New DIPG Advances: Promise for kids with brain stem gliomas | Dana-Farber Cancer Institute

Diffuse intrinsic pontine gliomas (DIPG) in children are very rare and lethal. Dana-Farber challenged the dogma about these brain stem gliomas, and finally, the study and treatment of DIPG is moving forward. Learn more about our discoveries at http://www.DiscoverCareBelieve.org.


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When I started in the field of neuro-oncology, there was one disease that was understood to have no therapeutic options whatsoever. When a child was diagnosed with a Diffuse Intrinsic Pontine Glioma, it was understood from the very beginning to the medical staff that this was a child that really had no hope.

Pontine Gliomas occur right here, in a critical part of the brain. This is what decides that your heart beats, that you've got blood pressure, that you breathe when you’re asleep. No one wanted to biopsy this, because to do so would damage child. And unfortunately, in the old days of the 1960s and ‘70s, biopsies did a ton of damage, and from that came the accepted dogma that you do not have to biopsy these tumors.

The problem is because you learn nothing about the tumor, there’s no way to improve the very problem you're dealing with. We made the first proposal for this concept of biopsy and molecular analysis win 2002. In 2003, we re-presented the same idea at multiple meetings—2004, 5, 6, 7, 8, 9—and time and time again, there was just sense that people were not ready to believe that the biology and technology had advanced.

Fortunately in 2009, some of our colleagues and friends came to the same conclusion and just went ahead and biopsied 24 kids and showed that—in fact, exactly as we predicted—they could do it safely. No damage to the kids. No damage to those critical centers. And we could do the kinds of analyses that begin to allow us to understand for the first time what these tumors are. For the first time, we have some idea of the enemy in front of us, and I think we can really begin to put together the structures and platforms that are going to be needed to deal with abnormalities that the individual children have.

We needed the advances in all of the neurosurgical techniques. We need all of the advances in the genomics and the molecular understanding. But we also needed, for example, the families that raised all of the money that actually allowed all of this clinical trial. Families are the ones that have stepped up and said, “Listen, this disease—as rare as it is—is critically important to us,” pushing the science, the medicine, the treatments forward, because they've only got one commitment: it is that disease.