The Most Effective Anti-Aging Drug Is Only Available to University Labs. Here’s Why.

Safe, Effective and Profitable. Rapamycin is two out of three: Here's why the most effective anti-aging drug is only available to university labs.

Safe, Effective and Profitable

Rapamycin is two out of three.

The American system of drug regulation is based on a demonstration of “safe and effective”.  But the funding for that demonstration is where a great bias creeps in.  The lion’s share of money spent on clinical trials — more than 90% of the $100 billion plus that is spent each year — comes from Big Pharma.  And Big Pharma’s priorities may not be fully congruent with yours and mine.  Each trial costs the better part of a billion dollars, and the success rate of the trials is at best one in four — closer to one in ten in the track records of some companies.  No company is going to invest that kind of money in a drug that they can’t control, so they can mark up the price and recoup the cost of their lottery ticket.

Imagine if we funded defense technology the same way.  “The Air Force is interested in a new fighter plane.  Boeing and General Dynamics, you’re invited to design and build prototypes at your own expense.  Then we’ll ask you to pay for testing it, and if it passes, we’ll buy it from you for 50 times your R&D budget.”

There are many things wrong with this system.  It results in drug prices that hit people when they’re down.  It concentrates a great deal of spending in areas where the risk is lowest, and the reward to the company — not necessarily the patient — is the greatest.  So we get a stream of me-too drugs, marginal improvements on the antihistamines and statin drugs of last year, while there is no money to test approaches that are truly innovative, and therefore high-risk.

Among the many ways that this system distorts funding priorities, perhaps the biggest is a neglect of natural products and nature-derived products.  The rules say that chemicals derived from plants cannot be patented. No patent, no monopoly. No monopoly, no megaprofit. No megaprofit, means no company willing to make the investment.

The Most Promising Life-Extension Candidate

Rapamycin was discovered in the 1990s, refined from bacteria in the soil found on Easter Island in the Pacific.  The bacteria use it as a potent anti-fungal agent, and it was approved by FDA to fight fungal infections in humans. But the side-effects included suppression of the body’s own defenses, and most doctors considered the drug too hot to handle.  Then, an off-label application was developed for use with organ transplant patients, where suppression of the immune system is part of the plan.

As a naturally-occurring chemical, rapamycin can’t be patented; but via a loophole, the uses and applications and formulations can be patented.  Pfizer was granted a patent in 1999 that runs out this year.  But in the years since their patent, rapamycin has been the subject of a great deal of hot, new science.  The mechanism of action was found to work through a biochemical receptor that was named TOR, for “target of rapamycin.”  And–surprise!–TOR turned out to play an outsized role in our biology, one that had never been suspected before.  TOR is a signal molecule with a whole cascade of effects on growth, development and aging.  Geneticists speak of the “TOR Pathway,” and it is mapped in ever greater detail.

Then, in 2009, it was discovered almost accidentally that a short course of rapamycin could extend the lives of lab mice, even when it is administered pretty late in their lives.  Among anti-aging drugs, rapamycin became a rock star overnight.

Last month, two encouraging reports came out of the research on rapamycin.  A short-term test in marmoset monkeys seemed to show that “immune suppression” was a bogeyman.  There were no adverse effects, even from continuous, long-term administration. And just last month, 40 aging dogs in a limited trial of rapamycin seemed to show improved health without troubling side-effects.


Rapamycin is presently the best candidate we have for a drug to extend life in humans.  It is expected to extend “health span” as well as lifespan, lowering incidence of cancer, heart disease and stroke. But is it “safe and effective” for use in people?  We may never know, because its patent has run out, and there is no company motivated to invest the cost of a human trial.

There are some things that capitalism does really well, but allocating money for pharmaceutical research isn’t one of them.

You can buy rapamycin from a number of lab supply houses, but only if you provide the address of a university lab, and certify that the purchase is for research purposes only.

(But you can buy my new book from your favorite online outlet or your neighborhood bookstore. It will change the way you take care of yourself, and change the way you think about your relationship to nature. )