It’s not a cancer wonder cure. It doesn’t help every child. However, Dr. Jim Whitlock says a radical new therapy that appears to kill the most common childhood cancer is one of the most remarkable advances he has seen in his 35-plus years of practice.
“We can now save children we couldn’t save before,” said Whitlock, division head of haematology and oncology at Toronto’s Hospital for Sick Children.
The drug, Kymriah, also comes with a colossal but secret price tag. While its “scientific ingenuity is marvellous,” McGill University biological scientist Jonathan Jarry recently wrote, the therapy, at potentially hundreds of thousands of dollars per dose, is also raising “the age-old question: what is the price of a human life?”
This month, Ontario became the second province after Quebec to announce it will cover the cost of Kymriah for eligible patients. The drug is part of a revolutionary therapy known as chimeric antigen receptor-T-cell (CAR-T) therapy that has been approved by Health Canada to treat two life-threatening blood cancers: acute lymphoblastic leukemia (or ALL) in children, and a form of non-Hodgkin’s lymphoma in adults.
We can now save children we couldn’t save before
Cameron Lahti was diagnosed with ALL in 2010, just weeks before turning four. He survived six-and-a-half years of chemotherapy, plus 10 rounds of radiation, relapsing twice. He had blood and skin infections. “His system was just completely breaking down,” his mother Debbie said. Finally, in July 2016, the Newmarket, Ont., boy took part in a clinical trial of CAR-T cell therapy at The Children’s Hospital of Philadelphia. He arrived in Philadelphia in a wheelchair. He was 10 years old. Three months after doing CAR-T cell therapy, Cameron did a cross-country run for his school. Today, three-and-half years out, he remains cancer free. “It’s the longest he’s been cancer free since he was diagnosed,” Debbie said.
Most children with ALL are cured the first time around with standard intensive chemotherapy. “In 2019, in a first-world country like Canada, about 90 per cent of those children will be cured. But 10 per cent won’t, and since ALL is the most common of children’s cancers, even 10 per cent of children who relapse represent a substantial population of patients,” Whitlock said.
Chemotherapy, for its part, is also a kind of controlled poisoning, he said. While effective, it carries short and long-term toxicities: low blood counts requiring transfusions, nausea, vomiting, hair loss, mouth sores. “The list goes on and on,” Whitlock said.
The novel treatment involves extracting T-cells, white blood cells floating in a person’s bloodstream, and genetically modifying those cells. The cells are sent to a special lab, where a bit of DNA is added to the cells that instruct them to better target and kill cancer cells that express a particular protein. The genetically engineered T cells are then infused back into the person, where they multiply madly and can survive in the body for years.
Sick Kids and Montreal’s Sainte-Justine hospital were part of a global trial published last year in the New England Journal of Medicine that reported an overall remission rate of 81 percent with at least three months of follow-up in children and adults given a single infusion of tisagenlecleucel (the generic name for Kymriah) who otherwise had no chance for cure. “We want to believe, although I think it’s probably too early to say for sure, that those children are cured,” Whitlock said.
The approach isn’t benign. The therapy can launch a scary phenomenon known as cytokine release syndrome, “which is basically your immune system freaking out,” Jarry, of McGill’s Office for Science and Society, said in an interview. Symptoms, which include high fever, nausea, rapid heartbeat, low blood pressure and problems breathing, can require admission to an ICU. Hospitals have to keep an expensive antibody on hand to reverse it.
It’s going to be harder to discover new therapies
There can also be neurological complications like confusion, stupor and difficulty speaking, Jarry said.
In addition, the Novartis drug has a Canadian list price of $ 450,000.
In a statement to the Post, Novartis said it has “worked collaboratively with Cancer Care Ontario on behalf of all provinces to reach a mutually agreed-upon confidential negotiated price where the clinical value of Kymriah and health system sustainability were taken into consideration.”
Ontario added that the discounted price to be paid by the provinces “improves the cost effectiveness of Kymriah and helps support cancer system sustainability.”
CAR-T cell therapy is available in Ontario at Sick Kids and Hamilton Health Sciences. Princess Margaret Cancer Centre and the Ottawa Hospital are working towards offering the therapy in 2020.
While it’s a one-time infusion, “it’s not something you give at home and overnight the person has full remission from cancer,” Jarry noted. CAR-T cell therapy requires a hospital stay for several weeks, meaning the final bill will be much larger.
However, the high-ticket price is part of a larger question, Jarry said. “How much are we willing to pay to survive disease, and how much are governments willing to pay?”
Newer drugs are more intricate than older ones, Jarry said, industry has to recoup the research and development costs of failed drugs — “which is most drugs” — and, when it comes to cancer drugs, the low-hanging fruit has already been found. “The ones that were easy to find, we found them. It’s going to be harder to discover new therapies.”
While CAR-T therapy should be celebrated as a “fantastic achievement,” he added, it doesn’t work well for solid tumours like brain, breast or colorectal cancers. And there is a price to be paid in terms of side effects and cost.
“But when your disease is a death sentence,” Jarry said, “less than perfect solutions are not to be dismissed.”
• Email: skirkey@postmedia.com | Twitter: sharon_kirkey