MCW clinical research teams launched a phase 3 study, led locally by John Charlson, MD, Associate Professor of Hematology and Oncology, to evaluate whether adding ADI-PEG20, an enzyme that breaks down arginine, to standard chemotherapy can improve outcomes for patients with advanced or metastatic leiomyosarcoma (LMS). This global trial builds on promising earlier findings and aims to determine if this approach helps control tumors longer than standard treatment alone.
“Earlier research suggests that combining ADI-PEG20 with gemcitabine and docetaxel may improve outcomes, particularly for patients with advanced LMS,” said Dr. Charlson. “This study is an important step in understanding how well it works and whether it should become part of standard care.”
LMS is a rare and aggressive soft tissue cancer that often depends on arginine, a nutrient essential for cell growth. While healthy cells can produce arginine, many sarcomas cannot and must absorb it from their surroundings. ADI-PEG20 works by removing this external supply, essentially starving the cancer cells while leaving normal cells unaffected.
The MCW Cancer Center continues to lead sarcoma research and treatment innovation. Dr. Charlson and the sarcoma team recently played a key role in the FDA approval of afamitresgene autoleucel (afami-cel)—the first engineered T-cell therapy for a solid tumor—by providing patients access to the groundbreaking treatment and contributing key data. As research progresses, the Cancer Center is at the forefront of developing and delivering new treatment options for rare and aggressive cancers, ensuring patients have access to the latest advancements in care.
“If this combination works, it could change the way we treat this disease,” said Dr. Charlson. “We’re looking at every patient who comes through our clinic to see if this trial could be the right fit.”
The Cancer Center research team expects to enroll 5 to 10 patients in the trial.