Cystic fibrosis (CF) is the most common fatal genetic disease, affecting 4,000 Canadians and 80,000 people throughout the world. The debilitating disease causes difficulties in breathing, recurrent lung infections and digestive disorders before killing those who suffer from it, at a median age of 35 in Canada. Currently, treatments can ease the symptoms of the disease, but there is no cure. Newer drugs today can address the underlying genetic defect that causes CF, but only some patients respond positively to them, while others do not – and there is no way for clinicians to know in advance which category a patient will fall into. Given the side effects of these drugs and their cost (more than $300,000/year per patient for a drug that needs to be administered lifelong), there is a pressing need for robust predictors of who will respond to what treatment.
Dr. Felix Ratjen of the Hospital for Sick Children and his team are developing predictive tools that will help clinicians determine the right medicine for the right patient. The team will examine how genetic factors, which can be assessed from a non-invasive blood test, can help predict individual treatment responses. They will also examine if drug testing on patient-derived tissue samples can be used to inform the potential clinical response to drugs by each patient. The team will work with industry partners, patient organizations and the Ontario Ministry of Health and Long-Term Care to integrate these strategies into patient care once they have been shown to be effective.
The result of the team’s work will be a paradigm shift toward individualized treatment for CF, assistance for clinicians in making treatment decisions, guidance for policymakers on reimbursement for the most cost-effective care and better health outcomes for patients.