New research funded by Brain Tumour Research is helping to refine a game-changing approach that could transform how glioblastoma is treated in the future. At the Scottish Brain Tumour Research Centre of Excellence, scientists are advancing innovative gene therapy techniques designed to target this aggressive disease with greater precision and reduced side effects.
Glioblastoma – the most common high-grade brain tumour in adults – carries a devastating prognosis, with survival rates that have barely improved in decades. Patients and families continue to face limited treatment options and far too little hope.
As part of the Scottish Centre of Excellence award, we have invested in gene therapy development to help researchers make these therapies more effective against glioblastoma and safer and gentler for patients. Their goal is to create future treatments that deliver powerful, targeted action while minimising harm.
What is gene therapy?
Gene therapy is an innovative treatment that delivers a set of genetic instructions (payloads) directly into cells. Once inside, these instructions help the cell produce proteins that can benefit patients in one of three ways:
- Kill cancer cells by creating toxic substances
- Replace faulty proteins to restore lost functions
- Stimulate the immune system to fight back
To deliver these instructions, scientists often use specially engineered viruses. These viruses are stripped of harmful components and repurposed as delivery vehicles (known as “vectors”) to carry therapeutic genes into the patient’s cells.
The challenge: targeting only cancer cells
One of the biggest hurdles of gene therapy is ensuring these therapies act only on cancer cells, leaving healthy cells unharmed. To do this, researchers use a few clever strategies:
- Cancer-specific delivery vehicles – designed to seek out and insert DNA only into cancerous cells
- DNA “on switches” – designed to make sure the treatment only activates inside cancer cells. They work like a puzzle piece that fits a unique DNA sequence found only in those cells
- Identifying new payloads that can target the cancer cells specifically, without disrupting normal cells
The more precise we make these systems, the safer and more effective gene therapy becomes. Our funding is supporting this third approach, aiming to identify specific vulnerabilities which might be an Achilles heel of the brain cancer.
What difference will this make for patients?
Our Scottish Centre of Excellence is home to worldclass gene therapy expertise. It is the birthplace of Trogenix, the biotech company cofounded by Professor Steve Pollard (Co-Director of our Centre), which secured a £70 million investment in October 2025 to advance its gene therapy work for glioblastoma and other aggressive solid tumours, and progress these treatments into clinical trials. The first clinical trial involving a glioblastoma patient is expected to begin in early 2026.
Now, with funding from Brain Tumour Research, scientists at the Centre will build upon this expertise. They are developing new ways to make such gene therapies even more effective while reducing potential side effects and opening this type of approach to diverse forms of brain cancer.
Optimising gene therapy delivery and precision
PhD student, Isabella Jameson Morris (below), funded by Brain Tumour Research, is identifying sections of DNA that are only open in glioblastoma cells to create highly specific landing sites for gene therapies to act on.
By understanding how DNA is packaged in glioblastoma cells, Isabella is aiming to make gene therapy more targeted, which could reduce side effects and improve treatment effectiveness.
This approach could also help tailor gene therapies for other aggressive tumours, such as paediatric high-grade glioma, and selected low-grade tumours.
With continued investment and collaboration, this research is moving us closer to smarter, safer gene therapies for glioblastoma. It is hoped it will enter clinical trials within five years.
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