Take a piece of paper--folding it makes a paper plane, an envelope, a complex and delicate origami flower, or a screwed-up mess. Take a sequence of amino acids--folding it makes a functional protein, or something that leads to dysfunction and disease. Misfolded proteins are behind a number of neurological disorders, including amyotrophic lateral sclerosis (ALS, or Lou Gehrig's Disease). Toronto-based Amorfix Life Sciences is developing a biomarker test for ALS based on its discovery of misfolded SOD1 (superoxide dismutase) in the blood of ALS patients.
ALS diagnosis is currently based on clinical findings and imaging, which can cause delays. "The availability of a reliable blood test would represent an important leap in the management of this devastating disease, allowing clinicians to conduct better clinical studies, and eventually begin treatment earlier," said Amorfix Chief Scientific Officer Dr. Neil Cashman.
As well as a diagnostic for ALS based on misfolded SOD1, Amorfix is developing a therapeutic antibody and therapeutic vaccine. ALS is incurable and fatal, with 80% of people dying within two to five years. Being able to diagnose and treat it earlier may improve the outcome, improving lifespan and quality of life, cutting costs for healthcare providers, allowing people to have longer working lives and reducing distress for families.
- read the release