PA Hospital is the first Queensland hospital to join a world-first international cancer trial, to treat patients with CAR-T cell therapy from the start of their myeloma diagnosis.
Haematologist and site study lead, Dr Emad Abro (second from right) says the trial is the first of its kind to treat patients from early diagnosis, and the first to replace bone marrow transplant, marking a pivotal step forward in advancing blood cancer treatment.
“CAR-T (Chimeric Antigen Receptor) cell therapy has been previously trialled in Australia with patients who have relapsed, but this is the first time in Australia that we have a study targeting patients at the start of their cancer treatment,” he said.
The therapy works by separating and collecting the patients’ healthy T-cells via apheresis, before shipping the cells overseas to a special lab where they are genetically reprogrammed to recognise the signalling molecules on cancer cells and fight them.
After four months of standard cancer treatment, CAR-T patients are reinfused with the reprogrammed CAR-T cells and transitioned to the maintenance treatment phase, where they will be monitored over the next 15 years.
“The goal is to control the cancer with the initial chemotherapy and follow it up with the CAR-T treatment to suppress the sub-microscopic disease, which we sometimes can’t even detect,” Dr Abro explained.
“The first goal is to get the patient in remission and cleared of symptoms of the cancer. Our next goal is to maintain remission and suppress the symptoms for as long as possible.”
Thanks to the targeted, single-dose nature of CAR-T therapy, Dr Abro says the treatment could both lower the risk of long-term side effects experienced with other more toxic treatments, and help improve access to similar treatments for a larger demographic of cancer patients.
“The benefit of the CAR-T therapy is that it is a targeted, one-off treatment that is unlikely to have the same long-term risks as other treatments like bone marrow transplant or chemotherapy, which cause a lot of side effects and can be very toxic. Factors like age and comorbidities can affect a patient’s ability to sustain bone marrow transplant, which has an associated 5 to 10 per cent mortality rate.
“The beauty of CAR-T is that it can be adjusted in future to attack different cancer cells, making it a very targeted approach, unlike chemotherapy or bone marrow transplants which have a lot of effects in the body.”
Michael Johns is the fifth patient recruited for the PAH study, after being diagnosed with Myeloma at just 58 this April.
“I’m still in a bit of shock at my diagnosis because I’ve had a really good run of health in my life, but every week I get more information and it gives me optimism for a long remission and quality of life,” he said.
Michael says the support he receives at his weekly PAH clinics has made a world of difference as he navigates treatment while continuing to work part-time in his role as a payroll manager in mining.
“I’m in awe of the care, the skill, the professionalism and the information provided by everyone here at PAH. My experience has been fantastic, and while no one ever wants to have cancer, PA has provided the limousine of treatment.
“Every piece of the treatment has been explained logically, it’s not been invasive in any way, and that’s helped me stay mentally positive because I’m well informed all the way along. I’m really optimistic about the future, and this trial is feeding that optimism that I’ll have a good, happy and healthy retirement.”
While the long-term outcomes of CAR-T cell therapy are yet to be quantified, Dr Abro says the possibilities are promising.
“This study is very important in that it has a very long follow-up, and that enables us to track how well the treatment works, as well as the long-term safety and wellbeing of the patients,” he explained.
“Myeloma is not yet a curable disease, but we are now developing the tools that bring a lot of optimism. With treatments like the CAR-T and some of the other new medicines coming on board, we may potentially cure some patients.”
Dr Abro says the trial is a strong reminder of the important link between clinical trials and progressing cancer treatment.
“We don’t know if this will be the study to achieve a cure, but it’s a big step in the right direction. With tools like this plus additional studies, we can find combinations of treatments that will actually improve patient outcomes.
“Progressing our patients’ treatment through clinical trials is not just something we aspire to, but something we believe should be the standard of care.”
Background
Myeloma is a rare type of blood cancer that develops from plasma cells in the bone marrow, affecting the bones, kidneys, and blood. The causes of myeloma are unknown. Certain chemicals, high levels of radiation (such as from working in a nuclear power plant) and viruses (such as HIV) have been linked to an increased risk of myeloma.
Myeloma is not considered hereditary, so it is rare for more than one person in a family to be affected. There is no current cure for myeloma. Treatment is the best way to delay disease progression, though outcomes vary from person to person. Life expectancy after diagnosis varies depending on factors such as age, disease progression at time of diagnosis, and treatments.
CAR- (Chimeric Antigen Receptors) T cell therapy is a type of cancer immunotherapy treatment that genetically alters immune cells called T-cells in a lab, enabling them to identify and destroy cancer cells more effectively. The benefits of CAR-T therapy include faster recovery time (less than 1 month versus an average of 3 months for bone marrow transplant); reduced risk of long-term side effects; and the benefit of a one-off treatment.
CAR-T therapy is currently approved for use in Australia for four diseases:
- B-cell acute lymphoblastic leukaemia (ALL)
- Adult diffuse large B-cell lymphoma (DLBCL)
- Mantle cell lymphoma (MCL)
- Multiple Myeloma
The CAR-T trial
PAH is the first Queensland hospital selected for an international CAR-T trial that will treat patients from early diagnosis. CARTITUDE-6 is the first longitudinal study over 15 years to replace bone marrow transplant with CAR-T therapy. The PAH trial launched in early 2024 and has recruited five patients to date. Six to eight eligible patients are being recruited for the PAH study. At PAH, patients undergo a four-week screening process to determine suitability, before being randomly allocated to one of two branches: the CAR-T group or the bone marrow transplant group.