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In a groundbreaking development for the treatment of Cystic Fibrosis (CF), a new study conducted by researchers at RCSI University of Medicine and Health Sciences and Children’s Health Ireland has demonstrated the safety and efficacy of a CF drug in newborns as young as four weeks old.
The study, published in the Journal of Cystic Fibrosis, marks a significant milestone in CF treatment, with the drug Ivacaftor (Kalydeko) proving to be safe and effective in addressing the underlying defect that causes CF in newborns. This achievement is hailed as a monumental moment in CF research by leading experts in the field.
“This is a huge moment in Cystic Fibrosis,” stated Paul McNally, Associate Professor of Paediatrics at RCSI and Consultant in Respiratory Medicine at CHI, and one of the lead researchers of the study. “Through this study, Ivacaftor has been shown to be safe and effective all the way down to four weeks of age. This is an important development because almost all children are diagnosed through newborn screening at around this time.”
Cystic Fibrosis, an inherited disease primarily affecting the lungs and digestive system, presents a significant health challenge worldwide. Notably, Ireland has the highest incidence of CF globally, with over 1,400 children and adults living with the condition, and more than 30 new cases diagnosed annually through the newborn screening program.
Ivacaftor, the first drug designed to target the basic defect in CF, was initially approved for adults and later for older and younger children. However, this recent research suggests that it is safe and effective for newborns aged as little as four weeks old.
Experts in CF anticipate that initiating treatment at an earlier stage could potentially slow down or even halt the progression of the condition in children. This aligns with ongoing international research efforts led by RCSI and Children’s Health Ireland, focusing on early intervention strategies.
Debbie Moss, mother to siblings Kara (aged 5) and Isaac (aged 2), who participated in the study, expressed gratitude for the research efforts: “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.”
Ivacaftor, manufactured by Vertex Pharmaceuticals, targets a genetic mutation present in approximately 4% of individuals with CF globally, and around 10% in Ireland. The pharmaceutical company is currently seeking an extension to the marketing authorization for Ivacaftor for use in newborns down to one month of age, further underscoring the potential impact of this groundbreaking research on CF treatment protocols.
With the promise of early intervention and targeted therapies, the findings of this study offer renewed hope for families affected by CF, signaling a significant advancement in the management of this complex genetic disorder.
