Cheshire parents in fight for Batten Disease treatment

The parents of two children with an extremely rare and life limiting condition say they want a new treatment to be made available on the NHS.

A Cheshire Couple are campaignign to overturn a decision by the National Institute for Health and Care Excellence (NICE) to deny funding for a "miracle" treatment that has benefitted two of their children who have CNL2 Batten's Disease.

Batten's Disease is always fatal and children diagnosed will also suffer from dementia along with worsening seizures and a loss of sight and motor function. Around five children a year are diagnosed with CNL2 Batten's Disease.

Mike and Lucy Carroll's children Ollie and Amelia were both diagnosed with the condition in 2015 when Ollie was 4 and Amelia just 2. 

Ollie and Amelia were accepted onto a compassionate use program for a trial of an enzyme replacement treatment being carried out at Great Ormond Street Hospital. The trial has benefitted both. Ollie's seizures have stopped since then and Amelia's mum told us about how her daughter is doing.

"Amelia started this treatment before the signs of Batten's disease and she still has no signs of the disease, yet the disease should have taken away almost all of her abilities yet she is still absolutely thriving. She's singing, dancing, she does lots of after school activities as well as attending a mainstream school."

The treatment is approved for use in America and some European countries but not the UK as NICE acknolwedged that the treatment improved the quality of life of children with the disease but questioned its long-term effectiveness.

The Carrolls are currently in talks with NICE, NHS England, the Batten's Disease Family Association and pharmaceautical company BioMarin to push for the funding of this treatment as there are 10 children in the UK with the disease who could potentially be left without treatment if the treatment is not recommended by NICE. 

Ollie and Amelia's dad Mike criticized the length of time the discussions are taking saying "The whole process needs speeding up, or the drugs company or NICE need to come to a decision where [they] will treat these children until there is a decision"

You can read more about the Carroll's story at www.olliesarmy.co.uk