"People have shown that rapamycin extends life span again and again and again. ... I view it as the ultimate preventative medicine"
"Sometimes, the reviewers would call me names," he says. That started to change in 2009, when a large National Institutes of Health- funded study established that rapamycin and its derivatives helped mice live longer. The NIH scientists started mice on the drug at 20 months, or late middle age in mouse terms (mice typically live two to three years). Male mice on rapamycin lived 9 percent longer. The females' life span was extended by 14 percent. This is roughly the equivalent of giving 60-year-old women a pill that would enable them to see their 95th birthday.
Rapamycin works at a fundamental level of cell biology. In the early 1990s, scientists at Novartis's predecessor, Sandoz, discovered that a rapamycin molecule inhibits a key cellular pathway regulating growth and metabolism. This pathway was eventually dubbed "target of rapamycin," or TOR, and it's found in everything from yeast to humans (it's known as mTOR in mammals).
MTOR is like the circuit breaker in a factory: When it's activated, the cell grows and divides, consuming nutrients and producing proteins. When mTOR is turned down, the "factory" switches into more of a conservation mode, as the cell cleans house and recycles old proteins via a process called autophagy. One reason caloric restriction extends life span in animals, researchers believe, is because it slows down this mTOR pathway and cranks up autophagy. Rapamycin does the same thing, only without the gnawing hunger.
Rapamycin has been found to reduce age-related bone loss, reverse cardiac aging, and reduce chronic inflammation in mice. It's even been shown to reverse Alzheimer's disease in them. The Novartis study was the first to examine rapamycin's effect on aging-related parameters in healthy older people. "It's a landmark study," says the Buck Institute's Kennedy. "It's the kind of study we need more of."
"The difference between rapamycin and resveratrol is that rapamycin really works as advertised and resveratrol doesn't," says the University of Washington's Kaeberlein.
Some of the effects reminds one of telomere lengthening but Bill Andrews (whose lab tests for TA) said, "Rapamycin doesn't induce telomerase expression."