Drug could stop mucus production in cystic fibrosis sufferers
Somer Love's typical day is a lot different from most people's.
Yes, she runs errands, hangs out with her family and friends and plays with her two pugs, Oscar and Lily.
But her day also includes taking more than 50 drugs and supplements, and dedicating nearly three hours to treatments that keep down the level of mucus that can fill her lungs because of cystic fibrosis.
"Most girls wake up early to curl their hair. I wake up to take a bunch of drugs," said Love, 30, who often punctuates the end of her sentences with laughter.
A new drug trial underway at the University of Utah could significantly cut back on Love's daily medical routine and improve her health and longevity.
The drug, currently called VX-770, would not do what most cystic fibrosis drugs currently do: treat the symptoms of digestion problems and mucus build-up in lungs, which can lead to infections and severe lung damage.
Instead, the drug is designed to fix the problem on the cellular level, and prevent mucus from forming in the first place.
"It's going to shake things up," Love said. "It's going to change cystic fibrosis as we know it."
Researcher Theodore Liou, a specialist in pulmonary critical care at the University of Utah Hospital, takes a more cautious tone, noting past drugs have been successful but not as revolutionary as patients have hoped. Still, he's optimistic.
"We need better therapies not only to give people longer lives but better quality of life," Liou said. "As new therapies are developed, some of the old ones will fall away and people will be less burdened."
What is cystic fibrosis? » Cystic fibrosis is a genetic condition that causes cells to improperly manufacture a protein. The flawed protein results in the production of a thick, sticky mucus.
The mucus clogs and damages lungs and breaks down ducts in the pancreas, causing digestion problems.
It also coats the inside of the lungs, collecting bacteria that a healthy lung would expel. That leads to infection and triggers the immune system.
"Unfortunately, the immune cells' weapons [against the bacteria] are just as lethal against human cells," Liou said. "Most of the harm done to the lungs is actually collateral damage."
That means many people with cystic fibrosis progress from difficulty walking up stairs to using oxygen and a wheelchair.
There are different genetic mutations that cause cystic fibrosis. The trial for VX-770 focuses on a mutation that affects only about 2 percent of people. Researchers hope they can adapt the drug to work for a much wider population.
If the drug stops cells from producing mucus, it won't reverse any existing lung damage. But for those with minimal damage -- younger patients, or those with milder cases -- the drug could provide the closest thing to a cure.
Dreams becoming reality » The Cystic Fibrosis Foundation has provided millions of dollars in funding to keep research centers such as the one at the U. stocked with personnel and equipment such as centrifuges and freezers. It has invested more than $75 million to see VX-770 through the testing stages.
The foundation has played a major role in developing all four drugs currently on the market to treat cystic fibrosis, and the foundation's leaders anticipate submitting 13 new drug applications with the Food and Drug Administration by 2012. Four of those, including VX-770, are in the third and final stage of trials.
Robert Beall, CEO and president of the Cystic Fibrosis Foundation, believes VX-770 and similar drugs will add decades to patients' lives.
"This pill is causing great hope," he said. "The dreams that you and I have for our children can become a reality for CF patients."
If the drug proves effective and safe, the quality of life will improve for patients like Love, whose care regimen is essentially a part-time job. In a voice husky from damage to her lungs, she said she's used to the drug regimen that's kept her alive since she was diagnosed at 11 months old.
But she'd love to do less. And in any case, she said, such trials get researchers one step closer to a cure.
"Every single new drug trial has the potential of adding years to my life," Love said. "Right now, I'm just taking it one day at a time."
Cystic fibrosis affects about 30,000 Americans and about 70,000 people worldwide. A new drug, dubbed VX-770, is being tested at University Hospital in Salt Lake City in three trials:
A 48-week trial for patients age 12 years and older who carry a rare cystic fibrosis mutation, called G551D.
A 48-week trial for patients ages 6 to 11 years who carry the same rare mutation.
A 16-week trial to evaluate the drug's impact on patients age 12 years and older who have the most common mutation, called Delta F508.
To be considered for participation in the University Hospital trials, patients must know which genetic mutation they have. Some insurance plans pay for testing, which can cost up to $3,000, but many do not. Call 801-587-7458 for more information.
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