In a study published in JAMA Oncology in November 2022, researchers from America and Europe collaborated to demonstrate “clinically meaningful and statistically significant extension of survival” for patients with glioblastoma. The results are described as “astonishing” by Professor Keyoumars Ashkan, of King’s College Hospital in London. The team aim to “offer fresh hope to patients battling with glioblastoma”.  

A deadly threat 

The Brain Tumour Charity describes glioblastoma as the “most common high grade primary brain tumour in adults”. They are grade 4 brain tumours that are fast growing and likely to spread, and often return despite treatment. Brain Tumour Research suggests that they “almost never spread outside of the brain, spine, or central nervous system to other parts of the body”. They are also “complex”, making them harder to treat.  

“The average survival time is devastatingly short.” 

However, some patients survive longer than a year or up to 5 years, and the reasons for this are unknown. Current treatment options include surgery, radiotherapy, and chemotherapy. Unfortunately, the reported recurrence rate is nearly 100% and the study describes “dismal patient survival”.  

A “fresh hope” 

The study objective was to investigate whether adding the autologous tumour lysate-loaded dendritic cell vaccine (DCVax-L) to standard of care (SOC) results in extended survival among patients. It was a phase III, prospective, externally controlled nonrandomised trial that compared “overall survival” (OS) in patients with newly diagnosed glioblastoma (nGBM) and recurrent glioblastoma (rGBM) with “contemporaneous matched external control patients treated with SOC.” 

Conducted at 94 sites in 4 countries, the trial took place between August 2007 and November 2015, with data analysis in 2020/2021. 331 patients between the ages of 18 and 70 years were enrolled. Following surgery and a collection of tumour tissue for the vaccine manufacturing, patients received the vaccine or a placebo on days 0, 10, and 20, and then in months 2, 4, 8, 12, 18, 24, and 30 as well as “monthly temozolomide as SOC”.  

“Each DCVax-L dose comprised 2.5 million DCs injected intradermally in the upper arm, alternating arms between treatment visits”.  

The data suggest a “20% relative reduction in risk of death at any point in time for patients with nGBM” who are receiving the vaccine, and this benefit “increased over time”. Furthermore, for rGBM a 42% relative reduction is risk was indicated, with the benefit once again continuing over time.  

Delivering the vaccine 

Professor Ashkan describes the vaccine as a “personalised solution, working with the patient’s immune system, which is the most intelligent system known to man”. Although the vaccine is not yet available to patients on the NHS, the US company that manufactures it, Northwest Biotherapeutics, intends to seek approval.  

Dr Karen Noble is Brain Tumour Research’s director of research, policy, and innovation. She states that this is the “first emerging therapy proven effective in treating glioblastoma since temozolomide chemotherapy in 2005”. 

“What the brain tumour community hopes is for it to become affordable, possibly becoming standard of care – so available on the NHS.”  

For Dr Henry Stennett of Cancer Research UK, this is “particularly exciting” because it can “improve outcomes for people who don’t usually respond well to therapy.” Although “stringent regulatory” steps need to be taken, it is a “big step forward”.  

We expect to hear more about other updates in developing a glioblastoma therapeutic vaccine at the World Vaccine and Immunotherapy Congress in San Diego later this month. To join us there, get your tickets now.