Services Provided by the Childhood Brain Tumor Foundation

  • Annual support for scientific and clinical research
  • Tri-annual newsletter with reporting of funded research, events, educational articles, and personal stories
  • Family Retreat Day and/or conferences
  • Childhood Cancer Ombudsman Program, (CCOP) to help with health insurance analysis and application, employment, and educational difficulties that may arise during or after treatment
  • Related Articles and Stories
  • Fund-raisers, Events
  • Provides additional specific information about brain tumors or treatments upon request
  • Sponsorship for the International Symposium on Pediatric Neuro-Oncology, 1996, 2000, 2002, 2004, 2006, 2008, 2010, 2012, and [2014]
  • Sponsorship for the Society for Neuro-Oncology annually since 2000 for the International Program.


The Childhood Brain Tumor Foundation and Children’s National Medical Center partnered again in the collaboration of the Childhood Cancer Survivorship Conference, Focus on Brain Tumors, on Sunday, May 22, 2011.  The comprehensive conference included excellent topics and expert speakers from the region who shared their knowledge.  

If you are interested in any information that was covered, please contact the foundation to inquire about any of the available copies of Power Point presentations,  This email address is being protected from spambots. You need JavaScript enabled to view it..

We look forward to future collaborations that provide vital information to families.

On November 10, 2012, the Childhood Brain Tumor Foundation held its annual fall Casino Gala since inception.  The event included plenty of activity, live band, silent auction, buffet dinner, open-bar, and our casino games.  The CBTF Board of Directors and Advisory were pleased to recognize three oustanding volunteers this year. 

We are deeply appreciative for the sponsorship support given by Whittles Physical Therapy, Second Chance Band/UVFD/JKS Spirit Fund, Children's National Foundation, and other anonymous sponsors.  It is through the support of businesses, friends, families, medical professionals, and general supporters that we will continue forward with our vital program initiatives.  Together, with all of our supporters, we will make a difference and we will help fund important research initiatives to find a cure.

Thank you to all of our event supporters! Thanks to all who support our organization every day!

Please click on the following links to read about CBTF funded grants (Unless otherwise noted, most studies are funded for up to two years):

Additional summaries are available in our fall newsletter editions.

Clinical and biological relevance of PI3K/AKT/mTOR

pathway activation in pilocytic astrocytoma

Fausto J. Rodriguez M.D., Johns Hopkins School of Medicine

(Report from funding, 2011-2012)

This study has been published in two journals.


Pilocytic astrocytomas (PA) are low grade neoplasms that represent the most frequent gliomas in children and your adults. Most of these tumors have an excellent outcome after gross total resection. However a subset progress clinically despite standard treatments, particularly those that arise in anatomic locations where a complete resection is not possible. Therefore, a basic understanding of the molecular mechanisms responsible for PA biology is needed in order to provide novel curative treatments.


The most frequent, consistent molecular abnormality in PA is activation of the MAPK pathway, usually as a result of activation of BRAF. However, several lines of evidence    suggest that other signaling pathways may also play a role in the biology of these tumors. One such pathway is PI3K/AKT/mTOR which contributes to cellular proliferation and growth, and may be targeted with currently available drugs. In the past year, with the support of CBTF, we have tested numerous components of the mTOR pathway using tissue microarrays of surgically obtained PA tissue (n=114). Our data suggests that mTOR is active in a significant subset of these tumors, based on moderate to strong pS6 immunostaining in up to approximately 60% of tumors tested.


We currently know that mTOR in the cell exists in association with other proteins in two   different complexes: mTORC1 and mTORC2. One of our preliminary observations is that   Raptor (a component of mTORC1) is increased to a greater extent than Rictor (a component of mTORC2) in sporadic PA, while the converse was noticed in diffuse and high grade gliomas. In follow-up studies, we are analyzing the prevalence of these alterations by clinicopathologic subgroups, including tumors arising in different parts of the brain.  Additionally we are studying the mechanisms responsible for mTOR activation in pediatric low grade gliomas, and the effect of pharmacologic inhibition of the pathway on tumor growth in model systems. 


We are grateful to the CBTF and Tennis for Tumors for their kind support for these studies.                                             CBTF thanks Dr. Rodriguez for his very informative progress report.



Clinical and biological relevance of PI3K/AKT/mTOR pathway activation

 in pilocytic astrocytomas

 (new 2011-2012, one-year)

PI:  Fausto J. Rodriguez M.D., Johns Hopkins Medical Institute


Patients with pilocytic astrocytoma (PA) generally have favorable outcomes following surgical intervention, although a subset may cause significant morbidity or mortality, despite lack of atypical histologic features. Unlike diffuse gliomas, increased proliferation, invasive growth pattern, and/or necrosis do not always predict aggressive behavior in PA, although we have previously shown that frank anaplastic histologic change in the form of brisk mitotic activity with or without necrosis may portend a worse survival. Insight into the molecular alterations and pathways underlying aggressive behavior in pediatric low grade gliomas is needed, to decrease the morbidity and mortality associated with these tumors and conventional therapies. The importance of the PI3K/AKT/mTOR signaling axis has been highlighted in diffuse and high grade gliomas, and there is increasing interest in pharmacological targeting of this pathway in the pediatric setting, given recent successes reported with some low grade gliomas such as subependymal giant cell astrocytoma.    However, little is known about the role of AKT/mTOR signaling in PA, the most common primary glioma in children. Some studies have highlighted a role for mTOR activation in NF1-associated PA, and we have also reported preliminary data suggesting that it mediates phenotypic variations in NF1-associated low grade gliomas with unusual morphologies.  In addition, we have recently documented an association with phospho-AKT (ser473) and specific aggressive PA subtypes, but its clinical significance, in particular relationship to outcome, remains to be tested in a formal manner to justify specific therapeutic approaches. The goal of this proposal is to examine the role of PI3K/AKT/mTOR signaling in PA to determine if therapies targeting this pathway should be tested in children.

We are grateful to the CBTF and Tennis for Tumors for their kind

support of this grant.

The short and long-term effects of biological agents on oligodendrocyte lineage and progenitor cells in the developing brain

PI:  Joseph Scafidi, DO – Children’s Research Institute, Washington, DC

(update on progress)  Grant duration, Aug. 2011--Sept. 2013


The brain of children is subject to rapid growth and development. White matter is the part of the brain that is vital for communication and processing of information. White matter comprises nearly half of the human brain, and its growth and    development occurs specifically after birth and continues throughout childhood. During this critical period of white matter maturation, it is vulnerable to drugs or other agents that affect its proliferation and growth and can result in injury. Injuries to the white matter lead to life-long cognitive and sensori-motor delays.


Children diagnosed with brain tumors are treated with combination therapies, which often include chemotherapy, radiation and specific targeted biological therapeutic agents, which are aimed at arresting tumor growth. The same pathways involved in uncontrolled tumor growth are part of complex mechanisms imperative for normal white matter growth and maturation. Using a multidisciplinary approach, the Scafidi laboratory is studying how these specific targeted biological agents affect  normal white matter development at different developmental ages. Specifically, their studies are focused on the cellular, biochemical and  behavioral effects these agents have on the developing brain and whether its effects are developmentally age specific.   Understanding the effects these agents have on the developing brain will further our knowledge of normal brain   development and the long-term consequences of these drugs.  (Oral presentations were made at two international meeting regarding this research.)


Sincere appreciation to the Childhood Brain Tumor Foundation for its dedication to funding vital research initiatives!      



The Short and Long-term Effects of Biological Agents on Oligodendrocyte Lineage and Progenitor Cells in Developing Brain (new, first-year)

PI:  Joseph Scafidi, DO, Children’s Research Institute


Childhood is a critical time because of rapid growth and development of the brain. During this period, the brain’s white matter, composed of myelin, is the last structure to fully mature. There is growing evidence that a disturbance in white matter development contributes to significant developmental delays. There is a large gap in knowledge in how newer molecularly   targeted therapies used to treat pediatric brain tumors affect brain development, specifically white matter, during this critical period. This study, using a multidisciplinary approach, will assess and characterize the effect newly developed molecularly   targeted agents have on myelin producing cells and determine whether this is dependent on developmental stage of the brain. This study will provide a better understanding of white matter development and establish new methods to assess the effects these therapies have on the developing brain.

Please click on the links below to read grant summaries and abstracts:


  • A Multi-Institutional Phase II Clinical Trial of a Novel Salvage Induction Chemotherapy Regimen-Gemcitibine Paclitaxel and Oxaliplatin (GemPOx) Followed by a Single High does Chemotherapy (HDC) and Autologous Hematopoietic Progenitor Cell Rescue (AuHPCR) for Patients with Recurrent or Progressive Intracranial Germ Cell Tumors, by Jonathan Finlay, M.D. Children's Hospital Los Angeles, CA, and Co-Principal Investigator Neha Patel, M.D., University of Wisconsin-Madison Children's Hospital

  • Hedgehog pathway activation in juvenile pilocytic astrocytoma, by Dr. Michael Cooper, Vanderbilt University, TN

  • 2008/2007

  • ABT-888, an oral poly(ADP-ribose) polymerase (PARP) inhibitor, enhances chemotherapy and radiation against pediatric brain tumors, by Dr. Jack Su, Pediatric Hematology-Oncology, Texas Children's Cancer Center, Baylor College of Medicine

  • A role of a transcription factor, FoXM1, in cancer stem cells in pediatric brain tumors, by Ichiro Nakano, MD, UCLA, Los Angeles

  • Post transcriptional regulation of gene expression in astrocytomas, by Frederico Bolognani, MD, PhD, University of New Mexico

  • Targeting novel angiogenic factors related to AKT kinase activity in ependymoma, by Dr. Gary W. Tye and Dr. Timothy E. Van Meter, Virginia Commonwealth University

  • 2007/2006

  • Determination of TP73 Expression and Function in Medulloblastoma, by John Y.H. Kim, MD, Baylor College of Medicine, Houston, TX
  • "Role of efbB2 in the sensitivity of EGFR signaling to guinzoline based EGFR inhibitors in glioma" (2007) and "Characterizing cell resistance to small molecular inhibitors of EGFR in malignant gliomas" (2006), by Hui-Kuo Shu, MD, Emory University, Atlanta, GA

  • "MicroRNAs targeting in medulloblastoma" (2007) and "MicroRNAs targeting in medulloblastoma" (2006), by Anna Krichevsky, MD, Brigham and Womens, Boston, MA

  • 2002

  • Identification of an alternatively spliced RNA for the Ras suppressor RSU-1 in human gliomas, by Suryaprabha Chunduru, Hiroyuki Kawami, Richard Gullick, William J. Monacci, Gerard Dougherty, and Mary Lou Cutler

    Please click on the following links to read researcher comments:

  • Why Research is Important to Me, by John Y.H. Kim, MD, Baylor College of Medicine, Houston, TX

  • Why I do Brain Tumor Research, by Dr. Jack Su, Pediatric Hematology-Oncology, Texas Children's Cancer Center, Baylor College of Medicine

  • Researching Childhood Brain Tumors, by Gary Tye, MD, Virginia Commonwealth University
  • Nothing is more frightening than to learn that your child has a brain tumor. These serious tumors are unfortunately all too common; in fact, malignant brain tumors are the most frequent type of solid cancer seen in childhood. To make matters more difficult, conventional radiation therapy - a mainstay of treatment for adults - cannot be used freely in young children because of the risk of cognitive problems in later life. However, the relatively new technique of radiosurgery allows many children with brain tumors to be treated with radiation in a safe and effective manner. Here we discuss the role of this new technology in the treatment of childhood brain tumors.

    The Problem of Radiation for Children

    The three mainstays of treatment for adult tumors - surgery, chemotherapy and radiation therapy - are also used to fight tumors in children. Complex combinations of these options are often chosen, with success depending on the type of tumor and the individual details. These regimens require the guidance of pediatric experts - pediatric oncologists, surgeons and radiation oncologists - because childhood tumors behave differently than adult tumors.

    Because radiation controls many tumors in adults, physicians were once hopeful that children could also benefit from this treatment. However, children respond differently to radiation than adults. For children under the age of three years, their brains are in critical stages of development and are simply too fragile to withstand radiation without harm. Within several years, the radiation can lead to severe developmental delay, memory and cognition deficits, and structural changes in brain tissue. By later life, many are devastated.

    Older children, those greater than five years of age, are less prone to these complications, and radiation therapy remains an important part of their treatment. Pediatric oncologists, nevertheless, carefully consider the potential for harmful effects of radiation in all children. And most oncologists usually withhold radiation from younger children, relying instead on surgery and chemotherapy to control the tumor until the child is old enough to withstand radiation therapy. Many feel that therapy is compromised by the omission of radiation, but they are still reluctant to add this treatment until the child is older. Unfortunately, the cost of sparing these children the later ravages of radiation is the denial of a potentially life-saving treatment.

    A Partial Solution: Radiosurgery and the Gamma Knife

    In the 1950's, the Swedish neurosurgeon Lars Leksell had a groundbreaking idea. Why not devise a machine that could focus the radiation onto the tumor alone, sparing the surrounding brain from the offending radiation? Such a device might hold immense benefits for children, since the minimal exposure to the surrounding brain meant that the risk of devastating side effects would be small.

    Leksell's idea brought about the Gamma Knife®, which is actually not a knife at all. Instead, it is a large spherical metal helmet containing 201 holes, which guide the radiation to a single spot at the helmet's center. Since that one spot receives more than 200 times than the dose to the surrounding brain, the result is the desired effect of focusing.

    The Gamma Knife® is used differently than standard machines for radiotherapy. Instead of a fractionated plan consisting of small doses given daily for many weeks to kill tumor cells without harming normal tissue, Gamma Knife® treatments consist of a single, powerful dose given in one sitting designed to annihilate any tumor within the targeted area. Since the Gamma Knife® is so precisely focused, this strategy of radiosurgery has been spectacularly successful in treating a variety of adult brain tumors. However, the Gamma Knife® cannot be used for young children because its use requires attachment of a metal frame to the head, a frame too heavy and too tight for the fragile infant skull. For older children with thicker skulls, Gamma Knife® radiosurgery remains a good option with success rates similar to those seen for adults.

    A New Approach: the CyberKnife®

    The CyberKnife® offers the precision of radiosurgery without the requirement of a head frame. Consisting of a robotically guided source of radiation, the CyberKnife® maintains the required precision by taking x-ray images of the patient throughout treatment to update its position: the robot senses any movement of the patient's head and automatically compensates. In fact, extensive testing has shown that the precision of the CyberKnife® approaches that of the Gamma Knife®. This means that radiosurgery can be delivered with the CyberKnife® without a head frame, so that radiosurgery can be offered to the infants and young children that have been denied this option in the past.

    Limitations of Radiosurgery and Advantages
    of the CyberKnife®

    Although the Gamma Knife is a powerful tool for the treatment of many brain tumors in children, not all brain tumors are suitable for Gamma Knife radiosurgery. For reasons that are unclear, the risk of complications increase if the tumor is greater than an inch in diameter or if the tumor is located in fragile locations of the brain such as the brainstem or thalamus. In such cases, a single, large radiosurgical dose may be too dangerous even if the repeated, small doses of a fractionated plan may be ineffective. In an effort to achieve the best of each, many centers offer hypofractionated plans in which a large dose of radiation is given in 3 to 7 smaller daily doses. These new plans have not been fully studied but have theoretical appeal and preliminary reports have been encouraging. They are not easily accomplished with the Gamma Knife® since the patients are rarely able to wear the head frame for 3 to 7 days. Hypofractionation can easily be obtained with the CyberKnife® since no frame is required to maintain the needed precision. For children with larger tumors or tumors located in sensitive areas, hypofractionation with the CyberKnife® is an option and extends beyond what was previously available.

    When to Use What: Gamma Knife®, CyberKnife® or Other?

    Deciding between these many options is not easy. The experts do not always agree, so you will need the advice of your doctors. Here is one approach.

    Children less than three years of age. Because of the risk of serious cognitive problems in later life, the use of conventional fractionated radiation therapy to treat young children is uncommon.

    Radiosurgery with the CyberKnife®, however, remains an option for these fragile patients because the focused fields of the CyberKnife® minimize radiation exposure to normal brain tissue. Furthermore, a rigid headframe is not required. Young children and even babies with delicate skulls can therefore be offered CyberKnife® radiosurgery.

    Older Children. The options for older children include conventional radiotherapy, Gamma Knife® radiosurgery, and CyberKnife®. Radiotherapy may be given if the tumor is too large for radiosurgery or if the tumor infiltrates into a large volume of brain. Single dose radiosurgery can be given with either the Gamma Knife® or the CyberKnife®, although the CyberKnife® may be chosen for some children to avoid the discomfort of the head frame or the use of general anesthesia. Hypofractionation with the CyberKnife® may be chosen if the tumor is large or has been treated in the past with radiation.

    What About Tumors of the Spine?

    Since the CyberKnife® does not require a headframe, it can deliver radiation to any structure in the body, including the spine. Experience has been promising but early, and most of the patients receiving radiosurgery for tumors of the spine have been adults. However, the technique is possible in infants and children; consult with your doctors and your CyberKnife® center.

    The Baylor Radiosurgery Center

    The Baylor Radiosurgery Center is a facility on the campus of Baylor University Medical Center in Dallas that is dedicated to radiosurgery and houses both a Gamma Knife® and a CyberKnife®. The physicians on the medical staff at the Baylor Radiosurgery Center pioneered the use of CyberKnife® radiosurgery in infants, and work closely with the pediatric oncology community in Dallas. If you wish to obtain more information about the services offered at the Baylor Radiosurgery Center, please call us at 214-820-HOPE.

    Physicians are employees of HealthTexas Provider Network and are neither employees nor agents of Baylor Health Care System, Baylor University Medical Center at Dallas or Baylor Health Care System's subsidiary, community or
    affiliated medical centers.

    This article was written for the Childhood Brain Tumor Foundation, Germantown, Maryland,

    14th ispno 114th ispno 2The Childhood Brain Tumor Foundation was delighted to be a silver sponsor of the International Symposium on Pediatric Neuro-Oncology (ISPNO), held from June 20 through June 23, 2010, in Vienna, Austria. The ISPNO is held every two years alternating in and out of the United States. This important meeting brings together basic scientists, translational researcher, clinicians, and other medical professionals to share new developments, research initiatives, outcomes from clinical trials, progress in research initiatives, and late effects. The Co-chairs for the 14th International Symposium on Pediatric Neuro-Oncology were Irene Slavc, M.D. and Thomas Czech, M.D. They warmly welcomed the attendees and opened the conference during a special session on Functional Systems of the Brain. This article includes selective topics from the conference.

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