Brain cancer drug candidate fast-tracked in clinical trials

Worldwide, an estimated 300,000 people are diagnosed with a primary brain tumour each year, with very little hope of an effective treatment. The good news is that an experimental drug for advanced solid tumours, including aggressive glioblastoma, has now passed the first phase of clinical trials with flying colours, raising hopes for an effective new treatment.

The first phase, which started in June 2021 and was conducted by Adelaide biotech company Aucentra Therapeutics, involved patients with glioblastoma as well as cervical, colon, gastrointestinal, pancreatic and uterine cancers, ensuring that the drug, known as Auceliciclib, was safe at different dosages. In the second stage, the drug’s effectiveness against solid tumours will be tested.

“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,” said Professor Shudong Wang from the University of South Australia (UniSA), which developed the drug. The second phase, in combination with the chemotherapy drug Temozolomide, will be focused on glioblastoma patients, who have a life expectancy 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,” Wang said.

“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 preclinical models that it can cross the blood–brain barrier, which makes it an ideal drug candidate for brain cancer. It also has two key advantages over other drugs in development: it is said to be more target-specific, reaching cancer cells in the brain more effectively, and 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. But Wang said progress has been limited by funding, with the trials costing more than $15 million.

“Aucentra Therapeutics has played a critical role in raising the capital needed to undertake the clinical trials, but we need a lot more support,” she said.

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.

Wang and Aucentra Therapeutics are looking to recruit up to 50 glioblastoma patients for the second phase. For more information on eligibility for the clinical trials, visit https://www.aucentra.com/.

Image credit: iStock.com/wildpixel

Please follow us and share on Twitter and Facebook. You can also subscribe for FREE to our weekly newsletters and bimonthly magazine.