Omaha World - Herald (Mar 25, 06:42 PM)  Cystic fibrosis sufferers deal with thick, sticky mucus that clogs the lungs and leads to chronic, life-threatening lung infections.

But a study using gene therapy is providing hope for some afflicted with the disease.

The University of Nebraska Medical Center and its hospital partner, the Nebraska Medical Center, are one of 12 sites nationwide participating in the study.

Treatment in the study involves administering a combination of a virus called AAV and a normal copy of the CFTR gene, said Dr. John Colombo, professor of pediatric pulmonology and director of the Nebraska Cystic Fibrosis Center at UNMC.

The gene makes a protein that keeps proper water balance in the bronchial tubes and in linings of other internal organs so they remain wet and clean.

Cystic fibrosis is caused by a defective gene that gives the wrong instructions, resulting in cells that can't make the protein work properly.

Participants in the study inhale an aerosol form of the drug. The hope, Colombo said, is that the normal copy of the gene will go into the cells that need them, creating more normal mucus secretions.

The treatment doesn't fix existing lung damage but could prevent future damage.

"We know it works in the lab and petri dishes, but we don't know about people yet," Colombo said.

For cystic fibrosis suffers Chad Humston, 17, and Kate Howard, 16, the study could provide an easier life.

"Just to make me a little more happy," Humston said. "Any time I get a chance to get healthier I jump right on it."

About 30,000 people in the United States suffer cystic fibrosis. The disease also causes pancreatic problems.

Participants in the study are given two doses, 30 days apart, of either the aerosolized drug or a placebo. Patients then are monitored for six months to determine the drug's safety and effectiveness.

Colombo said seven patients, including Humston and Howard, have received the two doses so far. The goal is to have 12 to 15 local participants.

The Cystic Fibrosis Foundation and Target Genetics Corp. of Seattle, the drug's maker, are administering the 100-patient study.

Humston, of Giltner, Neb., went for his last in-person monitoring session about a month ago and Howard, of Sioux City, Iowa, completed her final one Thursday.

Colombo said they both reported improved lung function.

Howard's mom, Lonnel, said cystic fibrosis is a disease that doesn't allow you to forget it for even one day.

Daily treatments include chest physical therapy - vigorous clapping on the back and chest to dislodge mucus from the lungs - and other treatments such as aerosolized antibiotics or mucus- thinning drugs. The average life expectancy for those afflicted with the disease is 33.

"The possibility of being able to be normal, Kate has no idea," her mother said. "I'm excited for her."

Howard said her daughter has had her best lung function since enrolling in the study, but it's unknown whether it's a direct result from the treatment.

Humston, who plans to attend community college this fall, said he's optimistic a cure will be found eventually.

"Time will tell everything," he said.

A third phase of the study could start as early as a year from now, depending on the results from this second phase. Colombo said a third phase would involve the study of several hundred patients.

The promise of gene therapy leading to a cure for cystic fibrosis is unknown at this point.

"It would be extremely optimistic to think that this is the final bullet," Colombo said. "It probably isn't, but probably is a big step toward that. Especially if it shows efficacy as an earlier, smaller study did appear to."