BURLINGTON, VT and LEBANON, NH – New translational research, published in Nature Medicine, demonstrates a novel biological therapeutic candidate for regressing pulmonary fibrosis in a difficult-to-treat preclinical model of the disease, providing much-needed hope for the roughly 150,000 Americans suffering from this devastating condition.
A chronic, progressive disease marked by lung scarring that impedes breathing and the flow of oxygen to the bloodstream, idiopathic pulmonary fibrosis (IPF) results in approximately 40,000 deaths each year in the U.S. alone. Believed to be associated with aging, the disease is diagnosed annually in approximately 40,000 individuals between 40 and 70 years of age. Survival is typically only three to five years after diagnosis.
This study included an examination of IPF patient lung tissue, transgenic mouse models and direct administration of a therapy based off an enzyme called glutaredoxin-1 – or GLRX – to the airways of murine models of IPF. GLRX repairs damaged proteins and is inactivated in the lungs of IPF patients and results in poor lung function. Senior study author Yvonne Janssen-Heininger, Ph.D., and first author Vikas Anathy, Ph.D., of the University of Vermont’s Larner College of Medicine, and colleagues are inventors on a patent for GLRX, granted to the University of Vermont.
Though the exact mechanisms responsible for IPF have yet to be determined, oxidative stress is believed to play a critical role in modifying and altering the function of proteins or enzymes in various diseases, including IPF. However, the therapeutic potential for reversing the oxidative stress mediated modification of proteins was unknown. Janssen-Heininger and colleagues have discovered that GLRX is capable of reversing the oxidation of proteins, preventing the death of lung cells and ultimately, regressing pulmonary fibrosis in experimental models. The researchers believe that these positive effects raise the potential of GLRX as a therapeutic agent for IPF, which currently has limited treatment options.
“This provides a strong rationale to develop inhaled glutaredoxin as a possible therapy for patients with lung fibrosis,” says Janssen-Heininger. She and the study authors state that further investigation will be needed to gain a deeper understanding of the activity associated with GLRX. This therapy is being developed in collaboration with Celdara Medical, LLC, of Lebanon, NH.
Jake Reder, Ph.D., Celdara Medical’s CEO, commented, “Having worked with Yvonne and her team for almost three years, we’re very happy to see these important results published. Our parallel translational initiative, done in collaboration between Celdara Medical and her group at the University of Vermont, has gone a long way to validating the clinical application and potential of this strategy, while significantly improving manufacturing and formulation, and also leading to the discovery of improved compositions of matter. We are continuing to advance this therapy toward the clinic and are beginning discussions with both strategic and financial partners with the resources to increase both development speed and probability of success. Our goal remains clear: to help patients turn the tide on this deadly disease.”
This work was supported by the National Heart, Lung and Blood Institute of the National Institutes of Health under award numbers R44 HL129593-01 and R35HL135828.
University of Vermont Contact: Jennifer Nachbur, (802) 656-7875
Celdara Medical Contact: press@celdaramedical.com
About Celdara Medical, LLC
Celdara Medical gives hope and health to patients by transforming academic innovations into medicines with the potential to cure the world’s most challenging diseases. Celdara Medical is a recognized leader with a rich stable of discoveries, developed in concert with premiere research institutions in the US, EU, and beyond. We secure lasting partnerships with inventors and their institutions, and provide the developmental, financial, and business acumen to bridge the gaps between discovery and clinical impact. With robust funding options, operations in greater Boston, NYC, and Washington, DC, growing affiliates in Seattle and Indianapolis, a wealth of opportunities in our pipeline, and partnerships with industry leaders worldwide, Celdara Medical navigates the path from science to medicine, accelerating innovation to improve human health.