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Pediatric Brain Tumor Trials

Infusion of MTX110 into the Fourth Ventricle in Patients With Recurrent Medulloblastoma

The Children’s Neuroscience Center’s newest pilot study seeks to enroll qualified participants with recurrent medulloblastoma. This novel trial places MTX110, a new formulation of panobinostat, directly into the fourth ventricle of the brain. The research study is led by David Sandberg, MD, FAANS, FACS, FAAP, Director of Pediatric Neurosurgery at Children’s Memorial Hermann Hospital and Dr. Marnie Rose Professor of Pediatric Neurosurgery at the University of Texas Health Science Center at Houston (UTHealth).

MTX110 is a chemotherapy drug that has shown promise in laboratory models of medulloblastoma, the most common malignant brain tumor in children. Treatments that are currently available for recurrent medulloblastoma are often associated with considerable toxicity, and when tumors recur despite these treatments, survival rates are low. Dr. Sandberg’s novel trial places the chemotherapy directly into the fourth ventricle, in an effort to deliver the treatment only to the affected area while avoiding the surrounding healthy tissue.

The pilot study, which has been approved by the Food and Drug Administration, will enroll five (5) patients with recurrent medulloblastoma at Children’s Memorial Hermann Hospital. MTX110 is a new formulation of panobinostat from Midatech Pharma PLC. This study is listed at ClinicalTrials.gov under NCT04315064. For more information, please contact Bangning.Yu@uth.tmc.edu or call (713) 500-7363.

Infusion of 5-AZA Pilot Study (Recurrent Posterior Fossa Ependymoma)

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 “Infusion of 5-Azacytidine (5-AZA) into the Fourth Ventricle or Resection Cavity in Children with Recurrent Posterior Fossa Ependymoma: A Pilot Study.” It is open to patients who are age 1 to 21 years old with recurrent ependymoma that originated in the posterior fossa of the brain.

The study employs a novel means of treating ependymoma brain tumors that originate from the fourth ventricle: infusion of a chemotherapy agent directly into the fourth ventricle rather than systemic intravenous delivery. The agent being infused, 5-AZA, has never been infused into the brain in humans but has been shown to effectively kill ependymoma cells in the laboratory. There will be no simultaneous systemic chemotherapy.

This study is listed at ClinicalTrials.gov, NCT # 02940483, under “Brain Tumor Recurrent.” If you would like additional information about this study, please contact Bangning Yu, M.D., Ph.D., by email at Bangning.Yu@uth.tmc.edu or call (713) 500-7363.

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 ClinicalTrials.gov, NCT # 02905110, under “Brain Tumor Recurrent.” If you would like additional information about this study, please email Bangning.Yu@uth.tmc.edu 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 please email Bangning.Yu@uth.tmc.edu or call (713) 500-7363.