Diffuse intrinsic pontine glioma (DIPG) is the most deadly solid tumor in children, with rapid progression, poor response to therapy, and average survival less than one year from diagnosis. We describe a hypothesis-driven research plan to elucidate the mechanism by which Tenascin-C (TNC), an extracellular matrix (ECM) protein implicated in neural stem cell fate and guidance of migrating neurons during brain development, contributes to pediatric brainstem glioma (DIPG) formation and progression. The role of TNC in pediatric gliomagenesis has not previously been explored. TNC detection in cerebrospinal fluid (CSF) or blood serum of DIPG patients could potentially serve as a novel biomarker of disease in lieu of tumor tissue biopsy for more accurate clinical diagnosis, tumor subtyping for targeted therapy, and measuring response to treatment. Further, TNC-targeted small molecule (nucleic acid) therapeutics could potentially overcome the molecular and anatomic obstacles that currently limit efficacy of standard oncologic treatments in DIPG.
|Effective start/end date||7/1/15 → 12/31/16|
- Cure Starts Now (Award Letter 5/11/15)