Unprecedented success in trialling new adult leukaemia therapy

A new, potentially life-saving drug has raised new hope for patients in advanced stages of chronic lymphocytic leukaemia – one of the most common types of adult leukaemia in Australia.

In many cases this cancer becomes resistant to traditional treatment methods such as chemotherapy. This is because of its high levels of a “pro-survival” protein called BCL-2 that render cancer cells, according to Walter and Eliza Hall Institute haematologist Prof. Andrew Roberts “basically indestructible”.

This new drug, currently in phase one clinical trials, targets this BCL-2 protein and breaks down the leukaemia cancer cells with-in the patient’s body creating a positive outlook for many patients battling the advanced stages of this disease.

Results from the trial reveal the cancer has become completely undetectable in almost a quarter of patients and in 61 per cent of cases the patient has gone into partial remission.

Peter MacCallum Cancer Centre chair of haematology, Professor John Seymour, said while trials were at an early stage, the drug’s success was unprecedented. “Patients on the trial were typically incurable, with an average life expectancy of up to 18 months, so to see complete clearance of cancer in nearly one quarter of these patients after taking this single therapy is incredibly encouraging,” he said.

The protein and its significance in leukaemia – as well as several other cancers including some lymphomas, breast and prostate, was first identified by scientists at the Walter and Eliza Hall Institute (WEHI) in 1988. Professor Andrew Roberts said it had long been a target of scientists trying to develop anti-cancer drugs.

The drug, taken as a pill, also shows promise for the treatment of these other types of cancer, which are also reliant on the BCL-2 protein.

Melbourne patients with advanced stages of chronic lymphocytic leukaemia were the first in the world to receive this new therapy developed in partnership with the WEHI. Trials have been run by the Royal Melbourne Hospital and the Peter MacCallum Cancer Centre. The ACRF is very proud to have provided significant funding to each of these three research centres in Melbourne.

A phase two trial to establish the drug’s safety and effectiveness in a larger group of patients in Australia, the US and Europe is under way and could lead to approval for wider use by regulatory authorities within three years.