A New Hope: Gene therapy trial for glioblastoma to begin in 2026

7 October 2025 | Blog, News, Support, Treatments

Our Head of Support Services, Lucy Wilkinson, explains the significance to glioblastoma patients of a clinical trial planned for next year.

A ground breaking gene therapy clinical trial for glioblastoma is expected to launch in early 2026, offering new hope in a field where treatment options are urgently needed.

Glioblastoma is one of the most aggressive and challenging brain tumours. Despite decades of research, there have been relatively few major advances in treatment over the past twenty years. Standard care, surgery, radiotherapy and chemotherapy remain largely the same, although there have been some important and encouraging developments.

That’s why scientists and clinicians are actively seeking new therapeutic approaches that can both kill tumour cells more precisely and reduce damage to healthy brain tissue.

Explaining gene therapy

Gene therapy refers to the delivery of genetic instructions into a patient’s cells, enabling them to produce proteins or molecules that help fight disease. These instructions are usually packaged into “vectors” (often engineered viruses that are harmless) which can enter cells and carry the genetic instructions into the cells.

The trial will be led by Biotech company Trogenix and based on research from the University of Edinburgh. It will combine two strategies – killing tumour cells and stimulating the immune system to clear remaining or hidden disease. Because the treatment is designed to kill tumour cells specifically, it is hoped that it will be associated with fewer side effects than conventional treatment.

If successful, the therapy might reduce the need for multiple rounds of treatment.

Gene therapy for glioblastoma is an exciting and hopeful area of research, offering the possibility of new treatment options in the future, which may extend to other cancer types. However, it’s important for patients and families to understand that these early trials face significant challenges and that many people may not be eligible to take part. Delivering the therapy effectively into the brain and throughout the tumour remains difficult and glioblastomas are highly variable, meaning some cells may not respond. These and other challenges must be overcome before such treatments become widely available.

Looking ahead

While there is still a long road ahead, the start of this trial marks an important step forward. Each new study helps researchers learn more about how to overcome the barriers that have limited progress for so long. If successful, it brings the wider glioblastoma community one step closer to more effective, personalised treatments and to a future where a diagnosis of glioblastoma comes with far more hope than fear.

Brain Tumour Support is here to support anyone affected by any type or grade of brain tumour. To find more information on our support services click here.