Cystic Fibrosis is a common and fatal genetic disorder in Caucasians. In the US alone, over 30,000 children and young adults are affected. Most of them will die prematurely from lung damage caused by the build-up of thick mucus, which becomes the host of chronic infections. In the last few decades scientific advances have been made, increasing median life expectancy from 10 years in 1968 to almost 30 years two decades later.
The use of compounds like triButyrate® was made possible as a result of the discovery of a gene mutation, responsible for 90% of all CF cases, in 1989. triButyrate® specifically appears to repair the molecular disorder, created by the CF gene mutation.
Since 1998, clinical studies, using triButyrate® as the active ingredient, have been ongoing, to further the development of a safe and effective cure for Cystic Fibrosis. According to Scientists, triButyrate® represents one of the most remarkable potentials yet in Cystic Fibrosis.
triButyrate® works as a chaperone for a protein called "The Cystic Fibrosis Transmembrane Conductance Regulator"(CFTR). The gene should ideally "write" instructions for the cell to produce the CFTR proteins. The protein itself is there to create a "channel" in the cell membrane to allow chloride ions to "exit" the cell. These channels through the cell membrane are crucial for the cell to function properly, through which chloride, sodium, calcium, as well as other substances move in and out of the cell, maintaining a proper balance.
Specifically in the lungs, this balance is crucial to maintain a mucus of proper composition, to be able to carry away congestion. Ideally the wet and thin mucus is easily removed by the cilia on the outside of the cells. On the other hand, without the proper transport in and out of the cell, the mucus becomes thick and sticky, and accumulates. As a result of this imbalance, bacteria invade the mucus, promoting infections and interfering with breathing.
Because of the gene mutation, the protein is not assembled correctly, and is unable to do its proper function. The cell rejects the disabled CFTR, and as a result not enough channels are created, for chloride transportation out of the cell. Sodium, on the other hand, flows into the cell through its own channels, accumulates and upsets the balance in the cells.
triButyrate® seems to attack the cause of the disease, rather than just treat the symptoms, correcting the defective chloride channel.
All rights to the product and trademark triButyrate® are owned by
Fyrlklövern Scandinavia AB.