The U.S. Food and Drug Administration (FDA) has officially approved the use of the drug ISM8969 within the framework of clinical trials and other studies involving humans.
The drug was recently developed with the help of artificial intelligence technologies to combat inflammation and neurodegenerative diseases, according to a spokesperson for the biotechnology company InSilico Medicine.
"Our AI-redesigned drug development process has enabled us to create a molecule that can efficiently penetrate brain tissue and is expected to have great potential in treating Parkinson's disease, " said Carol Sattler, the company's assistant president.
"We are delighted to have received FDA authorization, which will enable us to move to the next stage of drug development and begin clinical trials with human participants."
According to a statement released by the company, the experimental drug ISM8969 is a short molecule that inhibits the activity of the NLRP3 protein in brain cells and other tissues in the human body. This protein plays a key role in the development of chronic inflammation in the nervous system, which is believed to contribute to the progression of diseases such as Parkinson's and Alzheimer's , as well as other neurodegenerative diseases.
Attempts to treat these diseases face significant challenges, most notably the inability of most current drugs to cross the blood-brain barrier , a specialized tissue that separates the brain from the bloodstream and allows only small molecules and certain types of immune cells to pass through. Artificial intelligence techniques have enabled scientists to design a molecular structure for a drug in a very short period (less than two years) that can effectively cross this barrier while simultaneously inhibiting the NLRP3 protein.
Subsequent experiments on mice showed that administering this drug significantly slowed the progression of Parkinson's disease, increased physical strength, and improved limb control. The AI-developed molecule also significantly outperformed existing counterparts, making it a promising candidate for a new drug to treat neurodegenerative diseases.
Scientists indicated that FDA approval would allow for the commencement of Phase 1 clinical trials in the near future. Company representatives also explained that the drug has attracted the interest of a Chinese pharmaceutical company, which has signed a collaboration agreement with InSilico Medicine to conduct subsequent phases of clinical trials and for the potential application of the first ISM8969-based drugs in clinical practice.
