Hundreds of millions of dollars are spent on cancer research in Australia each year.
But there is frustration among blood cancer patients, specialists and advocacy groups as to why a TGA-approved therapy, widely accepted as an effective treatment option, has proved so difficult to access for people, the lives of to whom it is at risk.
A rapid cancer treatment, CAR-T therapy re-engineers the patient’s immune system to fight cancer cells.
The therapy is also expensive, with costs believed to be over $500,000 per patient.
But with CAR-T seen as a “last chance” treatment for some patients, there are fears that lives could be lost as behind-the-scenes discussions over funding deals drag on.
‘Do it fast’
After twice delaying support due to cost concerns, the independent Medical Services Advisory Committee (MSAC) has now recommended public funding for a form of CAR-T therapy to treat multiple myeloma – a type of blood cancer – after four previous lines of treatment.
The MSAC advises the government on whether new medical services and technologies should be funded by the taxpayer.
But it will still be some time before the treatment becomes available, with further complex – and largely opaque – negotiations over terms of use still to be held between pharmaceutical company Janssen, the states and the Commonwealth.
The Leukemia Foundation’s head of blood cancer partnerships, Tim Murphy, said the MSAC decision was a “huge step” for people who “have no other [treatment] option”.
But he warned against longer delays.
“We ask every single party involved in these negotiations to do it quickly,” he said.
“Diligently, thoroughly – but quickly – because people’s lives are at stake.”
A spokesman from Janssen’s parent company, Johnson & Johnson, said it would “continue negotiations” to bring the “highly specialized therapy” to Australia.
Health Minister Mark Butler said he had “no doubt” that CAR-T therapy would be a “game changer for cancer treatment”.
“Bringing any new health technology to Australia doesn’t happen overnight,” he said.
“But our government is aware of the challenge and is working methodically to make it a reality.
“The Commonwealth will need to work closely with states to make this treatment more accessible.”
‘disappointing’ reception
Seven years after being officially diagnosed with multiple myeloma, Glenn Beasley sees CAR-T therapy as cause for optimism.
“There are other treatment options currently available to me, but I think CAR-T … would be the best option at this stage,” said the father of two.
“People are getting deep forgiveness from it, even when they’ve had a number of previous lines of therapy.”
Having undergone several treatments, including two stem cell transplants, the North Sydney resident knows time is of the essence.
He wanted CAR-T to be available as soon as possible.
“I just can’t understand why it has to take so long,” he said.
“I hope now that the negotiations can proceed in a faster way … to make this a reality.”
The potential of the domestic product
The prohibitive cost of CAR-T has led to a number of Australian blood cancer patients moving to the US to receive the treatment.
But from her laboratory in Brisbane, hematologist and clinical scientist Siok Tey has been working to make CAR-T more accessible to Australian cancer patients.
A group leader at the QIMR Berghofer Institute of Medical Research, her team’s clinical trial, using locally produced CAR-T cells, has seen a number of patients with relapsed lymphoma achieve remission.
“The results we’ve seen have been very promising,” Dr Tey said.
Dr Tey said Australia had the medical expertise to research and develop home-grown CAR-T cells that were cheaper and potentially more effective than commercial products currently on the market.
“We can keep costs down and that’s really important,” she said.
“We want all Australians who could benefit from treatment to be able to get it.”
For subsidized CAR-T treatments, payment is split between the Commonwealth and state governments.
Dr Tey said if the government invested a fraction of what it spends on buying commercial products into designing and manufacturing CAR-T cells on Australian shores, the benefits could soon be realized.
“I’m not talking about waiting 10 years,” she said.
“I think within the next two or three years we can have a very strong local program.”
Mr Butler said steps were being taken to expand Australia’s CAR-T capacity, including $80 million for the Peter MacCallum Cancer Institute’s Center of Excellence in Cellular Immunotherapy.
“The center is intended to increase patient access to clinical trials and CAR-T therapies and create new jobs in specialized cell manufacturing,” he said.
‘People are dying’
Brisbane mum Bernadette Savanoff was in “complete shock” when she was diagnosed with myeloma in late 2016.
After a series of different treatments, she continues to live with the disease, saying it was now in a “dormant” state.
In recent years it has kept a close eye on CAR-T therapy, which is subsidized in Australia for some types of leukemia and lymphoma.
After the latest MSAC verdict, Ms. Savanoff was relieved that it was now emerging as a realistic treatment option for herself and other myeloma sufferers.
“To have the recommendation be funded and accessed for the myeloma community is absolutely amazing,” she said.
But watching the MSAC twice deny Janssen’s proposal brought her no joy.
“It’s devastating… I know people who have died in that waiting process,” she said.
“I understand fiscal responsibility, but why are we debating so long and so much when people are dying?”
Editor’s note: Reporter Hayden Smith previously underwent CAR-T treatment for relapsed lymphoma through a clinical trial.