Researchers optimistic about new cystic fibrosis treatment

[Guest guest]

August 05, 2003, Tuesday

HEADLINE: Researchers optimistic about new cystic fibrosis treatment

SOURCE: Scripps-McClatchy Western Service

BYLINE: SUE VORENBERG

Life for cystic fibrosis patients is punctuated by pain,

breathlessness and

continuous hospital visits, and it usually ends at a very young age.

A new study at the University of New Mexico's College of Pharmacy

seeks to

change that - giving patients new hope for much longer, almost normal,

pain-free

lives.

The study will investigate how a common bacterium called pseudomonas

aeruginosa plays a major role in the disease and what might be done to

destroy

it, said Graham Timmins, an assistant professor at the college.

" Cystic fibrosis is severely aggravated by a bacteria that's present

everywhere in the environment; it's ubiquitous, " Timmins said. " Because

of a

genetic defect in CF patients, they are susceptible to being colonized by

this

bacteria while others are not. If you could prevent the lung inflammation

that

the bacteria causes at the start in young kids, perhaps you could give

them a

normal lifespan. That's speculation, of course, but it's not

unrealistic. "

About 65 children and 50 adults across New Mexico have cystic

fibrosis. It is

a rare genetic disease that damages the body's mucus production

facilities.

That, in turn, hurts organs' ability to move fluids. Cystic fibrosis can

cause

digestion and pancreas problems, but its biggest impact is on the lungs,

Timmins

said.

" The disease lets the bacteria get hold, and that causes infections,

which

cause scarring, and the cycle keeps repeating, " Timmins said. " By the

teenage

years, a lot of these people are permanently colonized. The lungs get

very slimy

and difficult to breathe through or treat. The complications take people

on a

long, slow downward phase. "

Treatments have gotten better than the past, but patients have to

endure

continual chest poundings and lung suctioning - sometimes two to three

hours

daily - so they can breathe, said Perkett, director of the

Cystic

Fibrosis Center at UNM Hospital.

" At this point, there is no specific treatment for CF, " Perkett said.

" It's

always fatal, usually from lung failure. Supportive care has, at least,

improved

the lifespan. Twenty years ago patients lived to about age 7. Now they

live to

about age 32.

" These patients still have very difficult lives. It's hard for them to

do

normal activities because they have to adapt to horrible hospital

schedules. The

patients and their families are just incredible. They put up with so

much. "

Researchers think the bacteria catch hold so easily because patients

don't

produce enough nitric oxide in their lungs. The gas, which is naturally

produced

in the lungs, kills the bacteria in healthy people, preventing them from

colonizing or causing problems.

" We all get exposed to bacteria, but our lungs have ways of clearing

them, "

Perkett said. " With CF, the lungs can't clear them. It's like they get

stuck in

glue. If we could learn more about how to get that bacteria out, then the

lung

wouldn't be damaged or scarred. People would need much less therapy. "

In July, Timmins and a group of UNM researchers got an $80,000 grant

from the

National Cystic Fibrosis Foundation to study exactly how much nitric

oxide is

produced in the lungs of CF patients and to learn how much it takes to

effectively kill the bacteria.

" There have been some clinical trials nationally where people have

used

nitric oxide and nitric oxide-like drugs on CF patients, but they haven't

been

enormously successful, " Timmins said. " We think that by understanding the

bug

and how it works, we can find a way to stop it from defending itself, so

we can

find new ways to kill it. "

In the next two years, Timmins and others will use specialized imaging

equipment to measure nitric oxide in patients' lungs. Other studies have

looked

at only amounts in patients' breath, but that amount likely changes as

some of

it is absorbed by the bacteria, he said.

" We want to know the exact levels before the nitric oxide hits the

bacteria, "

Timmins said. " On the other side we want to see how the bacteria fights

against

that. With luck, that information will tell us the proper way to stop the

problem before it takes hold. Perhaps one day these people will be able

to live

normal, healthy lives. "

(Contact Sue Vorenberg of The Tribune in Albuquerque, N.M., at

http://www.abqtrib.com.)