New Brain Tumor Vaccine to Be Tested in Humans

December 1, 2010

For patients with low-grade gliomas, or slow growing brain tumors, a shot in the arm might soon lead to a new treatment therapy. A groundbreaking, first in humans vaccine will be tested in an early phase clinical trial that will soon begin at Wake Forest University Baptist Medical Center. Eighteen patients will be the first in the world to receive it.

“This study is looking at a very new form of treatment called a preventative brain tumor vaccine. The idea is to treat the low-grade glioma and to prevent it from growing back,” said Edward Shaw, M.D., a radiation oncologist.  “In this early phase study, we are looking to see whether the patient develops an immune response against this kind of brain tumor, a necessary step for the vaccine to work.”

The trial is a bi-institutional pilot study, funded by the National Cancer Institute, and shared with the University of Pittsburgh (UP). Wake Forest Baptist and UP will enroll nine patients each. The vaccine will be administered to adult patients who have been diagnosed and had surgery for the removal of a low-grade brain tumor. They will receive the vaccine every three weeks for six months. A simple blood test will determine whether an immune response has developed.

Brain tumors are relatively uncommon when compared to other types of cancer, Shaw points out, but they are the most common cause of cancer death in children. Low-grade gliomas are the most common primary brain tumors and usually affect children and young adults in their 20s, 30s and 40s. Low-grade gliomas often recur after surgery and radiation and may progress to a higher grade, more aggressive brain tumor. When they do, there is no curative treatment.

“If we can prevent the tumor from growing back or from transforming to a more malignant form, the hope is that we can cure more low-grade glioma patients,” Shaw said. 


The vaccine is the culmination of years of research and an interesting partnership that brings together three great scientific minds and their individual areas of expertise. Wake Forest Baptist has long been a leader, nationally and internationally, in the diagnosis and treatment of low-grade gliomas, and Shaw has led many of the research studies involving this type of tumor for the last 25 years. Shaw teamed up with Waldemar Debinski, Ph.D., director of the Brain Tumor Center of Excellence at Wake Forest Baptist, who discovered several of the glioma-associated antigens or molecules recognized by the immune system as foreign and targeted by the vaccine. 

The vaccine was developed by UP’s Hideho Okada, M.D., Ph.D., the third side of this scientific triangle, and is based in part on Debinski’s discoveries.

Okada’s expertise lies in immunological therapy and gene therapy for brain tumors. Okada has a long track record of inventive research in the field of tumor vaccines. Shaw and Okada are co-principal investigators for the study. Okada was the first to identify and fully characterize cytotoxic T-lymphocyte (CTL) epitopes in glioma-associated antigens, enabling him to develop novel vaccines. CTLs are the warriors that the immune system uses to fight infections and cancer.

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