“New England Journal of Medicine found the drug (Kalydeco) improved lung function and helped reduce the likelihood of a pulmonary exacerbation – temporary but serious lung function problems like coughing and shortness of breath – in CF suffers by 55 percent, HealthPop reported.”
For people who are candidates for this medication, it has changed CF forever. Patients have been able to slow down and even stop some of their medications because the results they are getting from this drug have been so significant. For young children who start this drug early in life, they may never see any damage to their lungs from Cystic Fibrosis. That is amazing. It is wonderful to read stories and posts from people whose children have been helped by this new medication.
But… it is also painful. The reason? Kalydeco is only effective in 4% of the CF population. It is only effect for people who have a specific genetic mutation (G551D). My daughter does NOT have this mutation. Therefore, this medication is not her ‘cure’. This makes it difficult to read all the news about Kalydeco. I am genuinely happy for those that it helps. But there are times when I have to shield myself from reading about those on this medication. There are times when it is just too much for me knowing that we could be so close to such a life changing medication and then also so far away.
The hope in the medical community is that some day there will be an additional medication that will be given in conjunction with Kalydeco to help people with other mutations. There are trials being conducted right now on a combination for people with 2 copies of Delta F508. If all goes well, it is being projected that those people could have the combination in a few years. Emma does NOT have 2 copies of Delta F508, so again… it will not be her cure.
There are projections that Emma group, people with 1 copy of Delta F508 (and another mutation) may see a drug come to market in 5 years… that could be close to a cure. But again… it is all speculation. There are so many things that need to go right for that to happen. There are so many opportunities where something could go wrong.
So we keep fighting. We keep hoping. Most days we read the stories of people on Kalydeco and we are happy for them. Some days the waiting is too painful and we can only hope that a cure comes in time for Emma. The reality is… for our family, nothing else matters.