Appeal to fund MEK Inhibitor Drug Trial
Neurofibromatosis Type 1 (NF1) is a dominant genetic disorder that affects as many as 1 in every 2,500 births. It can be inherited from a parent, but in 50% of cases a sufferer will be the first in their family to develop the condition. It is a cancer predisposition syndrome that causes tumours to grow on the nerves through the body and is both progressive and unpredictable.
A common complication of the NF1 condition is the development of tumours called Plexiform Neurofibroma’s. Whilst they are usually slow growing, their complex shape and where they may be located in the body, mean they often cannot be removed surgically. Another relatively common tumour occurring is 15% of children is the development of an optic pathway glioma. Due to their location in the optic nerve they cannot be removed and may cause vision loss in about half of the children who develop one (or similar).
There is no cure for NF1 and treatment options are limited. In instances where surgery is not available due to the tumour’s shape or location, patients will often face recurrent cycles of chemotherapy. This has significant side-effects, including but not limited to chronic nausea, significant weight loss, hair loss and a compromised immune system; and typically, has only managed to slow the growth of tumours.
SO WHAT IS A MEK INHIBITOR?
HEAR FROM DR GEOFF McCOWAGE TO LEARN MORE ABOUT THE TRIAL AND WHY IT IS SO IMPORTANT.
At a very basic level, MEK Inhibitors are given as a pill to target the biochemical pathway that tells a tumour cell to “stop dividing”.
Recently MEK Inhibitors have proven very successful in clinical trials conducted internationally, involving children with plexiform neurofibroma’s aged 2-18 years. The preliminary results of the Australian arm of one of those studies showed that over 55% experienced an average reduction in tumour size of 40%. Another 20% experienced stabilisation of their tumours, rather than the normal pattern of growth. All but two children tolerated the drug with only minor side effects.
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 involving 60 children across ten paediatric and adolescent/young adult cancer centres in Australia and New Zealand.
This is a huge step forward in treating a symptom of NF1, which can be life-limiting and life-threatening!
This appeal is the first step to delivering better treatment outcomes for sufferers, just like Xavier. Please take the time to read his story to better understand the struggles that families like his go through every day.
Our goal is to raise an additional $100,000 to help fund the first year of this trial. It seems like a big number, and it is, but when we look at the cost to families (not only the financial burden, but the emotional toll this condition takes), this number pails in comparison.
Donate today to help us, help those living with NF1.