An innovative gene therapy could achieve complete tumour elimination in brain cancer, data shows. 

Biotechnology company Trogenix has announced the publication of breakthrough preclinical data, reporting complete tumour eradication and durable protection in a state-of-the-art aggressive brain cancer model that closely mimics human glioblastoma (GBM). 

The results, published in Nature, come from Trogenix’s proprietary Synthetic Super-Enhancers (SSEs), engineered genetic constructs acting as selective transcriptional switches. These switches are designed to harness glioblastoma stem cells’ own unique transcriptional machinery.  

Key findings of the study show that, when delivered as a single treatment dose, SSEs led to complete tumour clearance in 83% of treated cases over two to three weeks.  

“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,” said Professor Steve Pollard, Chief Scientific Officer of Trogenix. 

“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 GBM. 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.” 

Glioblastoma survival rates ‘unacceptable’ 

The SSEs, delivered using adeno-associated virus vectors (AAVs), specifically target SOX2 and SOX9-driven gene networks that are activated in patient glioblastoma stem cells.  

Results show the approach meant no further tumour regrowth was reported after initial treatment, nor toxicity over the next 11 months. The SSEs also unlocked precision immune activation to eradicate tumours. 

SSE activity was validated using fresh patient GBM tissue samples, demonstrating selective expression in tumour cells but not in the healthy normal brain cells. This precision targeting of glioblastoma cells while sparing normal tissue minimises off-target toxicities, representing a key validation step before advancing to patient trials. 

The research supports an innovative approach to potentially achieving curative responses with long-term immunological protection against GBM, the most lethal form of brain cancer. 

“Around 3,200 people are diagnosed with glioblastoma every year in the UK, of which just 160 will survive for five years or more,” commented Dr Iain Foulkes, Chief Executive of Cancer Research Horizons. 

“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.”