For CF Patients and Families
In the future we predict that your CF treatment will be as individual as you.
The Program for Individualized CF Therapy (CFIT) was started in 2015 when Cystic Fibrosis Canada, SickKids Foundation and The Hospital for Sick Children joined forces to create a program to accelerate precision medicine for Cystic Fibrosis (CF) patients. What is precision medicine? It is disease treatment that is based on your genetics or cell response.
In 1989 SickKids researchers discovered that CF is a genetic disorder caused by mutations in the Cystic Fibrosis Transmembrane Regulator Gene (CFTR). Some new drugs (CFTR protein modulators) are now approved for certain mutations. However, for many mutations we do not yet have effective drugs available, and even for those where a drug is available, such as the common F508del mutation, the current drugs result in variability in improvement in lung function from patient to patient. Some patients respond reasonably well, whereas others do not respond at all. Our challenge is to understand and better predict who (both adults and children) will improve best on which CF modulator drug, where drugs are available, and support development for new drugs in mutations where there is currently no effective treatment.
To this end, the CFIT Program is creating a bank of nasal epithelial cells and stem cells (iPSCs) from 100 different CF patients. With these cell samples available as models for researchers, new therapies can be tested and perfected to treat CF patients in the future. To maximize the work of the program, different initiatives for researchers such as workshops, grant competitions and an annual meeting are hosted.
How you can help
We thank all patients and families for your interest. As 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 are recruiting 100 CF patients representative of the Canadian population with respect to their CFTR mutation (and a small number with relevant mutations internationally). 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. A small blood sample will be reprogrammed to generate induced pluripotent stem cells (iPSCs). The stem cells can be differentiated into CF-affected cell types, such as lung epithelium, lung organoids, bile ductal cysts, pancreatic cells and virtually any other CF-affected cell type. The resource of nasal cells and stem cells are being used as the basis for assays to determine patient-specific effects of existing drugs and new research compounds for individual CF patients.