The Wistar Institute

“Development of MicroRNA Inhibitors for the Treatment of Glioblastomas”

The research proposed in Dr. Huang’s project is focused on identifying chemotherapeutic drugs for the treatment of glioblastoma that target novel biological markers called microRNAs.  MicroRNAs (miRNAs) are single-stranded noncoding RNAs that represent a novel class of recently discovered gene regulators. It is believed that miRNAs might play a significant role in the development of brain tumors.  

Recently, several miRNAs have been discovered to be deregulated in malignant cells or brain tumor tissue samples. Despite the identification of some components of the miRNA pathway, small organic molecules that modulate the miRNA pathway have not yet been found.

The goal of this investigation is to identify lead compounds that target miRNAs by screening chemical libraries in a cell-based assay.  Compound hits identified in the screen will be optimized through structure-activity analysis.  The efficacy of these compounds will be tested for their ability to induce cell death in glioblastoma cells in culture and in a mouse model.

The most promising compounds resulting from this study will be sent for evaluation of drug potential to the Accelerate Brain Cancer Cure co-sponsored Preclinical Screening Program at the Preston Robert Tisch Brain Tumor Center at Duke University.  Dr Huang and his team expect that the anticipated discoveries will lead to new therapies and significant improvements in the lives of patients diagnosed with glioblastoma.

This project represents collaboration between Dr. Qihong Huang at the Wistar Institute and Dr. Alexander Deiters in the Department of Chemistry at North Carolina State University (NCSU).  Dr. Huang and the two teams expect to deliver highly effective chemotherapeutics and novel biological targets that will greatly advance the molecular understanding of brain tumor development and quickly provide new treatment options.





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