Biologic Research & Development
Seeking Prevention/Treatment of Clostridium difficile Infection (CDI)
As the name implies, biologics are medical agents (such as proteins, cells, or DNA) derived from biological sources used to prevent or treat disease. With advances in biotechnology, biotherapeutic agents are being investigated and/or developed to treat various medical conditions, including:
• Diabetes (human recombinant insulin)
• Anemia (human recombinant erythropoietin)
• Breast Cancer (trastuzumab, monoclonal antibody)
• Cutaneous T-Cell Lymphoma (denileukin difitox, bio-engineered protein)
• CDI (multiple biologics)
• Pertussis/whooping cough (monoclonal antibody)
• Rheumatoid Arthritis (multiple biologics)
• Psoriatic Arthritis (multiple biologics)
• Psoriasis (multiple biologics)
Extensive biologic R&D resources are directed toward preventing/treating CDI. These biologic agents can be described as either biopharmaceuticals, prebiotics or probiotics/live biotherapeutics.
Created by biological process, rather than chemical synthesis, biopharmaceuticals, are medicinal products for the treatment, prevention, or cure of disease in humans. Biologics are generally derived from living material, i.e. microorganisms or cell culture. This is a significant contrast to chemically synthesized small molecule drugs that have a defined, and clearly characterized structure. Types of biopharmaceuticals include:
• Monoclonal antibodies
• Therapeutic enzymes
Prebiotics are a special type of naturally-occurring fiber that facilitates growth of beneficial (protective) bacteria already living in the colon; they promote an environment that is inhospitable to harmful bacteria. They are found in whole grains, garlic, onions, banana, artichokes and honey. Commercially available prebiotic fiber supplements are commonly used to insure sufficient fiber is ingested to promote a healthy colonic environment.
Probiotics / Live Biotherapeutics
The FDA describes probiotics as live microorganisms which, when administered in adequate amounts, confer a health benefit on the host. Probiotics are live microbes (bacteria or yeast) and can be regulated in the United States as both foods and drugs. Examples of probiotics classified as food are dietary supplements and yogurts.
The FDA classifies probiotics as “Live Biotherapeutics” when intended and/or marketed for disease prevention or treatment (probiotics for clinical use); they would require the FDA approval process that is required for drugs and biopharmaceuticals. Even though there have been some promising reports from probiotic biotherapeutic research, strong scientific evidence to support therapeutic use is not available. Currently, the FDA has not approved probiotics for any therapeutic claims.
Biopharmaceutical R&D Projects Seeking CDI Prevention/Treatment
• Vaccine to prevent CDI – Clostridium difficile toxoid vaccine
• Monoclonal antibody combination to prevent recurrent CDI
o Neutralize Clostridium difficile Toxin A and Toxin B
• Beta-lactamase enzyme to prevent CDI following parenteral beta-lactam therapy in hospitalized patients
• Live biotherapeutics to prevent CDI recurrence
o non-toxigenic Clostridium difficile spores in oral dose
o fecal microbiota transplantation
o defined compositions of fecal bacteria
Brian Andresen, Chairperson of Biologic Research and Development Committee (*) *Michael Kaleko, MD, PhD, Senior Vice President, Reseaerch & Development Synthetic Biologics *Joseph Sliman, MD, MPH, Senior Vice President, Clinical & Regulatory Affairs, Synthetic Biolgics *Andy Bristol, PhD, Vice President, Research & Development, Synthetic Biologics *Lewis Barrett, Senior Vice President, Commercial Strategy, Synthetic Biologics