A drug being developed to treat a serious lung disease may also help to slow the ageing process, according to a team of researchers whose findings were published in Nature Biotechnology and presented at a conference in Paris last week.
In the small clinical study, patients who took rentosertib – developed by Insilico Medicine in Massachusetts – showed changes in blood proteins that made them appear biologically younger, reports the New York Post. Several tests estimated that after treatment, the patients were biologically about three to four years younger.
“There is a promising trend in age reversal in addition to treating the disease, and it came as a bonus for disease treatment,” said first author Alex Zhavoronkov, PhD, founder and co-CEO of Insilico Medicine.
“It basically means that you are predicted to be younger using a validated set of ageing clocks. We don’t know what that means yet.”
Presenting the results at the Nature Redefining Healthcare in the Age of AI conference at Sorbonne University, the researchers cautioned, however, that this does not yet prove the drug can actually reverse ageing or help people live longer.
“It’s early days,” Zhavoronkov noted. “The drug is still investigational.”
What is rentosertib?
Rentosertib is an experimental drug, discovered with the help of artificial intelligence, that is being developed to treat idiopathic pulmonary fibrosis (IPF), a serious disease causing scarring in the lungs.
In an unexpected outcome, the drug was found to change patterns of proteins in the blood in ways that made patients appear an average of three to four years younger, in one case up to six years.
Six different ageing tests showed the same general pattern, with patients who took rentosertib “scoring” biologically younger than those who received a placebo.
The strongest effect was seen after four weeks in patients taking 30mg of the drug twice daily.
When researchers compared the results with data from more than 55 000 people in the UK Biobank, they found that the drug changed several blood proteins in the opposite direction from what is normally seen with ageing.
The teams also found signs that the drug may affect ageing at cellular level, as it changed processes involved in how cells function and respond to damage.
Zhavoronkov said the company’s approach uses AI to search for what he calls “molecular criminals” – biological targets that may contribute to both ageing and disease.
The study involved researchers from Insilico Medicine as well as Harvard Medical School, Stanford University, the Broad Institute, RWTH Aachen University, Peking University and Westlake University.
Study limitations
Zhavoronkov stressed that the findings are preliminary. “This is a pilot,” he said. “It is early and exploratory.”
The study had some limitations, including the small sample size: just 42 patients were analysed over a 12-week period.
The participants all had IPF, which can change many of the same proteins researchers use to measure biological ageing. This means it’s possible that the improvements in the participants’ lung disease was related to the changes in blood proteins.
The findings don’t prove that the drug actually slows ageing, keeps people healthier longer or extends their lives, the researchers acknowledged.
The results were based only on changes in proteins in their blood, and do not yet support the use of rentosertib as an anti-ageing treatment.
Risks and side effects
The risks and side effects associated with rentosertib were found to be “manageable”,” according to Dr Carol Ann Satler, senior vice-president of clinical development for Insilico Medicine.
The most common potential side effects included mild diarrhoea, slightly lower potassium levels in the blood and temporary increases in liver enzymes.
“Once you stop the drug, essentially, they return to normal,” Satler added
In the next trial, the doctor said: “We’ll be looking to adjust the doses of the drug based on the side effect profile so that people can stay on it longer.”
Rentosertib has now progressed into a year-long phase 3 study of more than 300 patients in China, according to Insilico.
Larger and longer trials, including healthy older adults and populations without IPF, are needed to determine whether the drug truly has anti-ageing effects and whether they could lead to better health.
While more research lies ahead, Zhavoronkov said the findings have given him more confidence to continue pursuing drugs that could target both age-related diseases and ageing itself.
“Everybody agrees that living longer should be a fundamental human right for everyone,” he said.
Study details
Integration of proteomic ageing clocks in a phase 2a clinical trial supports simultaneous geroprotective assessment
Alex Zhavoronkov, Fedor Galkin, Shan Chen et al.
Published in Nature Biotechnology on 7 September 2026
Abstract
Drugs for ageing-related diseases may modulate ageing itself, but standard clinical trial designs cannot detect such effects. Ageing clocks could close this gap, but epigenetic models often yield inconsistent, hard-to-interpret results. In contrast, proteomic clocks, by tracking the immediate effectors of biological change, may excel in providing aging biomarkers or mechanistic insight. Here we compare six proteomic clocks (ProtAge, OrganAgemortality, OrganAgechrono, PAC, ipfP3GPT and PAOPAC) on serum proteomes from a published 12-week phase 2a trial of the candidate anti-fibrotic drug rentosertib in idiopathic pulmonary fibrosis. We measure the variance between the clocks and find that all six clocks consistently predicted lower biological age in treated arms. However, proteomic clocks alone cannot deconvolute ageing- and disease-specific effects. We addressed this issue indirectly through pathway analyses that identified potential anti-ageing shifts in senescence and metabolic processes alongside the anti-fibrotic activity of rentosertib. This work supports the goal of dual-purpose clinical trial designs that integrate ageing endpoints into studies for specific disease indications.
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