Earlier this month, Vertex Pharmaceuticals released big news regarding a new development in cystic fibrosis treatment.
The pharmaceutical company announced that their new, innovative technique in treating the disease had demonstrated substantial lung improvement in patients, and the company believes this treatment could potentially benefit 90 percent of total cystic fibrosis patients!
Cystic fibrosis, or CF, is a rare disorder that causes thick mucus to build up in the lungs, leading to not only severe breathing problems, but also progressive respiratory and digestive system damage. To learn more about CF, click here.
For the nearly quarter of a million cystic fibrosis patients in the world who do not respond to any current medications, this could be their miracle treatment.
After this announcement, Vertex Pharmaceutical’s shares skyrocketed by roughly 25 percent in after-market trading on Tuesday, reflecting others’ enthusiasm and optimism in the company.
In just a few earlier studies, Vertex Pharmaceuticals tested their “triple combo treatment” in roughly 100 cystic fibrosis patients. These patients were also among those with the most serious and resistant mutations.
The results from these studies exceeded what researchers had hoped. By gauging how much air a patient could exhale, they were able to verify substantial and much better improvement than originally expected.
Dr. Steve Rowe, director of the cystic fibrosis research program, assisted in running one of these Vertex Pharmaceutical trials. When he saw the results, he was also pleasantly shocked at the magnitude of improvement in patients, saying that the trial was an exemplar of “precision medicine” working.
This breakthrough, that uses three drugs in combination rather than two, to treat cystic fibrosis came from a Vertex lab three to four years ago. Luckily, results from early clinical research confirmed lab evidence which suggested this, in that three compounds could bind to misfolded proteins and fix them. This would consequently allow these cells to return to normal function, fixing the underlying cause of CF.
Chief executive, Jeffrey Leidin, was very pleased with their results, saying
“Everything we saw in the lab played out in the clinic,” he said, “[which is] one of those very rare moments in biotech.”
He is so encouraged by the results that he claims Vertex’s work could potentially change the course of cystic fibrosis in a fundamental way.
At the current moment, Vertex Pharmaceuticals has two approved cystic fibrosis drugs on the market, called Kalydeco and Orkambi. However, less than 40 percent of the total number of cystic fibrosis patients are eligible to take these treatments, roughly only 30,000 patients in total, so Vertex is looking forward to using this new three drug combination treatment to cover the many, many patients that need it.
However, it is still unrealistic to think that this is the end all be all miracle drug for cystic fibrosis. Still, about 5 to 10 percent of the world’s cystic fibrosis patients will not be able to form enough of the necessary protein to be able to receive this triple-combo treatment.
But don’t worry, because Vertex is working on those select few also.
In Leidin’s concluding remarks, he shares that the company will not stop seeking out cystic fibrosis treatment until there is something for everyone.
To read more about Vertex Pharmaceutical’s influential new project, click this.
What are your thoughts about ongoing research for rare diseases? Share your thoughts, and your hopes, with the Patient Worthy community!
To learn more about CF, check out our partners at www.cysticlife.org and the Strawfie Challenge.
