APPEAL TO FUND MEK INHIBITOR RESEARCH TRIAL
When we speak to families of children with Neurofibromatosis (NF), what they want is hope; hope that a cure can be found or at the very least, the promise of better treatment options. This research trial may not offer a cure, but it certainly has the potential to offer more effective treatment solutions for those living with NF1.
It is for this reason, amongst hundreds others that we are committed to the funding of a potentially life-changing clinical trial in Australia.
ABOUT THE TRIAL
The Australian and New Zealand Children’s Haematology and Oncology Group (ANZCHOG) have approached the Children’s Tumour Foundation (CTF) to support their efforts to run a Phase II MEK Inhibitor clinical trial across ten paediatric and adolescent/young adult cancer centres in Australia and New Zealand.
This will involve 60 Australian children and will investigate the effectiveness of Trametinib for the treatment of plexiform neurofibromas and optic pathway gliomas. In addition to monitoring the expected reduction in tumour size, it will also monitor the improvement in pain, function and quality of life as well as brain function and social development.
The trial will take place over five years with an expanded age range of children/young adults between the ages of 3 months and 25 years and will also consider the impact of the drug on learning and behaviour over time.
Once the trial is completed, the results will contribute to a body of evidence that will support any advocacy efforts to make this drug available to all NF1 sufferers as a front-line treatment with the Federal Government.
We want to ensure that families do not have to suffer through 12 months or more of harsh chemotherapy that often kills more than just the tumour cells in question. This trial brings with it the opportunity to demonstrate to the international NF research community Australia’s potential to significantly aid sufferers of NF, and to potentially attract future clinical trials.
But most importantly, what this particular trial offers is the opportunity to treat 60 children from Australia who have been diagnosed with NF1. However, the results of the trial have far more reach and are potentially life-changing for all children diagnosed with this genetic condition worldwide.
ANCHOG have estimated the costs (excluding the cost of the drug) to be close to $1 million dollars. This figure considers the following:
- Identification, selection and ethics review,
- Registration, scanning, paperwork and peer review; and
- The many specialists and allied health professionals involved in the trial
Half of this funding has been secured independently, but the remaining $500,000 still needs to be secured.
As part of our 2019 Tax Appeal, we raised an amazing $90,052 towards this figure, but we can't stop there. ALL FUNDS DONATED THROUGH THIS APPEALS PAGE WILL BE DEDICATED TO THE MEK INHIBITOR TRIAL.
READ XAVIER'S STORY TO LEARN WHY A TRIAL LIKE THIS IS SO IMPORTANT