THURSDAY, OCTOBER 17, 2024

How Groundbreaking NIH Research is Expanding to Birth-to-Four-Year-Olds

For nearly the first decade of the National Institutes of Health’s “All of Us” Research Program — aimed at increasing diversity in genetic research — a major component was missing: kids.

“Children are approximately 24% of our population in the U.S. and 100% of our future,” Dr. Sara Van Driest, director of pediatrics for NIH’s All of Us Program, told hosts Mark Masselli and Margaret Flinter. “In order to provide them with the very best care for the future and have them benefit from this resource, we need to include them.”

The journey is personal for Katrina Yamazaki, Ph.D., principal investigator for Community Health Center, Inc., in Connecticut, a partner organization in All of Us. Yamazaki and her husband adopted three boys through the foster care system.

“We don’t know a whole lot about their … biological families’ medical history,” Yamazaki said. “The idea that this program will one day be able to provide some of [that] missing information to me and my husband, in order to become health advocates for our children is really important to me.”

The NIH in August began limited enrollment in the program for children age 4 and under.

“We started with that youngest age group so we can follow them the longest,” Van Driest said.

Community Health Center, Inc., for its part, is partnering with community-based organizations such as the Hartford Public Library to build trust, raise awareness of the project and make a fun atmosphere through activities.

All of Us intends to change what might be seen as a “one-size-fits-all” approach to health care. It aims to encompass 1 million individuals of diverse backgrounds but doesn’t focus on particular diseases or conditions, Van Driest said. The diversity, too, goes beyond culture, touching geography, age and socioeconomic status.

“One of the goals of research is to connect the dots,” she said. Given the scope of the project, “there will be so many dots that we’ll be able to connect,” Van Driest said.

 “If we fail to include a group of individuals or an aspect of diversity, we miss out on that uniqueness. That limits us in what we’re able to understand about humanity in general,” she said. “It also limits research and learning about that group of individuals. And it limits downstream how clinicians can care about individuals and give them the very best possible outcomes.”

THURSDAY, MARCH 13, 2025

Michael J. Fox’s Foundation Makes Parkinson’s Breakthrough

Emmy award-winning actor Michael J. Fox shocked the world when he announced over 25 years ago that he had been diagnosed with Parkinson’s disease. Since then, he’s devoted his talents to eliminating Parkinson’s in our lifetime.

Researchers with The Michael J. Fox Foundation now have impressive progress to report. They’ve developed a biomarker test to measure the protein alpha synuclein that’s helping to diagnose Parkinson’s with more than 90% accuracy, even proving effective at identifying people who may later develop the disease.

“I think this is going to be a game changer for clinical trials and for patient care in the future,” says Samantha Hutten, Ph.D., a neuroscientist and vice president of translational research at the foundation. “But right now it is really a research tool that can be used to inform research and also how we design clinical trials.”

A positive test, for example, may not be able to tell patients which type of alpha synuclein disease they have, whether it’s Parkinson’s, Lewy body dementia or multiple systems atrophy.

“I think there’s huge excitement around it, particularly about the impact on clinical trials,” says Rebecca Miller, Ph.D., an associate professor at Yale University’s School of Medicine who was diagnosed with Parkinson’s in 2013 at age 38. “We’re all looking for a drug that will really be disease-modifying.”

Hutten tells hosts Mark Masselli and Margaret Flinter that the biomarker test is an “all-comers” study, meaning patients can participate if they’ve been diagnosed or have family with Parkinson’s, or have risk factors like loss of sense of smell or REM sleep behavior disorder.

The test comes at a crucial time: the global rate of Parkinson’s has doubled since Fox first revealed his condition.