A growing number of children with cancer are receiving the news that their families long for – a future free of this heartbreaking disease.
For too many children, however, this kind of future remains out of reach. A clinical trial funded by The Kids’ Cancer Project is treating a group of these young people who have almost no chance of experiencing five more years of life without a new medical breakthrough.
Children, adolescents and young adults with relapsed solid tumours, such as osteosarcoma, neuroblastoma and malignant rhabdoid tumours, generally have a five-year survival rate of less than 20 per cent. The prognosis for the 10 young patients currently enrolled in the North Trial, which is being conducted across 14 hospitals in Australia and New Zealand, is even worse.
“In this group of relapse patients, you could argue that their five-year survival rate is closer to zero because once they have relapsed post traditional treatment, their prognosis is dismal,” says Dr Paul Wood, a paediatric oncologist at Monash Health in Victoria who is leading the trial.
“In this clinical trial, we are targeting groups that have traditionally had a very poor prognosis, in the hope that this new treatment can make a difference.
Trialling a new treatment
This new treatment involves the use of a drug called Panobinostat, which is often used in adult patients with cancers such as multiple myeloma. Patients in the North Trial receive a low dose of the drug every day to promote a process called differentiation.
“For cancer to form, the normal cellular processes have to be overwritten or hijacked by the cancer cell,” explains Dr Wood, who is an Honorary Clinical Associate at the Hudson Institute of Medical Research and Associate Investigator, Paediatric Precision Medicine Program.
“What this drug is trying to do is switch all off those abnormal processes and allow normal pathways to become active again. We are trying to get the malignant cancer cells to transform into non-malignant benign tissue.”
Dr Wood explains that those enrolled in the North Trial must first undergo chemotherapy as a means of stabilising tumor growth.
“We've chosen a very difficult-to-treat patient population,” he says. “These patients have relapsed and have already had what is considered standard care, so it’s clear that they have a very resistant tumor that has gone through the ringer and managed to come out the other side and still be viable. If we were to just use Panobinostat as a single agent by itself, it would no doubt fail.”
Vital research funding
Dr Wood says medicine was his natural career choice. His father was a doctor and he excelled in biology in school. A desire to help children live a full life saw him choose paediatric oncology as a specialisation.
“The outcomes of paediatric cancers are much better than adult cancers, overall,” he says. “There are still these sub-populations that don't do so well, but overall we have good success."
"It means that then you can work with these families during treatment and see these kids grow up and go into adulthood and go to school and do all of these normal things, which is very rewarding," said Dr Wood.
"Paediatric oncology also has a very strong research component and there are a lot of other areas of medicine where we don't get to potentially make a difference with research.”
Dr Wood says potentially life-saving research would not be possible without funding and that The Kids’ Cancer Project has been essential to his study.
“None of this research can take place without funding,” he says. “It helps us to monitor the study, make sure we are doing it safely and make sure all the trial sites are compliant. If we don't do all these things, the population will lose trust in us as researchers and lose trust in our ability to look after their children.”
It’s been 10 months since the first child was enrolled in the North Trial and Dr Wood describes progress as “encouraging”.
“The trial hasn’t finished so we can’t give out results, but we are seeing some encouraging outcomes,” he says. “We have certain milestones that we have to reach for the trial to continue and, at the moment, we are achieving those milestones. The main one is that patients on the drug are still surviving.”
These milestones are bringing hope that a cancer-free future for these kids is well within their reach.