Professor David Ziegler
The Kids’ Cancer Project investment of over $700,000 for access to the drug DFMO (difluoromethylornithine) and Phase One of a clinical trial for children with relapsed high-risk neuroblastoma over a decade ago was our first investment into Professor David Ziegler’s groundbreaking neuroblastoma research in 2014.
This week after significant advocacy, the Federal Health Minister, Mark Butler, announced that every Australian child and young adult with high-risk neuroblastoma will now have free access at Australian hospitals to DFMO, thanks to funding from the Australian Government.
Neuroblastoma is the most common extra-cranial solid tumour affecting very young children, usually from one to five years of age. High-risk (HR) neuroblastoma remains highly resistant to standard chemotherapeutics delivering a dismal outcome for these little patients.
Owen Finegan, CEO, The Kids’ Cancer Project stated that the Federal Governments approval for interim funding for DFMO is truly life-changing for families affected and the drug will be made available in Australia on the PBS for hundreds of dollars rather than a treatment plan, travel and accommodation in the USA that was costing close to five hundred thousand dollars. Children with high-risk neuroblastoma now have a better chance of survival without parents having to travel with a sick child or worry about the financial stress involved.
This financial year The Kids’ Cancer Project has also partnered with like-minded charities Tour De Cure and Neuroblastoma Australia to ensure this pioneering research project into DFMO and access to the drug can translate to the next exciting stage – a clinical trial using a knockout drug combination.
DFMO will be used in combination with a new compound, AMXT-1501. Preliminary research has found that when the two agents are combined with standard neuroblastoma chemotherapy, outstanding responses have resulted.
A clinical trial of the DFMO and AMXT-1501 combination is currently underway for adults with cancer, and this has also paved the way for a clinical trial of DFMO plus AMXT-1501 combined with chemotherapy, in children with HR neuroblastoma.
The goal of these trials will be to lay the groundwork necessary to incorporate this highly promising and novel therapeutic strategy into the standard frontline treatment for every child with HR neuroblastoma.
Finegan said while time, funding and patience are key factors in delivering groundbreaking research outcomes, like-minded partnerships also deliver outstanding results. “I’m proud of the leadership shown in every aspect of this decade-long project.
From the research team to the funders our goal has been to make a difference to kids with neuroblastoma by providing access to new treatments and cutting-edge clinical trials so they too can have a bright future ahead of them.”
The investment of The Kids' Cancer Project and its partners is significant in enabling a range of projects to be conducted at the same time, representing a diverse yet comprehensive undertaking across our charity’s four pillars of research excellence: Discover, Translate, Support, and Build.
