Early clinical trials of the drug Venetoclax on 116 patients with advanced chronic lymphocytic leukaemia in Melbourne and the United States has seen the cancer reduced by at least half in nearly 80 per cent of cases.
In a small number of patients the cancer can no longer be detected at all and researchers are hopeful this drug in combination with other treatments could eventually pave the way for a cure.
The results of the trials conducted over four years at the Royal Melbourne Hospital and Peter MacCallum Cancer Centre, in conjunction with the Walter and Eliza Hall Institute, will today be published in the New England Journal of Medicine.
Rodney Jacobs, a 63-year-old record shop owner from Melbourne, was referred to the trial in 2012 after traditional therapies including chemotherapy proved ineffective in his fight against leukaemia.
“It was looking pretty bleak at that stage,” he said. “The results have been nothing short of stunning … I’ve had little or no side effects and I slowly got myself back to work with my business and slowly but surely I have a relatively normal life.”
Professor Andrew Roberts, a clinical haematologist at The Royal Melbourne Hospital and cancer researcher at the Walter and Eliza Hall Institute, which has a financial interest in the drug, said it was designed to kill a protein called BCL-2, which promotes cancer cell survival.
“I think this drug is going to be very significant and I think these results are significant in themselves because they show in real people exactly what the potential of this can be,” he said.
“Many of the people who achieved a complete remission are still in a complete remission more than a year later.”
The BCL-2 protein was first discovered by researchers at the Walter and Eliza Hall Institute in Melbourne back in the 1980s but they didn’t yet know how to inhibit it, said Professor John Seymour, Chair of the Haematology Service at Peter MacCallum Cancer Centre.
“Here we are a bit under 30 years later in collaboration with WEHI and pharmaceutical companies here and in the US having proved that’s achievable,” said Professor Seymour, who was also a lead researcher on the study.
“This is a completely new class of drugs and there is no other drug or medicine previously available that has had the ability to inhibit this BCL-2 protein,” he said, adding that the drug also has the potential to inhibit other forms of leukaemia and cancer.
“That potential for broader application and the fact that this is a completely new class of drugs takes this beyond simply being a very good outcome for patients,” Professor Seymour said.
The research was funded by drug companies AbbVie? and Genentech?.
Health Minister Jill Hennessy said the results of the trial were a sign of things to come from the $1 billion Victorian Comprehensive Cancer Centre.
“Bringing our best and brightest together under one roof will mean more cutting edge clinical trials, more world-first discoveries and better support and treatment for Victorian cancer patients and their families,” she said.