Drug treatment for rare blood cancer will save more lives

Ponatinib, the medication used to treat a rare and deadly form of cancer, has been made available on the PBS. Picture: Michele Mossop
Ponatinib, the medication used to treat a rare and deadly form of cancer, has been made available on the PBS. Picture: Michele Mossop

A drug aimed at treating an aggressive disease that affects the blood and bone marrow is set to save more lives, with the medication now available for more patients on the Pharmaceutical Benefits Scheme (PBS).

People who suffer from Chronic Myeloid Leukaemia (CML) can access ICLUSIG, also known as ponatinib. As of this month it is now available to all Philadelphia-positive Acute Lymphoblastic Leukaemia (PH+ ALL) patients, who are intolerant or resistant to other therapies.

The rare condition develops when the body produces too many of the white blood cells known as granulocytes. As cells grow, they gradually crowd bone marrow, interfering with normal blood cell production and become ineffective in fighting infections.

Each year in Australia about 330 people are diagnosed with CML. It is commonly diagnosed in adults older than 40 and develops slowly.

It is thought to arise from an acquired mutation in one or more of the genes. Most patients have a poor prognosis.

Ponatinib was initially made available through the PBS from November, 2015. Without subsidised access, patients would otherwise pay up to $66,500 per year for this treatment.

Without this expanded offering under the PBS, patients would otherwise pay about $53,000 per year for this treatment.

Leading Australian leukaemia authority, Professor Timothy Hughes, welcomed the new listing as “a major step forward.”

“These patients really have no prospect of long-term survival with current therapies and this PBS listing presents a really exciting new opportunity,” he said.

“While outcomes for Ph+ ALL patients have improved a lot, we still have a very high incidence of relapse and resistance to [other drugs] imatinib and dasatinib.

“Ponatinib has broad coverage against the resistant forms of leukaemia. It really does add to our capacity to provide more people with a stable, long-term response and, in some cases, the prospect of long-term remission.”

Chief executive of the Leukaemia Foundation, Bill Petch, says access to the latest medicines is a vital step to help improve, extend and ultimately save more lives.

“Our priority is to ensure that all Australians living with a blood cancer including leukaemia, lymphoma and myeloma have access to the best therapies and treatments available,” he said.

Comments