Graphic image of brain.

Graphic image by goa novi, Shutterstock.

Stanford Medicine Scope - October 31st, 2016 - by Erin Digitale

There’s a fascinating new story in Scientific American about how precision-medicine techniques are helping scientists crack the mysteries of the deadliest brain tumor. The tumor, a form of pediatric cancer called diffuse intrinsic pontine glioma, is truly devastating: It has a one-percent survival rate five years after diagnosis, and that figure hasn’t budged in 40 years. As the story explains, frustration with the dearth of treatment options for DIPG led one Stanford scientist to devote her research to studying the disease:

Michelle Monje, an assistant professor of neurology at Stanford University, first encountered DIPG around 2002 as an MD/PhD student there. Working with her clinical mentor to care for a nine-year-old girl dying of DIPG, it was “the first time I’d come upon a disease we had no idea how to treat,” Monje says. “I felt so close to this patient and was devastated by my inability to help her.”

Back then there was little molecular data on DIPG. No animal models. No cell cultures. Generating such research tools requires tumor samples from patients. Yet since MRI scans can reliably diagnose typical DIPG and getting brain stem tissue is not trivial, biopsies were rarely done. With precious little tumor tissue to study in the lab, Monje says, research progress on DIPG had stalled for decades.

The story goes on to explain how Monje and other scientists around the world are using the latest research methods to look for new approaches to DIPG treatment. For instance, they’re characterizing the gene mutations that drive the tumor, developing cell-line and animal models of DIPG to test therapies in the lab, and searching for forms of chemotherapy that could fight the disease. One of the fruits of this work is a clinical trial Monje is leading to test a drug called panobinostat; mouse studies suggest it could lengthen the lives of DIPG patients.

I’ve been reporting on Monje’s DIPG work since its early days, and I found it very interesting and helpful to read about how her discoveries fit into the growing picture of DIPG science.

Originally published at Stanford Medicine Scope Blog