About:

In 2019, ANZCHOG approached the CTF to support its efforts to run a Phase II MEK Inhibitor clinical trial across multiple paediatric and adolescent/young adult cancer centres in Australia and New Zealand. 

The trial is intended to improve treatment options for people with NF1, specifically those with complex tumours. Approximately 60 Australian and New Zealand NF1 patients between the ages of 3 months and 25 years will be recruited to investigate the effectiveness of the drug Trametinib for the treatment of plexiform neurofibromas and optic pathway gliomas.

In addition to monitoring the expected reduction in tumour size, the trial will also monitor the improvement in pain, function and quality of life, as well as brain function and social development. Running for five years, the trial will also consider the impact of the drug on learning and behaviour over time.

Once the trial is complete, the results will contribute to a body of evidence to support advocacy efforts to make this drug available to everyone impacted by NF1 as a front-line treatment that is funded under the Federal Government PBS. 

The Children's Tumour Foundation have invested $100,000 into the study, with another $50,000 to be secured. In June 2020, the Federal Government announced a significant investment ($760k) into childhood brain cancer clinical trials from the Medical Research Future Fund (MRFF) and is the first ever government funding to be granted to NF research. 

This is huge news for the NF community and means full funding of the trial is now within reach!