‘Revolutionary’ cancer drug using genetically modified cells approved (Kymriah)

Sydney Morning Herald (By Esther Han)

19 December 2018 — 12:00am


A revolutionary cancer therapy that supercharges a patient’s immune cells to hunt and destroy cancer cells has been approved for use in Australia, ushering in a new era in medicine.

Desperate patients with aggressive blood cancers who have not responded to conventional treatments have been heading overseas to receive a shot of the “custom-made” drug and experiencing “miraculous” results.

Daniel Clarke, left, travelled to the US in September to undergo CAR-T therapy for lymphoma cancer.

In a last-ditch attempt, Daniel Clarke, 45, and his family dropped everything and travelled to Boston so that he could undergo CAR-T therapy for diffuse large B cell lymphoma (DLBCL). One month later, scans showed the cancer was gone.

“I felt like someone had just handed my life back to me,” Mr Clarke, a Qantas engineer from the Sutherland Shire, said.

“It has all happened so quickly. Late September we came here not knowing what to expect, hoping for the best, fearing the worst. Then come November I was in complete remission.”

The Therapeutic Goods Administration (TGA) has approved CAR-T therapy – the first of its kind – for use in paediatric and young adult patients with B-cell precursor acute lymphoblastic leukaemia (ALL) and adult patients with DLBCL who have failed other treatments, including chemotherapy.

CAR-T therapy involves extracting a patient’s own beleaguered immune cells and genetically re-engineering them before infusing them back into the body. The single-shot “living drug” has generated enormous excitement in the medical world.

TGA’s approval was based on two global clinical trials that showed an incredible 62 per cent relapse-free survival rate at 24 months in paediatric ALL patients and a 43 per cent probability of overall survival at 18 months in adult DLBCL patients.

Pharmaceutical giant Novartis, which owns the therapy, commercially known as Kymriah, is increasing lab capabilities to meet growing demand, with Europe, Canada and Switzerland giving their stamp of approval.

“We are focused on ramping up capacity at our US and Switzerland facilities and we recently announced a collaboration agreement for additional manufacturing capacity with Fraunhofer (Germany) and CellforCure (France),” Lauren Carey from Novartis said.

Daniel Clarke, here with his children Sarah, Josh and Katelyn, is now in remission.

“These additional manufacturing facilities are intended to support production on a global scale.”

This type of cell therapy is so new, it does not fit into existing categories such as “drugs” and therefore ineligible for funding via the Pharmaceutical Benefits Scheme. While it is approved and will become available at three treatment centres, patients potentially face $598,000 in costs.

Federal Health Minister Greg Hunt said the government’s goal is to make Australia one of the global treatment centres for CAR-T therapy.

He said he was working closely with state and territory ministers to provide access to patients through the public system as soon as possible.

“I propose that CAR-T therapies such as Kymriah be funded in accordance with the mechanisms provided in the National Health Reform Agreement,” he wrote in a letter to his state counterparts this week.

Michael Dickinson at Peter MacCallum Cancer Centre, who helped lead a clinical trial, said it was “revolutionary” because previously this patient group had no other options.

“When we saw the change, it was a eureka moment. The durability of the treatment and scalability are exciting,” Dr Dickinson said.

Federal Health Minister Greg Hunt. CREDIT: LUKAS COCH

“I still see them and they’re living normal lives – one’s a businessman and another is herding cattle.”

Richard Vines, founder of Rare Cancers Australia, has helped raise $100,000 for three families so they can travel to the US, including the Clarkes.

“Sadly, there were two kids who got so sick so quickly they couldn’t get on a plane and they died,” he said.

“If the treatment had been here, they may have had chance. We’re thrilled it’s now here.”

Leanne Clarke, Daniel’s wife, said the treatment was “nothing short of a miracle”.

“Quite simply, it means my children get to celebrate this Christmas and hopefully many, many more with their dad,” she said. “There could be no greater gift.”

The three treatment centres are Royal Prince Alfred Hospital in Sydney, and Peter MacCallum Cancer Centre and Royal Melbourne Children’s Hospital in Melbourne.