Protein inhibitor offers potential to counter advance of brain tumours
24 Aug 2025
Drugs that prevent the body from clearing parts of brain cells damaged by cancer could help slow tumour progression, according to a new study.
Researchers led by UCL with funding from the Brain Tumour Charity, Cancer Research UK and the Oli Hilsdon Foundation, reported in Nature on the results of using protein-targeting inhibitors on glioblastomas in mice.
The target was SARM1, a protein which destroys and clears damaged nerve cell axons.
Study revealed that blocking the protein’s role in axon degeneration led to the development of less aggressive tumours in the mice, extended lifespan and allowed the brain to function normally for most of their remaining life.
Those mice that did not receive the inhibitor went on to develop more aggressive tumours and became more disabled, found the scientists.
Professor Simona Parrinello of the UCL Cancer Institute and study senior author said the work revealed new means to potentially delay or prevent glioblastoma progressing to more advanced states.
“This is especially important as current therapies do not work well for glioblastoma, which is extremely difficult to treat, in part because it is typically diagnosed when it is already very advanced,” she explained.
While glioblastomas are the commonest type of brain cancer, with some 3,000 diagnosed in the UK annually, there are limited human samples of early stage tumours. Owing to the difficulty obtaining evidence from patients, mice gene-edited to grow the tumours were used for the research.
The research team discovered that the brain’s white matter regions, being rich in axons, were a key area of tumour expansion.
The study’s senior co-author Mr Ciaran Hill, also of UCL Cancer Institute and a consultant neurosurgeon at UCLH, said: “Our findings show that there is an early stage of this disease that we might be able to treat more effectively.
“By interfering with the brain’s response to injury before the disease becomes intractable, we can potentially change how tumours behave, locking them in a more benign state.”
Parrinello added that SARM1 inhibitors currently trialled for other neurodegenerative diseases could be tested to tackle glioblastomas. However, she cautioned, more work was needed to confirm whether inhibitors could be applied to human subjects with glioblastoma.
Pic: Pixabay
