Mayo Clinic Launches First-in-Human Trial of Targeted Radiopharmaceutical for Liver Cancer
Mayo Clinic has become the first institution to administer an investigational radioactive medicine to a patient with hepatocellular carcinoma (HCC), the most common type of liver cancer. The therapy is a targeted radiopharmaceutical theranostic (RPT) designed to hone in on a novel protein called glypican-3 (GPC3).
The RPT combines two functions: a diagnostic imaging agent to locate cancer cells and a therapeutic agent to target and destroy them. “Whole-body GPC3 targeted molecular imaging shows high localization to the tumor and minimal accumulation in normal tissues, indicating that the therapeutic radiation effects will be limited to sites of disease,” said Ephraim Parent, M.D., Ph.D., radiologist and division chair of Nuclear Medicine at Mayo Clinic in Florida and co-principal investigator of the trial.
GPC3 is considered a promising treatment target because it is highly expressed in HCC tissue but absent in healthy adult livers. Normally active only during fetal development, the protein “reawakens” in liver cancer, fueling tumor growth and helping cancer cells evade the immune system. Its unique presence on cancer cells makes it an attractive target for therapy.
The investigational therapy being tested uses alpha particle–emitting RPTs, which can deliver highly potent radiation directly to tumor cells while sparing healthy tissue. The Phase 1/1b clinical trial is being conducted at all three Mayo Clinic sites in Rochester, Minnesota; Phoenix, Arizona; and Jacksonville, Florida, where the first patient was treated.
“Being able to offer access to this novel radiopharmaceutical approach reflects our dedication to pushing the boundaries of liver cancer care,” said Lionel Kankeu Fonkoua, M.D., a medical oncologist at Mayo Clinic in Rochester and principal investigator of the study. “At Mayo Clinic, we are committed to advancing innovative therapies that expand possibilities for those facing this devastating disease.”
Mayo Clinic in Florida previously pioneered the use of an investigational alpha-emitting radiopharmaceutical in a U.S. treatment setting for a patient with metastatic breast cancer. Now, with this liver cancer trial, the institution has marked another milestone.
The investigational drug is being developed by RayzeBio Inc., a Bristol Myers Squibb company, which is sponsoring the Phase 1/1b study.