Dr. Keith Ligon at the Dana Farber Cancer Institute is determined to discover the mutated ‘driver genes’ that sustain the unregulated growth of pediatric diffuse astrocytomas (PDAs). These tumors are a clinically problematic subset of the larger family of pediatric low-grade astrocytoma family. They are especially difficult because they are rare and represent a major clinical problem in pediatric neuro-oncology due to their heterogeneous pathology and unpredictable clinical behavior. The multi-year project ‘Evaluation of MYBL1 fusions oncogene in PDA”, funded by the PLGA Foundation, represents a significant step to help address the unmet need for discovery in this subset of tumors and in the fullness of time, these genes could serve as targets for drug development….read more…

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The Dana Farber Cancer Institute’s Dr. Keith Ligon’s MYBL1 project is designed as a three year initiative. Based on recent genomic studies at the DFCI, several new copy number aberrations appear to represent the first definitions of new subclasses within the PDA group. These aberrations may be useful for targeting therapy or predicting tumor behavior. The MYBL1 transcription factor is one such gene and by understanding this novel oncogene, and its potential for treating diffuse astrocytoma.

The project will specifically accelerate understanding the biology of normal MYBL1 and MYBL1tr in brain development and PDAs while also establishing MYBL1tr as a biomarker. This will happen through two specific aims:

  1. Construction of transgenic mouse models with genetic loss of function or gain of function in the brain. The loss of fuction mouse model will be a conditional knowckout of wt MYBL1
  2. Evaluation of the outcomes of patients found to have MYBL1 using IHC and bioinformatics approaches.

Funding for this project was raised by the PLGA Foundation and the hundreds of generous supporters who believe that more effective treatments and a cure for all PLGA children is close at hand.