A groundbreaking clinical trial has found that low-dose, intermittent use of rapamycin—a drug traditionally used in transplant medicine—may offer measurable health benefits for older adults, enhancing aspects of physical function and emotional well-being. The findings were published in the latest issue of Aging (Aging-US).
The study, titled “Influence of rapamycin on safety and healthspan metrics after one year: PEARL trial results,” was led by researchers Mauricio Moel and Stefanie L. Morgan from AgelessRx. It represents the most comprehensive investigation to date of rapamycin’s effects on healthy aging in humans.
While medical advances have extended human lifespan, many older adults still face significant declines in health, mobility, and independence. This gap between living longer and living well has spurred interest in therapies that address aging itself. Rapamycin, an FDA-approved drug known for its role in suppressing immune responses in organ transplant recipients, has shown promise in animal models for delaying age-related decline. Until now, however, its impact on healthy human aging remained largely unexplored.
The PEARL (Participatory Evaluation of Aging with Rapamycin for Longevity) trial enrolled 114 healthy adults between the ages of 50 and 85 in a randomized, double-blind, placebo-controlled study lasting 48 weeks. Participants were assigned to receive either a placebo or a weekly dose of 5 mg or 10 mg of compounded rapamycin.
The trial’s primary objective was to assess changes in visceral fat. Secondary outcomes included lean muscle mass, various blood biomarkers, and self-reported quality-of-life measures.
Although the study did not find significant reductions in visceral fat, other outcomes were encouraging. Women in the 10 mg group experienced notable increases in lean muscle and reported reductions in bodily pain. Participants taking 5 mg weekly showed improvements in general health perception and emotional well-being based on validated health surveys.
Importantly, rapamycin was well-tolerated, with minimal side effects. The most common minor complaint was mild gastrointestinal discomfort. Serious adverse events occurred at similar rates across all study groups, including the placebo.
“Our findings provide evidence that these rapamycin regimens are well tolerated with minimal adverse effects when administered for at least one year within normative aging individuals,” the researchers wrote.
The study’s authors acknowledged certain limitations, including the small sample size and the health-conscious nature of the participant group, which may have constrained the detection of broader effects. Additionally, the compounded rapamycin formulation used in the study had lower bioavailability than commercial versions, possibly limiting its impact.
Still, the PEARL trial marks an important step toward understanding how rapamycin might be used to promote healthy aging in humans. The results build on a growing body of evidence suggesting that pharmacological interventions could one day help extend not just lifespan, but healthspan.
Larger and more diverse clinical trials will be needed to confirm these findings and optimize dosing strategies. But for now, the study offers a hopeful glimpse into a future where aging itself becomes a manageable health condition.
