New hope for chil­dren with ter­mi­nal dis­ease

Macclesfield Express - - YOUR PICTURES - 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 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. ol­ 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.sur­vey­mon­­ent-2016.

Lucy and Mike Carroll with chil­dren Amelia aged 3 and Ol­lie 5 of Poyn­ton

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