A £70 million investment has been announced to speed up the development of a promising new treatment for glioblastoma and other aggressive solid tumours, such as colorectal cancer liver metastases.
The funding has been raised by Trogenix Ltd, a biotech company co-founded by Professor Steve Pollard, Director of our Scottish Brain Tumour Research Centre of Excellence (below), to further its work on gene therapy for solid tumours and progress them into clinical trials.
This pioneering treatment is initially being tested in glioblastoma, with the aim of a clinical trial opening in early 2026. It is hoped that the treatment can then be rolled out in other cancer types.
What is gene therapy?
Gene therapy involves delivering a set of genetic instructions into cells.
Once inside a patient’s cells, these new instructions are used to produce proteins. These new proteins might create a substance that is toxic and kills the cell, replace a malfunctioning protein to restore function that has been lost, and much more.
To deliver the genetic material, scientists often use specially engineered viruses. These viruses are stripped of their harmful components and repurposed as delivery vehicles, or 'vectors', to carry the therapeutic genes (instructions to make proteins) into the patient’s cells.
How can gene therapy be used to treat glioblastoma?
At the University of Edinburgh, and now at Trogenix, scientists have developed a gene therapy that uses a harmless viral vector to not only deliver instructions to make a toxic substance that kills glioblastoma cells but deliver instructions that wake and educate the immune system to clear the remaining disease. This approach is known as viral immunotherapy. They are anticipated to trial the treatment in their first glioblastoma patient at the beginning of 2026.
So, how does the potential treatment work?
A harmless virus, called an adeno-associated virus (AAV), is injected directly into the tumour. It’s been engineered to seek out glioblastoma cells and infiltrate them.
Inside the AAV is a specially designed piece of DNA, which is split into three parts:
- Part one is known as a synthetic super enhancer (SSE) and acts like a sensor. It only switches on inside aggressive glioblastoma cells and, in turn, activates parts two and three. The SSE remains switched off in healthy cells, ensuring treatment targets the right place
- Part two of the DNA produces an enzyme that converts a separate drug, taken by mouth, into a toxic one which kills the infected cell and its neighbours
- Part three makes a powerful immune signal called IL-12 that wakes up the immune system and tells it to attack the tumour
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. Crucially these events train other immune cells to hunt down any remaining cancer cells, including infiltrating or dormant ones.
This approach doesn’t just kill the tumour cells. It teaches the immune system to remember it, so if the cancer tries to come back, the body is ready to fight it off.
Why is this promising news for glioblastoma patients?
Glioblastoma is one of the most difficult cancers to treat. It is incredibly aggressive with a prognosis of just 12-18 months.
This new approach offers hope for a 'one-and-done' treatment; a single therapy that not only kills the tumour but also prevents it from coming back. The treatment is also designed to specifically attack cancer cells, sparing healthy cells, meaning it would reduce the chance of side effects in patients.
This work is still in early stages, and we look forward to seeing the results once clinical trials begin.
What is next?
Having raised the funds, researchers are advancing towards clinical trials, with first patient dosing anticipated in early 2026.
Dr Karen Noble, Director of Research, Policy, and Innovation at Brain Tumour Research said, “Patients with brain tumours need innovative new treatment options, so we are delighted to see investment in a gene therapy clinical trial for glioblastoma. Trogenix – led by researchers from the Brain Tumour Research Centre of Excellence in Scotland – will look to selectively destroy glioblastoma cells while also stimulating the immune system to provide long-term protection against tumour recurrence.
"While dedicated researchers play their role in working towards a cure for brain tumours, it's important that the life science landscape moves with them. That's why we are calling on government for increased investment in brain tumour research and wider access to clinical trials for people living with these life changing diseases."
Who is behind the research?
Trogenix was created in 2023 based on co-founder Professor Steve Pollard’s groundbreaking research at the University of Edinburgh – including its UK Centre for Mammalian Synthetic Biology and Institute for Regeneration and Repair – and the Cancer Research UK Scotland Centre.
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