Big Night Oot just what doc or­dered

Char­ity funds own sci­en­tist with event pro­ceeds

Lennox Herald - - LIFESAVER - Lor­raine Weir

A char­ity founded by a fam­ily whose daugh­ter was di­ag­nosed with life­lim­it­ing cys­tic fi­bro­sis when she was just six weeks old is now fund­ing its very own sci­en­tist.

Flut­terby FUNdrais­ers, started by Bon­hill woman Mag­gie Gal­lacher and daugh­ter Kelli, has raised over a quar­ter of a mil­lion pounds since form­ing in 2007.

And now cash col­lected by the char­ity will go di­rectly to a doc­tor re­search­ing po­ten­tially life-chang­ing gene ther­apy.

The char­ity, which gained of­fi­cial sta­tus in 2014, will fund the work of clin­i­cal re­search fel­low, Dr Aarash Saleh, who works with the Na­tional Heart and Lung In­sti­tute at Im­pe­rial Col­lege in Lon­don.

They have been work­ing closely with the Gene Ther­apy Con­sor­tium who they have been able to do­nate £256,759 to across their re­search pro­grammes based in Lon­don, Ed­in­burgh and Ox­ford.

An­nounc­ing the ex­cit­ing de­vel­op­ment at the or­gan­i­sa­tion’s flag­ship fundraiser on Fri­day night, Mag­gie, whose daugh­ter Kelli has CF, said: “We are de­lighted to an­nounce a new part­ner­ship, ce­ment­ing that re­la­tion­ship as we take on the spon­sor­ship of Dr Saleh, who will be work­ing on the new and ex­cit­ing wave two of re­search.

“We feel that be­ing able to put a face to the project will give the char­ity and our sup­port­ers a much more per­sonal touch and will al­low us to give you more reg­u­lar and in­for­ma­tive up­dates on what is in­volved in the day to day pro­cesses of the re­search team.

“Money will be go­ing straight to his work which is pro­vid­ing so much hope to peo­ple with CF.”

In 2015, a trial, which was 20 years in the mak­ing, showed hope for those with the chronic lung con­di­tion.

The re­sults of the UK Gene Ther­apy Con­sor­tium’s ex­per­i­ment proved those given treat­ment re­plac­ing the faulty gene re­spon­si­ble for cys­tic fi­bro­sis over a year had im­proved lung func­tion.

Now, early work by Dr Saleh, on the sec­ond wave, is show­ing real prom­ise.

He said: “We are now plan­ning to per­form the first ever trial test­ing our next gen­er­a­tion gene ther­apy.

“This treat­ment has been shown to be con­sid­er­ably more ef­fec­tive at de­liv­er­ing its healthy copy of the CFTR gene com­pared to the vec­tor used in the 2015 trial.

“Thanks to the funds raised by Flut­terby FUNdrais­ers, I have been able to make sig­nif­i­cant progress as­sess­ing a new tech­nol­ogy that al­lows us to de­tect very low lev­els of spe­cific genes present in cells with un­prece­dented ac­cu­racy so that we can de­ter­mine the num­ber of vi­ral par­ti­cles that have been trans­ferred to cells.

“So far our data have con­firmed ear­lier ex­cit­ing re­sults. Fund­ing from Flut­terby FUNdrais­ers has also al­lowed me to per­form ex­per­i­ments re­fin­ing our safety mea­sure­ments.

“This will give us a more ac­cu­rate way of con­firm­ing the safety of this prod­uct com­pared to pre­vi­ous tri­als and will po­ten­tially im­pact whether pa­tients need to stay in hos­pi­tal af­ter a dose of vi­ral gene ther­apy and if so for how long.”

Dr Gerry McLach­lan, who works at the Cen­tre for Ge­nomic and Ex­per­i­men­tal Medicine at Ed­in­burgh Univer­sity with the Scot­tish team of the UK Gene Ther­apy Con­sor­tium at­tended on Fri­day night and wel­comed the news that the char­ity will be fund­ing the work of his col­league.

He said: “The money raised tonight and in the fu­ture will help to fund Dr Aarash’s stud­ies and these kind of things are in­valu­able.

“Meet­ing pa­tients and fam­i­lies like Kelli and Mag­gie and see­ing their de­vo­tion to sup­port our re­search is fan­tas­tic.

“Pa­tients take part in our tri­als and have faith in us. We are so, so grate­ful and really it’s the ef­forts of the CF pa­tients and their fam­i­lies that make it so much more than just a job for us.”

This year’s Big­ger Night Oot for CF saw 370 peo­ple head to the new venue of the Ersk­ine Bridge Ho­tel for an evening of ex­cit­ing en­ter­tain­ment from a host of acts who gave their time for free in­clud­ing Take This, Lit­tle Mix Magic and Alan­nah Farmer.

It was the 11th an­nual event to be hosted by Flut­terby FUNdrais­ers coin­ing in £11,052 for re­search into the next wave of gene ther­apy tri­als.

Mag­gie added: “We are all truly hum­bled by your sup­port and gen­eros­ity and thank you to each and ev­ery per­son in­volved. On top of the money raised, we also re­ceived two cheques — one from our branch in Wales for just un­der £9000 and an­other from our friends in Derry for £3000.”

Lead­ing the way Kelli with mum Mag­gie (above) re­ceived a cheque from sup­port­ers from Derry (left)

Re­search Dr Aarash Saleh

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