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Younger Patients Included in Clinical Trials Targeting Sarcoma: A novel immunotherapy approach for sarcoma

The current standard of care for patients with advanced refractory sarcoma centers on various types of systemic chemotherapy, depending on the type of sarcoma, the location of the disease and the extent of the metastatic deposits. “But typically we bring clinical trials into the discussion pretty early,” says Ajay Gupta, MD, MS, Department of Pediatric Oncology at Roswell Park.

A clinical trial underway at Roswell Park aims to expand treatment options for those patients with a novel approach to immunotherapy. Investigators hope the strategy will overcome past limitations: Most immunotherapies are designed to work in highly mutated tumors, and sarcomas tend not to fall into that category, Dr. Gupta explains.

He serves as the site principal investigator for a clinical trial that will evaluate the safety and efficacy of mecbotamab vedotin (BA3011), an antibody-drug conjugate (ADC) that attaches to the cell surface receptor AXL. Dr. Gupta explains that BA3011 is a conditionally active biologic (CAB) that activates only in low pH environments. “The tumor microenvironment (TME) tends to be acidic, so the drug is activated only in the TME and deposits its toxic payload there.”

That payload, monomethyl auristatin E (MMAE) — which Dr. Gupta compares to a targeted version of the chemotherapy drug vincristine — works by blocking cell division. Because it targets AXL-positive cells specifically, healthy cells are generally spared its toxicity.

Phase 2 CAB-AXL-ADC Safety and Efficacy Study in Adult and Adolescent Patients with Sarcoma (NCT03425279)” is enrolling patients age 12 and older at 34 centers, including Roswell Park. The phase 1 part of the study, which compared BA3011 alone against BA3011 in combination with nivolumab, has closed. Phase 2 comprises several arms evaluating different cadences and frequencies of BA3011 as a monotherapy.

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