Research

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Today, more than ever, the bio-medical research community is aggressively investigating new treatments for idiopathic pulmonary fibrosis (IPF). While the long term goal is to prevent and cure the disease, the main focus of present therapeutic approaches is to attempt to slow disease progression, and extend the life of patients with IPF. There are a variety of therapeutic modalities currently being studied. Some of these include:

  • Anti-fibrotic therapies that slow, or inhibit, the production of scar tissue and fibrosis. Presently this research is focusing on developing drugs that can inhibit the formation of “growth factor” proteins that stimulate pro-fibrotic pathways which ultimately result in lung fibrosis. Also research is focusing on “mesenchymal” stem cells which may alter this pathological process.
  • Endothelin receptor antagonists (ERAs), such as ambrisentan and bosentan, that may help IPF patients manage pulmonary arterial hypertension (PAH), a serious condition associated with IPF. In some experimental models, ERAs have been shown to inhibit the formation of collagen and scar tissue.
  • Stem cell therapies, which may help repopulate damaged lung tissue with more normal lung tissue.
  • Pulmonary vasodilators, such as sildenafil, that can improve oxygenation in IPF patients with PAH.
  • Some angiotensin receptor blocking agents, which are primarily use as blood pressure lowering medications, an example being losartan. These agents may function similarly to ERAs , allowing patients to oxygenate more effectively..
  • Genetic research to identify genes that may be associated with IPF and help identify individuals and families that are prone to IPF. These markers may also predict the rate of disease progression, and this may also lead to the development of therapies to block the expression of these genes.

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.

Types of Research

Basic Science – these studies are done to increase the fundamental understanding of the disease process on a molecular and cellular level. In the long term this type of research can provide the scientific basis for the development of effective treatments.

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.

Translational research – translating basic research findings into meaningful treatments and clinical application. This area of research is becoming increasingly prominent and the NIH is proposing the creation of a National Center for Advancing Translational Sciences (NCATS).

Clinical Research – studies that are performed to determines the safety and efficacy of medications, treatment regiments, and diagnostic procedures. The different phases of clinical trials for the development of new therapies are described below.

Quality of Life/Social Science – this is a subcategory of Clinical Research, which has often been ignored but has recently gained increased attention by the research community and the regulatory agencies. The term is used to refer to the general well-being of individuals undergoing specific treatment modalities, and not only includes medications but also supportive therapies (nutrition, exercise, respiratory therapy and psycho-social support).

Clinical trials investigate dosage, safety, efficacy and potential outcomes of drugs or treatments in disease-specific populations through controlled trials. There are typically three stages or phases of clinical trials that must be performed before a drug or treatment may be submitted to regulatory agencies (ie: U.S. Food & Drug Administration (FDA) in the USA) for approval. Prior to commencing a clinical trial, a drug must have demonstrated safety and efficacy in a laboratory model.

  • Phase I: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
  • Phase II: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
  • Phase III: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
  • Phase IV: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.

Types of Clinical Trials: There are two (2) different types of clinical trials, interventional and observational.

  • Interventional studies are those in which the research subjects are assigned by the investigator to a treatment or other intervention, and their outcomes are measured.
  • Observational studies are those in which individuals are observed (no intervention in the study) and their outcomes are measured by the investigators.

Benefits and Risks associated with participating in a Clinical Trial:

Benefits – Clinical trials that are well-designed and well-executed are the best approach for eligible participants to:

  • Play an active role in your own health care.
  • Gain access to new research treatments before they are widely available.
  • Obtain expert medical care at leading health care facilities during the trial.
  • Help others by contributing to medical research to find an effective treatment for PF.

Risks - There are risks to participating in clinical trials.

  • There may be unpleasant, serious or even life-threatening side effects to experimental treatment.
  • The experimental treatment may not be an effective treatment.
  • The study may require more of your time and attention than would taking a non-study treatment, including additional trips to the study site, hospital stays, completion of health diaries or complex dosage requirements.

Additional Resource Information on clinical trials can be found at http://clinicaltrials.gov/info/resources