rapamycin clinical trial results

Researchers report promising anti-aging rapamycin clinical trial results

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Researchers just reported promising rapamycin clinical trial results in the first of its kind test of the drug’s safety and anti-aging effects on healthy Senior adults. [This article first appeared on LongevityFacts. Author: Brady Hartman. ]

A rapamycin clinical trial using healthy adults recently completed and reported the drug to be safe over the short-term when used for anti-aging purposes.

This clinical trial of rapamycin was one of the first testing the compound’s safety as an anti-aging drug in healthy Seniors. The clinical trial consisted of 25 healthy older adults 70-95 years between the ages of 70 to 95. The study participants took either a placebo or 1mg rapamycin daily for eight weeks. The main finding of the study was that the drug was safe, without significant side effects. The researchers published their results on February 3 in the journal Experimental Gerontology and concluded

“Thus, based on the results of our pilot study, it appears that short-term RAPA [rapamycin] treatment can be used safely in older persons who are otherwise healthy.”

Longevity scientists hail rapamycin as the world’s most promising lifespan-extension drug. Already approved to treat several human disorders, researchers believe that rapamycin controls the rate of aging of mammals. The drug has extended the lifespan in every animal tested so far, from yeast to mice. In several experiments with mice, rapamycin increased the average lifespan of the rodents by 20 to 33%.

Rapamycin Clinical Trial With Healthy Seniors

The just-completed rapamycin clinical trial in Texas is one of the first performed in healthy patients. However, earlier studies tend to focus on unhealthy patients. In one clinical trial of rapamycin, for example, investigators gave the drug for 12 weeks to patients undergoing cardiac rehabilitation. In this earlier study, the rapamycin-treated test subjects showed significant improvements in inflammatory markers, but no improvement in frailty.

Why Did We Need a Rapamycin Clinical Trial?

In 2009, rapamycin made headline all over the worldwide headlines, when a team of researchers led by David Harrison, discovered that the drug significantly extended the average lifespan of mice. The researchers gave rapamycin to mice that were 20 months old, equivalent to the age of 60 in humans. At the conclusion of the experiment, Harrison’s team was shocked to find that rapamycin increased the average lifespan of the male mice by 28% and the females by 38%.

Since the famous Harrison report, rapamycin has shown promising results in the treatment of many other age-related diseases. Subsequent studies in mice showed that the drug blocked the progression of Alzheimer’s disease, atherosclerosis, muscular dystrophy, and cancer. Rapamycin affects one of the principal chemical pathways involved in aging, the mammalian target of rapamycin (mTOR) pathway.

Dr. Kellogg Leader of Rapamycin Clinical Trial

The current clinical trial of rapamycin was headed up by Dr. Dean Kellogg, MD Ph.D., a celebrity in the longevity field. Dr. Kellogg works at the University of Texas Health Science Center (UTHSC) and Barshop Institute. Dr. Dean Kellogg focuses on translating the results of successful mouse experiments with rapamycin into humans.

As reported in an earlier article on rapamycin, Dr. Kellog has been giving the drug to patients at least since mid-2015. Commenting on the anti-aging powers of rapamycin, Dr. Kellogg remarked

“I never really thought I would see a pharmacological agent that can alter the aging process, rapamycin appears to slow the aging process.” – Dr. Dean Kellogg, MD PhD

Rapamycin Clinical Trial Design

Three participants withdrew from the study, and11 rapamycin-treated subjects and 14 controls completed at least eight weeks of treatment and were included in the final results. The clinical trial investigators monitored the patients for detrimental effects of rapamycin treatment on metabolism, including both standard clinical laboratory assays and oral glucose tolerance tests.

Also, the researchers sought to see whether rapamycin treatment induced modifications in parameters typically associated with aging. These parameters included measures of cognitive function which the researchers assessed using three different tools. Moreover, the scientists measured physical performance using handgrip strength and 40-foot timed walks. Lastly, the researchers monitored changes in general parameters of healthy immune aging, including serum pro-inflammatory cytokine levels and blood cell subsets.

Adverse Effects

Five participants in the rapamycin group reported adverse side effects; these were limited to facial rash (1 participant), stomatitis (1 participant) and gastrointestinal issues (2 participants) whereas placebo-treated participants only reported stomatitis (1 participant). Although the test subjects reported no other adverse events, the researchers observed a statistically significant lowering of several blood parameters in the rapamycin-treatment group. These drops in blood levels included decreases in hemoglobin, hematocrit, red blood cell count, red blood cell distribution width, mean corpuscular volume, and mean corpuscular hemoglobin. However, the rapamycin clinical trial investigators at UTHSC reported that

“none of these changes manifested clinically significant effects during the short duration of this study.” Adding “Similarly, no changes were noted in any other clinical laboratory, cognitive, physical performance, or self-perceived health status measure over the study period.”

Monitoring for Immune System Changes

Rapamycin is currently FDA-approved for use in organ transplant patients, and in the treatment of a few types of cancer. Doctors prescribe the immune-suppressing drug to transplant patients to stop them from rejecting their organs. Because rapamycin suppresses the immune system, the drug has serious side effects, at the doses used in transplant patients.

The researchers also reported that “immune parameters were largely unchanged as well, possibly due to the advanced ages” of the test subjects. The researchers detected rapamycin-associated increases in a myeloid cell subset and TREGS, but reported that the “changes in most other PBMC cell subsets were not statistically significant.”

Watching for Insulin Resistance

Other experimenters have reported that rapamycin increases the onset of type 2 diabetes. The UTHSC trial investigators monitored their participants using the oral glucose tolerance test (OGTT) and stated that

“the OGTTs revealed no rapamycin-induced increase in blood glucose concentration, insulin secretion, and insulin sensitivity.”

A welcome sign, indicating that none of the patients developed diabetes or insulin sensitivity during this clinical trial of rapamycin. However, the researchers concluded that

“a larger trial with a larger samples size and longer treatment duration is warranted.”

Take Home Message

Rapamycin is showing great promise as a drug to ward off the ravages of aging. Hopefully, the recently completed clinical trial of rapamycin in San Antonio will be followed by more extensive and longer-lasting clinical trials that demonstrate the drug’s safety and effectiveness for anti-aging purposes.

Related Articles on Rapamycin

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Cover photo credit: STEEX/Stígur Karlsson via Getty Images.

Ellen Kraig, Leslie A. Linehan, Hanyu Liang, Terry Q. Romo, Qianqian Liu, Yubo Wu, Adriana D. Benavides, Tyler J. Curiel, Martin A. Javors, Nicolas Musi, Laura Chiodo, Wouter Koek, Jonathan A.L. Gelfond, Dean L. Kellogg Jr. “A randomized control trial to establish the feasibility and safety of rapamycin treatment in an older human cohort: Immunological, physical performance, and cognitive effects.” Experimental Gerontology, Available online 3 February 2018, ISSN 0531-5565, https://doi.org/10.1016/j.exger.2017.12.026. Link to article in Experimental Gerontology. (paywalled).

David E Harrison et al. “Rapamycin fed late in life extends lifespan in genetically heterogeneous mice. ” Nature, 2009, 460 (7253): 392–5. doi:10.1038/nature08221. PMC 2786175. PMID 19587680.  Link to article in Nature.

Arriola Apelo SI, Lamming DW. “Rapamycin: An InhibiTOR of Aging Emerges From the Soil of Easter Island.” The Journals of Gerontology. Series A, Biological Sciences, and Medical Sciences. 2016 Jul. Link to article in The Journals of Gerontology.


Diagnosis, Treatment, and Advice:  This article is intended for educational and informational purposes only and is not a substitute for qualified, professional medical advice.  The information and opinions provided herein should not be used during any medical emergency or for the diagnosis or treatment of any medical condition. Consult a qualified and licensed physician for the diagnosis and treatment of any and all medical conditions. Experimental therapies – such as rapamycin therapy for anti-aging purposes – carry a much higher risk than FDA-approved ones. Call 911, or an equivalent emergency hotline number, for all medical emergencies. As well, consult a licensed, qualified physician before changing your diet, supplement or exercise programs.
Photos, Endorsements, & External Links:  This article is not intended to endorse organizations, companies, or their products. Links to external websites, mention or depiction of company names or brands, are intended for illustration only and do not constitute endorsements.

4 Replies to “Researchers report promising anti-aging rapamycin clinical trial results”

  1. Alan Green M.D.

    Hi Brady,

    Study used wrong dose and wrong interval. Right dose 3-6 mg and right interval once a week.
    They used 1 mg daily.

    My prediction: Result: Bupkis

    However, 1 mg daily will probably have only mild side effects.

  2. Hyper trophy

    In the 2009 study, rapamycin extended the total lifespan of mice by 9% and 18% for male and female mice, respectively. The 28 and 38 percent increases were in life expectancy at the age of first exposure (600 days).

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