Rapamycin and the Search for Longevity

Rapamycin is a FDA-approved drug with potential to extend our natural lifespans by decades…. Why aren’t we using it?

The population of the 21st century is steadily getting older over time, with age-related diseases such as Alzheimer’s posing an ever-increasing burden to our society and healthcare services. As a result, there is now a need for us to search for new ways to preserve our health and wellbeing as we grow older than previous generations. That said, many would argue that the already readily available drug rapamycin (sold under the brand name Rapamune) may provide an answer to this problem, with the potential to not only prevent the onset of age-related disease, but to extend human lifespan itself. 

Discovery

First isolated in 1972, rapamycin was originally discovered  after microbiologist Georges Nógrády noticed that the inhabitants of Rapa Nui (otherwise known as Easter Island) did not seem to catch tetanus, despite walking barefoot across the isle. The immunosuppressive effects of the drug subsequently led to its approval by the FDA in 1999 for use against organ rejection following human organ transplants. During the 21st century, however, the discovery of rapamycin’s ability to extend the lifespan of animals, while suppressing the very diseases that plague our own health in later life, has led to an explosion of interest in how we as humans could use the drug to our advantage.

So how does it work? 

Within the body, rapamycin specifically inhibits the mTOR (mammalian target of rapamycin) signalling pathway; mTOR is an enzyme commonly associated with promoting the onset of numerous disease states including cancers, neurological diseases and diabetes. Clinical studies performed in elderly volunteers have also shown the drug to provide a protective effect against common infections, while trialled rapamycin hand creams have been shown to boost collagen levels and improve the quality and appearance of the skin.

That said, we cannot yet be sure that rapamycin will keep us “forever young”, as the famous Alphaville song goes. Trials studying the long term effects of the drug at doses smaller than used following organ transplants are few and far between, being exceedingly difficult to organise from both a practical and ethical standpoint. In addition, these trials have also tended to only use healthy volunteers. For this reason, our insight into how rapamycin could be used to treat common diseases of ageing, alongside any interactions the drug may have with concurrently prescribed medications, remains limited. 

While rapamycin remains safe for human use, self medication with the drug (as is common amongst anti-ageing enthusiasts) remains discouraged by scientists and doctors alike, in part also due to the potential unreliability of clandestine pills sold online.

 Is it ethical?

Were rapamycin found to be effective at slowing the onset of human ageing and associated disease states, we need to consider who would likely have access to the drug. Notable differences in life expectancy and quality of healthcare already exist between the wealthiest and most deprived demographics within the UK, with boys born in the most affluent areas outliving the poorest by 8.4 years as of 2018, and girls by 5.8. Might rapamycin only compound this problem? Prominent researchers within the field may disagree, with some like Mikhail Blagosklonny proposing the use of state-funded, anti-ageing clinics to provide rapamycin alongside tips to improve the way we already live with exercise and a healthy diet.

As it stands, however, rapamycin is neither a panacea nor a philosopher's stone. Without further research and evidence to substantiate its use as a pillar of anti-ageing medicine, or an answer to the ethical dilemmas that would create, we may never know if the proverbial fountain of youth has been at our fingertips all along.

Science and TechRomane Pare