Pediatric Brain Tumor Foundation
Working to eliminate the challenges
of childhood brain tumors
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Research Goals

Researchers funded by the PBTF are looking for answers to questions such as:

  • How do the tell-tale mutations in histone proteins contribute to the development of DIPG?
  • What is the role of epigenetics in DIPG and can we discern candidate drug targets on the basis of available genomic data?
  • What are the salient changes in DIPG the result from a specific mutation at a histone-coding allele (H3.3K27M) and how may they be exploited for therapeutic purposes?
  • What are the candidate cells of origin for DIPG?
  • Can we identify specific sets of genetic signatures that reliably sub-classify pediatric gliomas by prognosis and that foretell the best treatment options?
  • Might a subset of pediatric gliomas and CNS-PNETs represent overlapping diseases with a continuum of molecular and histologic features?
  • Can oligodendrocyte progenitor cells initiate gliomagenesis?
  • How does mutated MLL2 affect the growth of medulloblastoma?
  • Can we identify novel prognostic markers that distinguish high- and low-risk patients within the four medulloblastoma groups?
  • Can a more refined classification of MB using genomic approaches better capture intertumor heterogeneity and prognosis?
  • Can humanized mouse models for medulloblastoma be developed form engineered human cerebellar stem or progenitor cells that mis-express MYCN?
  • Can CSF provide more sensitive source for measuring levels of circulating tumor DNA as a biomarker of tumor status?

The primary aims of translational research projects include:

  • Development of next-generation designer recombinant immunotoxins (RITs) that  are less immunogenic.
  • Preclinical studies to enable advancement of a small-molecule inhibitor of Smoothened into clinical testing in humans.
  • Validate the utility of Wee 1 inhibitors as chemosensitizers.
  • Determine whether the neurocognitive deficits induced by radiation are reversed by treatment with the Mn porphyrin, MnTnBuOE-2-PyP+5, a small-molecule mimic of superoxide dismutase.
  • Determine the anti-tumor efficacy of medulloblastoma RNA-loaded, dendritic cell vaccine.
  • Preclinical studies in vivo to determine the impact of the poliovirus-based immunotherapy on tumor cell killing.
  • Evaluate the utility of a radiolabeled somatostatin binding protein for targeted delivery of radiation.
  • Phase I study of adoptive immunotherapy to mediate tumor regression.
  • Demonstrate that treatment with c-Met inhibitor could be an effective treatment strategy in the Shh subgroup of medulloblastoma.