Baylor College of Medicine researchers at Texas Children's Cancer Center and their collaborators at The Methodist Hospital have launched two phase I clinical studies using immunotherapy to treat glioblastoma multiforme, the most aggressive type of brain cancer in both children and adults.

"Brain tumors, particularly GBM, remain one of the major challenges in pediatric and adult cancer so there is a dire need for new and innovative therapies," said Dr. Nabil Ahmed, assistant professor of pediatrics – hematology/oncology and principal investigator of the studies. "Patients with GBM are typically treated with surgery followed by radiation therapy and chemotherapy but their outcome is very poor."

Current prognosis

Adult patients usually survive between one to two years after being diagnosed, and the prognosis for children is only slightly better, he said.

"GBMs grow progressively and uncontrollably, partially because glioma cells are able to escape destruction by the immune system," explained Ahmed, also a pediatric hematologist/oncologist at Texas Children's Cancer Center. "We have shown in preclinical studies that immune cells from GBM patients, called T cells, can be rendered specific for GBM and have potent anti-GBM activity."

Based on these results, the research team has developed two clinical studies. Both studies rely on taking T cells from the blood of GBM patients. In the laboratory, the T cells are then rendered specific for GBM before being reinfused into patients to attack different molecular targets on the patients' tumor. The first study targets a common virus called cytomegalovirus (CMV), which is present in the majority of GBMs, and the second study targets CMV as well as a molecule present on GBM cells called Human Epidermal Growth Factor Receptor 2 (HER2).

T-cell approach

"Similar T-cell therapies, developed by BCM investigators, targeting other viruses and types of cancers have shown promising results in clinical studies, and we hope that our T-cell therapy approach for GBM will be equally successful," Ahmed said.

These studies were developed by Ahmed and Dr. Stephen Gottschalk, associate professor of pediatrics – hematology/oncology at BCM, who are both members of the Texas Children's Cancer Center and the Center for Cell and Gene Therapy at BCM, in collaboration with Dr. Robert Grossman, Dr. Yvonne Kew, Dr. Pamela New and Dr. Suzanne Powell, all of The Methodist Hospital.

Grant funding for these studies came from The Clayton Foundation for Research, The Dana Foundation, The American Brain Tumor Association, The Simmons Family Foundation and The Alliance for Cancer Gene Therapy. Funding was also provided by private individuals.

The Texas Children's Cancer Center is a joint program of Baylor College of Medicine and Texas Children's Hospital, and it is the pediatric program of the NCI-designated Dan L Duncan Cancer Center at Baylor College of Medicine.