Scientists Call for National Microbiome Moonshot to Tackle Chronic Disease, Starting With Early Life

Twelve of the world's leading microbiome researchers were invited to a roundtable convened by the U.S. Department of Health and Human Services. Their task was to design an ambitious plan for research and public education on the trillions of microorganisms that live in and on the human body. The result, laid out in a commentary in Nature Medicine, is a call for a microbiome "moonshot" aimed at finding out how certain microbes drive chronic disease and how others might be used to repair it.

Two of the twelve are Rutgers microbiologists Martin Blaser and Maria Gloria Dominguez-Bello. For years they and other researchers have suspected that disruptions to the microbiome help explain why obesity, asthma and food allergies rose at nearly the same time. Antibiotics, cesarean births, infant formula and diets heavy in processed foods can all change those microbial communities, which help shape immunity and metabolism. The evidence for the connection has grown. But showing cause and effect, rather than mere correlation, requires large, lengthy controlled experiments in humans, and those need more funding than researchers now have.

The money is not there yet. HHS has not committed extra funds to microbiome research, though the steady stream of interesting study results was enough for the agency to host the roundtable. The plan also fits a stated priority of the department's leadership: Secretary Robert F. Kennedy Jr. called for making microbiome research a national moonshot at a symposium in May. The panel's document shows what such an effort would involve. Putting it into practice would depend on winning funding for specific study and education proposals that fit within the general plan.

The plan sets out 10 priorities, grouped into four areas: building scientific foundations, advancing clinical applications, pursuing high-impact opportunities, and providing education. One priority is standardizing research methods worldwide, so that results can be shared and combined to reflect populations beyond the wealthy Western nations where most studies have been done.

The authors single out pregnancy and early childhood, especially the first 1,000 days, from pregnancy through a child's second birthday. "The microbiome develops during this period, creating an opportunity to investigate whether protecting the initial development can improve health years later," said Dominguez-Bello, the Henry Rutgers professor of microbiome and health. Antibiotics in infancy and cesarean delivery alter microbiome development and correlate with metabolic and allergic conditions later in life. The authors propose following people from pregnancy through adolescence to test whether those disruptions actually cause later disease and, if they do, to test ways of preventing it.

The clinical record so far is narrow. The most practical applications are two treatments approved by the Food and Drug Administration, Rebyota and Vowst. Both contain microbes derived from donated human stool and are used to prevent recurrent Clostridioides difficile infection in adults after antibiotic treatment. Cancer is another direction, since gut microbes can influence how patients respond to immunotherapy. Researchers are also studying gut-brain links in Parkinson's and Alzheimer's diseases. The authors note that extending microbiome approaches to those conditions will require evidence specific to each one.

The plan also calls for new standards for evaluating and approving microbiome treatments while keeping safety standards intact. It proposes evaluating, and reporting to consumers, how medicines, food additives and environmental exposures affect the microbiome. Education is named as a step that can be taken almost immediately: aligning microbiome and nutrition science with medical training and school nutrition education.

Blaser, the commentary's lead author and director of Rutgers' Center for Advanced Biotechnology and Medicine, said, "We hope to move quickly, with detailed proposals for particular studies and programs in this next fiscal year." Dominguez-Bello described the agenda as short-term as well as medium- and long-term. What to watch is whether those detailed proposals arrive, and whether HHS attaches money to them.