Patients suffering from pulmonary fibrosis experienced a reduction in their estimated biological age after receiving a drug developed through artificial intelligence, known as rentosertib. This finding is detailed in a study published in Nature Biotechnology.
Rentosertib (ISM001-055) is an experimental treatment for idiopathic pulmonary fibrosis, created by Insilico Medicine. The drug's molecule was designed using AI; specifically, Insilico's PandaOmics platform identified the target protein TNIK, while the Chemistry42 platform generated the molecule to interact with it.
This research was conducted by Insilico Medicine in collaboration with scientists from Harvard Medical School, Stanford University, the Broad Institute, Peking University, and other institutions.
Study Findings
The phase 2a trial involved 71 participants, who were administered rentosertib for 12 weeks following one of three dosing regimens: 30 mg once daily, 30 mg twice daily, or a single dose of 60 mg, alongside a placebo group.
The assignment of treatments was randomized, and neither the participants nor the researchers were aware of who received the medication. The primary aim was to assess safety, along with evaluating changes in lung function. Initial results are expected to be released in June 2025.
The new analysis utilized complete data from 42 participants, including 11 who received a placebo. Researchers employed six proteomic "aging clocks"—algorithms that estimate biological age based on protein composition in blood samples.
The models indicated a general trend of reduced estimated age, although statistical significance varied depending on the dosing regimen and timing of measurements. The most consistent results were observed at the four-week mark among patients taking 30 mg twice daily.
According to Insilico, this group experienced a decrease of approximately three to four years, with one model suggesting a reduction of up to six years. These figures reflect changes in the "aging clocks" and do not imply a corresponding increase in lifespan.
Between the fourth and twelfth weeks, further reductions in estimated age did not achieve statistical significance.
Limitations of the Findings
The authors noted that they could not determine whether the changes in blood proteins were indicative of an effect on biological aging or simply a consequence of the treatment for fibrosis. The sample size was small, the observation period lasted only 12 weeks, and there were no healthy volunteers involved in the trial.
Further investigation is necessary to ensure the safety of the drug. In the initial trial, discontinuation was linked to liver function issues and diarrhea. Researchers emphasized the need for larger and longer-duration studies.
Insilico has reported that rentosertib has advanced to phase three of clinical trials in China, focusing on the treatment of pulmonary fibrosis.
In August, MIT Technology Review highlighted the challenges that pharmaceutical companies utilizing AI in drug development face when it comes to regulatory approval.