Pill that ‘melts’ leukaemia approved for use in Australia after clinical trial
A PILL that “melts” leukaemia cells has been approved for use in Australia after clinical trials of the daily treatment left one in five end-stage patients cancer-free.
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A PILL that “melts” leukaemia cells has been approved for use in Australia after clinical trials left one in five end-stage patients cancer-free.
Venetoclax, developed and tested in Melbourne, was fast-tracked for use outside clinical trials in the US and Europe last year, after successful first-in-human use in a type of chronic lymphocytic leukaemia.
About 70 CLL patients got the drug in a clinical trial in Melbourne. A positive response in controlling the cancer was seen in eight out of 10.
Now venetoclax has been approved by the Australian Therapeutic Goods Administration for those with the type of CLL hardest to treat.
Its development is based on the Walter and Eliza Hall Institute of Medical Research’s discovery in 1988 that the BCL-2 protein was vital for a cancer cell’s survival.
Institute director Professor Doug Hilton said the drug, developed with US pharmaceutical companies AbbVie and Genentech, worked like a “lethal arrow” to BCL-2’s heart.
Institute clinician and scientist Dr Mary Ann Anderson, who was involved in clinical trials at Royal Melbourne Hospital, said the drug appeared a “smart, more targeted approach” to killing cancer cells without side-effects.
“I’ve looked after a number of patients and in each of those cases, after taking the drug, lymph nodes shrunk dramatically. They felt better in a matter of weeks, and many managed not to need blood transfusions,” she said.
Because the drug is so new — it was given for the first time in 2011 — it is not yet known whether it is truly a long-term cure, or if patients can safely stop taking it at some point.
Dr Anderson said trials to discover this were under way. Its earlier use, and its use in combination with other anti-cancer drugs, and in other cancers, is also being tested.
John Higham, 65, began taking venetoclax as part of a clinical trial two years ago, after two unsuccessful courses of chemotherapy.
“When they started me on it, after I took one 100mg tablet my blood levels immediately returned to normal,” he said.
“I appear to have been cured just after taking one tablet,” Mr Higham said.
“I think it’s a miracle.
“Without this, I’d most certainly be dead.”
AbbVie’s submission to the Pharmaceutical Benefits Advisory Committee for a subsidy for the drug is to be considered at a meeting in March.
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