Research

Today, more than ever before, researchers are aggressively investigating new treatments for idiopathic pulmonary fibrosis (IPF). While the long term goal of IPF research is to prevent and cure the disease, present therapeutic approaches consist of attempts to slow disease progression, and to extend the life expectancy of patients with IPF.

There are a variety of therapeutic approaches currently being studied, including:

• Anti-fibrotic therapies that may slow, or inhibit, the body’s ability to produce scar tissue, or fibrosis.
• Endothelin receptor antagonists (ERA’s) that may help IPF patients manage pulmonary arterial hypertension (PAH), which is a serious condition commonly associated with IPF; also in experimental models, ERA’s have been shown to inhibit the formation of collagen and scar tissue.
• Inhibitors of “growth factor” proteins that may, alone, or in combination with other similar proteins, contribute to the formation of scar tissue, or fibrosis.
• Pulmonary vasodilators, such as sildenafil, that may help IPF patient process oxygen more efficiently.
• Some blood pressure lowering medications, such as losartan which is an angiotensin receptor blocker, which may function similar to ERA’s and allow patients to process oxygen more efficiently.
• Genetic research to identify genes that may be associated with IPF and help identify individuals and families that are prone to IPF. The markers may also predict the rate of disease progression.

While some studies are in advanced stages of development, others are in much earlier stages. There are also a variety of clinical trials that are actively seeking the participation of patients.

The Stages of Researching Potential Treatments for Disease

Basic Research, or Basic Science – studies that increase understanding of fundamental clinical hypotheses or principles: this can be thought of as a research hypothesis that arises out of curiosity. In the long term this type of research can become the basis for a drug to enter human study, or clinical trials. Basic research is often conducted at an academic, university level.

Epidemiological Research - the study of a disease within certain populations; research in this area can lead to better understanding of potential risk factors, including occupational or environmental exposures, that can lead to development of a disease.

Pathogenic Research – understanding the the chain of clinical events that may cause a disease to progress

Translational research – bringing scientific findings at a molecular or cellular level (usually basic research or “bench” research in a laboratory) into clinical application, or human studies.

Genetic Research - identifying the role of a gene, or genes, that are important in the development of a disease. Research in this area could uncover specific chromosomes (either present or absent) that may be predictive of the likelihood of developing a disease and the rate of disease progression. It is important to note that there may be interplay between environmental factors and gene expression. Similarly certain environmental factors could result in gene alterations or mutations. Better understanding of the genetic markers of a disease, could lead to earlier diagnosis and treatment, new novel therapies, and ultimately disease prevention.

Clinical Trials – investigating dosage, safety, efficacy and potential outcomes of drugs in disease-specific populations through controlled trials. There are typically three stages of clinical trials that must be performed before a drug may be submitted to the Food & Drug Administration (FDA) for approval. Prior to commencing a clinical trial, a drug must have demonstrated safety and efficacy in a laboratory model.

• Phase I: Initial human studies consisting of a small number of individuals (usually normal volunteers) to determine the safety, tolerability, side effects, metabolism and pharmacologic actions of a study drug at different doses. This is usually done in a controlled, well monitored environment.
• Phase II: Controlled clinical studies conducted to evaluate the effectiveness of the study drug for a particular indication (or very rarely more than one indication). This is done in a larger patient population (up to 300 patients). If a drug proves to be ineffective, have serious side effects, or is not well tolerated, it is usually discovered during Phase II.
• Phase III: These are controlled, randomized multi-center drug trials comparing the efficacy of standard therapy or placebo with the study drug. Almost always the trial is performed in a blind fashion, i.e. the patient and physician do not know if the patient is receiving the study medication, standard therapy, or a placebo. These are large (up to 3,000 patients), expensive, and time consuming studies. Occasionally there is overwhelming evidence of the efficacy of a new therapy; if this arises the study is prematurely terminated, and the therapy approved.
• Phase IV: Post-marketing (e.g. after the FDA has approved a drug) studies to delineate additional information including the drug's risks, benefits, and optimal use.

For more information on current research on pulmonary fibrosis: