Australian regulator approves drug that melts away chronic leukaemia

10 January 2017

Australia's Therapeutic Goods Administration (TGA) has approved a drug that claims to be capable of melting away cancer in some patients with advanced forms of chronic lymphocytic leukaemia.

The drug, Venetoclax, developed in Melbourne, will be marketed as Venclexta and had been approved for some stage-four patients of the disease.

The drug would be available to patients who had failed to respond to standard treatments, or for those who had not been able to undergo other therapies, such as chemotherapy.

The drug works by blocking the action of a protein, BCL-2, that enables survival of cancer cells. Researchers around the world had been looking for a way to stop the protein for over 30 years.

According to one of the developers of the drug, Professor David Huang, the BCL-2 molecule was found to be overactive in many types of cancers, particularly leukaemia.

"It was thought that if we specifically target BCL-2 we might be able to cure cancer," he said, reported.

"So for many years researchers were able to figure out [that] what BCL-2 did when it was overactive [was] it prevented cancer cells from dying.

"We designed the drug to specifically inhibit BCL-2 function and essentially it's designed to trigger the cancer cells to commit cell suicide."

He added that the drug could be taken as a tablet once a day.

Though it had been touted as a wonder drug that melted away cancer cell, patient John Higham said: ''To me, it's a miracle. I mean, I'm agnostic, but to me, it's a miracle. My life's been saved by this drug,'' he told SBS News.

In 2007, the 65-year-old was diagnosed with Chronic Lymphocytic Leukaemia, or CLL, and though two rounds of chemotherapy helped, it did not rid him of the advanced blood cancer.

He was then put on Venetoclax drug trial, a last-resort treatment reserved for patients who were not responding to traditional therapy.

According to Dr Mary Ann Anderson, haematologist at the Royal Melbourne Hospital and Victorian Comprehensive Cancer Centre, the effectiveness of the drug exceeded all expectations.

''The first patient in the world had a football [sized tumour], underneath his arm. Within a week, it was a golf ball, and within a month, we couldn't feel anything,'' reported.

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