By: Trish Adkins
It was the middle of the night when then 6-year-old Frankie woke up screaming that his head hurt. Within 20 minutes, he wasn’t screaming anymore — he was unresponsive as they rushed him to the emergency room. Frankie had a brain bleed, caused by a rare brain tumor called pilomyxoid astrocytoma. There were also tumors in his spine. Frankie tried several chemotherapies and other targeted treatments. Nothing worked. Then, finally, proton radiation — 30 rounds of it — did. The tumors in his spine were gone, and his brain tumor was stable.
But stability came at a steep price. Frankie developed Moyamoya disease, a progressive blood vessel disorder of the brain. It impacted his ability to concentrate, walk and caused strokes.
In April 2024, Frankie died. Not only had his tumor started growing again, but the harsh treatments were too much for his body. He was just 12 years old.
Sadly, Frankie’s story is not uncommon. Brain tumors are the deadliest form of pediatric cancer and one of the most common malignancies in children under 3 years old. There are literally hundreds of types and subtypes of brain tumors, each with their own genetic markers that challenge oncologists to find treatments that work. And because of their location in one of the most critical and sensitive structures of the brain, all treatments must be considered with the potential of devastating side effects.
“One of the keys to treatment is being able to do an effective surgery and remove as much of the cancer as possible and ideally all of it. But for pediatric brain tumors, even though the surgeons do incredible things, they can't always do that right. The tumors will be in parts of the brain that a child can't lose, so it can't just be taken out,” said Dr. Will Parsons. 
There are other challenges, too. The central nervous system has protective mechanisms to keep out things it views as “bad,” which could include treatments like chemotherapy. And there is the continual, pressing issue of trying to understand the biology and genetics of brain tumors.
“That’s where there's been incredible progress over the last 10 to 20 years,” said Dr. Parsons. Dr. Parsons points specifically to some of the progress made studying and classifying medulloblastoma tumors. What was once considered one type of tumor, has been split into several types defined more specifically by their molecular components (the cell type, the genes and proteins present in the tumor). This classification has guided some new insights into treatments that are less harsh, giving hope for all children with brain tumors.
“ALSF has funded many brain tumor genetics related projects, some of which have generally contributed in small ways to this new understanding of molecularly-defined tumor types,” said Dr. Parsons. Dr. Parsons was one of the first Alex’s Lemonade Stand Foundation (ALSF) Young Investigator Grant recipients in 2007. His work studying the genetics of pediatric brain tumors laid groundwork for future work that led to more accurate molecular descriptions of brain tumors.
ALSF has funded more than 160 brain tumor focused projects since 2006; with even more planned on the horizon. Right now, researchers are studying targeted treatments like CAR T immunotherapy to see if these therapies could offer cures without devastating side effects.
Researchers can also suffer from a lack of access — access to tumor models and data. Pediatric brain tumors are rare, as compared to their adult counter parts. Without quality information about the genetics of these tumors, research can be slowed. To address this, ALSF’s Childhood Cancer Data Lab established the OpenPBTA, which stands for the Open Pediatric Brain Tumor Atlas. The tumor database is open to all researchers without charge and includes the genomic data for 1,074 pediatric brain tumors. The OpenPBTA can give researchers more information that can power their research faster than ever before.
And all of this has been fueled by generous donations to ALSF.
“We’re still losing too many children to pediatric cancer. But personally for our family, it’s always been important to Frankie that we raise money and give back because it made him feel more empowered, like he’s doing something to fight back against the illness that has taken so much from him,” said Erica Avello, Frankie’s mom.
May is Brain Tumor Awareness month. You can learn more about pediatric brain tumors and the pressing need for more research, here (and you can share the facts on social media!) Alex’s Lemonade Stand Foundation founder Alex Scott believed that together, we would find cures for childhood cancer. In that spirit, you can sign up to be part of our monthly giving program, the One Cup at a Time Club! Learn more here.
