💡 New hope emerges for children battling devastating brain cancer.

Embryonal tumor with multilayered rosettes (ETMR) is one of the most aggressive pediatric brain cancers, typically diagnosed before age 3. With survival rates heartbreakingly low, new therapies are urgently needed. Thanks to the V Foundation funded research, we’re now a step closer.

In a groundbreaking study, Dr. Volker Hovestadt and team at Dana-Farber Cancer Institute used advanced single-cell mapping and spatial imaging to reveal the inner workings of ETMR at an unprecedented level. They found that this cancer mimics the earliest stages of brain development, with a mix of immature, stem-like cells and more mature neuron-like cells, locked in a broken developmental cycle.

It turns out that at the center of this dysfunction is a cluster of cancer-driving molecules called C19MC microRNAs, found mostly in the most aggressive, stem-like cells. These microRNAs act like a brake on normal development, keeping cells stuck in a dangerous “never-grow-up” state. 

Even more exciting: Dr Hovestadt and team identified how cells inside the tumor talk to each other using communication systems and pathways to fuel the cancer’s growth. When they disrupted these pathways with drugs already in development (and in some cases, already FDA-approved), they saw powerful anti-tumor effects in lab models and even helped one young patient. 

🎯 Why this research matters: The team’s work offers a blueprint for targeting ETMR at its roots: by blocking the signals that keep it growing and preventing healthy cell maturation. Their findings point to real, testable strategies for treatment, bringing new hope to the youngest of patients and their families who are facing this rare and devastating disease. 

💥 If you know the V Foundation, you won’t be surprised that investments from our incredible donors helped make this discovery possible. We believe in supporting bold science that could save lives too young to fight for themselves. 

If you’d like to read more about the Hovestadt lab, you can find them here: https://hovestadtlab.github.io/pages/Team/ and you can read their paper at Cellular hierarchies of embryonal tumors with multilayered rosettes are shaped by oncogenic microRNAs and receptor–ligand interactions | Nature Cancer.