By: Michael Coomber (c2022)
In 2017, the FDA approved a new class of onco-immunotherapies for the treatment of B-cell acute lymphoblastic leukemia and B-cell non-Hodgkin lymphomas. Initial trials of these CAR T-cell therapies produced remarkable rates of remission, often in treatment-resistant individuals, despite causing life-threatening adverse effects in a subset of patients. Advancements in CAR T-cell therapy have come a long way since these initial trials. In fact, CAR T-cell therapy is now acknowledged as one of the most promising therapies for hematological cancers.
CAR T-cell therapy is just one of a family of targeted immunotherapies known collectively as Adoptive Cell Transfer (ACT), which rely on the transplantation of immune cells to fight cancer. In the case of CAR T-cell therapy, these transfers are autologous, meaning that a patient’s T-cells are separated from the patient’s own blood, modified, and then transplanted back into the patient.
However, such therapies are expensive. For example, Kymriah (Tisagenlecleucel), a CAR T-cell treatment vetted by Health Canada late in September 2018, had an initial list price of $450, 000 in Canada. This steep cost drove the innovative—and often curative—treatment into a whirlwind of concerns surrounding ethics and affordability in healthcare and pharmaceutical systems. Advocates for the funding of the drug argued that such a promising therapy should not be withheld. Conversely, the high price of the treatment led others to argue that further negotiation was necessary before Kymriah could be equitably funded. Concerning the total cost, a Canadian Agency for Drugs and Technologies in Health (CADTH) expert panel reached estimates of $25.6- and $387.4-million for children and young adults with acute lymphoblastic leukemia (ALL) and adults with lymphoma respectively, over the next three years– concluding that funding the drug is likely only cost effective for the ALL cohort, unless price reductions were made.
Despite this controversy around funding, efforts are now underway to initiate treatment in Ontario. Last month, Novartis Pharmaceuticals, the company which released the treatment, announced the certification of two sites for their administration, located at SickKids Toronto and the Hamilton Health Sciences’ Juravinski Cancer Centre. An additional two sites in Ontario are in the process of certification, which is expected to be completed in 2020. Cancer Care Ontario has since published a set of guidelines for those seeking treatment at these centres. These developments come quickly after the Centre hospitalier universitaire Sainte-Justine and the Maisonneuve-Rosemont Hospitals in Quebec became the first to offer Kymriah (the first CAR T-cell therapy) in Canada. This certification represents another major milestone for the Juravinski Hospital’s stem cell transplant unit, which received a $25 million investment to expand the unit last May.
Results at these sites will likely be noteworthy and may serve to further inform the debate around the place of high-cost life-saving interventions in our healthcare system. Whether or not performance at these centres exceeds or disappoints expectations could very well colour opinions on the next controversial treatment.