Researchers work around the clock and the world to find new discoveries in terms of cystic fibrosis treatment. Thanks to foundations, donations, and ongoing research, cystic fibrosis has made leaps and bounds as far as treatment is concerned. There is one pharmaceutical company at the forefront of these leaps. They have been testing a triple therapy that has shown groundbreaking improvement of lung function in cystic fibrosis patients. The therapy is in its early stages, but the news brings hope to those affected.
Need for Innovation
Even though there are medications to treat cystic fibrosis, there is no cure for the condition and it is life-threatening, not to mention life-altering. The current medications for cystic fibrosis only work with certain mutations of the disease. Other medications work by treating the symptoms that the condition causes. For example, inhalers temporarily open airways enough to improve breathing slightly. The good news for these patients is there are companies that are working solely on the improvement of lung function. Vertex Pharmaceuticals is one of them. They have been working on a triple combination medication that is already showing positive results. It is extremely exciting news for science and cystic fibrosis.
Who Is Vertex?
Vertex Pharmaceuticals is a household name for patients with cystic fibrosis. The company has produced life-changing effects for these patients. They are dedicated to producing medications that help people with a life-threatening disease. Vertex was founded in 1989 and now has locations globally. Most of their 2000-plus staff is focused on research and development. Like so many, they started off very small and quickly grew to impressive heights. With offices in London, Australia, Canada, and their home, Boston, MA, they have great minds together all over the world. They are front-runners of medical advances, especially in cystic fibrosis.
The cystic fibrosis program at Vertex is referred to as the “All In For CF”, and they mean it! They explain the program’s “singular passion” to “bring new medicines to all people living with this rare, life-shortening disease”. From the year they were founded to the present, they have done just that. They haven’t stopped researching from the time the doors opened. They continuously studied the gene responsible for cystic fibrosis, the CFTR gene. They began collaborations with the Cystic Fibrosis Foundation in 2000. A year later, they opened a facility in California to focus on the discovery of medications for cystic fibrosis. The efforts continued and Vertex is now responsible for producing two of the most effective medications for treatment of the disease. They have held the biggest clinical trials in cystic fibrosis history. They are partnered with several other companies to include research on sodium channels and gene editing. It is a company that never stops. They come up with one medication and start studying the next. Companies like this are a great hope for a cure.
Ivacaftor, also known by its trade name Kalydeco, was the first drug of its kind. It was approved by the FDA in 2012 and was called a wonder drug. The drug works by improving the function of proteins to help prevent the buildup of mucus. It is the first drug that was able to treat the root cause of the condition and not just the symptoms. In 2015, Vertex had another important medication approved by the FDA, Orkambi. It is a combination medication for people with certain forms of cystic fibrosis. It works by bringing more proteins to the surface and making the ones at the surface more effective. This allows for a better balance of salt and water in the lungs. This is a big deal for cystic fibrosis patients.
The Triple Combo
The latest venture for super company Vertex is the triple therapy combination. The scientists have been working on combining medications to further improve lung function. This therapy would work for patients with one certain mutation and one minimal function mutation. The pill is a combination of the medications that exist, as well as another one of Vertex’s prodigy's Tezacaftor. Tezacaftor was formulated while trying to widen the range of cystic fibrosis mutations that the current medications can help. The studies have shown that when the three drugs are put together, not only does it work on more mutations, but it also improves lung function remarkably.
Lung function was reported at improvements of 9 percent to 12 percent. These preliminary results are getting everyone excited to see what happens next. Vertex plans to start more trials to test which combination is the most effective way to go. The CEO of Vertex, Jeffrey Leiden, told the Boston Globe of his excitement. He explains it as a rare moment. “This is the most significant advance to date in our ability to fundamentally change the course of the disease for up to 90 percent of all patients.” He goes on to say the most powerful statement that embodies the company’s values and mission: “We’re not stopping till we get something for everyone!”. With Phase I and Phase II trials showing success, the group plans to head into Phase III. It should begin the first half of next year. Trials can go on for up to 12 months, but if they find the right combination, it is going to be successful.
Vertex in the Community
Beyond the science, Vertex is a company very much about the community. They have created multiple programs to help patients and families with cystic fibrosis.
“Team Impact CF Project” is a program the company started that puts kids on an athletic team from their hometown college. These kids become signed members of these teams from draft to graduation day. The kid's team interacts with these athletes who are important role models. The two form bonds and sometimes a friendship that lasts beyond. These team interactions can last two or more years, with value lasting a lifetime. They also have an “All In For CF” scholarship program that helps kids and their families attend college. They hold fundraisers all over the world in the form of walks, bike rides, and marathons. They have awards for innovative scientists and young people who have an interest in cystic fibrosis treatment. These awards are hundreds of thousands of dollars. Another worth mentioning is the Circle of Care Program. Vertex provides grants that fund the start of community programs. These resources help patients with cystic fibrosis with social challenges, education on self-care, and support. The company website is pretty inspiring, to say the least. Vertex is far from a typical pharmaceutical company. There’s even a page where employees talk about why they are “all in for CF”, some close to the disease themselves.
The fact that a medication could be in place in two years that can treat 90 percent of the cystic fibrosis population is nothing short of amazing. People often think of pharmaceutical companies as pill pushers with a product that’s unnecessary, but companies like Vertex show a little more humanity than greed. The fact that they donate so much money to giving back to communities is admirable. They are not only a drug company. They are a foundation of people who live to help others live.