School of Medicine

Study sheds light on treatment options for devastating childhood brain cancer

A new study led by Washington University School of Medicine provides new guidance on the treatment of medulloblastoma, a pediatric brain cancer. Some aspects of radiation therapy may be reduced while still providing effective treatment. Shown is a proton therapy plan for a patient with medulloblastoma. An extra "boost" radiation dose to the back of the brain is shown in red. The preventive radiation treatment to the entire brain and spine is shown in green. (Image: School of Medicine)

Medulloblastoma is a rare but devastating childhood brain cancer. This cancer can spread through the spinal fluid and be deposited elsewhere in the brain or spine. Radiation therapy to the whole brain and spine followed by an extra radiation dose to the back of the brain prevents this spread and has been the standard of care. However, the radiation used to treat such tumors takes a toll on the brain, damaging cognitive function, especially in younger patients whose brains are just beginning to develop.

A national study led by Washington University School of Medicine in St. Louis and St. Jude Children’s Research Hospital suggests that children with what is called “average risk medulloblastoma” can receive a radiation “boost” to a smaller volume of the brain at the end of a six-week course of radiation treatment and still maintain the same disease control as those receiving radiation to a larger area. But the researchers also found that the dose of the preventive radiation treatments given to the whole brain and spine over the six-week regimen cannot be reduced without reducing survival. Further, the researchers showed that patients’ cancers responded differently to therapy depending on the biology of the tumors, setting the stage for future clinical trials of more targeted treatments.

Children with average risk medulloblastoma have five-year survival rates of 75% to 90%. In contrast, children with what’s called “high risk medulloblastoma” have five-year survival rates of 50% to 75%. Other factors — such as a child’s age and whether the tumor has spread — help determine the risk category. For this study, the researchers focused on patients with average risk medulloblastoma.

The findings appears online June 10 in the Journal of Clinical Oncology.

“Medulloblastoma is a devastating disease,” said first and corresponding author Jeff M. Michalski, MD, the Carlos A. Perez Distinguished Professor of Radiation Oncology at Washington University. “It is a malignant brain tumor that develops in the cerebellum, the back lower part of the brain that is important for coordinating movement, speech and balance. The radiation treatment for this tumor also can be challenging, especially in younger children whose brains are actively developing in these areas. There’s a balance between effectively treating the tumor without damaging children’s abilities to move, think and learn.”

Children with average risk medulloblastoma typically undergo surgery to remove as much of the tumor as possible. They also receive chemotherapy and radiation therapy to prevent the spread of the tumor to other parts of the brain and spine through the cerebrospinal fluid.

“We wanted to investigate whether we could safely reduce the amount of radiation these patients receive — sparing normal parts of the brain and lessening the side effects for children with this type of brain cancer — while also maintaining effective treatment,” said Michalski, also vice chair and director of clinical programs in the Department of Radiation Oncology. “We found that reducing the dose of radiation received over the six-week course of treatment had a negative impact on survival. But we also found that we could safely reduce the size of the volume of the brain that receives a radiation boost at the end of the treatment regimen. We hope such measures can help reduce the side effects of this treatment, especially in younger patients.”

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