Phase II clinical trial starts evaluating new drug for glioblastoma

A Phase II clinical trial has begun in the US to evaluate a new drug, GDC-0084, that targets the signalling pathway implicated in almost all (90%) of glioblastoma multiforme brain cancer cases.

GDC-0084 is under development by the Australian biotech company Kazia Therapeutics as an alternative treatment option for the two thirds of patients that are resistant to existing standard treatment (temozolomide).

Recruitment for the trial, which has started at the Stephenson Cancer Center at the University of Oklahoma, was announced by the company’s CEO and executive director, Dr James Garner. More US sites will join in recruitment for the trial in the coming weeks.

“The need for new therapies for glioblastoma remains immense - clinical trials of a variety of potential new therapies are ongoing, but we haven’t yet seen a very convincing new treatment option emerge,” said Garner. “One reason is that many of the drugs being tried are really intended for use in other tumour types, and are only tested in glioblastoma via small and speculative clinical trials.

“GDC-0084 was designed specifically as a treatment for brain cancer, so it has been very carefully optimised for this disease area. The work on the drug to date has been of an exceptionally high standard.

“We are excited to now have the trial underway, and look forward to working with the participating clinicians.”

The drug is being developed as an inhibitor of the PI3K signalling pathway, which is implicated in most cases of glioblastoma. It is distinguished from other brain cancer drugs currently in development by its ability to cross the blood-brain barrier.

It has been granted orphan drug designation by the US Food and Drug Administration (FDA) as a potential treatment for a rare disease with high unmet patient need.