Save Beau’s Vision -treatment fund to access tovorafenib

My name is Emma and I’m Beau Johnston’s mum. We are in a race against time to see if we can save Beau’s vision.

Beau was diagnosed with a Hypothalamic Optic Pathway Glioma (Brain tumour) BRAF KIAA fusion at 2. She had debulking brain surgery followed by 8 chemotherapy courses for progressive disease that left her visually impaired. Her recent scan has unfortunately shown a new tumour on her remaining functional nerve fibres.

There is a targeted therapy called Tovorafenib or Ojemda that has shown great promise for tumours like Beaus. We had been hoping to access to save on the long lasting side effects of Radiotherapy with risk of stroke, secondary cancers, vision loss, pituitary dysfunction and cognitive issues. Unfortunately the phase 2 trial is closed and Beau is not eligible for the phase 3 and there is currently no route for compassionate access through IPSEN. To access via the USA it is 35k USD per month for the drug alone. And 450k for the year which is unattainable for most and certainly for us in the timeframe required.