Cystic Fibrosis Studies
Since 1998 clinical studies have used Sodium Phenylbutyrate to further
the development of a safe and effective treatment for Cystic Fibrosis.
Sodium Phenylbutyrate appears to repair the molecular disorder created
by the CF gene mutation. According to Scientists, Sodium Phenylbutyrate
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 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
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 Phenylbutyrate, on the other
hand, flows into the cell through its own channels, accumulates and upsets
the balance in the cells.
Copies of clinical trials are available upon
request for doctors and medical researchers.