Newswise — In the first major study of its kind, principal investigator Benjamin Gaston, MD, a pediatric pulmonologist and researcher at Case Western Reserve University School of Medicine and University Hospitals Rainbow Babies & Children’s Hospital, has received a five-year, $13.3 million federal grant to develop optimized, personalized combinations of medications tailored to the unique needs of children with cystic fibrosis and severe asthma.

While there are a number of medications used to treat these conditions, until now they have not been combined in large numbers of patients with an aim of discovering which combinations work best for specific disease profiles. “Cells of cancer patients can be studied for response to different agents and combinations of agents in order to identify the best treatment approach,” said Gaston. “Here, we will take a similar approach to individualize therapy for severe airways diseases. The focus of our project is personalized medicine: identifying the most synergistic treatment method for each patient based on their own needs.” The investigators anticipate that this approach will be adopted over time by other pediatric medical centers throughout the country and around the world.

Under the grant, funded by the National Heart, Lung and Blood Institute of the National Institutes of Health, Gaston and colleagues will conduct highly specific lung function testing and laboratory analyses to identify patients most likely to respond to certain therapies.

For example, researchers will assess patients’ airway-cell pH levels. In cases of low pH levels, which are found in some patients with severe asthma and cystic fibrosis, the researchers can determine which combinations of medications (“pH modifiers”) work best, such as whether inhaled albuterol in buffered glycine is more effective than albuterol alone.

In a second example, individual patients’ cells will be grown in the laboratory and treated with different cystic fibrosis corrector agents: the most effective therapy or combination of therapies in the cell system could ultimately be used to improve lung function in patients.

The varying combinations of medications that will be assessed in these and other scenarios include new treatments developed, in part, by the lead investigators.

In addition to Gaston, CWRU investigators include project leaders Jonathan Stamler, MD, Director of the Harrington Discovery Institute at UH; and Mitchell Drumm, PhD as well as core directors Michael Konstan, MD; Ted Torphy, PhD; Calvin Cotton, PhD; and Mark Schlucter, PhD.

“This is a bold step by the National Heart, Lung and Blood Institute to change the way we think about cystic fibrosis and severe asthma treatment,” said Gaston. “My colleagues and I are grateful to the Institute for its support and honored by their faith in our capacity to translate new scientific discoveries into therapies for these patients.”

This study will be funded by National Heart, Lung and Blood Institute grant HL128192-01A1.


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