Promising results for potential new glioblastoma treatment

Alexa Copson 3 min read

A promising new treatment for glioblastoma has been shown to eliminate the tumour and prevent recurrence in animal studies, achieving what researchers “thought impossible”.

Trogenix has announced the publication of its breakthrough pre-clinical data in Nature, reporting complete tumour eradication in 83% of treated cases and durable protection in a brain cancer model that closely mimics human glioblastoma.

The publication, authored by scientists from the University of Edinburgh, UCL Cancer Institute and The Royal Infirmary of Edinburgh, focuses on Trogenix’s unique Synthetic Super-Enhancers (SSEs) technology. Trogenix, co-founded by Professor Steve Pollard, is a spin out from the University of Edinburgh and this work was funded by Cancer Research UK and the Biotechnology and Biological Sciences Research Council.

The gene therapy uses a harmless viral vector (a specially engineered virus which acts as a vehicle) to deliver genetic instructions specifically to glioblastoma tumours. Once inside the tumour cells, these genetic instructions prompt the cell to produce a toxic substance, and also trigger the immune system to attack glioblastoma cells. You can read more about this approach in our blog.

In the study, single dose of SSEs caused tumours to shrink within one to two weeks. In 83% of the treated cases, the tumour disappeared entirely over the subsequent two to three weeks. The research team also gathered evidence of precision immune activation, meaning it triggered the immune system to specifically eliminate the tumour while SSE activity remained undetected in healthy normal brain cells. Excitingly, over the following 11 months, tumours did not return after the initial treatment and there were no harmful side effects.

Professor Steve Pollard (pictured below), Study Lead at the University of Edinburgh 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.

“Our Synthetic Super-Enhancer technology combines the dual power of cancer cell killing and immune stimulation through a sophisticated ‘Trojan horse’ precision delivery method with the potential for transforming how we address glioblastoma. We can finally hit the tumour hard and early by using controlled gene therapy that has been designed to be highly selective for the most aggressive cancer cells. We are committed to move 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.”

Professor Steve Pollard

Dr Karen Noble, our Director of Research, Policy and Innovation, said: “Patients with brain tumours need innovative new treatment options, so we are delighted to see these promising pre-clinical results. Treatments for glioblastoma haven’t changed in more than 20 years so the potential of a therapy which could selectively destroy glioblastoma cells while also stimulating the immune system to provide long-term protection against tumour recurrence is hugely exciting.

“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 brain tumours.”

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Alexa Copson, Communications Manager
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