Recent Breakthrough Increases Efficacy of Cystic Fibrosis Drugs

For cystic fibrosis patients, treatments can be tough. This is why research for more effective therapies are crucial, and luckily, researchers have recently made a breakthrough that increases the efficacy of preexisting drugs, aiding those who were not seeing their desired results.

Quorum-sensing inhibitors

The researchers of this study were all from the University of Montreal Hospital Research Centre (CRCHUM), and were inspired to look into how treatments can be enhanced for those who are not seeing results.

Emmanuelle Brochiero is one of the leaders of the study, as well as a professor at Université de Montréal. He explains the group's work: "Adding molecules called quorum-sensing inhibitors to current drugs not only reduces bacterial production of certain harmful residues but also restores the efficacy of existing treatments on the cells of cystic fibrosis patients." These treatments include Orkambi and Kalydeco, which have been life-changing for some, while others saw limited or essentially no benefit. Instead of starting over and trying to create a new drug, they were interested in how they might increase the efficacy of these preexisting drugs, and bring those who haven't experienced benefits to the heightened level that others have experienced with the addition of supplements.

Their findings have been published in Frontiers of Cellular and Infection Microbiology.

Difficulty in research

While the scientists at University of Montreal Hospital Research Center have proven to be very successful, there is a reason that cystic fibrosis data is not necessarily abundant at the moment. The disease affects people very specifically, and the response of one person is very unlikely to closely simulate that of another. The result is that when researchers attempt to produce a study, overgeneralizations are prevalent, and finding the root cause of something for one patient will not be the same as for another. This is likely because there are over 2,000 potential gene mutations, each of which manifests itself in different ways, and offer different responses to varying drugs.

Researchers have previously attempted to find drugs that would be successful for each of the thousands of gene mutations, but are now beginning to realize that simply isn't feasible. Researchers like Brochiero are recognizing that a new approach is integral to make meaningful progress in the field.

Work in progress

Throughout the last ten years, Orkambi and Kalydeco have become available. For some, the drugs were extremely beneficial, for others they helped a bit, and many saw no visible change. This can become very depressing for those with cystic fibrosis after a while, as participating in clinical trials or beginning new treatment regimens offers hopes of significant improvements, but often they simply are not experienced as desired.

That is why the scientists looked into what was causing the variations in results. Normally, Kalydeco is taken by cystic fibrosis patients who have rare mutations , which are usually more than four percent of patients. Orkambi is taken by those who have more frequent mutations, at around eighty percent. While they were both breakthroughs at their time of approval, the limited efficacy for some has sparked a curiosity of the varying needs patients have.

Read on to learn more about this study and what its result mean for the future of CF treatment.