Dramatic Progress for Medulloblastoma Patients

by St. Baldrick's Foundation
August 2, 2021

Want to know how St. Baldrick’s donors are the saving lives of kids with a common brain tumor? This isn’t just an example of progress – it’s the biggest increase in survival rates many researchers have ever seen from one clinical trial! And that trial was supported by St. Baldrick’s.

Grace was diagnosed with medullobalstoma at age 5. She is an almost-14-year survivor.

Brain tumors are the leading cause of disease-related death in children, and medulloblastoma is the most common pediatric malignant brain tumor. Most are diagnosed before the age of 10.

St. Baldrick’s has supported a lot of research aimed at finding cures for medulloblastoma, but the biggest impact to date was reported on July 21, 2021, in the Journal of the American Medical Association (JAMA Oncology).

And it wasn’t because of a new drug.

Like so many advances in pediatric cancer, this progress came from using existing treatments in a new combination. In this case, that means adding a chemotherapy drug called carboplatin during radiation therapy.

The result?

The 5-year survival rate increased from 54% to 73% for children with high-risk group 3 medulloblastoma.

That’s a 19% increase – almost unheard of from one clinical trial!

And it emphasizes the need to know whether a child with medulloblastoma falls into this high-risk group at diagnosis, as this is the only group for which this more intense therapy made a significant difference. 

In real life terms, this means that in this type of medulloblastoma, 20 more kids of every 100 will survive than would have before this discovery.

St. Baldrick’s Support Made It Possible

The St. Baldrick’s Foundation is the biggest non-government supporter of the Children’s Oncology Group (COG), which conducted this significant clinical trial. 

COG Chair, Doug Hawkins, MD of Seattle Children’s Hospital, says, “The St. Baldrick’s Foundation was pivotal in the conduct of this study. By providing COG institutions with additional support for every patient enrolled on a study, St. Baldrick’s contributed to the study’s dramatic, practice-changing results.”

Also involved in this study was Dr. Paul Northcott, recipient of the prestigious St. Baldrick’s Robert J. Arceci Innovation Award.  “This is a critical advance that will have a profound impact on how children with high-risk group 3 tumors are clinically managed in the future,” he says.  Dr. Northcott’s lab led the molecular summarization of the clinical trial, and he adds that studies of this scale would not be possible without the kind of support St. Baldrick’s provides.

Much work remains to be done before every child with a brain tumor can be cured. But St. Baldrick’s supporters can be proud to play an important role in this exciting and dramatic advance.

Together, let’s keep supporting research to give kids a lifetime.


Read more on the St. Baldrick’s blog