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Preliminary results from GBM trial presented at a prestigious international conference


Associate Professor Mustafa Khasraw and Cure Brain Cancer Foundation CEO, Associate Professor Kerrie McDonald present VERTU findings at ASCO 2019.

Researchers from the Australian-based Cooperative Trials Group for Neuro-Oncology (COGNO) have presented the preliminary results from a Phase 2 clinical trial, VERTU, at the American Society for Clinical Oncology (ASCO) Conference in Chicago on Sunday June 2nd.  The VERTU trial investigated Veliparib as a new treatment for glioblastoma (GBM), the most common and deadly form of brain cancer.

VERTU is the first large randomised clinical trial conducted at 17 sites across Australia for newly diagnosed GBM patients. This impressive feat has been made possible through the collaboration between Cure Brain Cancer Foundation and the University of Sydney NHMRC Clinical Trials Centre. This is an investigator-initiated study. AbbVie provided product support. Cure Brain Cancer Foundation and Cancer Council New South Wales co-funded this trial with two grants totalling over $850,000 ($500,000 from Cure Brain Cancer Foundation), which were awarded following competitive application processes.


Veliparib is a drug that has the potential to improve cancer treatment in combination with standard treatment (chemotherapy and radiotherapy). Veliparib targets a protein important in cellular repair, and when this protein is inhibited it causes the cell’s repair mechanisms to fail, leading to cell death. VERTU compared a control group that received standard treatment with an experimental group that received veliparib with radiation, and then with chemotherapy at separate timepoints. This clinical trial aims to determine the efficacy, safety and tolerability of veliparib, as well as changes to the patient’s quality of life.


Preliminary results from 125 patients who accessed VERTU demonstrated this treatment is safe and tolerable, and it did not negatively impact on the patient’s quality of life. There was no difference clinically or through MRI results in the six-month progression-free survival* (6mPFS) between the control and experimental groups. However, further data will be gathered for later time points (9mPFS and overall survival) and the analysis will be reported later in the year.

“Australia has a relatively small population, which is why it is paramount that collaborations like this exist and continue to reach and help more patients,” Associate Professor Mustafa Khasraw, the principal investigator of the study, said. “We would like to thank the patients and their families for their participation in this study.”

*Progression free survival is the length of time during and after the treatment of a disease, such as cancer, that a patient lives with the disease but it does not get worse. 

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