Out of tragedy comes hope: brain tumours breakthrough

When a child is diagnosed with a brain tumour, the entire family is thrown into a whirlwind. While some brain tumours are treatable, others are not. The brain tumour called “diffuse intrinsic pontine glioma” is located in the brain stem, an area that is crucial to many functions of the body such as breathing, blood pressure, and heart rhythms. Because of this, surgery cannot be done to remove the tumour. Chemotherapy does not work. Radiation helps for a bit but not for long. This type of tumour only occurs in middle childhood, and most children pass away within one year of developing symptoms. Research has been difficult because the tumour cannot be biopsied, meaning there has been no tissue available to do studies on. But some researchers and parents of children who have died from this tumour are trying to change all that.

Doctors in neurological sciences at Stanford University asked parents of some of their deceased patients to donate the tumours so that they could do research on them. This remarkable gift has resulted in a breakthrough in the study of this lethal tumour. The researchers were able to use the donated tissue to grow the cells in the lab. They are the first in the world to succeed at this. They were also able to introduce the tumour into animal models so that further research of its biology and other testing can be done. Results from their study are published in the current Proceedings of the National Academy of Sciences.

“These donations opened up a world in terms of being able to study this tumour, understand the biology behind its growth, and develop therapies,” said Dr. Michelle Monje, a paediatric neurologist and primary author of the paper.

As a result of the donations and research, Dr. Monje’s team gained insight into the molecular pathways of this type of tumour. They found that the tumour actually follows a type of molecular pathway which occurs in other types of brain tumours. Several pharmaceutical companies have already been developing drugs to block this pathway in other tumours. Dr. Monje’s research means those same drugs might one day be used to treat diffuse intrinsic pontine gliomas.

“For the families who donated their children’s tumours to the Stanford research, their gift provides an opportunity to have some good come from a terrible personal tragedy,” said Erin Digitale, a communications representative at Stanford University.

For more information, see the "Overview of the Brain" and "Diffuse Pontine Gliomas" sections of the Brain Tumours Resource Centre.

Sherene Chen-See


Monje M, Mitra SS, Freret ME, et al. Hedgehog-responsive candidate cell of origin for diffuse intrinsic pontine glioma. PNAS early edition. www.pnas.org/cgi/doi/10.1073/pnas.1101657108.

Digitale E. Discoveries offer first new hope in three decades for lethal pediatric brain tumor. Stanford School of Medicine press release Feb 28, 2011. http://med.stanford.edu/ism/2011/february/pontine.html. Accessed Mar 10, 2011.