Concerns raised about expansion of CAR T-cell therapy to common cancers

The potential expansion of CAR T-cell therapy to more common cancers will lead to a ramping up of patient need that Australia is currently not prepared for, an expert has warned.

Speaking during the HSANZ symposium on T-cell therapeutics, Professor Michael Dickinson from the Peter MacCallum Cancer Centre and the Royal Melbourne Hospital said that there were currently two products available globally under commercial arrangements – tisagenlecleucel (Kymriah) and axicabtagene ciloleucel (Yescarta).

“There are over 11 jurisdictions around the world where these products are now available for patients but they are famously expensive and also famously complicated to implement at a site level,” Prof. Dickinson told Blood 2019 delegates in Perth.

In December 2018 Kymriah was approved by the TGA for the treatment of paediatric and young adult patients up to 25 years with B-cell precursor acute lymphoblastic leukaemia (ALL) that is refractory, in relapse post-transplant or in second or later relapse.

It was also indicated for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma after two or more lines of systemic therapy.

And in April 2019 the Health Minister announced that Kymriah would be available to Australian children and young adults with acute lymphoblastic leukaemia that has relapsed or is refractory to other treatments following a positive recommendation from MSAC.

Prof. Dickinson told delegates that the PeterMacCallum Cancer Centre and the Royal Children’s Hospital in Melbourne were currently the only centres in Australia to offer CAR T-cell treatment but this was soon to be expanded to include the Royal Prince Alfred, the Sydney Children’s Hospital and Westmead Hospital.

New indications equals increased demand

Both Kymriah and Yescarta were currently under consideration by MSAC for the third-line treatment of DLBCL, which, if approved would likely lead to a “massive ramping up” of patient need, Prof. Dickinson said.

“We may also see indications beyond large cell lymphoma including multiple myeloma, which then puts us into hundreds and hundreds of patients who may need access to CAR T-cell therapy,” he warned.

Europe and the US had made moves to address a growing demand by setting up new treatment centres that could deliver the technology but Australia was lagging behind, with sites delivering low volumes of therapy to a small number of patients.

“We hope there will be a massive expansion of CAR T-cell in Australia and globally but this will be very demanding for our healthcare system,” he said.

Key issues in the roll out of CAR T-cell therapt in Australia

According to Prof. Dickinson, the key issues in the role out of CAR T-cell therapy in Australia were many and varied. For patients, access to therapy was urgent but there were questions around how to select and triage patients.

There was also the issue of who should deliver the therapy, for example, should all centres be able to deliver therapy or should treatment be managed by centres of excellence?

One of the key issues for Australia, said Prof. Dickinson, was how to ensure equity of access, particularly given the long distances some patients had to travel.

But ensuring equity of access required equity of knowledge, yet Prof. Dickinson told delegates he had noticed an’ inequity of knowledge’ among doctors treating patients with DLBCL and ALL.

“[They] just didn’t know for months after the ALL indication that CAR T-cell therapy was available, just didn’t know that medical treatment overseas programme was available for patients who were fit enough to travel,” he said.

Other key issues that needed addressing included the need for clinician training, outcomes monitoring and the scalability and readiness for new indications that were likely to come on board.

Sending patients overseas

Prof. Dickinson said the Australia government had taken quite an unusual approach by allowing patients to access CAR T-cell therapy overseas through the medical treatment overseas programme. Over the past 8 months, 34 patients have been sent to centres in the US to receive CAR T-cell therapy at a cost of between $800 to $1 million per patient.

“Outcomes for patients are not more than a 40% durable complete remission, which means that most patients are subsequently relapsing and some patients have unfortunately died overseas or had difficulties being repatriated,” Prof. Dickinson told delegates.

“It’s absolutely fabulous that the government is providing this option… but distance itself creates enormous risks for these patients with very aggressive malignancies… it highlights that there is an enormous need to bring this therapy locally,” he added.

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