UTHealth and Children's Memorial Hermann begin two new clinical trials for pediatric brain cancer

Two new clinical trials for pediatric brain cancer have begun at The University of Texas Health Science Center at Houston (UTHealth) and Children's Memorial Hermann Hospital.

The pilot trials, approved by the U.S. Food and Drug Administration, both focus on the administration of chemotherapy agents directly into the fourth ventricle of the brain, the most common site for pediatric brain tumors and one that is difficult to access surgically.

"Administering chemotherapy directly to the site of the tumor can enable very high drug levels at the site of active disease while decreasing the likelihood of systemic toxicity," said David Sandberg, M.D., the Dr. Marnie Rose Professor in Pediatric Neurosurgery at McGovern Medical School at UTHealth, who pioneered the procedure. "Current treatment options can have serious side effects, and we need to do better. Radiation therapy can be harmful to the developing brain and chemotherapy in the high levels needed to cross the blood-brain barrier can cause many side effects as well as damage to organs throughout a child's body."

Both trials are designed to target brain tumors that develop in the posterior fossa portion of the brain that includes the cerebellum, brain stem and fourth ventricle.

One trial builds on Sandberg's previous research that investigated the infusion of methotrexate for pediatric brain tumors. Results of the research, published in October 2015 in the Journal of Neuro-Oncology, showed that some patients with recurrent medulloblastoma experienced a beneficial anti-tumor effect. The new study will look at combining methotrexate with another chemotherapy agent, etoposide, for infusion into the fourth ventricle for medulloblastoma, the most common malignant brain tumor in children. Children with other tumors such as atypical teratoid rhabdoid tumor (ATRT) are also eligible.

The second trial focuses on a different chemotherapy agent for a type of brain tumor called recurrent posterior fossa ependymoma. Ependymomas form from ependymal cells that line the ventricles and passageways in the brain and spinal cord, according to the National Cancer Institute.

The chemotherapy agent, 5-azacytidine (5-AZA), is infused directly into the fourth ventricle. The agent has never been injected into a human brain before but has been shown to be promising in treating ependymomas in the laboratory. No systemic chemotherapy will be given to clinical trial participants.

Both studies are open to patients age 1 to 21.