Clinicians ready for myeloma ‘cure’

14 September 2022

Leading Peter MacCallum haematologist Professor Simon Harrison is hoping to soon be “very, very busy”. While this may seem unusual for anyone to wish for – especially a leading cancer clinician – Professor Harrison is hoping that the approval and funding of a fourth CAR-T in Australia for a new and much larger patient population will result in an avalanche of work.

“I hope I’m going to be busy and see a lot of patients because this is going to be transformative,” Professor Harrison said. “We’ve gone from ‘put your life in order’ to a position where 100 per cent of the patients will be cured.”

The first patients treated with CAR-T have now been alive and cancer free for a decade, but until now the therapy has only targeted blood cancers with relatively small patient populations.

Yet the pending approval and potential MSAC recommendation of Janssen’s CARVYKTI (cilta-cel) would be a game-changer for Australian patients, says Professor Harrison, who predicts it will quadruple the number of patients eligible for CAR-T and most likely cause a short-term bottleneck, or “throttling”, with patients either waiting or denied access to treatment due to a lack of capacity in sponsor’s global CAR-T manufacturing sites.

But he says the therapy is so successful, there is no doubt it is coming and will be available to more and more patients as the evidence demonstrates its benefit earlier in the treatment cycle.

“In myeloma, CAR-T cells are here, they’re here to stay, and they’re coming up front very soon,” he said.

Professor Harrison was speaking at the Blood22 Conference in Sydney this week, the Director of the Centre of Excellence in Cellular Immunotherapy at Peter MacCallum saying cancer centres around the country were preparing to manage the patient workload resulting from the pending TGA approval of Carvykti, with Peter Mac planning to manage its backlog as outpatients for as long as possible.

He said while the high-cost therapy was likely to become more affordable over time, brought down by ‘economies of scale’ and by reducing the gap between when T-cells are harvested and when they’re returned to the patient, he believes the treatment is already worth the cost given treating a refractory multiple myeloma patient can cost $100,000 a year and still not offer cure.

“This therapy is coming and will almost certainly replace stem cell transplants,” he said referring to a common treatment option for multiple myeloma patients. “… If you’re getting deep, lasting remissions, that’s clearly more affordable than any remission that lasts a short period of time, which is where we are [currently] at.”

If approved and recommended, Carvykti will be the fourth CAR-T to be funded in Australia. However, the third – Gilead’s TECARTUS (brexucabtagene autoleucel) – was approved and recommended by MSAC in July 2021 yet is still not publicly funded.

Professor Harrison says studies showing myeloma patients treated with CAR-T in the second-line were getting longer and more sustained remission than from their original therapy despite being “destined to do badly” pointed to it inevitably being used earlier than its likely fourth-line approval.

He cited one patient who tolerated 13 lines of therapy over three years before receiving CAR-T, after which she went into complete remission and returned to work.

“That’s the kind of transformative, life-changing technologies we’re talking about,” he said. “The question is not can we afford it, but how do we afford it so as to maximise the benefit to our patients.”

While cell therapies are an exciting new option, he says another class of therapies is “hot on its heels” and offering hope particularly for patients who cannot tolerate cell therapies.

Bispecific antibodies are also proving the be game changers in a number of therapeutic areas, yet Janssen’s TECVAYLI (teclistamab), granted marketing approval in Europe last month, is the first bispecific for relapsing and refractory multiple myeloma.

Tecvayli is expected to have been filed with the TGA along the provisional pathway after being given a provisional designation earlier this year, the pending arrival of the bispecific expected to provide another new and important options for multiple myeloma patients.

©MedNews 2022