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The Time is Right for Pediatric Brain Tumor Advances

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Pediatric brain tumors continue to take a heartbreaking toll on far too many kids and their families each year. With no cure, and not even a standard of care for many pediatric brain tumor types, the current outlook for those diagnosed with this disease is simply unacceptable.

This year in the U.S., an estimated 4,620 infants, children, and adolescence will receive a brain tumor diagnosis. This rate of occurrence, unfortunately, now places brain tumors atop the list most common cancers in the pediatric population. Of these cases, approximately 20% will be given the diagnosis of an aggressive class of brain tumors known as pediatric high-grade gliomas (pHGGs). Tragically, pHGG patients are faced with five-year survival rates of only 15-30%. Even more devastating, a unique subgroup of pHGG patients diagnosed with a tumor known as diffuse intrinsic pontine glioma (DIPG) have a mean survival of only nine months…roughly the length of an average school year.

034Looking at these statistics and knowing that the survival figures have barely budged in nearly 40 years, one can’t be blamed for feeling discouraged. But, the bravery we see from children battling pediatric high-grade gliomas and other brain tumors gives the National Brain Tumor Society the inspiration we need to stand-up and demand more for these kids.

Thankfully, many great strides have been made in just the past few years in understanding pHGGs, including DIPG, and how to treat them. For the first time in history, we are finally in a position to push the creation of new, effective therapies of children with these aggressive brain tumors.

Building a New Foundation

One researcher or lab did not make all the discoveries, nor did one agency or foundation fund the research that got us to where we are today. The accumulation of findings from years of commitment and diligence from the research and patient community, in addition to a set of game-changing findings in 2012, has built a new foundation of scientific knowledge for which to combat pHGGs:

  • Studies conducted in 2007 and 2010 by Drs. Nada Jabado (McGill University/Montreal Children’s Hospital) and Suzie Baker (St. Jude Children’s Research Hospital) highlighted substantial differences in pHGGs compared with tumors from adult patients and suggested the existence of molecularly diverse subgroups within pHGGs.[i],[ii],[iii]
  • In 2011, Dr. Michelle Monje (Stanford School of Medicine) and colleagues developed DIPG models derived from tissue directly from DIPG patients.[iv] This helped transform the path of preclinical pediatric research from one that was accustomed to using adult models, to using models of the actual disease being studied.
  • In 2012, Drs. Baker and Jabado, along with their colleagues, made what is likely the most significant research finding to date in the field of pHGG.
    • Their separate, but complementary research, pointed to mutations in cells’ histones (proteins that form the structures around which DNA is wrapped) as unique drivers of pHGGs. [v],[vi] These studies were the first to report on the association of epigenetic mutations (mutations not caused by changes to the DNA sequence) in regulatory histones (which control how genes are switched on or off in a cell) with human tumors, and provided clear evidence that pHGGs are a distinct entity from adult HGGs.

The Blueprints For A Cure

These latest scientific discoveries are already being integrated into research, leading to improvements in how we study pHGGs, as well providing targets for innovative treatment exploration. Unfortunately, the pediatric drug discovery and development landscape is still riddled with additional barriers that are currently hindering us from fully capitalizing on these breakthroughs. These barriers include, but are not limited to inconsistent regulations around clinical trials and isolated research efforts.

NBTS_OC_0348Building off the solid foundation finally in place for pHGG research, the National Brain Tumor Society has created a framework and blueprint to move promising research closer to treatments that will benefit pediatric brain tumor patients. This effort is being facilitated through our newest pediatric initiative, Project Impact: Driving Discovery to a Cure for Pediatric Brain Tumors.

Project Impact has a unique approach to pediatric cancer research and drug development, which combines our strengths in galvanizing the global research community, while utilizing public policy to drive change and confront inefficiencies in pediatric drug discovery and development. By optimizing the entire R&D pipeline and breaking down existing barriers we will be able to accelerate new and effective treatments into the clinic for pHGGs patients.

In addition to the National Brain Tumor Society staff, this effort also includes legions of researchers, clinicians, nurses, and biopharmaceutical industry collaborators who are committed to not only solving the riddles of pediatric brain tumors, but also translating discoveries into increased survival. Furthermore, we also have over 30,000 advocates supporting our efforts on the public policy front.

We Can Succeed

Recently while attending a conference on Precision Medicine, one of the speakers said that developing a cancer treatment, from inception to market, is harder than landing a person on the moon. If that’s the case, look at what NASA has done recently; their New Horizons Mission has recently begun capturing unprecedented views of Pluto, the most distant planet in our solar system – some 7.66 billion miles beyond the Earth’s moon. Landing a person on the moon does not look all that hard anymore, does it? Given the latest advances and excitement around Project Impact, nor does developing effective treatments for kids living with a brain tumor today and those who will be diagnosed tomorrow. I believe that we are well own our way in our own mission.

Support Our Efforts

There has never been a better time for pediatric brain tumor research, and with September serving as Childhood Cancer Awareness Month there is also no better time to join the fight with the National Brain Tumor Society. Nearly all research funding for pediatric cancers currently comes from the National Cancer Institute (NCI), private foundations, and philanthropic sources like NBTS. Therefore, your support will be critical in not only advocating for policies that will help advance pediatric research and drug development, but also in directly funding pediatric brain tumor research by donating here!


 

[i] Faury et al. ,Molecular profiling identifies prognostic subgroups of pediatric glioblastoma and shows increased YB-1 expression in tumors. J. Clin. Oncol., 2007. 25: 1196–1208.

[ii] Haque, et al., Gene expression profiling from formalin-fixed paraffin-embedded tumors of pediatric glioblastoma. Clin. Cancer Res., 2007. 13: 6284–6292.

[iii] Paugh et al., Integrated molecular genetic profiling of pediatric high-grade gliomas reveals key differences with the adult disease. J. Clin. Oncol., 2010. 28: 3061–3068.

[iv] Monje et al., Hedgehog-responsive candidate cell of origin for diffuse intrinsic pontine glioma. Proceedings of the National Academy of Sciences, 2011. 108(11): 4453-4458.

[v] Wu et al., Somatic histone H3 alterations in pediatric diffuse intrinsic pontine gliomas and non-brainstem glioblastomas. Nature Genetics, 2012. 44: 251–253.

[vi] Schwartzentruber et al., Driver mutations in histone H3.3 and chromatin remodelling genes in paediatric glioblastoma. Nature, 2012. 482: 226–231.

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