New US drug hope for Ollie and Amelia STUART GREER
PARENTS of two terminally ill children have been given their first hope of a treatment that could help slow down the disease.
Lucy and Mike Carroll’s children Ollie, five, and Amelia, three, are two of only five children in the UK to have been chosen for a new drug trial to slow the effects of Battens Disease.
The rare genetic condition means as they get older they will lose their speech, mobility and eyesight and are unlikely to live past the age of 12.
Trials of a new drug called Cerliponase Alpha by US pharmaceutical company BioMarin have proved positive and are being expanded to the UK to only five children.
Mum Lucy, 31, from Poynton, said: “We are feeling a massive mix of emotion right now, full of joy, hope, worry but also sadness as we know that this ‘treatment’ has come too late for some children.
“Our hearts break for these families.
“We know this is not a cure and we know that this may not work but we have hope, we have something when we used to have nothing.
“We know this won’t stop Battens Disease one day taking our babies from us, but if this can help them and make their lives more comfortable and give them longer with us. Then we are just so grateful we have been given this opportunity when we feel so utterly useless.”
The CLN2 enzyme replacement therapy trial, which could start in the New Year, means the family will have to travel down to Great Ormond Street Hospital (GOSH) every two weeks, indefinitely.
Lucy added: “When Ollie and Amelia were first diagnosed with Battens Disease our first questions like every other parent were what do we do now?
“What treatment will they need? What will make them better? We sat in utter shock as the doctors shook their heads and told us there is no cure, there is no treatment.”
The tragedy facing the Carrolls triggered a huge reaction from their family, friends and the wider community who have been fundraising to support them and campaigning to raise awareness of the disease.
Last month Ollie won an Inspirational Child award at the WellChild awards and shared a heartwarming moment with Prince Harry. The touching image made headlines around the world.
Lucy and Mike, 32, have refused to give up hope of a future for Ollie and Amelia and lobbied for access to the new drug trials - even travelling to Paris to ask for help.
The family are now focussed on fundraising to cover the cost of the travel to London and accommodation during the trials.
Lucy said: “This could not have happened without the Batten Disease Family Association (BDFA), GOSH, the Batten Disease Support & Research Association (BDSRA) and BioMarin working together as a team.
“Together they have given hope to families, they have given hope to children when they were told they had no hope.”
For more on the Carroll’s story and to support their fundraising visit www.olliesarmy. co.uk.