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.
Tags: anti-HMGCR myopathy, antibodies, autoimmunity, childhood diseases, clinical research, clinical trials, immunology, intravenous immunoglobulins, IVIG, muscle, muscle diseases, muscular dystrophy, myopathy, statins
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.
Tags: 2017 NIH Director’s Transformative Research Award, All of Us, clinical trials, diabetes, digital platform, exercise, high blood pressure, light therapy, melatonin, N-of-1 trials, pain, personalized medicine, precision medicine, standard care, weight loss
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.
Tags: Clinical Center, clinical research, clinical trial recruitment, clinical trial registration, clinical trials, Discovery Channel, experimental medicine, First in Human, NIH Clinical Center, patients
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!