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From the CEO’s Desk: Pediatric Brain Tumors – Circling the Square

On September 12th the National Brain Tumor Society announced a major new campaign to defeat pediatric brain tumors which seeks to bridge the gap between the latest science happening in laboratories and how children with the deadliest cancer fare in the clinic. Here’s why:

Ask any leader or expert in the field of pediatric neuro-oncology, and they’ll tell you that we are currently amidst the most exciting time in the history of pediatric brain tumor research and drug discovery.

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NBS CEO David Arons speaks during a press conference at the National Press Club announcing Project Impact: A Campaign to Defeat Pediatric Brain Tumors

Yet, earlier this month, the CDC released a sobering and jarring new report that found that pediatric brain tumors are now the leading cause of cancer death in all children and young adults ages 0-19, accounting for three out of every 10 cancer deaths in kids during 2014. We had known for some time that pediatric brain tumors were the leading cause of death in children under 14 years-old, but the new report showed that these tumors now surpassed leukemia for this dubious distinction in all kids under 19 years-old.

The reason for this is simple: while pediatric leukemia patients have benefited greatly from many of the great strides made in the past five decades in science and medicine, the most malignant pediatric brain tumors – pediatric high-grade gliomas, including diffuse intrinsic pontine gliomas (DIPG) – have not.

So how can it be that the pediatric brain tumor community is currently amidst an incredibly exciting time when these tumors just became the biggest cause of cancer deaths in kids in the United States? How do we square this circle?

It starts with understanding why pediatric brain tumors have not benefited equitably from advances in our understanding of cancer.

29075033314_8135844d72_oFor the most part, the biggest dissimilarity is the difference between leukemias and the deadliest pediatric brain tumors – again, pediatric high-grade gliomas, which disproportionally weigh-on the mortality rate of pediatric brain tumors – in response to chemotherapies. Many leukemias patients respond very well to treatment with chemotherapies. Pediatric high-grade glioma patients tend to receive little or no benefit from chemotherapies. Further, certain leukemia patients are eligible for bone marrow transplants (leukemia affects the production of blood cells, which takes place in bone marrow) as a high-risk, high-reward treatment. Of course, there is no equivalent for brain tumor patients. This is what underpinned this month’s CDC report.

The lesson here is that the most aggressive pediatric brain tumors need to be attacked with more precision-based treatments, that unlike systemic therapies like chemotherapy, target the specific molecular alterations that are driving the tumors. Moreover, given the complex and resistant nature of these tumors, a single drug may not be enough and drug combinations will likely be needed including both targeted- and immuno-directed therapies.

However, until just a few years ago, pediatric neuro-oncologists were basing their research and drug discovery efforts off of targets that had been identified in adult high-grade glioma patients. For instance, adult glioblastoma (a high-grade glioma) and pediatric glioblastoma looked identical under a microscope, so it was rationally believed that they were biologically similar. Turns out – based on new research enabled by emerging technologies like cheaper, faster, and easier genome sequencing – recent studies have found that the molecular drivers of pediatric high-grade gliomas are quite distinct from their adult counterparts.

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(L-R): David Arons, Dr. Roger Packer of Children’s National Health System, Dr. Suzanne Baker of St. Jude Children’s Research Hospital, Danielle Leach of St. Baldrick’s Foundation, and Cord Schlobohm of NBTS

From these studies, subsequent research has revealed mutations that appear with a high frequency and are likely triggers of pediatric high-grade glioma growth. These mutations – to a protein known as a “Histone H3” – represent, for perhaps the first time, a true target for pediatric brain tumor researchers to attack. This is where the excitement stems from.

But identifying a target is one thing, being able to successfully exploit it in a clinical setting is another.

That’s where the Defeat Pediatric Brain Tumors Research Collaborative comes in. It is through this ambitious precision medicine model for translation pediatric brain tumor research that we circle the square

The Defeat Pediatric Brain Tumors Research Collaborative brings together an all-star team of the leading scientists and clinicians in the field of pediatric neuro-oncology to work together and take on the key stages of research that now left to close the gap between having identified our main target and finding a treatment that will act against it. With all of these top researchers working together – and working on the different projects still needed, simultaneously – not only can we close the gap, but we can close it much quicker than if the field continued to work individually in their own labs.

And keeping with the spirit of collaboration, this project is also open to philanthropic and advocacy partners, as well. St. Baldrick’s Foundation, the largest private funder of childhood cancer research grants, has already joined with as a partner for the Collaborative, and we’re hopeful others from the brain tumor and pediatric cancer communities might sign on as well.

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Dr. Suzanne Baker of St. Jude’s speaks during the Project Impact press conference

This is a project that children – and the families of children – with brain tumors, particularly high-grade gliomas, deserve. For decades these children have been sidelined in the march toward progress seen in the greater cancer community. For years the pediatric brain tumor community has been subjugated to the land of ‘nothing’ and ‘no.’ These kids have no options, no choice, no treatments available for them, and there was nothing their doctors could do – just like there was nothing they could’ve done differently to prevent this.

“No” and “nothing” can’t be our answers anymore. An effort like the Defeat Pediatric Brain Tumors Research Collaborative is poised to bring the community together to capitalize on the latest science and technology has to offer to make good on the promising developments in the field of pediatric brain tumor research and improve survival for children with this deadly disease.

If you want to join us in the fight, please visit www.braintumor.org/projectimpact.

With Sincere Gratitude,

David F. Arons, JD

Chief Executive Officer

National Brain Tumor Society

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