Well here is a big article on the latest CF treatment, but don't forget...it's a MICE deal, and SCLN is already in FastTrack FDA mode and Phase II trials:
Cystic Fibrosis Remedy Shows Promise in Tests on Mice
By THOMAS H. MAUGH II, Times Medical Writer
Boston researchers, in experiments with mice, have
developed what they believe could be the first effective
therapy for cystic fibrosis, a devastating genetic disease
marked by life-threatening accumulations of mucus in the
lungs and pancreas.
Human trials of the remedy, which involves administration
of high levels of a purified form of a common food supplement,
could begin early next year, they said Saturday at a Cystic
Fibrosis Foundation conference in Seattle.
Using mice with the same genetic defect found in humans
with the disease, researchers from Beth Israel Deaconess
Medical Center found that cell membranes in the organs most
affected by cystic fibrosis have a deficiency of a fatty acid
called docosahexaenoic acid, or DHA.
Giving the animals large quantities of DHA dramatically
eased disease symptoms, they reported. Because the mouse is
considered such a good model for the disease, the results
should hold in humans as well, the researchers said.
"This exciting research represents an entirely new strategy
to correct and possibly even prevent some of the ravages of
this disease," said Robert J. Beall, president and CEO of the
Cystic Fibrosis Foundation.
Although DHA is present in fish oils and nutritional
supplements, the researchers warned strongly against patients
taking such supplements on their own because other
components could be harmful.
"If we were to leave patients with any message, it is 'Don't
do it,' " Beall said. Cystic fibrosis victims who take high doses
of the supplements "could do themselves irreparable harm"
because of impurities in the commercial products, he added.
"Give us the time to do clinical trials properly."
Genzyme General of Cambridge, Mass., is now developing a
suitable form of DHA for human testing that would eliminate
such problems and is working as fast as possible to make it
available, said Dr. David Meeker, Genzyme's vice president for
medical affairs. DHA is isolated from cold-water fish.
Cystic fibrosis, commonly called CF, is a genetic disease
affecting about 30,000 children and adults in the United States,
making it the most common fatal genetic disorder. About 1,000
new cases are diagnosed each year, most in early childhood.
CF causes cells lining the lungs and the pancreas to secrete
an abnormally thick, sticky mucus. In the pancreas, the mucus
blocks bile ducts used in secreting enzymes that help the body
digest food. The mucus also clogs the lungs, impairing
breathing and leading to a high incidence of life-threatening
infections. About half of CF patients die before the age of 32.
The gene that causes CF was identified 10 years ago, and
that discovery has led to many new approaches to treatment.
Several gene therapy trials are now underway, for example, but
none so far shows signs of great effectiveness.
At least 11 new treatments for CF are now in clinical trials.
But if the new results reported in mice can be replicated in
humans, DHA promises to be the most effective of all.
One particular advantage is that DHA would be taken orally
to treat the whole body, while many of the other new
treatments are directed only at the lungs or the pancreas, said
Mary Lou McDowell, co-chairwoman of the National Cystic
Fibrosis Awareness Committee.
Dr. Juan Alvarez and Dr. Steven Freedman of Beth Israel, a
Harvard University teaching hospital, are specialists in
studying the metabolism of fatty acids, the long molecules
from which cellular membranes are constructed. Although
other scientists have examined fatty acids in the bloodstream
and found nothing unusual, Alvarez and Freedman decided to
look at the tissues most affected by CF--the lungs, the
pancreas and the intestine.
Using mice with the same genetic defect as humans, they
found that those tissues had an unusually high level of a fatty
acid called arachadonic acid, or AA, and unusually low levels
of DHA. DHA, in particular, plays a key role in the functioning
of cellular membranes, regulating the passage of materials into
and out of the cell. Because the two fatty acids are linked in
the cell, low levels of DHA lead to high levels of AA.
To see if this fatty acid imbalance was related to the
disease, they fed a group of CF mice high levels of DHA for a
week. After seven days, the AA/DHA ratio in the tissues had
returned to normal and many of the symptoms were brought
under control, Freedman said.
Bile ducts in the pancreas, for example, were no longer
clogged or blocked. The intestines absorbed food better. And
the lungs were no more susceptible to pneumonia than those
of healthy mice. The researchers observed no side effects from
the treatment, he added, and healthy mice given large doses of
DHA showed no changes.
Working with researchers from the University of
Massachusetts Medical Center, Alvarez and Freedman then
studied biopsy tissues from the nose and colon of six humans
with CF and six healthy controls. They found the same fatty
acid imbalance in the human patients, but not in the controls.
said. That finding strongly suggests that the results in mice
can be translated into humans.
DHA is a constituent of fish oil that is sold commercially as
an aid for cholesterol control, as well as of some other food
supplements. But Freedman cautioned against patients taking
such products in high doses.
"Not only can we make animals much better with pure DHA,
but we can also make them much sicker by giving them other
fatty acids," such as those found in the commercial products,
he said. And eicosapentanoic acid, the primary constituent of
fish oil, not only blocks the activity of DHA, but can suppress
the growth of children who consume large quantities, he
noted. The drug being developed by Genzyme would be pure DHA.
Information for patients, including a summary of the
presentation and a Q&A, can be found at the foundation's
Web site, cff.org.
Once should always remember that should Genzyme zoom tomorrow on the news it always represents a great time to short any stock on MICE news..as soon as people find out how long it will take to get to market they sell.
chris
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