Mouse model study shows promise in treatment of medulloblastoma and ependymoma

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Researchers have demonstrated a novel approach in mouse models that delivers appropriately-targeted chimeric antigen receptor CAR T-cell therapy directly into the cerebrospinal fluid that surrounds the tumor in an effort to treat medulloblastoma and ependymoma.

Researchers at Baylor College of Medicine, Texas Children’s Hospital and the Hospital for Sick Children reported their findings in Nature Medicine. The findings support further clinical studies to evaluate this strategy to treat pediatric brain cancers. A first-in-child clinical trial is recruiting patients at Texas Children’s Hospital and Baylor College of Medicine to test the safety and anti-tumor efficacy of this approach (NCT02442297).

“Recurrences of medulloblastoma and ependymoma can be disseminated throughout the lining of the brain and spinal cord, which are bathed in cerebrospinal fluid. This location offers the opportunity to deliver therapies into the cerebrospinal fluid compartment and could provide a better chance for the therapy to reach and eliminate the tumor than administering it through the bloodstream,” co-corresponding author Nabil Ahmed, associate professor of pediatrics and immunology, section of hematology-oncology at Baylor and Texas Children’s Hospital, said in a statement.

In the mouse model studies, CAR T cells were administered into the cerebrospinal fluid around the tumor or into the bloodstream of mice harboring multiple patient-derived medulloblastoma and ependymoma tumors. The tumor size and animal survival were studied for about 200 days.

The results showed that administering tumor-specific CAR T cells into the cerebrospinal fluid was more effective than administering them via the blood. The researchers found that combining immunotherapy with azacytidine was significantly more effective than either treatment alone.

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