So Macy Menzie could participate in vital clinical trials over 12 months, the seven-year-old had to fly from Hobart to Melbourne every three weeks.
Her condition, neurofibromatosis type 1, had resulted in a brain tumour in her optic pathway and traditional treatments were not working.
Read more: New State-Wide Children’s And Adolescent/Young Adult Cancer Clinical Trials Unit
Up until two years ago, seriously ill Tasmanian children like Macy were forced to travel to the mainland to take part in clinical trials. Then Associate Professor John Heath decided enough was enough.
“She essentially had the equivalent of a stroke,” explains Associate Professor John Heath, from Tasmania Health Service and the Royal Hobart Hospital. “She has a left-side weakness in her body and she’s got visual impairment. What we’re trying to do is to prevent her from going blind, and potentially it’s life threatening beyond that.”
In 2017, after Professor Heath played a key role in establishing a Children’s and Adolescent/Young Adult Cancer Clinical Trials Unit at Royal Hobart Hospital, Macy was finally able to dump her intense travel schedule to be treated locally.
The Clinical Trials Unit, now funded by a pledge of more than $300,000 over five years from The Kids’ Cancer Project, is ensuring that trailblazing science is being brought to Tasmanians. It means children and their families can avoid the need to travel for potentially life-saving treatment at a time that they can least afford to do so.
Video footage of the 2017 landmark funding announcement.
“I had worked at the Royal Children’s Hospital in Melbourne for many years and was aware of the number of patients travelling from Tasmania, where clinical trials were not available,” said Professor Heath.
“It’s been well established for many years that the health outcomes for children with cancer are very closely related to the availability of, and participation in, clinical trials. Those two factors made it clear that we needed to bring Tasmania into that clinical trial space,” he said.
Also beyond dispute, Professor Heath stated, is the fact that the best outcomes for seriously ill children occur when they are able to be treated locally and experience as little disruption to their lives as possible.
“You’re not just dealing with the treatment, you’re dealing with siblings and parents and friends,” Professor Heath said. “The ability to not have to travel is a big factor, because it means they can still attend school, they can sleep in their own bed, they can enjoy their usual support networks. Then, from a financial point of view, it means the parents can often continue to work in some capacity and not have to spend a great deal of money on travel and accommodation and eating out, etc."
In the two years since the Clinical Trials Unit has been established in Hobart, over 100 children have participated in clinical studies and five have had treatment included as part of that study.
But it’s not just about the children. The Unit also benefits the local medical community in terms of knowledge, exposure to leading research from around the globe and participation in broader national and international trials.
“The other part of it, which is not part of the clinical trials but is a corollary of it and has been, to a large extent, funded by The Kids’ Cancer Project, is that it has allowed us to participate on the national stage,” Professor Heath said.
“We are now a full member of ANZCHOG (the Australian and New Zealand Children’s Haematology/Oncology Group) and that allows us to participate in the exchange of knowledge, keeping up to date with things going on nationally and internationally, and contributing to policy. That’s an indirect benefit, but it’s an absolutely vital one.”
The Kids’ Cancer Project, Professor Heath said, has allowed Tasmania Health Service to match international best practice and, at the same time, benefited the children and families of Tasmania. The success of the Clinical Trials Unit and its involvement in vital research has also meant it has been able to apply for further funding from the National Health and Medical Research Council.
That’s important, because the Unit’s future success is all about funding, Professor Heath said.
“If we don’t have the money then we can’t employ people, so it could all end overnight,” he said. “That would be a disaster. If we’re able to continue then we’ll open more clinical trials, such as one that is currently coming down the line from Europe for the treatment of leukaemia.”
Macy Menzie is 10-years-old and no longer has to travel interstate for the medical treatment that will stop her from going completely blind.
The entire program has a very real effect on patients, Professor Heath said. Young Macy, for example, has been able to access through a compassionate-use program a number of revolutionary drugs that have not previously been listed.
“This is possible because we’ve been able to show the drug company that we will monitor her as though she’s on a clinical trial,” he said. “We can do this because we now have the infrastructure, and particularly an experienced clinical trials coordinator, to collect the data and interact with the company with regards to side effects, etc."
"So now a ten-year-old girl is benefiting from being on a drug that would be seen as cutting-edge in Paris, London or New York, but she’s able to do it at home in Hobart.”