Rapamycin: The Fountain of Youth?

The Holy Grail of science has been to discover a drug to slow down aging while extending the healthy years for humans.  Unfortunately, pharmacological history is littered with compounds and drugs that have failed to deliver on the anti-aging promise.

Today there is renewed hope in the scientific community about a drug that has shown to extend the life span of middle-aged mice.  After extensive studies in cells and animals, ground breaking research has been launched using humans in a closely-watched trial.

The drug is called Rapamycin, which was originally approved by the U.S. Food and Drug Administration (FDA) in 1999 for transplant patients. Rapamycin was used as an immunosuppressant to help prevent the body's rejection of organ transplants.

Although the drug has been around for less than two decades, it was discovered in the 1970's by a Canadian medical expedition to Easter Island, a remote dollop of land in the Pacific Ocean located about 2,100 miles off the coast of Chile.

Rapamycin, an eponym of the Polynesian name for Easter Island, is a naturally occurring bacterium found in the soil near the colossal stone head statues that dot the landscape.  The organism secrets a potent antifungal compound.  A Canadian doctor purified it and named it Rapamycin.

Years later it evolved into the standard drug treatment for organ transplant patients, but otherwise it languished in relative anonymity. A breakthrough study underwritten by the National Institutes of Health (NIH) in 2009 reignited interest in Rapamycin, this time as an anti-aging drug.

In the research, Rapamycin was given to male and female mice in late middle age.  The results were nothing short of astounding.  The male and female mice lived nine and 14 percent longer, respectively, than average.  This is equivalent to extending the life of a 60-year old woman to 95.

As often happens, the news media pounced on the discovery, leading to wild speculation about a "magic pill" that would be a bonanza for seniors.  Three magazines, Science, Nature and Time, called the findings one of the year's top discoveries.  The hype leapfrogged scientific caution.

Back in obscure labs where scientists toil inconspicuously, the job had only begun.  Exhaustive investigation at the University of Texas Health Science Center (UTHSC) lead to additional studies in animals and cells. The testing paved the way for the first trials to be conducted on humans.

This appeared to be the logical next step because Rapamycin showed promise as drug that could extend life by delaying the onset of aging-related diseases, such as cancer, heart disease and Alzheimer's.  The drug works by intervening in molecular processes that promote aging.

The Barshop Institute for Longevity and Aging Studies at UTHSC in San Antonio is currently partnering with the South Texas Veterans Health Care System in a clinical research study to determine if Rapamycin can be applied as a "possible intervention for aging" in humans.

Eight World War II military veterans have been recruited for the initial trial, most of them in their 90's.  The research is designed to assess the drug's impact on the volunteers' physical ability, immune system function, learning and memory.

The trial is ongoing, but there are already anecdotal stories of success. However, only a few volunteers have received the drug and additional research is needed to validate the findings.  At some point, plans are to expand the trial by opening the research to more participants.

But don't look for Rapamycin at your local pharmacy any time soon. Even if the trials are successful, the drug must receive the stamp of approval from the FDA to treat aging.  Since getting old is not considered a disease, that could present an obstacle for gaining FDA endorsement.

There are also ethical issues.  Would it be good for society if everyone lived to be 120?  Other concerns would include the cost and availability of the drug, not to mention the consequences for health insurance carriers, Medicare and Social Security programs.

Leaving that aside, it will be difficult for the government and the big pharmaceutical companies to ignore public pressure once the word oozes out about the trial's success.  Everyone wants to live longer while enjoying good health.  Rapamycin may yet turn out to be The Fountain of Youth.  

In the 16th century, Spanish explorer Juan Ponce de Leon went searching for the elusive Fountain of Youth.  He never located the mythical healing waters.  Perhaps, Ponce should have been looking on Easter Island.