The development of novel blood protein biomarkers to enable precision care in cystic fibrosis

Cystic fibrosis is the most common fatal genetic disease, affecting one out of every 3,600 children born in Canada. In 2013 alone, Canadians with cystic fibrosis spent about 25,000 days in hospital, mainly due to pulmonary exacerbations, which cause respiratory distress due to excessive mucus production, infection, and inflammation in the lungs. They are generally managed with two- to three-week courses of intravenous antibiotics and intensive chest physiotherapy. This is gruelling for patients and is costly for the health care system, at nearly $20,000 per episode.

A variety of therapies can prevent or treat pulmonary exacerbations, but to be effective, they need to be given at the right time. This research program aims to develop knowledge and tools that can improve the timing of prescribing and adjusting these therapies to make them more effective.

This program will examine hundreds of blood proteins concurrently, using well-characterized blood samples from cystic fibrosis patients. The proteins will be evaluated for their utility as biomarkers that can be used to predict pulmonary exacerbations and failure of a given course of treatment. Finding biomarkers like this will allow the development of simple blood tests to predict such events. Doctors could use these tests to personalize their courses of treatment to individual patients, thus reducing medical costs and improving patient outcomes.

The ultimate goal of this program is to enable more cost-effective, adaptive, personalized medical care for cystic fibrosis patients through mobilizing biomarker discovery research.