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Clinical trial testing novel treatment for recurrent posterior fossa ependymoma now open for enrollment

Pediatric neurosurgeon David I. Sandberg, MD, consults with a patient at Children's Memorial Hermann Hospital. (Photo by Children's Memorial Hermann Hospital)
Pediatric neurosurgeon David I. Sandberg, MD, consults with a patient at Children's Memorial Hermann Hospital. (Photo by Children's Memorial Hermann Hospital)

A new clinical trial analyzing whether infusions of two drugs directly into the brain can effectively treat children and adults with recurrent posterior fossa ependymoma, a malignant brain tumor, is now recruiting patients at The University of Texas Health Science Center at Houston (UTHealth Houston).

Led by David I. Sandberg, MD, professor and director of Pediatric Neurosurgery with McGovern Medical School at UTHealth Houston, the study aims to determine whether simultaneous infusions of two chemotherapy drugs — 5-Azacytidine (5-AZA) and trastuzumab — into the fourth ventricle of the brain or resection cavity in patients with recurrent posterior fossa ependymoma can safely shrink or kill the tumor.

An ependymoma is a rare type of primary brain or spinal cord tumor. They are found throughout the central nervous system, including in the back of the head, known as the posterior fossa.

“Recurrent ependymoma is a disease that has no cure, and the overwhelming majority of patients die of their disease despite efforts at salvage therapy,” said Sandberg, the Dr. Marnie Rose Professor in Pediatric Neurosurgery at McGovern Medical School. “We are trying to provide hope to patients by offering a novel treatment approach in which drugs are infused directly into the site of disease origin and recurrence. Our hope is that we can obtain high drug levels at the site of disease, thereby killing tumor cells there, without having high drug levels in the bloodstream, which causes side effects without any definite treatment effect.”

In the U.S., 200 new cases of ependymoma are found in children and adults annually, though it occurs more often in children than adults. The overall five-year survival rate for individuals with ependymoma is about 82%, which drops to 72% among children up to 19 years old.

Currently, an ependymoma is treated by surgical resection followed by radiation therapy. When tumors recur, additional surgery, additional radiation, or chemotherapy may be offered, but the success rate with each of these types of treatment is very low. Because of the high failure rate of current treatments, novel treatment approaches are necessary, Sandberg said.

He is optimistic about the combination of the two drugs for several reasons. In a previous small pilot trial, Sandberg’s team infused 5-AZA into the fourth ventricle of the brain in patients with recurrent ependymoma, and two patients, who had failed multiple prior types of treatment, had shrinkage of their recurrent tumor. The second drug, trastuzumab, is also extremely active in the laboratory against ependymoma cells.

“We are hopeful that the combination of these two powerful drugs will have a better response than either drug individually,” Sandberg said.

Patients who enroll in Sandberg’s trial will receive weekly 10 mg intraventricular 5-AZA infusions for six consecutive weeks. They will also receive weekly 21 mg intraventricular trastuzumab infusions for six consecutive weeks.

All patients will undergo an MRI of the brain and total spine within seven days after the final 5-AZA and trastuzumab infusion to determine their response to the treatment and to assess for any signal changes in the brain or spine caused by the infusions. Additionally, there will be 30-day and 90-day follow-up assessments by telephone or in person.

The trial will enroll 10 study participants. Eligible participants can be between 1 and 80 years old and must have histologically verified ependymoma that originated in the back of the head, with recurrence or progression anywhere in the brain and/or spine. Patients are also eligible if they have a residual tumor that has not been completely cleared despite prior treatments.

The study is listed at ClinicalTrials.gov under NCT04958486. For additional information about the study, please contact Bangning Yu, RN, at Bangning.Yu@uth.tmc.edu or 713-500-7363.

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