Pinpointing Critical Drug Targets for Autoimmune Disease

Pinpointing critical drug targets for autoimmune disease

Competition: EXPLORE Program 2011
Funding: $300,000 / 2 years
Beginning: December 2011
Completed project
This study aims to identify somatic mutation sufficient for the development of autoimmune diseases. The proof of concept will be done on patients with rheumatoid arthritis.

Overview

The aim of this project is to identify somatic mutation sufficient for the development of autoimmune diseases. The proof of concept will be done on patients with rheumatoid arthritis.

Autoimmune diseases affect millions of individuals in North America. They are a major health and economic burden as people with such conditions suffer from debilitating symptoms that can lead to life-long disability and mortality. Although progress has been made in the last few years, many autoimmune diseases lack effective, targeted therapies with acceptable safety profiles primarily due to a lack of knowledge on what selectively activated pathways are involved in the disease. Somatic mutations, in addition to hereditary mutations and environment effects, are considered a driving force in proliferative cell diseases such as cancer that are not too dissimilar to inflammatory disorders. If a single mutation in a pivotal gene were sufficient to cause autoimmune disease, this would highlight a potential drug target.

Based on this understanding, Dr. Richards and his team proposed that somatic mutations could in fact lead to uncontrolled cellular proliferation causing autoimmunity. They have secured joint-space aspirates from individuals having new-onset arthritis, in addition to their circulating white blood cells. They are presently comparing their genomes for evidence of mutations present in the inflammatory cells in the joint space, but absent from the circulating periphery.

Impact on the drug discovery process

  • Pioneer new autoimmune disease treatment through novel disease specific targets.

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