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A generic drug used to treat transplant patients has been found to help extend the lifespan of some animals. Image: Guido Mieth/Getty Images
A few years ago, Matt Kaeberlein was diagnosed with frozen shoulder. “It was really bad,” he recalls. The pain was so bad he couldn’t sleep well or throw a baseball. Doctors recommended physical therapy and said it could take a year for him to recover.
Frustrated, he decided to try rapamycin, a drug that several prominent scientists in the field of longevity research have begun taking in recent years in the hope of warding off age-related health problems. While it has not yet been tested in humans for anti-aging purposes, rapamycin has been shown to extend the lifespan of mice..
“I decided to give it a try,” says Koebellein. It was his “first foray into biohacking,” and he was very pleased with how it turned out: “Within two weeks, the pain was gone by 50 percent,” he says. And by the 10th week, his range of motion had returned and the pain was gone completely.
“And it never came back,” he says.
Kaeberlein knows a lot about rapamycin: He’s a biologist and co-founder of the Canine Aging Project, which studies how rapamycin affects the healthspan of dogs, and is the former director of the Healthy Aging and Longevity Institute at the University of Washington.
Rapamycin was first approved by the FDA for use in transplant patients in the late 1990s. At high doses, it appears to suppress the immune system, and at lower doses, it reduces inflammation, Kaeberlein said. It works by blocking a signaling pathway in the body called mTOR, which appears to be a key regulator of lifespan and aging.
Although the drug is not approved for pain relief or anti-aging, some doctors prescribe rapamycin off-label to prevent age-related symptoms. Kaeberlein and his colleagues have studied about 300 such patients taking low doses, and many of them report benefits.
But anecdotes are no substitute for science. Understanding the risks and benefits of a drug requires research. And that’s where dentists come in.
Dr. Jonathan Ahn of the University of Washington has received FDA approval to test rapamycin on patients with periodontal disease, a common condition that tends to progress with age. Ahn says there isn’t much that can be done to treat patients with periodontal disease other than cleaning and removing the plaque, the bacterial buildup. “All we do is put a bandaid on it,” he says. His goal is to find and treat the underlying cause of the disease.
There is already some evidence from transplant patients that rapamycin may help improve oral health, and as part of the study, An and his collaborators will also measure changes in participants’ microbiomes and circadian clocks.
The study will enroll participants over the age of 50 who have periodontal disease. They will take different doses of the drug intermittently for eight weeks, after which An will be able to determine whether the drug is safe and effective.
A beneficial effect from rapamycin would help demonstrate that it is possible to target the underlying cause of disease, he says. “Ultimately, it’s about targeting the biology of ageing,” he says.
Dr. Ahn sees periodontal disease as a kind of canary in the coal mine of age-related diseases. For example, periodontal disease has been linked to an increased risk of heart disease and may also be linked to dementia. Scientists say bacteria in the mouth associated with periodontal disease causes inflammation, which can lead to “cascading” damage to blood vessels, which can lead to heart and brain problems.
“We predict that if we can target that underlying biology, we may be able to address a lot of other underlying conditions as well,” Ang says.
Because rapamycin is a generic drug, pharmaceutical companies have little incentive to fund new research. An and his colleagues received a grant to conduct the trial, which could pave the way for further studies to see whether rapamycin can help prevent or slow the progression of other age-related diseases.
Eric Verdin, a physician who directs the Buck Institute on Aging, says his institute is raising funds to further study rapamycin. There are many unanswered questions, Verdin says, such as “what is the effect of different concentrations in a single dose?” Verdin also wants to explore the “molecular signature” of people taking rapamycin. Verdin wants to learn more about dosage and intervals, because many doctors who prescribe rapamycin off-label recommend repeatedly starting and stopping it.
Researchers are also working on other drugs that may have similar effects, spurring the development of new drugs and other interventions that target biological aging, including a new $100 million XPRIZE Healthspan competition supported by Hevolution and other funders aimed at accelerating research in this area.
For now, Peter Diamandis, a physician who writes about longevity and founder of XPRISE, is taking rapamycin. “I take six milligrams every Sunday night, so once a week,” Diamandis explains. He takes it for three weeks, then takes a month off. “I believe the rapamycin I’m using is safe, and that it does more good than bad,” Diamandis says.
Diamandis constantly monitors his body using a variety of health indicators, but he acknowledges that it’s hard to judge rapamycin’s effectiveness given everything else he does to stay healthy, including eating a healthy diet, cutting out sugar, exercising daily, and prioritizing sleep.
His plan is to support research into interventions and strategies that can help people live healthier lives while continuing their healthy lifestyle habits.
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This story was edited by Jane Greenhalgh