Charlotte Brain Tumor Patient
Is First In The Country To
Receive Experimental Treatment

Originally published Fall 1999

A new study is underway in Charlotte evaluating an experimental treatment of Glioblastoma, a type of incurable brain tumor. This experimental therapy is being offered jointly by Carolinas Medical Center, Presbyterian Hospital, and Carolina Neurosurgery and Spine Associates, and is part of a multinational clinical trial sponsored by Neurocrine Biosciences, Inc., a biotechnology company in San Diego, California. The first patient to be treated on this protocol in the United States received this therapy at Carolinas Medical Center on February 9, 1999. The therapy involves the administration of a bacterial toxin (IL-4 toxin) directly into recurrent brain tumors.

Anthony Asher, M.D., F.A.C.S.
Glioblastoma is the most common and deadliest form of brain tumor, accounting for approximately 6,500 cases in the United States each year. The average survival time with standard treatment of surgery, followed by radiation therapy to reduce the tumor size, is approximately 12 months. Chemotherapy can also be administered, but patient quality of life and overall survival rates remain low. Because of the highly aggressive nature of this disease, researchers are working to develop innovative therapies to improve patients’ survival.

Researchers at the Food and Drug Administration have discovered that malignant brain tumor cells have receptors on their surface for the Interleukin-4 (IL-4) molecule. IL-4 is a molecule that normally mediates certain immune responses. In an attempt to use this finding to develop anti-tumor therapies, the FDA researchers in collaboration with researchers from the National Institute of Health, bound the IL-4 molecule to a bacterial toxin in the laboratory. They found that once this IL-4 Pseudomonas-exotoxin bound to receptors on tumor cells, the toxin was taken inside the cells. Once inside the cells, the toxin molecule became active, killing the cancer cells by destroying their protein production capabilities. Brain cancer cells are killed while normal cells are spared because the malignant cells, but not normal brain cells or immune cells, express high levels of IL-4 receptors and therefore bind the IL-4 toxin.

In this study, patients with a recurrent malignant tumor will undergo a biopsy of their tumor to confirm the diagnosis of recurrent disease. Once the recurrence is confirmed, up to three silastic catheters will be placed in and around the tumor. The toxin will then be delivered through the catheters into the tumor bed via a micro infusion pump over a 96-hour period. Following the infusion, the catheters will be removed and patients will have serial MRI scans to assess the efficacy of the toxin.

Carolinas Medical Center and Presbyterian Hospital will be participating along with approximately a dozen major medical centers in the United States and Germany in this project. For more information on the IL-4 toxin you may contact Dr. Anthony Asher, Principal Investigator of the study at 1-800-344-6716
or click here.

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