Breakthrough! 'One and Done' Gene Therapy Wipes Out Brain Tumours

The fight against aggressive brain tumours has just received a monumental boost. Scientists have developed a groundbreaking gene therapy that could eradicate glioblastoma, the most lethal form of brain cancer, with a single dose. Early trials in mice saw 83 per cent of tumours completely eliminated, offering a glimmer of hope for thousands.

What's this 'Trojan horse' treatment?

The innovative gene therapy, developed by Professor Steve Pollard's team at the University of Edinburgh and spun out into biotech company Trogenix, uses a clever "Trojan horse" method. A harmless virus delivers a specially engineered piece of DNA directly into the tumour.

This DNA carries three vital instructions designed to destroy the cancer cells from within.

How does it work its magic?

First, a "synthetic super enhancer" acts like a sensor, ensuring the treatment is only switched on inside glioblastoma cells, targeting the right spot. Next, a protein converts a separate, inactive drug – taken as a tablet – into a toxic one, killing the cancer cell.

Finally, the DNA triggers a powerful immune signal, waking up the body's own defences to attack the tumour.

Long-term protection against recurrence

This creates a powerful two-pronged attack: the toxic drug eliminates local cancer cells, while the boosted immune system learns to fight back. Mice in the trial saw dramatic tumour shrinkage within two weeks, with complete clearance in 83 per cent of cases over three weeks.

Crucially, the process trains the immune system to detect and attack the cancer if it ever tries to return, providing long-term protection. Researchers found no tumour regrowth, even when mice were "re-challenged" with the cancer.

Professor Steve Pollard, Co-Founder and Chief Scientific Officer at Trogenix, called it a monumental achievement. "This pre-clinical work in an aggressive brain cancer model that closely mimics human glioblastoma has achieved what we thought impossible – complete tumour elimination and long-lasting protection against cancer recurrence without off-target toxicity using a single dose of a single agent. We are committed to moving these findings as quickly and safely as possible to patients and are optimistic that this can provide a new approach to tackling solid tumours. We look forward to starting our Phase I/II ADePT trial for glioblastoma this year."

Hope for human trials

This precision targeting was confirmed in fresh patient glioblastoma tissue samples, where the therapy only activated in tumour cells, leaving healthy ones untouched. This could significantly reduce side-effects.

The next exciting step will be early-stage clinical trials in humans, focusing initially on safety. These are due to begin in Spring 2026.

Around 3,200 people are diagnosed with glioblastoma every year in the UK. Just 160 will survive for five years or more. That number is unacceptable, and we urgently need better treatment options.

Dr Iain Foulkes, Chief Executive of Cancer Research Horizons, added: "This work lays the foundation for Trogenix's next steps into early-stage clinical trials, steps that will hopefully take us closer to a world where fewer people lose their lives to brain cancer."

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OFFICIAL SOURCE VERIFICATION: This report is based on official data from University Newsroom. Document: [Read the official report from University Newsroom](https://www.ed.ac.uk/news/gene-therapy-safely-eradicates-brain-tumours-in-mice) Source Link: https://www.ed.ac.uk/news/gene-therapy-safely-eradicates-brain-tumours-in-mice