Should trials for Glioblastoma be conducted differently?

Glioblastoma (GBM) is an aggressive form of cancer found in the brain. The cause of GBM is still unclear and a cure is yet to be established.

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Most patients with GBM will survive roughly 12 - 15 months following diagnosis, with very few surviving over five years. Even with maximum treatment GBM often recurs, making prognosis poor for most sufferers.

Current treatments

treatments for GBM include surgery, radiation therapy, chemotherapy or targeted therapy. Targeted therapy is the most recent treatment for GBM, involving medicines targeting parts of cancer cells marking them differently from normal cells.

Experimental treatments

GBM is particularly difficult to treat as tumors hide behind the blood-brain barrier, making it hard for drugs to pass. A tumor spreads throughout the brain, blending with healthy tissue and becoming difficult to separate from normal cells. Furthermore, GBM is heterogeneous; meaning it’s made up of various cells which respond to treatments differently.

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Currently, researchers are developing experimental treatments for GBM including immunotherapy and targeting signaling pathways controlling the growth of cancerous cells.

Unfortunately, negative results in trials have caused some drug companies to drop out following a lack of enthusiasm for upcoming research. With under 15% of GBM patients enrolled in adaptive phase 1 studies, a rethink is required for research to continue.


Erik P. Sulman, MD, PhD suggested provider bias, number of participants, costs to patients, exclusion of emerging therapies and technologies in trial design and abandoned trials contribute to the failures of current research, and that developing a new paradigm for studies should be considered.


Firstly, a common concern for potential participants is the delay between surgery and starting radiotherapy. Reassurance that waiting for test results is safe, and in some cases, a better outcome can even be obtained by delaying, may help people feel more confident that it’s safe to participate in studies.

Another suggestion is overlapping studies. Using an adaptive study design in early phases as utilised by, allows studies to make use of new information and technology to improve clinical trials as they progress.

That said, no matter how much adaptation goes into how trials for GBM are conducted, the bottom line is that rapid recruitment of participants is vital for any clinical trial and must be considered a priority.


Published by Sunil Pandey


Jan 16, 2019, 12:11:14 PM

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