Health

‘Groundbreaking’ cystic fibrosis drug safe and effective in newborns, new research shows

Though it is known that drugs like Kalydeco improve cystic fibrosis, the ‘big question’ is if they slow down or prevent decline when started earlier in life, researchers say

Siblings Kara and Isaac Moss participated in a landmark study that supports the use of a ground-breaking Cystic Fibrosis drug in infants from four weeks of age. Photograph: Patrick Bolger
Jack White
Fri Apr 12 2024 - 09:47

A “groundbreaking” drug that targets the underlying cause of cystic fibrosis has been shown to be safe and effective in newborns aged four weeks and above.

Until now, Ivacaftor – sold under the trade name Kalydeco – has only been available to those aged four months or older.

Ireland has the highest incidence of the progressive inherited disease in the world due to its high prevalence of Celtic genes which are more susceptible to mutations that cause it.

The drug works by targeting a genetic change seen in around 10 per cent of people with cystic fibrosis in Ireland.

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The new research carried out by RCSI University of Medicine and Health Sciences and Children’s Health Ireland has been described as a “huge moment” for the condition by Paul McNally, co-author of the study and associate professor of paediatrics at RCSI.

A longer-term study that is tracking the impact and efficacy of earlier intervention among 550 children from Ireland and the UK has recently begun

Mr McNally said the findings mean treatment can be started immediately after diagnosis, once an expansion is approved by the European Medicines Agency.

Though it is known that drugs like Kalydeco improve cystic fibrosis, the “big question” is if they slow down or prevent decline when started earlier in life, he said adding that the drugs hold “amazing promise”.

A longer-term study that is tracking the impact and efficacy of earlier intervention among 550 children from Ireland and the UK has recently begun.

Early findings in the use of the drug among children are promising including a lack of respiratory symptoms and restoration of pancreatic function.

“We’re hopefully looking at future generations of children who will have much, much milder disease than previous generations because they started on drugs much earlier,” he said.

Two-year-old Isaac Moss from Ashbourne, Co Meath was the first baby in the world to be diagnosed with cystic fibrosis from birth through the newborn screening programme, after which he was enrolled directly on to the trial.

His five-year-old sister Kara was part of an earlier phase of the study that paved the approval of the drug in older infants and led to the latest trial.

Their mother Debbie said Kara’s diagnosis was a “huge shock” and “devastating” at the time, but because her children have started Kalydeco at such a young age, “they’re the same as any other child”, she said.

“It gives a lot of hope for the kids’ lives,” she said.

* This article was amended on Friday, April 12th.