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

NicoleHamblett

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 mission is to provide hope, care and cures to help every child live the healthiest and most fulfilling life possible. Together, Seattle Children’s Hospital, Research Institute and Foundation deliver superior patient care, identify new discoveries and treatments through pediatric research, and raise funds to create better futures for patients.

Ranked as one of the top five children’s hospitals in the country by U.S. News & World Report, Seattle Children’s serves as the pediatric and adolescent academic medical center for Washington, Alaska, Montana and Idaho – the largest region of any children’s hospital in the country. As one of the nation's top five pediatric research centers, Seattle Children’s Research Institute is internationally recognized for its work in neurosciences, immunology, cancer, infectious disease, injury prevention and much more. Seattle Children’s Hospital and Research Foundation works with the Seattle Children’s Guild Association, the largest all-volunteer fundraising network for any hospital in the country, to gather community support and raise funds for uncompensated care and research.

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