4-Year Study On Cystic Fibrosis Patients Shows Efficacy

The combination of modulator therapies tezacaftor (TEZ) and ivacaftor (IVA) for cystic fibrosis (CF) has shown long-term safety as well as clinical benefit in a trial that lasted four years.

Both the CF modulators were assessed across the age group of 12 years and older. The participants of the study tolerated the combination regimen over a period of 96 weeks. These patients, apparently, had already participated in a 120-day trial when it came to the same combination.

As far as the recent study was concerned, the enhancements achieved throughout the earlier trial were maintained. Hence, the recent study shows tezacaftor, along with ivacaftor, could work in the long term.

The protein called cystic fibrosis transmembrane regulator, or CFTR, takes control of the movement of water when it comes to the lung tissues. This goes on to allow a thin and free-flowing production of the mucus.

Apparently, IVA happened to be the first small molecule that targeted the CFTR protein directly. It attaches itself to the CFTR protein at the surface of the cell, thereby holding the gates of the channel open and also keeping the channel of the protein open for a longer time so as to allow the water movement as well as decrease the mucus’ thickness. That said, this drug happens to be appropriate only for those people who have a mutation that goes on to produce proteins at the surface of the cell, which does not take place in patients with F508del.

Referred to as a corrector, TEZ binds itself to the CFTR protein and enables the protein to maintain its shape so that it can operate better. This is significant for those with F508del, as it can very well elevate the quantity of the protein present at the cell’s surface, where it can be acted upon by the IVA.

The IVA approval in 2012 made a significant breakthrough in treating the disease. It gave out a new class of CF drugs named modulators that addressed underlying problems in CF rather than just treating the sequelae of CF. When the patients were treated with a mix of CF modulators targeting the function as well as the structure of the protein, the effects were even greater.

While the clinical trial was still being conducted, a three-part section that comprised of TEZ, IVA, as well as elexacaftor was approved in 2019. It has since become the care standard.

As per the Medical University of South Carolina’s Dr. Patrick Flume, with TEZ and IVA, one can see a surge of around 2–3 percent in lung function in patients with CF. On the other hand, the triple therapy will go on to see more than a 15% improvement in the function of the lungs.

In conclusion, the findings also show that the mixed modulator therapy is well tolerated and safe in the long term, showing that two of the three drugs in the standard therapy can be used in a safe manner over time.