Are you up to date on recent developments in cystic fibrosis (CF) care? Thanks to advancements in medical science, the outlook for people with CF has improved a lot in recent decades. Scientists continue to develop new drugs and strategies to improve the lives of people with CF.
Let’s take a look at some of the latest developments.
Updated guidelines to improve personalized care
In 2017, experts from the Cystic Fibrosis Foundation released updated guidelines for diagnosing and classifying CF.
These guidelines might help doctors recommend more personalized approaches to treating CF.
Over the past decade, scientists have developed a better understanding of the genetic mutations that can cause CF. Researchers have also developed new drugs to treat people with certain types of genetic mutations. The new guidelines for diagnosing CF might help doctors determine who is most likely to benefit from certain treatments, based on their specific genes.
New drugs to treat the underlying cause of symptoms
CFTR modulators can benefit some people with CF, depending on their age and the specific types of genetic mutations they have. These drugs are designed to correct certain defects in CFTR proteins that cause symptoms of CF. While other types of medication can help relieve symptoms, CFTR modulators are the only kind of drug that is currently available to address the underlying cause.
Many different types of genetic mutations can cause defects in CFTR proteins. So far, CFTR modulators are only available to treat people with certain types of genetic mutations. As a result, some people with CF can’t currently benefit from treatment with CFTR modulators — but many others can.
To date, the U.S. Food and Drug Administration (FDA) has approved three CFTR modulator therapies for people in certain age groups with certain genetic mutations:
- ivacaftor (Kalydeco), approved in 2012
- lumacaftor/ivacaftor (Orkambi), approved in 2015
- tezacaftor/ivacaftor (Symdeko), approved in 2018
According to the Cystic Fibrosis Foundation, research is currently underway to assess the safety and efficacy of these drugs in other groups of people living with CF. Scientists are also working to develop other types of CFTR modulator therapies that might benefit more people with CF in the future.
To learn more about who might benefit from treatment with a CFTR modulator, speak with your doctor.
Ongoing research to develop new therapies
Researchers around the world are working hard to improve the lives of people with CF.
In addition to the treatment options that are currently available, scientists are currently trying to develop new types of:
- CFTR modulator therapies
- drugs to thin and loosen mucus
- drugs to reduce inflammation in the lungs
- drugs to fight bacterial and other microbial infections
- drugs that target mRNA molecules that code CFTR proteins
- genetic editing technologies for repairing mutations in the CFTR gene
Scientists are also testing the safety and efficacy of current treatment options for new groups of people with CF, including young children.
Some of these efforts are in the early stages, while others are farther along. The Cystic Fibrosis Foundation is a good place to find information about the latest research.
Progress is being made
CF can take a toll on the physical and mental health of people who have it, as well as those who care for them. Fortunately, ongoing advancements in CF research and care are making a difference.
According to the latest Patient Registry Annual Data Report from the Cystic Fibrosis Foundation, the life expectancy of people with CF continues to rise. The average lung function of people with CF has improved significantly over the past 20 years. Nutritional status has also improved, while the presence of harmful bacteria in the lungs has declined.
To achieve the best possible outcomes for your child and make the most of recent advancements in care, it’s critical to schedule regular checkups. Tell their care team about changes in their health and ask if you should make any changes to their treatment plan.
The takeaway
While more progress is needed, people with CF are living longer and healthier lives on average than ever before. Scientists continue to develop new therapies, including new CFTR modulators and other drugs to treat CF. To learn more about your child’s treatment options, speak to their doctor and other members of their care team.
