NIH Awards $2.1M for Collaborative Work on Potential Gene Therapy

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by Marta Figueiredo, PhD |

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A team of researchers from the Michigan State University (MSU) College of Human Medicine and Spectrum Health will use a $2.1 million grant from the National Institutes of Health (NIH) to advance preclinical research on a gene therapy for cystic fibrosis (CF).

The four-year award was given to the project’s principal investigator, Xiaopeng Li, PhD, an associate professor at MSU’s department of pediatrics and human development, whose research focuses on the role of the small airways in CF development.

Other members of the team are MSU researchers Jeremy Prokop, PhD, and Christopher Waters, PhD, and four pulmonologists. They are Reda Girgis, MD, Susan Millard, MD, and John Schuen, MD, all from the Helen DeVos Children’s Hospital Cystic Fibrosis Center and Spectrum Health, and Ryan Thomas, MD, of MSU.

This collaborative work is part of the Cystic Fibrosis Translational Research Program, which is sponsored by the MSU-Spectrum Health Alliance and the Hunt for a Cure Foundation.

“Marrying basic science and clinical medicine is a goal that is driving the collaboration between MSU and Helen DeVos Children’s Hospital/Spectrum Health,” Schuen, also the chair of the MSU cystic fibrosis translational research program, said in a university press release.

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“This relationship has already led to significant grant opportunities and published literature,” Schuen said, adding that “together, we will help uncover important discoveries at the cellular level that we hope will benefit our CF patients in the future.”

CF is caused by several types of mutations in the CFTR gene, which provides the instructions to produce CFTR, a protein “gate” key for salts and water movement through cells that produce mucus, sweat, saliva, tears, and digestive enzymes.

CFTR mutations cause CFTR to stop working properly, leading to the accumulation of thick and sticky mucus in organs that include the lungs. CFTR deficiency also impairs the production of bicarbonate, a substance necessary to maintain a proper pH, or acid-alkaline, balance, on the cell surface.

Low bicarbonate levels were previously shown to increase acidity (lower pH) of the small airways, altering mucus properties, promoting thick mucus obstruction, and increasing susceptibility to bacterial infections.

However, the significance of the small airways in CF patients remains poorly understood, Li said.

Now, the team of researchers will assess how CFTR mutations make small airways so vulnerable to life-threatening infections and the feasibility of a gene therapy approach that replaces the defective CFTR gene with a normal gene.

“One reason we got the grant is we have a lot of data to show this is a feasible approach to treating the disease,” Li said.

Notably, gene therapy has the potential to be an effective, and potentially curative, therapeutic approach for CF patients, Li said.

According to B. Keith English, MD, chair of the College of Human Medicine’s department of pediatrics and human development, this MSU-Spectrum Health collaboration highlights the importance of “our outstanding and expanding research partnership in the area of cystic fibrosis.”

The NIH in 2019 granted another team of MSU-Spectrum Health researchers $2.7 million to investigate the role of anaerobic bacteria in the development of flare-ups among CF patients. Such bacteria live without oxygen, and are frequently found in the lungs of people with CF.

CF is “a particularly devastating disease,” Li said. “Hopefully, one day we can apply this treatment [the gene therapy] to human patients.”