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Mustang Bio, City of Hope Initiate Clinical Trial of CAR T-cell Therapy in Brain Cancer Patients

NEW YORK – Mustang Bio and City of Hope announced on Monday that they have initiated a Phase I clinical trial evaluating Mustang's autologous CAR T-cell therapy, MB-101, in leptomeningeal brain tumors, including glioblastoma, ependymoma, and medulloblastoma.

The clinical trial will enroll roughly 30 patients into two arms, one consisting of glioblastoma patients and the other consisting of ependymoma or medulloblastoma patients. The researchers are primarily interested in assessing the safety of the treatment and the three-month survival rate, while secondary endpoints include overall survival, and CAR and endogenous T-cell levels.

Mustang's cell therapy consists of harvested immune cells engineered to contain CAR T cells directed at IL13Rα2, which the company believes to be an attractive target for CAR T-cell therapy, since it is overexpressed on most glioma cells but minimally expressed on normal tissue. The manufactured product will be administered via an intraventricular catheter surgically placed prior to treatment.

Patients will be treated at City of Hope, where the therapy was developed and previously evaluated in a small, physician-run study. That study demonstrated early yet promising safety and efficacy with MB-101, with one leptomeningeal glioblastoma patient experiencing a complete response.

"We believe these preliminary results warrant further evaluation of these CAR T cells," Lisa Feldman, the clinical trial's principal investigator at City of Hope, said in a statement. "We look forward to providing updates on the trial and to continue working closely with Mustang with the goal of bringing a safe and effective treatment option to patients with this life-threatening disease."

In addition to the brain cancer CAR T-cell therapy, Mustang Bio recently began dosing patients with certain hematologic cancers in a Phase I/II clinical trial of its CD123-targeted CAR T-cell therapy, MB-102.