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Eve4.2

Everolimus Research

Mostly mechanism / observationalLimited safety

7 peer-reviewed studies

What the evidence says

Mostly mechanism / observational

Most Everolimus studies are mechanism or observational rather than RCTs that measure a clinical effect — keep findings provisional.

Most evidence is from medium-quality randomised trials published 2008–2025 with a typical study size of 410 participants.

Based on 7 studies · 5 RCTs · 1,616 total participants

Confidence

High

By outcome

Aging & healthspan (emerging)
Mostly mechanism / observational5 studies
Immune & vaccine response
Mostly mechanism / observational4 studies
Oncology (approved use)
Too few graded studies2 studies
Safety profile
Too few graded studies1 study

Active research area

3 studies in the last 5 years

200820162025

1RCTn=410 · medium study2008

Everolimus prolonged progression-free survival relative to placebo in patients with metastatic renal cell carcinoma whose disease had progressed on VEGF-targeted therapy.

Motzer, Escudier, Oudard, Hutson, Porta, Bracarda · Lancet (London, England) (2008)

RECORD-1: pivotal phase III RCT of everolimus 10 mg/day vs placebo in metastatic renal cell carcinoma (n=410)
Significantly prolonged progression-free survival — the registration trial that established everolimus as an oncology drug
Confirms everolimus pharmacology, dosing, and the immunosuppressant safety profile in humans

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2RCTn=724 · large study2012

Everolimus combined with exemestane improved progression-free survival in HR-positive advanced breast cancer (BOLERO-2).

Baselga, Campone, Piccart, Burris, Rugo, Sahmoud · The New England journal of medicine (2012)

BOLERO-2: pivotal phase III RCT of everolimus + exemestane vs exemestane in HR-positive advanced breast cancer
More than doubled progression-free survival — a second registration-grade RCT for everolimus
Reinforces the human dosing and toxicity profile (stomatitis, pneumonitis, hyperglycemia) at oncology doses

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3RCTn=218 · medium study2014

RAD001 (everolimus) enhanced the response to the influenza vaccine by about 20% at doses that were relatively well tolerated.

Mannick, Del Giudice, Lattanzi, Valiante, Praestgaard, Huang · Science translational medicine (2014)

Randomized, placebo-controlled trial of low-dose everolimus (RAD001) in elderly volunteers
Improved the antibody response to influenza vaccination by ~20% and reduced PD-1 exhaustion marker on CD4/CD8 T cells
The best human signal that mTOR inhibition can partially reverse immunosenescence

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4RCTn=264 · medium study2018

A low-dose combination including everolimus (RAD001) that selectively inhibits TORC1 was associated with a significant decrease in the rate of infections reported by elderly subjects.

Mannick, Morris, Hockey, Roma, Beibel, Kulmatycki · Science translational medicine (2018)

Phase 2a randomized, placebo-controlled trial in 264 elderly subjects (low-dose BEZ235 + everolimus, selective TORC1 inhibition)
Significantly decreased the rate of infections (P=0.001) reported over the year after dosing, and up-regulated antiviral gene expression
Improved influenza-vaccine response — extends the immune-aging signal from biomarkers to a clinical infection endpoint

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5RCT2021

In the phase 3 PROTECT trial, RTB101 did not significantly reduce the percentage of older adults with clinically symptomatic respiratory illness.

Mannick, Teo, Bernardo, Quinn, Russell, Klickstein · The lancet. Healthy longevity (2021)

Phase 2b and phase 3 (PROTECT) randomized, placebo-controlled trials of the mTOR inhibitor RTB101 in adults aged ≥65 to prevent respiratory illness
The phase-3 trial MISSED its primary endpoint — RTB101 did not significantly reduce clinically symptomatic respiratory illness
Mandatory counter-evidence: the program's larger confirmatory effort failed to replicate the earlier infection-reduction signal

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6Systematic Review2022

Preclinical studies show a role for rapamycin and its analogues (rapalogues) in age-related musculoskeletal disorders, but the applicability to humans is unknown.

Lin, Salech, Lim, Vogrin, Duque · Aging clinical and experimental research (2022)

PRISMA systematic review of rapamycin and rapalogues (including everolimus) on age-related musculoskeletal disorders in humans
Strong preclinical mTORC1-inhibition rationale, but human evidence is sparse and inconclusive
Top-of-pyramid synthesis showing the human geroscience evidence base remains thin

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7Review2025

mTOR inhibitors such as rapamycin and everolimus are among the leading candidate geroprotectors, but no agent has yet been shown to extend human healthspan.

Kritchevsky, Cummings · JAMA (2025)

Translational review of the geroscience field and candidate geroprotective drugs
Frames mTOR inhibition (rapamycin/rapalogs) and the immune-aging trials within the broader evidence base
Reiterates that no drug has demonstrated human healthspan/lifespan extension

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View in Research Library

Everolimus Research

Mostly mechanism / observational

Efficacy of everolimus in advanced renal cell carcinoma: a double-blind, randomised, placebo-controlled phase III trial.

Everolimus in postmenopausal hormone-receptor-positive advanced breast cancer.

mTOR inhibition improves immune function in the elderly.

TORC1 inhibition enhances immune function and reduces infections in the elderly.

Targeting the biology of ageing with mTOR inhibitors to improve immune function in older adults: phase 2b and phase 3 randomised trials.

The effect of rapamycin and its analogues on age-related musculoskeletal diseases: a systematic review.

Geroscience: A Translational Review.