A single dose of an innovative gene therapy could eliminate hard-to-treat brain tumours and prevent them from coming back, according to a pre-clinical trial in mice, reported in Nature.
The innovative treatment is built on more than a decade of research led by Professor Steve Pollard, spun out into biotech company Trogenix in 2023.
More than 80 per cent of mice in the trial saw a complete elimination of their brain tumour, with no toxicity and no tumour recurrence.
The findings provide compelling evidence for a “one and done” approach to treating human glioblastoma, the most lethal form of brain cancer, experts say.
The innovative treatment is built on more than a decade of research led by Professor Steve Pollard at the University of Edinburgh. The technology was spun out into biotech company Trogenix in 2023, supported by Edinburgh Innovations, with the goal of speeding up development from the lab and into patient trials.
The technology is initially being tested in glioblastoma, but the Trogenix team hopes that the treatment can be used for other cancer types, targeting solid tumours.
Gene therapy
Using a ‘Trojan horse’ approach, the gene therapy – a specially engineered piece of DNA – is delivered to cells hidden inside a harmless virus, which is injected into the tumour.
The DNA contains three important instructions, designed to destroy the tumour.
The first – a synthetic super enhancer (SSE) – acts like a sensor, only switching on inside glioblastoma cells, ensuring the treatment targets the right place.
The second is a protein that converts a separate, inactive drug – taken as a tablet – into a toxic one, killing the glioblastoma cell.
The third triggers a powerful immune signal that wakes up the immune system and tells it to attack the tumour.
Lasting protection
The result is a two-pronged attack. The toxic drug kills cancer cells locally, while the immune signal stimulates the immune system to attack the tumour.
Mice treated with the gene therapy saw their tumours shrink dramatically within the first two weeks, with complete tumour clearance in 83 per cent of treated cases over the next two to three weeks.
The process also teaches the immune system to detect the cancer if it returns, providing long-term protection. Researchers found no tumour regrowth in mice after the initial treatment, and no detectable tumour formation in mice re-challenged with the cancer.
Precision targeting
Using fresh patient glioblastoma tissue samples, scientists were able to demonstrate that the gene therapy was only switched on in tumour cells, leaving nearby healthy cells untouched.
The SSE technology allows the treatment to specifically target cancer cells in a way previous therapies have not managed. This selectively could help to reduce the risk of side-effects in patients, experts say.
The next step will be early-stage human clinical trials, which are due to being in Spring 2026. The focus of these trials will be on the safety of the treatment.
The research, published in the journal Nature, was funded by Cancer Research UK and the Biotechnology and Biological Sciences Research Council (BBSRC).
Professor Steve Pollard of the University’s Institute for Regeneration and Repair, co-founder and Chief Scientific Officer of Trogenix, said:
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.”
Trogenix success
Trogenix emerged from groundbreaking research at the University of Edinburgh’s UK Centre for Mammalian Synthetic Biology and the Institute for Regeneration and Repair, and the Cancer Research UK Scotland Centre. It was co-founded by investor 4BIO Capital, including funds from Old College Capital, the University’s in-house venture investment fund.
In October 2025, the company raised £70 million in a Series A investment round led by IQ Capital, including Cancer Research Horizons’ biggest ever investment.
Dr Andrea Taylor, CEO of Edinburgh Innovations, said:
EI is proud to have helped build the bridge between Steve’s research and the clinic, supporting company formation and the protection of intellectual property that enables the promising technology he has developed to get closer to patients. We await the trial results with hope.”
Dr Iain Foulkes, Chief Executive of Cancer Research Horizons, said:
Around 3,200 people are diagnosed with glioblastoma every year in the UK, of which just 160 will survive for five years or more. That number is unacceptable and we urgently need better treatment options. 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. Cancer Research Horizons remains committed to supporting Trogenix's mission to target aggressive cancers, and we're excited to see if their promising precision science can benefit patients."
