Cystic Fibrosis (CF)

CF is a genetic disorder that affects the respiratory, digestive and
reproductive systems. There are approximately 30,000 people in the United
States with CF, 3,000 in Canada and 30,000 in other areas of the world. In
the U.S., there are approximately 2,500 new cases diagnosed each year.

Currently, there is no cure for CF, but there are many promising new
treatments in use and even more on the horizon. The median life expectancy
for a person with CF is now 32 and only thirty years ago, a CF patient was
not expected to reach adulthood! Many people even live into their fifties and
sixties.

>From <A HREF="http://www.CysticFibrosis.com">www.CysticFibrosis.com</A>:

"In the Pipeline

There are many new and exciting treatments and medications currently being
developed for cystic fibrosis. In order to effectively treat the symptoms of
the disease, researchers are constantly trying to understand what causes the
problems associated with it. The discoveries that result enable scientists to
create new methods of treating the condition. Some of these treatments fight
the body's reaction to the symptoms of CF, some treat the disease at the
cellular level, while some attack the very gene that causes CF in the first
place.

Most cystic fibrosis patients are treated with inhalable aerosol solutions.
People with CF inhale medications daily to open airways in the lungs, to thin
their abnormally thick mucus and to fight the bacteria that cause their
recurring lung infections. However, although treating these problems makes
the patient live a longer and more comfortable life, none of these control
the inflammation that occurs in the lungs, which is what ultimately causes
respiratory failure. Inflammation occurs in the airways as the body tries to
battle the infections. It is caused by a number of factors in the body, but
one of the most powerful is interleukin-8, (IL-8), which is very hard to
control medically. The good news is that there is a new inhalation aerosol in
the preclinical stages of research that has been shown to reduce the levels
of IL-8 produced by the lungs, which will hopefully control the dangerous
levels of inflammation common for those with CF.

In late 1999, another breakthrough occurred when researchers associated with
the CF Foundation were studying mice who were bred with the genetic defect
that causes CF. Like humans with the condition, the mice exhibited an
imbalance of fatty acids. There was a high level of arachidonic acid, (AA),
but very low levels of docosahexaenoic acid (DHA). The researchers tried
treating the mice with massive doses of DHA and noticed that the symptoms of
the disease in the lungs, intestines and pancreas were reversed. The
scientists were very hopeful that the same results could be achieved in
humans, as the fatty acid imbalance is so similar. However, they warn against
people attempting to "self-treat" by obtaining DHA and taking very high
doses. This compound is extremely dangerous in large doses. Any treatment
that is eventually created will have to be based on a safe derivative of DHA
that is not yet close to being discovered.

All of these potential treatments so far treat the symptoms of the disease,
but none has yet addressed the cause. Cystic fibrosis is caused by a defect
in the Cystic Fibrosis Transmembrane Regulator (CFTR) protein. The purpose of
this protein in the body is to transport chloride ions across the cell
membranes. In a person with CF, this protein is unable to transport chloride
across the membranes of cells lining many organs, especially the lungs and
pancreas. Without the proper chloride levels in these cells, the proper water
levels cannot be maintained. When there is not enough water, fluids become
very thick. This is what causes the lungs to produce such thick mucus in a
person with CF, which leads to recurring and debilitating lung infections.
But again, there is good news. There are two protein-repair therapies in
clinical trials that would correct the problem with the CFTR protein. In
fact, there is even a gene therapy being developed which may be able to
deliver a healthy CFTR gene to a CF patient.

Although none of these therapies or medications will be available by the end
of the calendar year, many exciting new strides are being made all the time
to continually give hope to CF patients and their families."

Important References and Abstracts
Investigative Therapies