

For over 60 years, the NIH Clinical Center—the world’s largest hospital dedicated to clinical research—has been at the forefront of developing treatments for our most deadly and damaging diseases. It’s here at our “House of Hope” in Bethesda, MD, where, among many other medical firsts, chemotherapy was first used to treat cancerous tumors, gene therapy underwent its first human tests, surgeons first successfully replaced the heart’s mitral valve, and the first anti-viral drug for HIV/AIDS met with early success.

Now, in a Discovery Channel documentary called First in Human, millions of people all around the globe will get a chance to see the doctors, nurses, and other staff of NIH’s remarkable research hospital in action. Narrated by Big Bang Theory star Jim Parsons, the three-part series debuts at 9 p.m.-11 p.m., ET, Thursday, August 10. The second and third segments will air at the same time on August 17 and 24. For a sneak peak, check out the video clip above!

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Find and Replace: DNA Editing Tool Shows Gene Therapy Promise

Neutrophil being edited with CRISPR/Cas9

Caption: This image represents an infection-fighting cell called a neutrophil. In this artist’s rendering,  the cell’s DNA is being “edited” to help restore its ability to fight bacterial invaders.
Credit: NIAID, NIH

For gene therapy research, the perennial challenge has been devising a reliable way to insert safely a working copy of a gene into relevant cells that can take over for a faulty one. But with the recent discovery of powerful gene editing tools, the landscape of opportunity is starting to change. Instead of threading the needle through the cell membrane with a bulky gene, researchers are starting to design ways to apply these tools in the nucleus—to edit out the disease-causing error in a gene and allow it to work correctly.

While the research is just getting under way, progress is already being made for a rare inherited immunodeficiency called chronic granulomatous disease (CGD). As published recently in Science Translational Medicine, a team of NIH researchers has shown with the help of the latest CRISPR/Cas9 gene-editing tools, they can correct a mutation in human blood-forming adult stem cells that triggers a common form of CGD. What’s more, they can do it without introducing any new and potentially disease-causing errors to the surrounding DNA sequence [1].

When those edited human cells were transplanted into mice, the cells correctly took up residence in the bone marrow and began producing fully functional white blood cells. The corrected cells persisted in the animal’s bone marrow and bloodstream for up to five months, providing proof of principle that this lifelong genetic condition and others like it could one day be cured without the risks and limitations of our current treatments.

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