Scientists at Queen Mary University of London and Barts Brain Tumour Centre have launched a pioneering clinical trial that could revolutionize treatment approaches for glioblastoma, the most common and aggressive primary brain tumor in adults.
The trial has already enrolled its first five patients with suspected high-grade tumors, marking a significant step forward in the quest to develop personalized treatments for this devastating disease that affects approximately 3,200 people annually in the UK, with patients typically surviving only 12 to 18 months after diagnosis.
Breakthrough in Understanding Tumor Biology
The trial builds on groundbreaking research published in BMC Biology that revealed crucial differences in chromatin remodeling between glioblastoma stem cells and healthy brain tissue. This process, which controls gene activation through DNA accessibility, appears to create unique patterns in each patient's tumor cells.
Professor Silvia Marino, who led the study, explains: "Brain tumor cells have a different combination of genes that are turned on or off when compared to healthy brain cells – a combination that is unique from patient to patient. This understanding has revealed new potential treatment targets and opened the door to personalized therapeutic approaches."
Novel Treatment Targets Identified
The research team has identified two promising drug targets - Smox and GABBR2 - which are believed to play a crucial role in promoting cancer growth. These discoveries provide new avenues for therapeutic intervention that could be tailored to individual patients based on their specific genetic profiles.
Trial Design and Future Impact
The ambitious trial aims to recruit 200 participants over the next five years. Researchers will analyze patients' healthy cells alongside their cancerous ones, searching for abnormally activated or suppressed genes that could be targeted with existing or new medications.
"While we're not yet at the stage of prescribing drugs based on these predictions, this trial will help validate our approach in the laboratory setting," Professor Marino notes. "We're optimistic that this could lead to personalized treatment options for patients in the near future."
Patient Perspective
Max Vardy, a 24-year-old glioblastoma patient from Surrey, represents the human impact of this research. Diagnosed last June and currently undergoing chemotherapy after completing radiotherapy, Vardy shares his perspective: "When I was told that my glioblastoma was terminal and that there are no curative treatments currently available, I was greatly saddened. This research gives people like me grounds for hope that there may be an effective treatment being developed."
Industry Response
Dan Knowles, chief executive of Brain Tumour Research, emphasizes the significance of this development: "This is a significant milestone for the brain tumor community, specifically patients living with the deadliest form of the disease. With continued investment into research for brain tumors, together we will find a cure."
