A research team from the University of Arizona (UA) College of Medicine – Tucson was given a $4.4 million grant from the U.S. Department of Defense to study two experimental treatments believed to delay or possibly reverse excessive scar tissue formation in the lungs of patients with idiopathic pulmonary fibrosis (IPF).
The money will be used to investigate whether the compounds are safe and effective when administered orally or inhaled.
“Before we give these drug candidates to human patients in clinical trials, we have to perform rigorous, controlled testing to evaluate their safety and efficacy,” Louise Hecker, PhD, the grant recipient and a UA associate professor of medicine, said in a University of Arizona news release.
The two experimental treatments to be tested were designed to specifically target a protein, the Nox4 enzyme. Previous studies conducted by Hecker and colleagues found that the Nox4 protein is present in significantly higher levels in the lungs of patients with IPF.
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The researchers hypothesized that the overactivation of this enzyme may be behind the excessive scar tissue formation, and thus targeting this enzyme could lead to the reduction of scar tissue.
This hypothesis is supported by an animal study also developed by Hecker showing that scar tissue growth can be reversed in a mouse model of IPF when Nox4 is blocked.
“Nox4 clearly plays a critical role in mediating scar tissue formation,” Hecker said. “It is an important and exciting target for us to pursue for a cure.”
UA President Robert C. Robbins, MD, said IPF is a “deadly disease with a very grim prognosis. Dr. Hecker has the groundwork for a potential cure that could save lives. I look forward to seeing what she discovers with the support from this grant.”
In 2016, Hecker received a $300,000 pilot grant from the UA BIO5 Institute, a research institute based at the University of Arizona, to support her research. The funds helped her team identify the two compounds that will now be tested in preclinical studies.
Hecker also received a $1 million grant from the U.S. Department of Veterans Affairs to help understand why IPF primarily affects older people.