Cutting-Edge Genomics Lead to Groundbreaking Clinical Trial for Brain Tumors in Young Kids

Our team at St. Jude Children’s Research Hospital recently published an extensive review article in the Journal of the National Comprehensive Cancer Network® (NCCN®) . This piece provides a comprehensive look into the evolving understanding and treatment of medulloblastoma, the most common malignant brain tumor diagnosed in children.

In our review, we trace the history of medulloblastoma treatment protocols and landmark studies involving infants and young children. For many years, doctors took a one-size-fits-all approach using surgery, craniospinal radiation, and chemotherapy. But they knew this carried significant risks for developing brains.

Efforts to delay or avoid radiation led to important discoveries. Outcomes varied significantly based on clinical and pathological factors, suggesting underlying biological differences.

The molecular revolution transformed medulloblastoma classification, identifying consensus subgroups with distinct clinical behavior. Patterns emerged – the distribution of molecular subgroups changes dramatically with age. Critically, diversity was uncovered within subgroups as well.

This is reviewed in detail in a different publication.

Integrating molecular insights led to more personalized treatment regimens tailored to each child’s specific tumor. Instead of blanket therapy, kids could receive targeted strategies adapted to their molecular subtypes and risk factors.

Our review laid the foundation for our team’s groundbreaking new clinical trial, SJiMB21, now underway at St. Jude. This trial incorporates next-generation molecular classification to precisely guide frontline treatment for our youngest patients with medulloblastoma.

Infants with a certain molecular subtype receive chemotherapy alone, aiming to maximize survival while avoiding radiation's neurocognitive impacts. Other molecular-based regimens are customized based on subgroup/subtype and disease attributes.

SJiMB21 represents a truly modern approach to clinical research, rapidly translating complex genomic discoveries into smarter, kinder patient care. We hope it will become a new standard for treating medulloblastoma and other childhood cancers.

Our work offers just a glimpse into the future of precision medicine and the progress being made for our most vulnerable patients. Though treatments remain limited today, the molecular revolution has ignited tremendous hope. We believe emerging science will illuminate the path to targeted therapies that cure kids while allowing them to thrive.

#PrecisionMedicine #Bioinformatics #BrainTumors