Okay, people in the CF Community, listen up!
A new targeted treatment for Cystic Fibrosis (CF) may be on the horizon sooner vs. later, and I’m pretty freaking excited about it! This could be a game-changer.
About three years ago, a clinical trial took place in the UK that involved gene therapy to treat patients with CF. Big deal, you say? Oh yeah. It was a big deal and I encourage you to read this article in the Daily Mail, superbly written by Diana Pilkington.
Here’s a quick overview of the article…
Meet 17-year-old Oli Dillon, who took part in the clinical trial. Oli has been living with cystic fibrosis (a rare genetic disease that attacks several organs, primarily the lungs) since he was a baby.
Like many sufferers of CF, Oli chooses to follow a strict regimen every day to help remove the buildup of mucus that forms in his lungs. If he doesn’t, his prognosis to try to stay relatively active would vastly diminish because CF is a progressive and life-threatening disease.
To try to control his symptoms, Oli takes multiple nebulizer treatments and does two sets of physiotherapy, in addition to taking nearly 50 pills—Every. Single. Day. The whole regimen takes hours. All this, while still going to school and pursuing his acting career.
Although he’s been hospitalized a few times for weeks on end, and he deals with stomach pain and digestive issues, he’s doing relatively well. Fortunately, he’s got great support from his mother and family, and when he qualified for the clinical trial, Oli jumped at the chance!
So the BIG TAKEAWAYS from the clinical trial are:
- CF patients were given healthy copies of a gene they lack – essentially, the gene was enclosed in a tiny bubble of fat that was administered through a nebulizer.
- The newly implanted gene’s mission is to prevent mucus from building up in the lungs.
- The gene therapy does not genetically modify the defective genes permanently, so patients must continue with the treatment, but researchers hope that the therapy will slow the progression of CF.
- Throughout the trial, patients given the treatment or the placebo did not experience bothersome side effects.
- Although the clinical trial didn’t meet its “end point” for the majority of patients, the lung function of some patients improved as much as 20 percent before the trial ended!
- While patients who received the placebo experienced a decline in lung function, patients who received the gene therapy did not experience a decline in lung function. Now THAT’s reason to celebrate!
- Scientists and researchers are continuing to study the results and hope to conduct a second clinical trial. (One never knows about the power of those “late responders” and outliers. Heck, some patients just need longer for their bodies to respond!) It is thought that more benefit may be achieved by giving patients higher doses of the gene therapy.
How did Oli do?
Although his lung function was only raised from 66.9 to 68.9 percent, he said he was coughing up more mucus (which is a good sign) right from the beginning and he noticed he still has more energy—even AFTER the trial!
So what are YOU going to do?
Have hope, people! Keep your eyes and ears open for the next clinical trial (we’ll keep you posted). Keep working with your doctors, continue taking your treatments, learn as much as you can, and be kind to yourself and others.
If you want to learn more about CF, visit the following websites:
- U.S. National Library of Medicine’s Genetics Home Reference
- Cystic Fibrosis Foundation
- National Organization for Rare Disorders (NORD)
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This month, PatientWorthy is teaming up with CysticLife.org to spread awareness of CF and support their collaboration with the Mayo Clinic for a community-driven research project investigating the benefits of exercise for CF patients. If you’re interested in contributing to the fund or want to become a CysticLife member, drop by CysticLife.org or donate here today! Don’t forget to share this post!