Irish siblings lead way21 in Cystic Fibrosis trial
Pair look forward to ‘brighter future’
TWO Irish siblings are at the centre of a major breakthrough for Cystic Fibrosis patients.
Kara and Isaac Moss can now look forward to a “brighter future” after they participated in a new trial that showed the medication they were taking massively alleviated their symptoms.
Ivacaftor (Kalydeko), is the first drug designed to treat the basic defect in Cystic Fibrosis.
It was originally approved for adults, then sequentially over several years for older and younger children.
Currently, it is approved for babies aged four months and older, however, this new research suggests that it is safe and effective for babies as young as four weeks of age.
The drug targets a genetic change seen in around 4% of people with Cystic Fibrosis worldwide and around 10% in Ireland.
Experts predict that the earlier treatments can begin, the more likely that the progression of the condition can be down or halted in children. Kara. five, was part of the earlier phase of the study, which was conducted by RCSI University of Medicine and Health Sciences and Children’s Health Ireland, before it was approved for children over four months.
Her brother Isaac, two, was the first baby with Cystic Fibrosis in the world to be diagnosed from birth, so he was immediately enrolled into its most recent trial.
Their mum Debbie said: “Both Kara and Isaac are doing really well and remarkably are not experiencing any of the typical symptoms of Cystic Fibrosis at the moment.
“Research studies like this one are so important to ensuring that children get access to the right treatments as early as possible. With the right medications, they can enjoy a healthy childhood and look forward to a brighter future.”
Paul Mcnally, Associate Professor of Paediatrics at RCSI and Consultant in Respiratory Medicine at CHI, said the approval of the drug for younger chilslowed dren is “a huge moment”. He is also one of the authors of the study, which was published in the Journal of Cystic Fibrosis.
Dr Mcnally added: “Over the years Ivacaftor, or Kalydeko, has been put through clinical trials in younger and younger children. Now, through this study, it is safe and effective all the way down to four weeks of age.
“The availability of a treatment that targets the underlying cause of the disease in newborns and can be started immediately at diagnosis will provide a huge sense of reassurance and hope for families.”
Ireland has the highest incidence of Cystic Fibrosis in the world, with approximately 1,400 children and adults living with the condition in this country.
Thirty cases of CF are diagnosed here each year, typically at four weeks of age through the newborn screening programme.
They’re not having any of typical symptoms of CF
MUM DEBBIE YESTERDAY