Brain Tumor 'Vaccine' Clinical Trials Open, Recruiting at UC

CINCINNATI—Researchers are currently seeking patients with glioblastoma multiforme (GBM) for two novel cancer vaccine clinical trials. This fast-growing form of brain tumor occurs in the central nervous system and accounts for 60 percent of the 17,000 new primary brain tumors diagnosed annually in the United States. 

"Despite advances in surgical technique, radiation and new chemotherapy drugs, survival rates for malignant brain tumors have remained disappointingly low over the past two decades,” explains Christopher McPherson, MD, director of surgical neuro-oncology for the UC Brain Tumor Center and assistant professor in the UC College of Medicine’s neurosurgery department. "Brain tumor vaccine therapy has emerged as a promising avenue for treating this disease more effectively, so we are excited to offer these trials in Greater Cincinnati.”

Led locally by McPherson and Rekha Chaudhary, MD, brain cancer vaccines are being offered for both newly diagnosed and relapsed GBM through separate trials. One of the vaccines targets a unique cellular mutation expressed in about 30 percent of brain tumors. 

"The experimental vaccine seeks to retrain the body’s natural defenses to identify and attack the cells involved in cancer formation, just as the immune system would attack foreign disease cells,” explains Chaudhary, a neuro-oncologist with UC Health and adjunct assistant professor at the UC College of Medicine. 

Celldex Vaccine Targeting EGFRvIII

This phase-3 study pairs an investigational vaccine (rindopepimut/GM-CSF) with standard therapy for GBM with radiation and chemotherapy to determine if the combination treatment approach could help brain tumor patients live longer than chemotherapy alone.  

The vaccine targets a specific mutation in the epidermal growth factor receptor (EGFR) called the EGFRvIII mutation, which is present in about 30 percent of patients with GBM.  

To qualify for the trial, patients must test positive for the specific mutation. Upon enrollment, the patient is randomized into one of two treatment arms to receive either the Celldex experimental vaccine or a blinded control. This trial is sponsored by Celldex Therapeutics, maker of the experimental vaccine.  

About 400 patients are expected to enroll in the trial nationwide, including 10 to 20 patients at the UC Brain Tumor Center, which is affiliated with both the UC Cancer Institute and UC Neuroscience Institute.

In addition, a phase 2 study, also sponsored by Celldex, offers the same investigational vaccine to patients with relapsed EGFRvIII-positive GBM. Patients enrolled in the study will be segmented into two treatment groups, based on previous treatment regimens. 

The first group will consist of patients who have experienced GBM recurrence and who have not previously received bevacizumab (chemotherapy drug). Patients within this group will be randomized to receive either the investigational vaccine or a blinded control in addition to their chemotherapy. The second group will include patients who have experienced GBM recurrence while on bevacizumab therapy. All patients in the second group will receive the investigational vaccine.

The study is expected to enroll about 95 patients from 20 centers across the United States, including the UC Brain Tumor Center. 

Chaudhary and McPherson have no financial interests in Celldex Therapeutics.

More Information 

»For more information on trial enrollment, call the UC Cancer Institute Clinical Trials Office at 513-584-7698. For specific trial information, visit cancer.uc.edu. Chaudhary and McPherson both treat patients at the Barrett Center and University Hospital as part of UC Health’s multidisciplinary neuro-oncology team. Both are affiliated with the UC Brain Tumor Center. 

The UC Cancer Institute and Brain Tumor Center are part of the Cincinnati Cancer Center, a joint cancer program involving the UC College of Medicine, Cincinnati Children’s Hospital Medical Center and UC Health. The collaborative initiative brings together interdisciplinary research teams of caring scientists and health professionals to research and develop new cures, while providing a continuum of care for children, adults and families with cancer.