Pinpoint Critical Drug Targets for Autoimmune Disease
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
For more info