Glioblastoma strikes 200,000 people each year worldwide, and has no cure
UVA Comprehensive Cancer Center researchers led by Hui Li have found a small molecule that blocks AVIL, a key cancer-driving gene in glioblastoma (GBM). In a study published in Science Translational Medicine, the molecule successfully shut down AVIL activity in cells and in mice, reducing tumour growth without harmful side effects.
The work is promising because AVIL is abundant in GBM tumours but barely present in healthy brain tissue, suggesting the treatment could be highly targeted. The compound also appears able to cross the blood–brain barrier and could potentially be taken as an oral pill.
While the results are encouraging, the molecule still needs significant optimisation and safety testing before it could enter human clinical trials. Li has also founded AVIL Therapeutics to develop AVIL inhibitors, and the team holds a patent related to the approach.