Rapamycin

Extracted Attributes

Sirolimus

Sirolimus, also known as rapamycin and sold under the brand name Rapamune among others, is a macrolide compound that is used to coat coronary stents, prevent organ transplant rejection, treat a rare lung disease called lymphangioleiomyomatosis, and treat perivascular epithelioid cell tumour (PEComa). It has immunosuppressant functions in humans and is especially useful in preventing the rejection of kidney transplants. It is a mammalian target of rapamycin (mTOR) kinase inhibitor that reduces the sensitivity of T cells and B cells to interleukin-2 (IL-2), inhibiting their activity. This compound also has a use in cardiovascular drug-eluting stent technologies to inhibit restenosis. It is produced by the bacterium Streptomyces hygroscopicus and was isolated for the first time in 1972, from samples of S. hygroscopicus found on Easter Island. The compound was originally named rapamycin after the native name of the island, Rapa Nui. Sirolimus was initially developed as an antifungal agent. However, this use was abandoned when it was discovered to have potent immunosuppressive and antiproliferative properties due to its ability to inhibit mTOR. It was approved by the US Food and Drug Administration (FDA) in 1999. Hyftor (sirolimus gel) was authorized for topical treatment of facial angiofibroma in the European Union in May 2023.

Web Search Results

• Rapamycin: Benefits, Side Effects, and Research - Lifespan.io

  El-Tanani, M.; Nsairat, H.; Aljabali, A.A.; Serrano-Aroca, Á.; Mishra, V.; Mishra, Y.; Naikoo, G.A.; Alshaer, W.; Tambuwala, M.M. Role of Mammalian Target of Rapamycin (MTOR) Signalling in Oncogenesis. Life Sci 2023, 323, 121662, doi:10.1016/J.LFS.2023.121662. Gao, X.; Zhao, F.; Wang, Y.; Ma, X.; Chai, H.; Han, J.; Fang, F. Discovery of Novel Hybrids of MTOR Inhibitor and NO Donor as Potential Anti-Tumor Therapeutics. Bioorg Med Chem 2023, 91, 117402, doi:10.1016/J.BMC.2023.117402. [...] Mandrioli, J.; D’Amico, R.; Zucchi, E.; De Biasi, S.; Banchelli, F.; Martinelli, I.; Simonini, C.; Lo Tartaro, D.; Vicini, R.; Fini, N.; et al. Randomized, Double-Blind, Placebo-Controlled Trial of Rapamycin in Amyotrophic Lateral Sclerosis. Nature Communications 2023 14:1 2023, 14, 1–14. Gianessi, L.; Magini, A.; Dominici, R.; Giovagnoli, S.; Dolcetta, D. A Thermostable Micellar Formulation of Rapalogs for Intracerebroventricular Delivery and the Therapy of Neurological Disorders. 2023. Yang, Y.Y.; Wang, W.L.; Hu, X.T.; Chen, X.; Ni, Y.; Lei, Y.H.; Qiu, Q.Y.; Tao, L.Y.; Luo, T.W.; Wang, N.Y. Design, [Synthesis and Biological Evaluation of Novel 9-Methyl-9H-Purine and Thieno[3, 2-d]Pyrimidine Derivatives as Potent MTOR Inhibitors]( Bioorg Chem 2023, 132, 106356. [...] Posansee, K.; Liangruksa, M.; Termsaithong, T.; Saparpakorn, P.; Hannongbua, S.; Laomettachit, T.; Sutthibutpong, T. Combined Deep Learning and Molecular Modeling Techniques on the Virtual Screening of New MTOR Inhibitors from the Thai Mushroom Database. ACS Omega 2023, 8, 38373–38385, doi:10.1021/ACSOMEGA.3C04827. Panwar, V.; Singh, A.; Bhatt, M.; Tonk, R.K.; Azizov, S.; Raza, A.S.; Sengupta, S.; Kumar, D.; Garg, M. Multifaceted Role of MTOR (Mammalian Target of Rapamycin) Signaling Pathway in Human Health and Disease. Signal Transduction and Targeted Therapy 2023 8:1 2023, 8, 1–25.

• Rapamycin for longevity: the pros, the cons, and future perspectives

  Rapamycin, an antibiotic discovered in the 1970s from Streptomyces hygroscopicus on Easter Island (Rapanui), 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 [...] 4. 1. Canpolat M., Gumus H., Kumandas S., Coskun A., Per H. (2018). The use of rapamycin in patients with tuberous sclerosis complex: long-term results. Epilepsy & Behav. 88, 357–364. 10.1016/j.yebeh.2018.09.020 - DOI - PubMed 5. 1. Chung C. L., Lawrence I., Hoffman M., Elgindi D., Nadhan K., Potnis M., et al. (2019). Topical rapamycin reduces markers of senescence and aging in human skin: an exploratory, prospective, randomized trial. GeroScience 41 (6), 861–869. 10.1007/s11357-019-00113-y - DOI - PMC - PubMed [...] 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. Currently, rapamycin (i.e., sirolimus and everolimus) is FDA approved for the prevention of transplant organ rejection and for anti-seizure therapy in Tuberous Sclerosis Complex (TSC; caused by variants in TSC1 or 2). We aim to summarize the mTOR pathway, the impact rapamycin has on the mTOR pathway, and the state of rapamycin use in the field of aging and longevity. Importantly, we will discuss the gaps in knowledge, pitfalls, and potential for the use of rapamycin to prevent aging/age-related disease and discuss the lessons learned from achieving FDA approval of

• Rapamycin for longevity: the pros, the cons, and future perspectives

  Rapamycin’s purported geroprotective effects are often attributed to its ability to induce autophagy, a cellular recycling process responsible for degrading protein aggregates and other damage-associated molecular patterns (DAMPs) (Rubinsztein et al., 2011; Arensman and Eng, 2018; Zinecker and Simon, 2022; Szőke et al., 2023; Tabibzadeh, 2023). mTORC1 normally inhibits autophagy by phosphorylating components of the Unc-51-like autophagy-activating kinases 1 (ULK1 complex), and its inhibition by rapamycin removes this suppression and initiates autophagosome formation (Kim et al., 2011; Park et al., 2023). Online proponents of anti-aging interventions claim that rapamycin-induced autophagy promotes longevity by maintaining proteostasis and reducing “toxic burden” in post-mitotic cells which [...] The success of rapamycin in mTORopathy models stems from clearly defined molecular etiology and robust biomarkers. Specifically, highly penetrant mutations driving mTOR hyperactivation renders the pathway an actionable target. In contrast, aging is a heterogeneous and multifactorial process without a single dominant pathway, and most preclinical studies do not incorporate genetic stratification or polygenic risk scores. This may explain why mTOR inhibition yields robust disease-modifying effects in monogenic epilepsy models but produces inconsistent outcomes in aging research. Understanding rapamycin’s efficacy in epilepsy may inform how to refine translational models of aging. [...] Rapamycin, an antibiotic discovered in the 1970s from Streptomyces hygroscopicus on Easter Island (Rapanui), 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 Shows Limited Evidence for Longevity Benefits in ...

  Rapamycin, originally developed as a drug to suppress the immune system, has gained interest as a possible anti-aging therapy. It works by blocking a key cellular pathway called mTOR, which plays a role in growth and metabolism. In animal studies, blocking this pathway has extended lifespan. However, the translation of these results to humans remains uncertain. The current study examined clinical trials and observational studies involving healthy adults who took low doses of rapamycin or similar drugs. “This paper has reviewed trials of low-dose mTOR inhibition therapy in human subjects.” [...] Rapamycin, originally developed as a drug to suppress the immune system, has gained interest as a possible anti-aging therapy. It works by blocking a key cellular pathway called mTOR, which plays a role in growth and metabolism. In animal studies, blocking this pathway has extended lifespan. However, the translation of these results to humans remains uncertain. The current study examined clinical trials and observational studies involving healthy adults who took low doses of rapamycin or similar drugs. “This paper has reviewed trials of low-dose mTOR inhibition therapy in human subjects.” [...] Aging # Rapamycin Shows Limited Evidence for Longevity Benefits in Healthy Adults “What emerges is a complex picture that remains insufficient to affirm or negate the longevity and healthspan extending benefits attributed to rapamycin.” Click here to listen to the press release BUFFALO, NY — September 24, 2025 — A new research paper was published in Volume 17, Issue 8 of Aging-US on August 7, 2025, titled, “What is the clinical evidence to support off-label rapamycin therapy in healthy adults?”

• What is rapamycin? - New Scientist

  Rapamycin was isolated in 1972 from a bacterium found on Easter Island, aka Rapa Nui – hence the name. For many years it was an obscure transplant drug but in the early 2000s was found to significantly extend the lifespan of worms, yeast, flies and mice. In one experiment, researchers gave rapamycin to a group of 20-month-old mice, equivalent to retirement-aged humans. They fed the mice small doses for three months, then took them off the drug and waited for them to die. Mice usually die aged around 30 months but the drugged ones lived an extra 2 months on average. The final survivor died more than two years after the start of the experiment, at the ripe old age of 3 years and 8 months – the equivalent of around 140 in human years. [...] Your body is ageing down one of four – or more – possible pathways. Figuring out your "ageotype" could help you zero in on the things you can do to stay healthier for longer Rapamycin is thought to exert its life-extending properties by mimicking the effect of caloric restriction, one of the most reliable ways to extend lifespan in non-human animals. It targets a signalling molecule called mTOR (an acronym for mechanistic target of rapamycin) which is an important node in our nutrient-sensing pathways. Lack of food switches mTOR off and activates emergency systems that enable us to survive periods of starvation.