The dominant view of the regulation of medicine within academia and government is more or less that (a) people should not have the right to choose their own risks and make their own mistakes, (b) the role of regulators is to remove as much risk as possible, and (c) that the high cost of medicine and slow pace of introduction of new drugs is a better problem to have than greater freedom for patients. This is the background against which one can find papers such as today’s open access discussion of rapamycin and the state of its use as a means to improve late life health and modestly slow degenerative aging.
Rapamycin has long been approved for use as an immunosuppressive drug, but of late has attracted far more attention for its ability to upregulate autophagy, slow aging, and extend life in animal studies. This has led to a significant degree of off-label prescription of rapamycin by physicians. Physicians have the discretion to prescribe any approved drug for any use that is defensible, but this only happens when there is a body of work to suggest that the novel use could be safe and useful.
Thus rapamycin is in the nebulous state occupied by many drugs that are prescribed off-label: animal studies indicate that it could be used in a novel way at a novel dose, in this case to slow aging at lower doses than its established immunosuppressive use, but little to no concrete human data exists to confirm that new use. That data is unlikely to emerge any time soon because clinical trials are expensive and genetic drugs cannot produce enough revenue to justify that cost. Meanwhile, a good fraction of academics and regulators are appalled by off-label use, as one might expect given their views on freedom, risk, and the purpose of regulators.
Rapamycin for longevity: the pros, the cons, and future perspectives
Rapamycin, an antibiotic discovered in the 1970s, has become a critical tool in biomedical research. Initially recognized for its potent antifungal and immunosuppressive properties, rapamycin has recently gained significant attention for anti-aging therapy and seizure treatment via mTOR pathway inhibition. The mechanistic target of the rapamycin (mTOR) pathway is an evolutionarily conserved metabolic signaling cascade that regulates cell division, growth, and survival. There is growing evidence that mTOR pathway activity accelerates aging and the development of age-related diseases including cancer, atherosclerosis, diabetes, and declining immune function. Therefore physicians and “biohackers” are using mTOR inhibition via rapamycin (and rapamycin analogs) off-label for prevention of age-related conditions despite not being widely recognized as a treatment by the broader clinical community.
As rapamycin gains popularity for its anti-aging potential, online longevity clinics have emerged offering access to the drug with minimal medical oversight. This semi-regulated availability raises ethical concerns regarding patient safety, misinformation, and the potential for serious harm. This is best illustrated by the widely publicized case of tech entrepreneur Bryan Johnson, who undertook an elaborate self-directed anti-aging regimen involving rapamycin, metformin, and over 100 daily supplements. Despite extensive physiological tracking, Johnson ultimately discontinued rapamycin and expressed regret over its use citing side effects such as elevated blood glucose, susceptibility to infection, and impaired healing. This case highlights the risks of bypassing peer-reviewed science in favor of anecdotal “biohacking” culture. Clinical literature has long documented rapamycin-associated toxicities that mirror the complaints reported by Johnson and others. The use of such a powerful immunosuppressant outside established indications, especially in otherwise healthy individuals, demands stronger ethical scrutiny and public education.
Lastly, while the FDA does not recognize aging as a disease, there is growing interest in approving therapeutics that enhance healthspan, or delay aging-related decline. However, FDA approvals are structured around specific, diagnosable indications, rather than generalized syndromes. Should rapamycin or related compounds demonstrate efficacy, they would be approved for specific indications (e.g., Alzheimer’s) rather than aging per se under the current approval standards. Nonetheless, even within this evolving framework, it is important to note that most off-label prescribing-despite it being common clinical practice-rarely achieves FDA approval, as only about 30% of off-label prescribing is supported by adequate scientific evidence despite any clinically observed positive outcomes. These regulatory and evidentiary constraints must be considered when evaluating rapamycin’s future clinical and research trajectory.
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