Childhood cancer doesn't discriminate. Neither should geography.
Dr. Jim Whitlock, Director of the Garron Family Cancer Centre and Division Head, Haematology/Oncology, gives his perspective on access to paediatric cancer clinical trials.
Dr. Jim Whitlock is the Director of the Garron Family Cancer Centre and Division Head, Haematology/Oncology/Bone Marrow Transplant at SickKids, and Chair of the C17 Council for Children's Cancer and Blood Disorders.
Antonia Palmer and Patrick Sullivan are parents and members of the AC2ORN (Advocacy for Canadian Childhood Oncology Research Network) Executive Committee.
Equal access to health care is a profoundly Canadian principle. Yet boundaries, both geographic and bureaucratic, become barriers to treatment for paediatric cancer in this country. This must change.
Research in paediatric cancer is accelerating, while the evolution of treatment has plateaued. Fifty years ago, it was considered unethical by some to treat kids with leukemia; they were going to die, regardless. Still, the first combination chemotherapy regimens were used to treat children, before being translated to adult treatment. Today, the survival rate for childhood cancers is over 80 per cent. It's a significant achievement, but at a steep price. Conventional treatments – chemo, radiation, and surgery – have late effects that manifest even 30 years post-diagnosis. Sometimes fatal, they include organ failure, cognitive impairment, sterility, and secondary cancers.
It's time to move past the slash of surgery, the burn of radiation, and the poison of chemotherapy. The way forward is threefold: first, shift the paradigm – from thinking about all Phase 1 and 2 clinical trials as 'experimental', to considering them ‘state-of-the-art’ – when they demonstrate successes; second, treat Canadian kids with cancer as citizens of a single country, rather than as residents of 13 distinct provinces and territories, bound by the circumstance of where they live; and third, ease the regulatory burden.
Getting kids into clinical trials is imperative, because that's where we're seeing success for the newest therapies that don't slash, burn and poison: consider the recent reports of a Phase 1 trial in the US studying a CAR T-cell therapy targeting a protein on the surface of acute lymphoblastic leukemia (ALL) cells. Complete remission was realized in 20 of 22 patients with refractory (hard-to-treat or relapsed) ALL, who had been heavily pre-treated, and were without other options. Phase 1 trials are primarily about dosing and side-effects, but this trial also had remarkable success in getting children into remission. Advances in the genetics of cancer also mean that the benefits of certain gene-based therapies – oral medications in the form of a pill – can be realized much earlier in the trial process. Here in Canada, a first-in-children trial of a new drug, which is now standard of care in adults with melanoma, is producing remarkable results in children with brain tumours – children who have limited options with approved treatments.
So, as early-phase clinical trials move from the experimental to the effective, governments have to move with them. Provincial and territorial health jurisdictions in Canada do a good job paying the cost of out-of-province treatment that's considered 'standard of care', such as bone marrow transplants. But Phase 1 and 2 clinical trials are typically classified as 'experimental', and often aren't covered by provincial health care plans – meaning that kids who need access to a clinical trial in another province may not be able to get there.
Kids in the most desperate need of new treatment options need to be in appropriate clinical trials – whether the child is in BC and the trial in Ontario, or vice versa. The provinces must end what amounts to denial of access by agreeing to cover, in-province or out, the standard aspects of medical care that patients in clinical trials require – not the costs of experimental drugs or tests – just those aspects of treatment which a child with recurrent or progressive cancer would need to undergo, regardless of whether they are part of a clinical trial or not.
We don't ask or propose that every province fund every clinical trial. What's needed is a pan-Canadian mechanism that identifies, in a measured, responsible way, what's sufficiently promising to be eligible for provincial funding. What's working needs to get working faster: the progression of a paediatric cancer, especially relapsed cancer, is often faster, and more deadly, than a three-year Health Canada approval timeline. The number of paediatric patients is small. But the number of 'life years' impacted by – and lost to – childhood cancer is great.
No Canadian child with cancer should be a victim of geography.