According to a story from globalnews.ca, Andre and Joshua Laroque are nine and seven years old respectively. The brothers, being close in age, spend a lot of time together and share a lot of similar hobbies. They also share the same rare disease diagnosis: cystic fibrosis. However, currently only Andre has access to a disease-modifying treatment because he was able to participate in a drug trial. Now, parents Sasha Haughian and Jamie want Joshua to get access to the same treatment too.
About Cystic Fibrosis
Cystic fibrosis is a type of genetic disorder which can have impacts throughout the body, but it is most characterized by the build up of abnormally thick, sticky mucus in the lungs. This mucus becomes a fertile breeding ground and habitat for potentially infectious bacteria. Many patients must take antibiotics for much of their lives. This disorder is caused by mutations of the CFTR gene. Symptoms of cystic fibrosis include progressive decline in lung function, lung and sinus infections, coughing up mucus, fatty stool, poor growth, infertility in males, clubbed digits, and digestive problems. Treatment includes antibiotics and medications or procedures intended to maintain lung function. Lung transplant is an option when lung function declines severely. Life expectancy ranges into the 40s and 50s with good care but recently approved treatments have the potential to improve outcomes. To learn more about cystic fibrosis, click here.
Andre and Joshua’s Story
When Andre started the trial, Joshua fell below the specified age range. The drug that has been helping Andre is called Symdeko. Meanwhile, Joshua continues to see declines. A related drug called Orkambi is available in Canada, but only under certain circumstances, and the extreme costs makes it impossible for the family to purchase out of pocket. Orkambi is only permitted for use in patients between age six and seventeen; they must have also seen at least a 20 percent decline in function in a six month period while using their current therapies.
These limitations are based on decisions from the Canadian Agency for Drugs and Technologies in Health which determined that Orkambi’s impact was not good enough for broader coverage. Still, the constraints serve to limit the usefulness of the drug even further and don’t account for the potential impacts of early treatment.
While there is also a new treatment recently approved in the US that promises to help the vast majority of cystic fibrosis patients in a big way, it is unclear how long it will take for approval in Canada. Since the disorder is progressive in nature and ultimately lethal, every moment spent without treatment puts Joshua’s life at greater and greater risk.
Until regulations change, he’s stuck.