Breathe - 10 Years Later
The SickKids Breathe Program was a five-year research program established in 2004. Almost ten years later, the impact of this program has been immense.
The goal of the SickKids Cystic Fibrosis (CF) Centre Breathe Program was to develop new tools and approaches targeting the basic defect in the most common CF-causing mutation: ∆F508-CFTR. The program had two foci. One group of researchers focused on therapy discovery: the identification or invention and characterization of novel therapeutic agents. The other group focused on the development of clinical tools to enable meaningful evaluation of the efficacy of CF therapies.
The Breathe funding enabled the transformation of our basic and clinical studies of Cystic Fibrosis. With Breathe funding, we were able to translate our basic understanding of CF disease mechanisms into therapy discovery platforms and new clinical tools for CF therapy testing. Breathe funding created a new high-throughput assay platform for identifying potentiators and correctors of the primary defects caused by CF gene mutations, enabled the development of our mouse facility into a preclinical drug testing resource and provided new tools for assessing the activity of CF drugs such as KALYDECO. The successes at SickKids enabled by Breathe led to the integration of new clinical tools in ongoing trials of the Vertex modulators and new academic and pharmaceutical partnerships across the country and globally (Quebec to British Columbia, United States, United Kingdom and Germany). All of these collaborations focus on the discovery and implementation of new CF therapies.
The momentum provided by Breathe led to our new efforts to further transform our drug development to better treat every CF patient (including the majority with the F508del mutation). We want to optimize therapies to best treat each and every patient. We envision a novel drug discovery and implementation program that will facilitate patient- specific treatments and clinical trials. This will be a new uniquely Canadian collaborative model for treating CF patients, ensuring the CF Canada researchers continue to lead in promoting the health and well-being of CF patients.
For more information see the Breathe website.