Abstract
Introduction
More than 80 autoimmune disorders have been identifi ed, all involving aberrant immune responses to self-antigens. The emergence of biologic agents, such as the interferons and the TNF-α inhibitors, has greatly improved the treatment outlook for some of the more common autoimmune diseases. These agents have signifi cant drawbacks, however, including side effects and high costs. Pharmaceutical companies large and small have already begun to focus their efforts on the molecular events that trigger autoimmunity, a discovery process that, it is hoped, will reveal new drug targets that suppress undesired autoimmune infl ammatory responses in some of the most common diseases.
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- Understanding the etiology of the different autoimmune diseases will allow more successful diagnosis and treatment options. How will the identifi cation of genetic markers help to create new diagnostic tools? Will these tools offer a greater chance for successful treatment, or even a cure?
- Biologic agents have greatly improved the treatment of many autoimmune diseases, but these agents are expensive and carry the risk of signifi cant side effects. What new methods are being studied as both treatments and curatives?
- Earlier identifi cation of autoimmune diseases could greatly improve the quality of life for patients. What' s in store for the future of autoimmune R&D? What companies already have trials under way?
Scope
- Pathophysiology and etiology: autoimmune disease is caused by antibodies that attack normal tissue; the diseases can be organ-specifi c or systemic; most autoimmune diseases are caused by genetic and/or environmental factors.
- Epidemiology: 2-8% of the U.S. population has some type of autoimmune disease, and the prevalence of these diseases is increasing.
- Novel approaches to treatment: targeted biologics, new cell technologies, and novel treatment methods are the focus of current drug discovery programs.
- Unmet need creates opportunity for new therapies: interferon-beta (for MS) and the TNF-alpha inhibitors offer signifi cant benefi t over more traditional corticosteroid and DMARD treatments.
- Potential curative therapies on the horizon: cell therapies like stem cell replacement and tolerization vaccines represent potential for cures.
Table of Contents
- Executive Summary
- Strategic Considerations
- Introduction
- Overview of Autoimmune Diseases
- Pathophysiology and Etiology
- Epidemiology
- Novel Targets and New Approaches to Treating Autoimmune Disease
- Novel Targets
- Sphingosine-1-Phosphate Receptor Modulators
- Janus Kinases
- Matrix Metalloproteinases
- Tolerance Induction
- PDE Inhibitors
- Novel Treatment Methods
- Antisense
- Hematopoietic Stem Cell Transplant
- Cell Therapy for the Treatment of Type 1 Diabetes
- Novel Targets
- New Approaches to Drug-Target and Biomarker Discovery for Autoimmune
Diseases
- Genetic Analysis
- Proteomics: Mass Spectrometry, Protein and Antibody Arrays
- Mass Spectrometry
- Microarrays
- Flow Cytometry
- Lipidomics
- Imaging
- Company Profi les
- Alba Therapeutic
- Bayhill Therapeutics
- BioSeek
- MultiCell Technologies
- Novocell
- Osiris Therapeutics
- Osprey Pharmaceuticals
- Outlook
- Ongoing Unmet Need Creates Opportunity
- Potential Curative Therapies on the Horizon
- Other Future Yields of Today' s Autoimmune R&D
Tables:
- 1. Overview of Select Autoimmune Diseases
- 2. Select Drug Classes Used to Treat Autoimmune Diseases
- 3. Select Novel Targets for the Treatment of Autoimmune Disease
- 4. Select Novel Approaches to Autoimmune Therapy
- 5. Select Companies Investigating Autoimmune Disease, 2008
Figures:
- 1. Prevalent Cases of Select Autoimmune Diseases in the United States, 2007
- 2. Sites of Action of Current and Experimental Immunosuppressive Agents
