Please read a letter below from the parents of Ollie and Amelia, together we can help them.
We are appealing for your help to start a trial/compassionate use program which if successful will save the eyesight of children with CLN2 Batten Disease. £250,000 needs to be raised in the shortest possible time.
Lucy and Mike Carroll have 2 children with CLN2 Batten Disease. Ollie (9) and Amelia (7) CLN2 late Infantile Batten disease is an ultra-rare disease that progresses rapidly with seizures, loss of movement, speech, and swallow, accompanied with blindness.
There is no cure for CLN2 Batten Disease. Following years of campaigning by Lucy, Mike and others, NHS England agreed, in September 2019, to fund a treatment which is proven to slow down the progression of this disease via a managed access agreement for a five year period which is delivered at Great Ormond Street Children’s Hospital, London. Eighteen children are currently receiving this treatment.
The treatment involves a four hour enzyme infusion delivered directly into the brain. This is carried out by a specialist team every two weeks.
There is growing evidence that the treatment slows the progress of the disease and improves quality of life. Unfortunately it does not prevent the inevitable loss of eyesight in children with CLN2 Batten Disease.
Amelia (7) had treatment on compassionate grounds ahead of the managed agreement and is able to lead an active life, meet friends, attend school and eat normally without needing a feeding tube. Her eyesight, however, is deteriorating and like other CLN2 Batten Disease children she will become blind. This is very frightening for any child to have to experience and extremely distressing for their parents and siblings. Every effort should be made to prevent this from happening if this is possible.
Recent research in animals shows that there is hope that this progression towards blindness can be slowed or prevented by injecting the same enzyme into the eyes of children with CLN2 Batten Disease.
Specialist staff at Great Ormond Street Hospital want to carry out a trial in order to investigate the effectiveness of this approach but money is needed for this to go ahead and currently the NHS is not in a position to provide the necessary funding.
Children do not have the time available for a long wait whilst decisions are made over money. They are continuously progressing towards becoming blind. Something needs to be done and quickly.
Consequently, Lucy and Mike have again taken the initiative to raise awareness and to try and secure the £250K needed to start the trial.
They are looking for sponsors, donations and any other support that will help so that this trial can start as quickly as possible.