The Pediatric Brain Cancer Fight Wages On

Nov 27, 2018 | Blog, Brain Cancer, Pediatric Cancer, Pediatrics

TrialSite News was recently contacted by a reader in the United States with a request for a summary of pediatric brain tumor clinical research status. It is a topic painfully close to home as one of our founders lost a family member to brain cancer. According to the National Brain Tumor Society (NBTS) brain tumors represent the leading cause of cancer-related death in children 0-19—passing Leukemia. This same organization reports that they represent the second most generic form of cancer-related death for young persons under 22. NBTS reports that there is no consistent standard of care for children—which is an unacceptable situation. However, in this summary we will not review standard of care, but rather look at the clinical research landscape.

About 2,000 children and adolescents 20 and under are diagnosed with malignant brain tumors annually in the United States (U.S.). Apparently, at least in the U.S., the incident rates are growing, but many point to improved diagnosis and reporting as MRIs become widely available. The approximate pediatric survival rate for the central nervous system (CNS) cancer is 60 percent. For children two and under, approximately 70 percent of brain tumors are either medulloblastomas, ependymomas, and low-grade gliomas. Rarer forms of brain tumors—typically seen in infants include teratomas and atypical teratoid rhabdoid tumors. Germ cell tumors including teratomas make up just 3 percent of pediatric primary brain tumors; however, worldwide incidence may vary.

Other sources, such as Dana Farber Boston Children’s report that brain tumors occur in only five of every 100,000 children. The Boston oncology specialist notes brain tumors can be benign (non-cancerous) or malignant (cancerous), but both types can be life threatening. It is noted that children with brain tumors often face a better prognosis than adults with comparable conditions.  The majority of children and adolescents with brain tumors do survive into adulthood. Advances in neurosurgery, radiotherapy and chemotherapy have improved overall survival and cure rates for children fighting brain tumors. However, the treatment-related modality remains high and considerable long-term sequelae are prevalent. Growing advances in pediatric tumor knowledge—tumor biology—represents hope for finding new “molecular targeted” drugs with the potential to augment, supplement or possibly one day replace conventional chemotherapy and radiotherapy. Well designed, rigorous and methodical clinical trials ongoing are needed to assess the safety and efficacy of emerging approaches.

Top Cancer Hospitals in the U.S.

It is important to keep track of the elite cancer hospitals in the U.S. Each year the U.S. News & World Report release its annual list of the top 48 hospitals in the United States.  The organization includes specialization rankings as well.  Approximately 4,800 hospitals are considered. Of the top cancer hospitals in the U.S. each meets the National Cancer Institute’s standard of care and hence receiving the official “Comprehensive Cancer Center’ designation.  In 2018, 60 hospitals have received this status. The top 5 include:

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The Mayo Clinic, the third most prestigious cancer hospital in the United States (and in the world) provides an overview of standard of care for pediatric brain tumors today including the following options:

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Pediatric Brain Tumor Research

In the past children frequently have had to accept medicines and treatments based on what is known to work with adults. However, children are unique—they are not adults. We should not just simply do a “hand me down” approach to treating children with brain tumors. Rather, there needs to be a move to design and plan clinical studies for specific children and adolescent conditions when possible. Pediatric trials can be considered different. Pediatric brain tumor clinical trials are rare—as we will see below. To hammer this point home, see the National Institute of Health’s Organization of Rare Disease in the new “No More Hand-Me-Down Research” video. As we have discussed historically, there are limited numbers of clinical trials open to pediatric brain tumor children. Organizations such as A Kids’ Brain Tumor Care Foundation work tirelessly to changing the paradigm in clinical research when it comes to pediatric brain tumor trials.

Overall, TrialSite News believes the future holds great promise, but we have not reached daylight yet. By all measures, considerable investment has gone into clinical trials for pediatric brain tumors. However, according to a recent comprehensive analysis of pediatric brain tumor clinical research, significant therapeutic improvements lag in progress for most CNS malignancies. A comprehensive review of pediatric brain tumor clinical trials from 2000-2017 was published in Neuro-Oncology, Volume 20 June 2018. What follows is a summary of these findings.

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Out of the 182 total clinical trials, those that included newly diagnosed patients totalled 73 (40 percent); recurrent/refractory/progressive at 83 (45.6 percent) and other equaled 2 (1 percent) disease settings. As reported by authors Hwang et al. the clear majority of the 182 pediatric trials have been phase 1 at 81 (40 percent) or phase 2 88 (44 percent).  A breakdown of trials by tumor/cancer type include:

  • DIPG 23 percent
  • High grade glioma 22 percent
  • Medulloblastoma 19 percent
  • Ependymoma 15 percent
  • Low grade glioma 9 percent
  • Other 12 percent

Hwang et al. reported that as of January 2018, 42 percent of the pediatric brain tumor trials were complete; 56 percent were active and not recruiting or neither at 2 percent.  Apparently out of the entire universe of 182 studied, only four trials were withdrawn.

TrialSite News research team did a brief review of for a summary of any pediatric tumor phase 2 or 3 trials. We provide a summary of findings with a focus on approach, research centers, and any themes with sponsors. Key search parameters included the following:

  • Status (not yet recruiting, recruiting, enrolling by invitation, active)
  • Phase (2 or 3)
  • Condition (pediatric brain tumor)
  • Institutions (commercial, academic & government)

We found 13 phase 2 trials. The completion dates range from 2019 to 2026. Most of the studies are either funded by private foundation sources or government agencies (e.g. NCI); few are commercially sponsored clinical trials. Out of the 13 trials, all involved a major academic medical center and/or research hospital as a sponsor. The National Institutes of Health (NIH) National Cancer Institute (NCI) is co-sponsoring six of the studies and commercial sponsors are involved with two of the studies. The conditions under observation range from Brain Neoplasm and Brain Tumor/Low Grade to Medulloblastoma and general brain tumors.

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Below we summarize the lead clinical researchers addressing brain tumor trials. For our reader that requested this research (as with any other reader) we are happy to broker introductions to these research sites and investigators. It is the least that we can do to help a parent or family member that has a child battling a brain tumor.

Select Leading Pediatric Brain Tumor Investigators

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Brain cancer is now the leading cause of cancer deaths in children. When combined with Leukemia, between 1999 and 2014 more than 50 percent of all cancer deaths among children and adolescents ages 1-19 were attributable to brain cancer. Despite considerable investments in clinical trials for pediatric brain tumors, advanced therapeutic improvements have been slow for most central nervous system malignancies as was thoroughly reported by Eugene Hwang, et. al. They identified and analyzed 182 clinical trials involving pediatric brain tumors between 2000 and 2017.

TrialSite News provides a link to a summary of this important research paper herein. We explore a subset of clinical trials focused on “pediatric brain tumors” and identified 13 phases 2 and 3 studies. There are an additional 26 phase 1 studies with the same criteria which we did not review in the table above  The phase 2 and 3 studies we were able to review focused on a diverse range of treatments from improved detection to assessment of the safety and efficacy of innovative approaches, as well as experimental drugs. TrialSite News was formed to bring transparency and openness to clinical research with a focus on the research site/center. We stand ready to support our readers identify clinical trials landscape in key therapeutic areas such as pediatric brain tumors.


Daniel O’Connor

Mr. O’Connor has spent nearly 20 years providing technology and services to the clinical trials and health technology industry. An entrepreneur, he has been instrumental in building a few different ventures focusing on FDA 21 Part 11 enterprise document management, technology-enabled patient recruitment services, clinical document and safety data exchange, as well as population health and community care coordination for at-risk populations. Mr. O’Connor has built a comprehensive research site data base and intelligent clinical research site news curation engine with TrialSite News. He earned his combined MA and JD from the University of California (Los Angeles and Hastings College of the Law).