Cystic Fibrosis…
For a mother, those words are disheartening, confusing, and so, so frightening.
For a child, those words mean countless daily medications, extensive hospital visits, and a shortened life of feeling like you are breathing through a straw with your nose plugged.
This life is all too familiar to families afflicted by cystic fibrosis, like Emma Hughes and her son, Chester.
Their days are dominated by managing the symptoms of CF, a debilitating condition in which patients have much thicker and stickier mucus than normal, leading to breathing problems, frequent infections, respiratory failure, and irreversible lung damage.
The PatientWorthy team has been following Chester’s struggle, the Orkambi fight (The Irish Fight, Community Fight, and Celebrities’ Support) and has partnered with BreatheWithMe Strawfie Challenge, to help advocate and support them.
Since Chester was born in May 2015, he has been admitted to the hospital numerous times, for weeks on end for IV antibiotics, and he is currently in pseudomonas eradication treatment.
Though this is the reality of Chester’s cystic fibrosis diagnosis, Vertex, a revolutionary pharmaceutical company saw a need for a more comprehensive and effective treatment option for patients like Chester, so the company developed, tested, and manufactured Orkambi (ivacaftor/lumacaftor).
Orkambi is a second precision medicine that works on the root cause of cystic fibrosis by treating those with two copies of the DF508 mutation, rather than just treating the severity of the symptoms of the disease. Orkambi could be a life-altering and frankly, a potentially life-saving option for patients with this mutation.
Emma has seen Orkambi being used in other countries for those with just one copy of the DF508 gene. She is hopeful that Chester, who has one DF508 gene, could also benefit from Orkambi treatment. The story would end there, happily, if Chester lived in the United States, or any other country that offers this treatment, but he doesn’t. Chester lives in the United Kingdom, where Orkambi was made unavailable on the NHS healthcare system on cost grounds in June of 2016, even though it was licensed and recognized as effective since November of 2015.
For the past year, Emma, other CF families, The Strawfie Challenge and The Cystic Fibrosis Trust in the U.K. have been breathing strongly and fighting tirelessly to bring Orkambi to the people that need it the most, people like Chester.
Today is an accumulation for this past year’s frustration, and protests are being held across London, Wales, Scotland, and Northern Ireland against the NHS’s Orkambi decision.
Yet, these protests and the larger problem are not only confined to the borders of the U.K., but they also call on the entire worldwide rare disease community. The people with cystic fibrosis in the U.K. represent only 0.02% of the entire population, so they need others to empathize and rally support behind them.
Emma and Chester in particular are asking for your help during this day of protests. The internet can connect us all, even across the pond, and it can also garner support in a more extensive way than many other platforms. Remember the ALS ice bucket challenge?
Now it’s cystic fibrosis’s turn in the limelight.
Emma started a blog and is on board with the #OrkambiNow social media campaign. She and the rest of the UK CF community, are asking you to tag the cause and take a selfie holding a sign that says #OrkambiNow today, June 26th to post on your social media pages.
The Cystic Fibrosis Trust has also provided a downloadable speech bubble that people can use to write in why they support Orkambi and share online. Click here to access this resource!
Emma told us:
“People with CF fight everyday just to survive, so it’s only right we fight for them. They didn’t choose this battle, but they are doing it with sheer determination. As the mother of a child with CF, who at one point didn’t know if he would make it, I am passionate about this fight. the negotiations between the NHS and Vertex need to start in order to give a chance to those who need Orkambi now. Too many parents are burying their children when there are precision medicines out there that can add to the life expectancy and quality of life. It’s just wrong.”
Help us help Emma and Chester, and the rest of the UK CF community, prove that where you live shouldn’t dictate the quality of your treatment options. This issue affects us all, whether you’re a neighbor, friend, parent, or child affected by cystic fibrosis or any other rare disease.
Click here to read more about cystic fibrosis from our other CF partner, Cystic Life.
