Bold opening: A potential breakthrough in brain cancer care is finally on the horizon, and the world is watching closely to see if this can change outcomes for glioblastoma patients. But here’s where it gets controversial: this is a first-in-US trial testing immunotherapy in newly diagnosed cases, a bold step in a field that has seen little progress for years.
A world-first clinical trial in the United States has begun to test whether immunotherapy can revolutionise treatment for glioblastoma, the same aggressive brain cancer faced by renowned pathologist and researcher Richard Scolyer. Led by Australian medical oncologist Professor Mustafa Khasraw of Duke University in North Carolina, the trial aims to determine if immunotherapy drugs can meaningfully improve survival for patients with this deadly disease.
“I’m incredibly excited that this trial has commenced,” Scolyer said. “It takes a long time to get trials approved, but being able to recruit patients in the United States—and hopefully here soon—means we can test whether these drugs make a real difference.”
Duke will collaborate with four other cancer centers in Los Angeles, San Francisco, Houston, and New York, as part of a two-stage study design.
In the first stage, researchers will assess safety in a small group of newly diagnosed patients. If that phase goes well, the second stage will enroll a larger cohort. Participants will be randomly assigned to receive one or two immunotherapy drugs (Nivolumab and Relatlimab) before surgery, followed by a combination with standard treatments like radiotherapy and chemotherapy after surgery.
Scolyer was diagnosed in mid-2023 with IDH-wild-type glioblastoma, a highly aggressive form of brain cancer. Although the prognosis was grim, colleague and Melanoma Institute Australia oncologist Professor Georgina Long proposed leveraging immunotherapy—an approach she had used successfully in advanced melanoma—as a potential strategy for glioblastoma.
The plan was risky, given that conventional brain cancer treatments had seen little change for nearly two decades. “Even if it shaved a few months off my life, it could yield crucial information quickly,” Scolyer explained. “Being alive and functioning well long after diagnosis gives hope.”
Scolyer, who shared the 2024 Australian of the Year award with Long, faced a setback when his tumor recurred after an operation last March. Yet he has defied expectations by continuing his work and training for a charity bike ride in Tasmania, even after fracturing two ribs in a fall.
Khasraw describes the GIANT trial (Glioblastoma Immunotherapy Advancement with Nivolumab and Relatlimab) as a “team science” effort, uniting clinicians and scientists from multiple institutions worldwide to bring innovative therapies to patients.
So far, three patients have been enrolled, with a fourth at early screening. While Scolyer’s ongoing survival is encouraging, Khasraw cautions that it’s too soon to draw conclusions about the treatment’s effectiveness from one patient’s experience. “I’m a scientist,” he noted. “I’m driven by data, and meaningful evidence requires solid trial results.”
Nevertheless, he views the trial as a positive step for glioblastoma care, describing it as a platform to generate convincing data that could improve outcomes at the population level. The goal is not a single miracle patient but improved results for many patients.
Researchers expect to have more definitive information within about two years regarding whether the immunotherapy approach can be transformative for glioblastoma.
A peer-reviewed Nature Medicine paper published last year by Georgina Long and colleagues helped lay the groundwork for this trial, which Australia will run through the Brain Cancer Centre. The Australian leadership team is headed by Associate Professor Jim Whittle of Melbourne’s Peter MacCallum Cancer Centre, co-head of research strategy at the Brain Cancer Centre.
The Australian trial will enroll only newly diagnosed glioblastoma patients, with the rationale that immunotherapy may be more effective before radiotherapy or chemotherapy have altered the tumor environment.
The GIANT trial embodies a broader push toward immunotherapy in brain cancer, reflecting a shift from conventional approaches toward strategies that mobilize the body’s own immune defenses. As researchers collaborate across borders, clinicians and patients alike hope this could mark a turning point in a disease that has long defied durable cures.
Question for readers: Do you think early immunotherapy before standard treatments could become the new standard of care for glioblastoma, or should these approaches remain experimental until more definitive data emerges? Share your thoughts in the comments.
