Researchers turn fatal brain cancer into treatable disease for children

Some children with a rare brain tumor that is considered almost universally fatal can be saved if they receive radiation therapy followed by tandem (given one after the other) cycles of high-dose chemotherapy. This finding, from researchers at St. Jude Children's Research Hospital, is published in the March 1 issue of the Journal of Clinical Oncology (JCO).

The tumor, called atypical teratoid/rhabdoid tumor (ATRT), is a rare aggressive cancer that arises either in the cerebellum or cerebral cortex and often spreads through the central nervous system. The cerebellum is the lower, back part of the brain that controls balance.

The St. Jude team showed that, while children with ATRT under three years of age have a dismal prognosis, children older than 3 can be cured by upfront treatment with radiation followed by chemotherapy using so-called alkylating agents (cyclophosphamide, cisplatin, vincristine and etoposide), according to Amar Gajjar, M.D., director of St. Jude Neuro-oncology. In this case, physicians first surgically removed as much of the tumor as possible before treating children with radiation followed by chemotherapy.

The success in raising the survival rate among these children reflects the long-standing leadership of St. Jude in addressing the needs of children who suffer from rare cancers, as well as more common malignancies, according to Gajjar.

"Effective therapy for patients with ATRT has remained elusive until now, at least for children older than 3 years," said Gajjar, senior author of the JCO report. "The fact that we found that upfront radiation is so effective, especially in older children, suggests that this should become a standard approach to ATRT therapy."

Gajjar's team studied the medical records of 31 patients, 22 of whom were younger than 3 years old. All patients had surgery, and 30 received subsequent chemotherapy. Most patients who were 3 years old or older received postoperative radiation to their brains and spines.

The rates of two-year event-free survival (EFS, 78 percent) and overall survival (OS, 89 percent) among children aged three and older were significantly better than those for younger patients (EFS, 11 percent; OS, 17 percent). Event-free survival is the time after treatment that a person remains free of severe treatment side effects, recurrence or progression of cancer, or death from treatment side effects or the cancer itself.

The study also demonstrated that older children who experienced relapse of ATRT could be saved using ICE chemotherapy (ifosfamide, carboplatin- and etoposide).

Other authors of the report are Tanya M. Tekautz, Christine E. Fuller, Susan Blaney, Maryam Fouladi, Alberto Broniscer, Thomas E. Merchant, Matthew Krasin, James Dalton, Gregory Hale, Larry E. Kun, Dana Wallace and Richard J. Gilbertson.