‘Watershed’ in cancer therapy raises hope

Australian cancer researchers have been buoyed by the approval in the US of a “revolutionary” cancer therapy that works by genetically altering a patients’ cells to fight the disease.


The Food and Drug Administration (FDA) this week approved the drug CTL019 developed by Novartis Pharmaceutical, the first gene therapy to hit the US market.

Peter Orchard, CEO of CanTeen – the national support organisation for young people living with cancer – has hailed the decision a “watershed” for cancer treatment.

“It’s part of a new way of cancer treatments that could genuinely start to impact survival rates in some of those nastier cancers that have really been difficult to deal with,” Mr Orchard told AAP on Friday.

Dr Emily Blyth – a haematologist and bone marrow transplant physician at Westmead Hospital – says an important step has been taken towards making this lifesaving therapy available in Australia.

Although that is still “heartbreakingly” a long way off.

“It’s getting access for Australian patients that now becomes the challenge,” Dr Blyth said.

The treatment involves collecting t-cells, a type of immune cell, from the patient that are then genetically engineered to produce special receptors designed to recognise and kill the cancer cells.

Haematologist and cancer researcher Dr Kenneth Micklethwaite at the Westmead Institute – who is leading a trial of a very similar therapy in Australia with Dr Blyth – says the therapy works by harnessing the power of the immune system to fight the disease.

He said the results of international clinical trials have been “remarkable”.

“They’ve seen patients who have very active leukaemia that’s completely resistant to all other therapies, nothing else has worked, and they’ve given them these cells and their leukaemia has responded very promptly and just melted away,” Dr Micklethwaite said.

The potent personalised treatment, however, is not cheap.

Novartis set the price for its one-time infusion of so-called “CAR-T cells” at $475,000.

Mr Orchard says for an Australian hoping to access this treatment overseas could cost them upwards of $1 million.

For this reason, efforts by CanTeen are now under way to bring this sort of cutting-edge treatment to Australia in the hope of improving survival rates among the 1,000 young people diagnosed with cancer every year in the country.

Last month, CanTeen put out a call for expression of interest to researchers after receiving $5 million in funding from the federal government to conduct clinical trials for sarcoma, leukaemia and brain tumours.

“We would love to see it tried more widely here in Australia, it’s a new frontier of treatments for cancer that we just really need to explore now and use to give people hope where there has been no hope,” Mr Orchard said.

Dr Blyth has called on the nation’s doctors to advocate for their patients “because that’s how we get them access to the world’s best treatments.”

“Who wants to tell a patient ‘look this is a available for someone who lives in the US it’s not available for you’, it’s just too heartbreaking,” said Dr Blyth.