Integrative Continuum: Accelerating Therapeutic Advances in Rare Autoimmune Diseases

Autoimmune diseases are chronic, life threatening, and of burgeoning public health concern. They rank among the 10 most common causes of death in women, and some have incidence rates surpassing those of heart disease and cancer. Emerging information regarding molecular and cellular mechanisms afford...

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Bibliographic Details
Published in:Annual review of pharmacology and toxicology 2012-01, Vol.52 (1), p.523-547
Main Authors: VAN HERLE, Katja, BEHNE, Jacinta M, VAN HERLE, Andre, BLASCHKE, Terrence F, SMITH, Terry J, YEAMAN, Michael R
Format: Article
Language:English
Subjects:
Animals
Autoimmune Diseases - drug therapy
Autoimmune Diseases - immunology
Biological and medical sciences
Cerebrospinal fluid. Meninges. Spinal cord
Clinical Trials as Topic
Disease Models, Animal
Diseases of visual field, optic nerve, optic chiasma and optic tracts
Female
Humans
Medical sciences
Nervous system (semeiology, syndromes)
Neurology
Neuromyelitis Optica - drug therapy
Neuromyelitis Optica - epidemiology
Neuromyelitis Optica - immunology
Ophthalmology
Public Health
Rare Diseases - drug therapy
Rare Diseases - epidemiology
Rare Diseases - immunology
United States
United States Food and Drug Administration
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Summary:Autoimmune diseases are chronic, life threatening, and of burgeoning public health concern. They rank among the 10 most common causes of death in women, and some have incidence rates surpassing those of heart disease and cancer. Emerging information regarding molecular and cellular mechanisms affords opportunities for the discovery of novel therapeutic strategies or the repurposing of FDA-approved pharmacologic agents. Yet, obstacles to drug development amplify as an inverse function of the incidence of rare autoimmune disease; challenges include heterogeneous clinical presentation, paucity of definitive biomarkers, and poorly validated measures of therapeutic response. An integrative continuum model to address these challenges is being applied to neuromyelitis optica (NMO)-a potentially devastating neurodegenerative process that has had limited therapeutic options. This model links target discovery with pharmacologic application to accelerate improved clinical efficacy. The application of such innovative strategies may help researchers overcome barriers to therapeutic advances in NMO and other rare autoimmune diseases.
ISSN:0362-1642
1545-4304
DOI:10.1146/annurev-pharmtox-010611-134628