Novel approach could lead to the treatment of devastating brain tumors

A glimmer of hope for finding a solution to Hox gene dysregulation.


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Glioblastoma multiforme (GBM) is the most common devastating brain tumor in adults. It mainly arises from the glial cells in the brain. People who suffer from Glioblastoma Multiforme have a five percent survival rate over five years. This figure has not been improved for decades.

A new study by the University of Surrey has shown that a short chain of amino acids (the HTL-001 peptide) effectively targets and inhibits the function of a family of genes responsible for the growth of GBM – Hox genes.

Hox genes play a vital role in the healthy growth of brain tissue. These genes are ordinarily silenced at birth after vigorous activity in the growing embryo. Inappropriate activation of the genes leads to the progression of cancer. Hox gene dysregulation has long been recognized in GBM.

Scientists first tested the peptide to determine its safety. The peptide passed the safety tests and is suitable for patient trials. These trials are now being considered in GBM and other cancers.

This seven-year research project suggests that the peptide could be a new approach to treating one of the most common and devasting forms of brain cancer in adults – Glioblastoma Multiforme (GBM).

Professor Susan Short, a co-author of the study from the University of Leeds, said: “We desperately need new treatment avenues for these aggressive brain tumors. Targeting developmental genes like the HOX genes that are abnormally switched on in the tumor cells could be a novel and effective way to stop glioblastomas growing and becoming life-threatening.”

James Culverwell, CEO of HOX Therapeutics, said“HOX Therapeutics is excited to be associated with this project, and we hope that with our continuing support, this research will eventually lead to novel and effective treatments for both brain and other cancers where HOX gene over-expression is a clear therapeutic target.” 

Journal Reference:

  1. Arunachalam, E., Rogers, W., Simpson, G.R. et al. HOX and PBX gene dysregulation as a therapeutic target in glioblastoma multiforme. BMC Cancer 22, 400 (2022). DOI: 10.1186/s12885-022-09466-8