‘There is a lot of hope’ for CF babies, as new drug proves effective and safe for infants

Kara and Isaac Moss with associate professor of paediatrics at RCSI and consultant in respiratory medicine at CHI Paul McNally.

There is “a lot of hope” now for babies born with cystic fibrosis (CF), according to the mother of an Irish baby who was the first in the world to be treated as a four-week old with a ground-breaking approach.

Isaac Moss, now 2, took part in a global study examining whether a drug already in use for children and adults is safe for babies — through CHI at Crumlin Hospital.

His mother Debbie said: “I think it gives a lot of hope, especially for parents who just have a newborn and they get the diagnosis.” 

Isaac’s sister Kara, now 6, also accessed the treatment during an earlier trial stage for children.

Kara’s diagnosis had been “a huge shock” to Debbie and her husband Wayne.

“It’s so devastating, it’s so upsetting and you don’t know what the future holds for her,” she said.

These drugs are just so effective, it’s just amazing. There is a lot of hope there, it’s just good.

She recalled getting the phone call about Kara, and "that devastation we would have had because we didn’t know what her life would be like”. 

Kara and Isaac Moss participated in a landmark study which supports use of a ground-breaking cystic fibrosis drug in infants from four weeks of age. Picture: Patrick Bolger

However she said: "Now it is just completely changed because it is more about the hope for their life. Kara will live a normal life, she will live to be a normal age.” 

The drug, ivacaftor (Kalydeco), is the first designed to treat the basic defect in cystic fibrosis.

“Kalydeco made a huge difference, and to us it helped us make a decision to have another child [after Kara] because to be honest we knew the risk was one in four of having another child with CF,” she said.

It has made their daily life easier as the tablet is only taken twice a day.

“It was very successful for Kara, and it did mean we could reduce down some of the other medication she had been on,” her mother said.

“For us, it made things a lot more manageable. I’d say within a few months we would have seen Kara’s drugs reducing.” 

The couple feel “so lucky” to have accessed this trial in Ireland.

“Research studies like this one are so important to ensuring that children get access to the right treatments as early as possible,” she said.

With the right medications, they can enjoy a healthy childhood and look forward to a brighter future.

Associate professor of paediatrics at the Royal College of Surgeons Ireland and consultant in respiratory medicine at CHI Paul McNally said: “This is a huge moment in cystic fibrosis.”

He is one of the study authors, published in the Journal of Cystic Fibrosis, showing how Kalydeko is effective and safe for babies.

“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,” he said.

The study, ‘Safety and efficacy of Ivacaftor in infants aged one to less than four months with Cystic Fibrosis’, also involved researchers from America and England.