New hope for children with terminal disease
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 delay 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 Batten 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.
The drug will be administered to children through a port directly in the brain in a four hour infusion.
Lucy added: “When Ollie and Amelia were first diagnosed with Batten 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. I can’t describe how it feels to hear those words, we are forever haunted by those words.”
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. Before, we had no hope, none what so ever.”
For more on the Carrolls story and to support their fundraising visit www. olliesarmy.co.uk. PARENTS and carers are in with a chance to win a family ticket to the panto in return for filling in a council questionnaire.
A family ticket for four to see a Christmas performance of Aladdin is on offer to parents who give their views on improving access to childcare, such as day nurseries, pre-schools, childminders and out of school clubs.Those who take part in an online questionnaire will be entered into a free prize draw to win a family ticket for four to see ‘Aladdin at Crewe’s Lyceum Theatre.
The findings of the survey will be published next year as part of Cheshire East’s childcare sufficiency assessment.
Councillor Liz Durham, cabinet member for children and families, said: “We hope that parents and carers will have five or 10 minutes to spare to take part in this research by completing the online questionnaire, which is anonymous and confidential. You will not be identified in any report and we thank you in advance for your help in this research, it is much appreciated.”
The questionnaire can be accessed at www.surveymonkey.co.uk/r/csa-parent-2016.
Lucy and Mike Carroll with children Amelia aged 3 and Ollie 5 of Poynton