NIH Awards $9.8 Million to Seattle Children’s for Cystic Fibrosis Research


Researchers to conduct clinical trials, manage national data coordinating center

Seattle Children’s has been awarded nearly $10 million over the next five years from the National Heart, Lung, and Blood Institute of the National Institutes of Health to develop treatment for cystic fibrosis (CF) patients who suffer from early Pseudomonas aeruginosa infection.

More than 45 sites across the country will take part in the research and related clinical trials, which is using the acronym OPTIMIZE, which stands for “OPTIMIZing Treatment for Early Pseudomonas aeruginosa Infection in Cystic Fibrosis.”

Pseudomonas aeruginosa is an opportunistic bacterium that targets people weakened by lung disease, including CF, and other serious medical conditions, such as severe burns and immunodeficiencies. Two things set it apart from many other bacteria: An eerie green color and its resistance to antibiotics. Pseudomonas aeruginosa infections don’t immediately make patients sick, so they haven’t always been treated with antibiotics right away. Recently, evidence has shown that these infections are easier to eradicate with antibiotics when treated very early, and that this response decreases as the infection becomes more established.

Researchers will target new approaches to attack this infection, trying to reduce its impact on respiratory symptoms and improve on clearing it from the body. They hope that these new approaches will ultimately contain or prevent the infection from becoming chronic. The research builds on a previous study led by Seattle Children’s of early Pseudomonas aeruginosa infection in which researchers identified a treatment approach that was successful in eradicating the infection in a majority of children. In this study, about one-third of children had a recurrence within about one year and those children were subsequently at higher risk of increased respiratory symptoms.

“We hope to determine whether new approaches to therapy for Pseudomonas aeruginosa will keep more of our patients symptom and infection free,” said Nicole Hamblett, PhD, lead investigator at Seattle Children’s coordinating the new OPTIMIZE trial.

It’s long been known that many combinations of bacteria inhabit the lungs of patients with CF. The thick mucus that clogs their lungs offers a fertile breeding ground, and bacteria that become established in these secretions can cause chronic and destructive infections. Most research has focused on Pseudomonas aeruginosa because it commonly infects people with CF, is virulent, and is very difficult to eradicate.

“If Pseudomonas aeruginosa was on the TV show ‘Survivor’ it would win every time,” said Bonnie Ramsey, MD, a pediatric pulmonologist who leads CF research at Seattle Children’s and who will serve as lead clinical investigator for the 45 sites across the country participating in the trial. “Once this infection gets established, it’s almost impossible to totally clear from a patient’s lungs.”

The research project is supported by the National Heart, Lung, and Blood Institute of the National Institutes of Health under Awards 1U01HL114623-01A1 and 1U01HL114589-01A1.

About Seattle Children’s

Seattle Children’s Hospital, Foundation and Research Institute together deliver superior patient care, advance new discoveries and treatments through pediatric research, and raise funds to create better futures for patients. Consistently ranked as one of the top 10 children’s hospitals in the country by U.S. News & World Report, Seattle Children’s Hospital specializes in meeting the unique physical, emotional and developmental needs of children from infancy through young adulthood. Through the collaboration of physicians in nearly 60 pediatric subspecialties, Seattle Children’s Hospital provides inpatient, outpatient, diagnostic, surgical, rehabilitative, behavioral, and emergency and outreach services to families from around the world.

Located in downtown Seattle’s biotech corridor, Seattle Children’s Research Institute is pushing the boundaries of medical research to find cures for pediatric diseases and improve outcomes for children all over the world. Internationally recognized investigators and staff at the research institute are advancing new discoveries in cancer, genetics, immunology, pathology, infectious disease, injury prevention, bioethics and much more.

Seattle Children’s Hospital and Research Foundation and Seattle Children’s Hospital Guild Association work together to gather community support and raise funds for uncompensated care, clinical care and research. The foundation receives nearly 80,000 gifts each year, from lemonade stand proceeds to corporate sponsorships. Seattle Children’s Hospital Guild Association is the largest all-volunteer fundraising network for any hospital in the country, serving as the umbrella organization for 450 groups of people who turn an activity they love into a fundraiser. Support from the foundation and guild association makes it possible for Seattle Children’s care and research teams to improve the health and well-being of all kids.

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