Baby Molly Hamilton.

Baby Molly Hamilton.

Meet Molly

On Feb. 17, 2008, Erin and Bill Hamilton welcomed their daughter Molly into the world. She appeared to be a perfectly healthy, 9-pound baby girl, but a newborn screening test revealed Molly had cystic fibrosis.

“We were devastated,” Erin said. “We didn’t know anything about cystic fibrosis and had no idea how this disease would affect her life.”

Life-changing research

Dr. Bonnie Ramsey, director of the Center for Clinical and Translational Research at Seattle Children’s Research Institute and a national leader in cystic fibrosis research.

Dr. Bonnie Ramsey, a national leader in cystic fibrosis research.

Cystic fibrosis is an inherited condition that affects about 30,000 people in the United States. It causes thick, sticky mucus to build in the lungs, digestive tract and other areas of the body. It’s also a disease that has become the life’s work of Dr. Bonnie Ramsey, director of the Center for Clinical and Translational Research at Seattle Children’s Research Institute.

In 1938, when cystic fibrosis was first recognized as a disease, babies with this condition rarely lived past one year. Thanks to researchers like Ramsey, children with this disease can now live comfortably into adulthood.

“It is so rewarding to see how research impacts the lives of these children,” Ramsey said. “My goal is to keep conducting research studies until there are successful treatments for all patients with cystic fibrosis.”

Molly was just two years old the first time Ramsey’s research made a difference in her life. A decade earlier, Ramsey had developed TOBI (tobramycin inhalation solution), the first aerosol antibiotic designed to improve lung function for patients. The drug was approved by the FDA in 1997.

The medication TIBO allowed Molly to live a more active life.

The medication TIBO allowed Molly to live a more active life.

In 2010, Molly was treated with TOBI as part of a clinical research study at Seattle Children’s. The medication thinned her lung mucus and reduced a persistent cough, helping her to breathe. By the time Molly was 3, she was living an active life – fishing with her dad and wakeboarding.

The turning point

A year later, Ramsey confirmed that an oral medicine, Kalydeco, improves lung function in the 4 percent of patients with cystic fibrosis who have a genetic mutation called G551D. Molly was part of that 4 percent. When Kalydeco was approved by the FDA in 2012, Molly was eligible for the treatment. Just a month after she started taking the medication, her lifelong cough and other nagging symptoms disappeared.

Molly's family celebrated the success of the drug Kalydeco by going to Disneyland.

Molly’s family celebrated the success of the drug Kalydeco by going to Disneyland.

“The change was incredible,” Erin Hamilton said. “Before Kalydeco, she was waking up three to four times a night in coughing fits. She’d had terrible digestion problems and her skin always tasted salty. Her digestive problems were better in just a week. After 10 days, her skin was no longer salty. It was an absolute miracle.”

Looking ahead

Ramsey is currently leading a worldwide study to test if Kalydeco, in combination with a second drug, Lumacaftor, works in cystic fibrosis patients who have the most common mutation, Delta F508.

Molly, meanwhile, is an active 6-year-old. She plays basketball and soccer, swims and rides horses. She loves to be outside and play with her older brother, Will. She continues to see Ramsey in Seattle Children’s Cystic Fibrosis Clinic.

Molly and her family today.

Molly and her family today.

“There is a lightness and a silliness about her that was always missing,” Erin Hamilton said. “We now believe that our daughter will live a long, normal life.”

Media who are interested in interviewing Dr. Bonnie Ramsey should contact Seattle Children’s PR team at 206-987-4500 or at press@seattlechildrens.org.

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