A drug being developed to treat a serious lung disease may also help to slow the aging process, researchers say.
In a small clinical study, patients who took rentosertib – developed by Insilico Medicine in Massachusetts – showed changes in blood proteins that made them appear biologically younger.
Several tests estimated that after treatment, the patients were biologically about three to four years younger.
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“There is a promising trend in age reversal in addition to treating the disease, and it came as a bonus for disease treatment,” first author Alex Zhavoronkov, PhD, founder and co-CEO of Insilico Medicine, told Fox News Digital.
“It basically means that you are predicted to be younger using a validated set of aging clocks. We don’t know what that means yet.”
The results were published in Nature Biotechnology and presented Monday at the Nature conference at Sorbonne University in Paris.
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The researchers caution, however, that this does not yet prove the drug can actually reverse aging 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 that causes 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 aging 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 30 mg of the drug twice a day.
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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 aging.
The researchers also found signs that the drug may affect aging at the cellular level, as it changed processes involved in how cells function and respond to damage.
“Everybody agrees that living longer should be a fundamental human right for everyone.”
Zhavoronkov said the company’s approach uses AI to search for what he calls “molecular criminals” — biological targets that may contribute to both aging and disease.
The study – which was published in Nature Biotechnology – 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 analyzed over a 12-week period.
The participants all had IPF, which can change many of the same proteins researchers use to measure biological aging. 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 aging, 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-aging 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 diarrhea, slightly lower potassium levels in the blood and temporary increases in liver enzymes.
“Once you stop the drug, essentially, they return to normal,” Satler told Fox News Digital.
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.”
Looking ahead
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-aging 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 aging itself.
“Everybody agrees that living longer should be a fundamental human right for everyone,” he said.