Scientists have known that the cancer drug ONC201 blocks cells from proliferating and kills tumors in cell and animal models—but they haven’t known exactly how it works, or what its molecular target is.
Nevertheless, multiple clinical trials of the drug, in various cancer types, are underway. In 2018, ONC201, made by the Philadelphia-based company Oncoceutics, received a fast track designation from the Food and Drug Administration, meaning it gets an expedited review, for the treatment of certain gliomas in adults.
Now, two studies published independently this month reveal the drug’s mechanism of action: ONC201 works by activating ClpP, an enzyme that chews up misfolded proteins in mitochondria.
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