Related Experiment Video
Updated: Mar 11, 2026
06:15
Tumor Treating Field Therapy in Combination with Bevacizumab for the Treatment of Recurrent Glioblastoma
Published on: October 27, 2014
27.9K
First-in-Class Small Molecule Inhibitor of Oncogene AVIL in Glioblastoma
Zhongqiu Xie1, Sophia Xie1, Hui Li1,2
1Department of Pathology, School of Medicine, University of Virginia, Charlottesville, Virginia, USA.
DNA and Cell Biology
|March 10, 2026
Summary
Researchers identified the actin-regulatory protein AVIL as a key driver in glioblastoma multiforme (GBM). Targeting AVIL with a novel compound showed significant promise for treating this aggressive brain cancer.
Area of Science:
- Neuro-oncology
- Molecular Biology
- Cancer Therapeutics
Background:
- Glioblastoma multiforme (GBM) is an aggressive primary brain tumor with poor prognosis.
- Current therapies offer limited survival benefits, necessitating novel therapeutic targets.
- The actin-regulatory protein AVIL was investigated for its role in GBM pathogenesis.
Purpose of the Study:
- To investigate the role of AVIL in glioblastoma multiforme (GBM) development and progression.
- To identify and evaluate a novel therapeutic strategy targeting AVIL in GBM.
Main Methods:
- Genomic and transcriptomic analyses of GBM datasets (REMBRANDT, TCGA, CGGA).
- Functional studies including gene silencing, overexpression, and *in vitro*/*in vivo* xenograft models.
- Small molecule screening to identify AVIL-specific inhibitors; preclinical efficacy testing of identified compound.
Main Results:
- Recurrent AVIL amplification and consistent overexpression in GBM across molecular subtypes and glioma stem cells (GSCs).
- AVIL is essential for glioma genesis, driving proliferation, migration, and transformation via actin cytoskeleton remodeling and FOXM1-LIN28B pathway activation.
- A selective AVIL inhibitor (compound A) demonstrated potent GBM cell growth inhibition, antitumor efficacy in preclinical models, and favorable pharmacokinetic properties.
Conclusions:
- AVIL is a critical oncogenic driver in glioblastoma multiforme.
- Targeting AVIL represents a promising therapeutic strategy for GBM.
- A first-in-class AVIL inhibitor shows significant translational potential for precision neuro-oncology.
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