The Australian medical community is excited about the United States’ approval of a new non-invasive leukaemia cancer treatment that is already being trialled locally.
The CAR-T cell treatment, developed by Novartis Pharmaceuticals and the University of Pennsylvania, is the first form of gene therapy to be introduced to the US market.
It involves the leukaemia patient being hooked up to a machine for a couple of hours while certain immune cells are extracted. Over a period of up to 12 days, the cells are then reprogrammed in a lab to detect and kill the cancer cells.
The breakthrough has been welcomed by Australian medical practitioners and researchers as a promising sign not only for leukaemia, but for the future treatment of many other forms of cancer.
Local researchers expect it could be available in Australia within a few years.
Professor Joe Trapani, the executive director of cancer research at the Peter MacCallum Cancer Centre, was involved with the very first CAR-T clinical trial on patients in Australia in 2010.
He told The New Daily the Peter MacCallum Centre was among the first groups in the world to reprogram cells in this way, with initial lab-based trials dating back to the mid 1990s.
“This treatment is definitely not false hope for cancer patients, this is beyond promising,” Professor Trapani said.
“It means the US Food and Drug Administration is completely convinced by the evidence that this is an effective cancer therapy.
“Before CAR-T cells, we’ve been able to save about 70 per cent of children with leukaemia, but those other 30 per cent of children who don’t respond to chemo have died. This will help prevent deaths.”
Professor Trapani said that at this stage CAR-T would not be used as an initial treatment but as an option for those who don’t respond to existing forms of cancer treatment such as chemotherapy and radiotherapy.
His current research is focused on similar reprogramming of cells but for the detection of lung cancer cells.
“I’m optimistic we will see similar products in a few years’ time to treat other forms of cancer,” the professor said.
Senior paediatric oncologist Dr Geoff McCowage at The Children’s Hospital at Westmead, NSW, said a similar CAR-T cell therapy was being developed in Sydney and hoped to treat patients within the next 12 months.
He said the hospital was also developing a different set of CAR-T cells which aim to one day treat people with brain tumours and bone cancers.
“I expect in the next year or so that the treatment will become commercially available to Australian patients, being treated here,” Dr McCowage said.
“Before that, it may be that Australian patients travel overseas to access the therapy.”
Are there any health risks?
Dr McCowage noted that some deaths occurred during the trials but said the benefits of the treatment by far outweighed any possible dangers.
“In part these (deaths) may have occurred because the patients were dangerously unwell with the leukaemia,” he said.
“In addition, what was found was that the CAR T-cells were so active, so effective, that the leukaemia was killed off very quickly which led to massive changes in the body chemistry.
“So toxicities remain, and they can be major, but they are largely manageable and we consider them acceptable given the serious nature of the disease and the benefits that are being achieved.
“It must be remembered that the current chemotherapies and bone marrow transplants are also very toxic, with major side effects.
“CAR T-cells are not worse than these.”
A spokeswoman for the Therapeutic Goods Administration (TGA) confirmed it had approved use of CAR-T for “clinical trials” in Australia in recent years.
An Australian representative for Novartis Pharmaceuticals, which sponsored the US-approved treatment, would not comment on whether it planned to seek approval from the TGA to make the treatment commercially available in Australia.
If approved, it would mark the first cell-based gene therapy for cancer treatment approved for use in Australia.
Novartis indicated it would charge $US475,000 ($A598,453) for the treatment, custom-made for each patient.