National CAR T-cell network heralds a big win for patients

Chimeric antigen receptor (CAR) T-cell therapy is a type of immunotherapy where a patient’s own T-cells are genetically altered to directly target cancer cells.1 By reprogramming autologous T-cells to express CARs, the receptors combine the effector functions of T-cells with the ability of antibodies to recognise tumour surface antigens with high specificity.1 In January 2020, Health Minister The Hon Greg Hunt, announced the expansion of access to tisagenleucleucel (Kymriah®) for relapsed or refractory diffuse large B-cell lymphoma (DBLCL) in addition to refractory,  in relapse post-transplant or second or later relapse for B-cell precursor acute lymphoblastic leukaemia (ALL).2,3 Already, more than 100 patients have been treated in Australia, and we caught up with some of the personnel at the Melbourne and Sydney sites to hear how its all going.

The arrival of CAR T-cell therapy in Australia has demonstrated the importance of equitable access to care

“This process has made me appreciate how equal access to care is a fundamental part of our health system. Before now, our patients who were eligible for CAR T-cell therapy had to travel overseas to access this life-saving treatment. The Medical Treatment Overseas Program (MTOP) was available, but due to the nature of the treatment requiring a carer and frequent ongoing follow-up before and after treatment, in reality the logistics was too much for many patients. Families would be faced with having to relocate overseas for months, which could mean leaving your job, school or other family members behind,” explained Professor Emily Blyth, Deputy Director of the Sydney Cellular Therapies Laboratory at Westmead Hospital, Sydney. “Now in Sydney we’ve had the green light to get set up to deliver CAR T-cell therapy, which has been a massive infrastructure development process, but one met with universal enthusiasm. In Sydney, one site (Royal Prince Alfred Hospital) that is accepting referrals for adult patients. Westmead Cellular Therapies is in the process of being set up as an approved treatment site.

With the COVID-19 pandemic in full swing by the time the Sydney sites were due for auditing, Prof. Blyth explained how the drive to make this treatment accessible to patients as soon as possible meant everything kept ticking along. “As we were ready for our Westmead centre to be audited, the pandemic hit. But testament to the resilience and innovation of all involved in setting up the centres we were able to do it remotely and move a step closer to campus sign-off. It’s been a hugely collaborative process that has been designed for personalisation and scale.”

One case illustrates how the system has been set up to serve the patient

Prof. Blyth said one case epitomised the collaborative process for her and was a moment that “proved this system is going to work well.”

“Before our centres were open in Sydney, we had a patient who was eligible for CAR T-cell therapy and was referred to Melbourne. He was seen by the clinicians via Telehealth and was just about to fly down when the borders closed. Very quickly we were able to communicate with the State teams and deemed treatment of this patient was a priority. Although the Peter MacCallum Cancer Centre was still treating patients, at that stage the border to Queensland remained open to New South Wales, so we organised for the patient to have their cells collected at the newly opened Brisbane site and the necessary exemptions to have the patient treated at the Sydney centre. The management of this single patient shows me how well everyone pulls together and because of the national patient prioritisation, every case is known to everyone – so we can always work out the most efficient way to manage patients.”

Dr Michael Dickinson, Clinical Haematologist at the Peter MacCallum Cancer Centre in Melbourne also recalled how this case was “the best example of how we all collaborate to get treatment closer to home. Every Tuesday we work through the patient cases together, and it’s very exciting to see that what we’ve been asked to do here is working. We’ve set up a national network to deliver patient care that’s both efficient and centred around the patient.”

Dr Dickinson also reflected on how collaborative Novartis has been in helping centres overcome logistical challenges as well. “It’s been a very positive experience, with everyone doing their bit. Now we’ve managed to improve access to innovative therapies – which while it was there in theory, was not accessible for all those who needed it in reality. We’re overcoming the barrier of geography with each centre we set up, and the system is being built in such a collaborative way that we’re all learning from each other – which means we’re progressing fast. Even through the pandemic we’ve continued to treat patients and as Dr Blyth noted, we’ve put our minds together to make the experience as easy on the patient as possible. That’s something we’re incredibly proud of.”

CAR T-cell therapy is a multidisciplinary affair. For nurses involved in patient care, Nicole O’Leary, Haematology Nurse Consultant at the Peter MacCallum Cancer Centre explained how the CAR T-cell experience has been extremely rewarding.

“Patients eligible for CAR T-cell therapy are already extremely complicated from a disease perspective, but you combine that with the logistical and financial pressures they had to face before access in Australia and it’s on a whole other level. Thinking about the ALL population, you see patients from young families where they are working full time and faced with relocating overseas for treatment in an unfamiliar environment. Now the distance to travel is less and the systems and support are familiar, which takes a lot of pressure off families.”

Ms O’Leary explained how beyond the treatment being subsidised, there are other financial programs available for patients who have to relocate. “In some areas we’ve seen organisations and the manufacturer offer financial assistance programs for some patients and their carers who still have to travel and find accommodation close to the treating hospital, which can be game changing for some. I have seen and heard so many amazing stories of patient access to this treatment which is now the standard of care for ALL and DLBCL – which is incredible.”

Teams at the centres of excellence are ready for patients to be referred early

Prof. Blyth, Dr Dickinson and Ms O’Leary appealed for clinicians around the country to be aware of just how ready they were to see patients as early as possible. “There shouldn’t be a worry that you’re referring someone too early. The wonderful thing is that your patient will  be considered by a single clinician at a single site.” Ms O’Leary also noted how in the USA, where CAR T-cell has been established for some time, still struggles with patients presenting at the earliest moment. “We’ve done all of this great work to streamline access to CAR T-cell therapy across the country. We’re ready. We’re waiting.”



  1. Feins S, et al. Am J Hematol 2019;94:S3-S9.
  2. Australian Government. Department of Health. Ministers. The Hon Greg Hunt MP. Expanded access to cutting edge CAR T-cell therapy, 28 January 2020.
  3. Kymriah (tisagenleucleucel) suspension Approved Product Information, 19 February 2020.

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