Glioblastoma patients have been left in limbo, with drug development stalled due to a combination of complex biology, a fearsomely aggressive disease and a risk-averse pharma industry. But momentum in the field has been building, writes Henry Scowcroft, with a pioneering new platform trial set to offer more options to UK patients and kick-start progress.
As a young boy, Nader Sanai watched on as his aunt died from a glioblastoma brain tumour. She was in her late twenties. It left his family devastated.
“It’s a terrifying disease,” says Sanai, now a neurosurgeon based in Phoenix, Arizona, where he runs the Ivy Brain Tumour Institute. “It’s not just about losing the ability to do certain things like walk or move, or communicate. You lose the very elements of yourself – your own self-identity. It’s your whole thought process that gets degraded.”
His aunt’s ordeal and death left a lifelong impression on him, motivating him to go to medical school and ultimately specialise in neurosurgery, to try to make a difference with people facing the same fate. But progress in the field has been limited.
“What’s just completely absurd to me is that my aunt’s experience 40 years ago is almost identical to what my patients go through today,” he says. “I feel embarrassed, quite honestly, to acknowledge that because I’m part of this field, and I feel a sense of responsibility to change that.”
There have, Sanai acknowledges, been improvements in surgery and radiotherapy techniques. “These fields have become really quite well developed. But there have been few to any bright spots in terms of new medical therapies.”
This lack of progress in drug development stands in stark contrast to other cancer types. For example, between 2000 and 2023, the US FDA approved 26 new breast cancer treatments. For glioblastoma, just two new drugs – temozolomide and bevacizumab – have been licensed over the same period.
Credit: Cancer Research UK | In depth