Mary's Page

38th Annual Tower Climb & Run

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Raised: $3,360.00

Goal: $4,000.00

My CF Climb Story

2019 was a monumental year in the Cystic Fibrosis community! In October the FDA approved Trikafta, a new breakthrough therapy. Trikafta is a combination of three drugs that target the defective CFTR protein. In normal cells, the CFTR protein acts as a channel that allows cells to release chloride. But in people with CF, this protein is defective and the cells do not release the chloride. The result is an improper salt balance in the cells and thick, sticky mucus in the lungs that leads to recurrent lung infections. CF patients often require hospitalization to receive powerful IV antibiotics to treat these infections. The only medicines available in the past were to treat the symptoms of the disease, while the holy grail of CF research has been to develop therapies that correct the underlying defect. Trikafta has proven to partially correct the defective protein and so far, appears to be having a dramatic effect on the lives of many CF patients.

For my son Andrew, 2019 marked another year of zero CF related hospital days and no IV antibiotics. He was already benefitting from Trikafta’s predecessor therapy, which was a double combination drug called Symdeko. He began taking Trikafta at Thanksgiving and we are cautiously optimistic that he will not have any adverse effects and that he will benefit even more than he did from Symdeko. Trikafta added a third compound to Symdeko, and the results from clinical trials ware impressive. For people with his mutations, pulmonary function increased by an average of 10 percentage points. Perhaps even more exciting was that the sweat chloride levels decreased on average from about 90 to about 45. (Under 30 indicates no CF, over 60 indicates CF, in between indicates CF is a possibility). This is a strong indication that Trikafta partially corrects the faulty protein in CF! Additionally, Trikafta is effective in 90% of the CF population, while Symdeko was only helpful to 50%.

We, along with so many in the CF community are beyond grateful for the donors, scientist and health care professionals that made this possible! There is still a lot of work to be done to continue to improve the lives and life expectancy of CF patients. 10% of CF patients get no benefit from these breakthrough therapies and for those that do, CF is still wreaking havoc on their organs, albeit hopefully more slowly. The Cystic Fibrosis Foundation is committed to leave no CF patient behind. Per CEO, Michael P. Boyle “We firmly believe that our most important and challenging work is still ahead. We see an unprecedented opportunity to identify and advance treatments for the underlying cause of CF and deliver a cure for every person with the disease, and will use every tool available to advance the research that will get us there.”

Thank you for reading my update and for your continued support!!





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