An experimental brain cancer drug has been fast-tracked in clinical trials. 

The drug to fight aggressive brain cancer has proved so successful in the first trial on humans that University of South Australia scientists will fast-track the second stage. 

The development provides a glimmer of hope for glioblastoma patients, who face a survival time of only 12 to 18 months after diagnosis.

The first phase, which started in June 2021, involved patients with glioblastoma, as well as cervical, colon, gastrointestinal, pancreatic and uterine cancers, ensuring that Auceliciclib was safe at different dosages.

In the second stage the drug’s effectiveness against solid tumours will be tested.

Researchers are now recruiting up to 50 glioblastoma patients for the second phase, trialling the drug Auceliciclib.

“Phase one usually takes up to two years if there are any safety concerns with a new drug, but we didn’t experience any issues with Auceliciclib, which is very encouraging,” says University of South Australia’s Professor Shudong Wang.

The second phase, in combination with the chemotherapy drug Temozolomide, will be focused on glioblastoma patients, whose life expectancy is very limited, with a survival time of just 12-18 months after diagnosis.

“Despite surgery, chemotherapy and radiotherapy, glioblastoma is an incurable cancer. One reason is due to late diagnosis where the tumour has already spread in a way that makes surgical removal very difficult,” Prof Wang says.

“Also, there are very few existing drugs that can cross the blood-brain barrier. The brain does an excellent job of protecting its most vital organ from toxins and pathogens. The downside is that it keeps out vital medication.”

Auceliciclib has demonstrated in pre-clinical models that it can cross the blood-brain barrier, which makes it an ideal drug candidate for brain cancer.

Worldwide, an estimated 300,000 people were diagnosed with a primary brain tumour in 2020, with very little hope of an effective treatment.

Auceliciclib has two key advantages over other drugs in development. It is more target-specific, reaching cancer cells in the brain more effectively, and is less toxic.

If the drug proves successful in the clinical trial, it will also be an important breakthrough for brain tumours metastasized from other cancers including breast and lung.

Clinical trials are currently being undertaken in Adelaide, Sydney and Melbourne. Contingent on funding, the team hopes to expand the trial sites to all capital cities across Australia.

A separate trial is ongoing for Auceliciclib as a monotherapy for patients with a range of late-stage cancers, including breast, lung, ovarian and colorectal.