Pulmonary Fibrosis: Novel Therapeutic Development
Abstract
Excessive scar tissue in the lungs is known as pulmonary fibrosis. Pulmonary fibrosis occurs in many lung diseases that affect active military personnel, Veterans, and the general population. In some cases, the causes of a lung disease are unknown. This defines a pulmonary disease called idiopathic pulmonary fibrosis (IPF) that is characterized by excessive fibrosis and affects more than 5 million people worldwide and more than 200,000 people in the U.S. Pulmonary fibrosis affects the lung function, making it difficult for the afflicted individual to breathe normally or to be able to perform normal physical activities. Some patients may need to receive supplemental oxygen to preserve a minimal quality of life. Excessive pulmonary fibrosis is also associated with chronic irritants like cigarette smoke in diseases known as COPD (chronic obstructive pulmonary disease) or emphysema. Military personnel exposed to lung irritants due to the environmental conditions of their deployment may also become afflicted with pulmonary fibrosis. We have discovered that a specific biological step is critical for the production of excessive fibrosis in IPF, and we have designed a novel drug to block that step. We have found that we could prevent progression and induce regression of the excessive pulmonary fibrosis of IPF with our drug. Although our drug has excellent efficacy and safety profiles in a physiologically relevant rodent model of human IPF, it is necessary to complete all the U.S. Food and Drug Administration (FDA) safety requirements before we can test the drug’s safety and efficacy in human clinical studies. Completing these required FDA safety studies will get us an Investigational New Drug (IND) designation for our therapeutic. Our request of funds to the Congressionally Directed Medical Research Programs (CDMRP) is with the intention of using these funds to obtain an FDA approval for an IND, giving us the ability to use the drug in clinical safety studies, in patients with IPF. This IND designation and the clinical human safety studies, to follow the successful completion of this proposal, will allow us ultimately to pursue clinical human efficacy studies and bring this novel drug into clinical practice for the treatment of IPF. There are currently two medications to treat pulmonary fibrosis, but they are only partially effective, do not cure IPF, have many adverse effects, and are extremely expensive. If the CDMRP provides the funding needed for our program, it will advance us to the IND stage of development and provide us the impetus necessary to develop an effective and safe “smart drug” that will have a low cost of production and that will be readily accessible to military personnel, Veterans, and the U.S. population with significant chronic fibrotic lung diseases, including IPF. The development to the IND stage is a significant and decisive step in the overall development of a novel medication as with an IND the funding opportunities for further development (human safety and efficacy studies) are greatly increased. The achievement of an IND designation by the FDA will capture the interest of venture and angel investors and more importantly, garner us a greater potential for industry partnerships in the development of this therapeutic. In addition, an IND designation assures all parties that the compound is safe and efficacious in animal models and is likely to be safe and efficacious in humans. An IND designation also positions our therapeutic to take advantage of increased federal funding opportunities that are more accessible for a therapeutic at this advanced stage of development. It also provides assurances that the compound can be manufactured well and safely and has sufficient stability to be used as a medicine. This all increases our chances to develop our drug for IPF and investigate its probable value to treat additional chronic fibrotic diseases, including excessive scarring of
Document Details
- Document Type
- DoD Grant Award
- Publication Date
- Mar 10, 2021
- Source ID
- W81XWH2010719
Entities
People
- Mario Chojkier
Organizations
- United States Army
- Xfibra (United States)