The mandate of the Program for Individualized CF Therapy is to change the future course of disease for CF patients by determining the best medication on an individual patient basis. We will recruit up to 100 CF patients representative of the Canadian population with respect to their CFTR mutation. Each will be fully characterized with respect to genomic and clinical phenotypic data. Blood and primary nasal epithelial cells will be obtained from each patient. Induced pluripotent stem (iPS) cells will be derived from blood samples and differentiated into CF-relevant cell types, such as ciliated lung epithelium, lung organoids, bile ductal cysts and pancreatic progenitors. The resource of cell lines and differentiated progeny will form the basis of validated cell and tissue-based assays for assessing the effectiveness of existing or novel drug combinations against individual CF patients and their affected tissues.
The Centre will also provide patient-specific pre-clinical and clinical tests to enhance the effectiveness of future clinical trials. Data collected will be available nationally for broad-based screening and the technologies will also be provided to investigators who wish to analyze their own specific patients in the same manner. Ultimately it is the hope to make this resource available internationally as well. The unique capacity of these resources will accelerate our progress toward personalized medicine for CF patients. Find out more about the resources available in the platforms section.