A drug can slow down aging. Here’s how it will be tested on humans: Gunshots

A few years ago, Matt Kaeberlein diagnosed with frozen shoulder. “It was really bad,” he recalls. He wasn’t sleeping well and couldn’t throw a ball because of the pain. His doctor recommended physical therapy and told him he could take a year to become better.

Feeling frustrated, he decided to try rapamycin. In recent years, some high profile longevity scientists have started taking the drug in hopes of preventing age-related health problems. So far, it hasn’t been tested in people taking it for antiaging, but rapamycin has been shown to do prolongs the lifespan of mice.

“I decided to give it a try,” says Kaeberlein. It was his “first foray into biohacking” and he was very pleased with what happened next. “Within two weeks, 50% of the pain was gone,” he says. And at the end of 10 weeks, he had regained range of motion and the pain was completely gone.

“And he hasn’t come back,” he says.

Kaeberlein is no stranger to rapamycin. He is a biologist and its co-founder Dog Aging Project to study how rapamycin affects the health span of dogs. He is also its former manager Healthy Aging and Longevity Research Institute at the University of Washington.

Rapamycin was first approved by the FDA for use in transplant patients in the late 1990s. In high doses it suppresses the immune system. In low doses, Kaeberlein says it seems to help fight inflammation. It works by inhibiting a signaling pathway in the body called mTOR — which appears to be a key adjuster of life span and aging.

The drug is not approved for pain or antiaging, but some doctors prescribe rapamycin off-label with the goal of preventing age-related conditions. Kaeberlein et al about 300 were interviewed of these patients, taking low doses, and many report benefits.

But anecdotes are no substitute for science. To understand the risks and benefits of a drug, research is needed. And that’s where a dentist comes in.

Dr. Jonathan A, at the University of Washington, has received FDA approval to test rapamycin in patients with gum disease—a common condition that tends to accelerate with age. When he treats patients with gum disease, he says there’s not much he can do beyond cleaning and removing plaque—a buildup of bacteria. “All we do is put a bandage on,” he says. Its goal is to find and treat the underlying cause of the disease.

There is already some evidence from transplant patients that rapamycin can help improve oral health. And as part of the study, An and his colleagues will also measure changes in the participants’ microbiomes and their biological clocks.

The study will register participants over 50 years of age who have gum disease. They will take the drug, in various doses, at intervals for 8 weeks. An will then be able to determine whether the drug is safe and effective.

If rapamycin has a beneficial effect, he says, it will help prove that it is possible to target the root cause of the disease. “It’s really about targeting the biology of aging,” he says.

Dr. An believes that gum disease may be a kind of canary in the mine of age-related diseases. For example, gum disease is associated with a higher risk of heart disease, and maybe dementia as well. Scientists say it’s possible that bacteria in the mouth linked to periodontal disease cause inflammation, which can cause a “cascade” of damage to blood vessels, leading to heart or brain problems.

“If we can target this underlying biology, we predict that it may address many of these other underlying conditions,” says An.

Rapamycin is a generic drug, so drug companies have little incentive to fund new research. An and his collaborators have obtained a grant to conduct the trial, which could open the door to further studies to determine whether rapamycin can help prevent or slow other age-related diseases.

Eric Verdin, a physician who leads the Buck Institute for Research on Aging, says his group is raising money for more research on rapamycin. He says there are many unanswered questions, for example “what is the effect of different concentrations in a single dose?” And he wants to look for a “molecular signature” in people taking rapamycin. She wants to know more about dosages and intervals, as many off-label prescribers recommend taking the drug on and off.

Researchers are also working on other drugs that may work in similar ways, and there is a push for new drugs—or other interventions—that target biological aging. There is a new $100 millionXPRIZE Healthspan Competitionwith the aim of accelerating research in the field supported by Development and other funders.

Currently founder of XPRIZE Petros Diamantis, A doctor who writes about longevity says he takes rapamycin. “I do six milligrams every Sunday night, so once a week,” for three months, he explains. Then he takes a month off. “I believe that rapamycin – the way I use it – is safe and has more upside potential than downside,” he says.

Diamandis constantly monitors his body with many health metrics and acknowledges that it is difficult to determine the effect of rapamycin, considering all the other things he does to stay healthy, such as eating well, eliminating sugar, exercising daily and the priority of sleep.

His plan is to continue healthy lifestyle habits while supporting research into interventions and strategies that can help people add more healthy years to their lives.

Find Allison Aubrey on Instagram at @allison.aubrey and in X @AubreyNPR.

This story was edited by Jane Greenhalgh