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Pediatric Brain Tumor Trials

Combination Intraventricular Chemotherapy Pilot Study:

Methotrexate and Etoposide Infusions into the Fourth Ventricle or Resection Cavity in Children with Recurrent Posterior Fossa Brain Tumors

Lead Physician: David Sandberg, M.D.

The Children’s Neuroscience Center has a new brain tumor trial that is open to qualified participants. The research study is being conducted by David Sandberg, M.D., Professor and Director of Pediatric Neurosurgery at McGovern Medical School at UTHealth and Children’s Memorial Hermann Hospital.

The study is called “Combination Intraventricular Chemotherapy Pilot Study:  Methotrexate and Etoposide Infusions into the Fourth Ventricle or Resection Cavity in Children with Recurrent Posterior Fossa Brain Tumors.” It is open to patients who are age 1 to 21 years old with recurrent medulloblastoma (PNET), recurrent ependymoma, and recurrent atypical teratoid/rhabdoid tumors involving the brain and/or spine.

The study employs a novel means of treating malignant tumors that originate from the fourth ventricle: infusion of two chemotherapy agents directly into the fourth ventricle rather than systemic intravenous delivery. There will be no simultaneous systemic chemotherapy.

This study is listed at, NCT # 02905110, under “Brain Tumor Recurrent.” If you would like additional information about this study, please contact Marcia Kerr, RN by email at, or call (713) 500-7363.

Phase I Dose-Escalation Trial

A phase I dose-escalation trial is now open to qualified participants. The trial is the only study in the world investigating the direct administration of methotrexate into the fourth ventricle of the brain for the treatment of children with recurrent malignant fourth ventricular brain tumors.

The clinical trial is led by David Sandberg, M.D., FAANS, FACS, FAAP, director of pediatric neurosurgery at Children’s Memorial Hermann Hospital, the Memorial Hermann Mischer Neuroscience Institute at the Texas Medical Center, and the McGovern Medical School at UTHealth. Dr. Sandberg has pioneered the use of direct infusions into the fourth ventricle of the brain to treat children with recurrent malignant brain tumors in this location. A pilot clinical trial completed in August 2015 demonstrated that some patients with recurrent medulloblastoma experience a beneficial anti-tumor effect both within the fourth ventricle and at distant sites.

Delivering chemotherapeutic agents directly to the site of disease is particularly advantageous for children because it enables high drug concentrations at the site of disease origin while minimizing the side effects of chemotherapy by decreasing systemic drug exposure.

To learn more about the trial, contact Marcia Kerr at (713) 500-7363 or via email at