An update on Amy for all those who were wondering about her declining health. There is some bright news in our lives today.
Amy’s lung function is currently under 30% the predicted value. That’s about the point when I was listed for a transplant. However, she is mostly stable so unless she has a sudden crash, she isn’t eligible for the lung transplant program. She feels healthy in the sense that she has no acute infection but her lungs are terrible from years of scarring and any cold, flu, or virus (!) could kill her.
And she’s starting Trikafta today! Trikafta is a triple combination drug that is for those with the most common CF genetic mutation. It’s a breakthrough that was on a lot of Top 10 most exciting scientific discoveries lists at the end of last year. Those in CF community have been promised life changing breakthroughs before but these new meds actually seem to be delivering on the promise. They're taken for life but the hope is that it'll make CF a manageable disease that lets people live until old age.
The drug is not approved in Canada. Vertex, the pharmaceutical company, hasn’t submitted it for governmental review yet. Advocates in the CF community and within CF Canada are working hard on reaching an agreement between Vertex and Health Canada to have this orphan drug approved but it’s moving slow and people with CF are dying in the meantime.
Everyone in government is currently busy trying to prevent the general population from flooding the hospitals with COVID19 cases – but when Trikafta does get approved and as long as it’s affordable, it will most benefit those who still have their relatively healthy lungs. It’ll increase quality of life, reduce hospital stays. Provide people with CF the opportunity to plan for the future instead of wondering if they’ll live past 35. Trikafta has been approved in the UK and the US and those who have access to the drug before their lungs are "critical"are having lung function increases of 20-30%. It’s working wonders. To use the word infuriating that a medication exists that can dramatically change peoples lives but it’s just out of reach is putting it mildly.
Back to the positive, Amy is getting it! She has it. She has it in her hands.
She was approved through Vertex’s compassion care program which is providing Trikafta for Canadians with CF who have critically low lung functions. This does mean though, that the 20-30% increase in lung function that other people are seeing will probably not happen with her.
The doctor’s best guess was a 5% increase due to all the previous damage to her lung tissue. But 5% is still 5%. And if it stabilizes her lungs, that would be crucial to her not crashing out. We don’t know yet how well it’ll work. Today is Day 1 and we’re all hopeful and celebrating that Amy has the chance to try it.
