Preclinical development of monoclonal antibodies for the treatment of AL cardiac amyloidosis
Amyloid cardiomyopathy is an under-recognized cause of heart failure and is caused by normal proteins in the bloodstream going bad (known as amyloid) and accumulating in the heart. This accumulation of amyloid in the heart tissue causes the wall of the heart to become rigid and ineffective at pumping blood to the rest of the body, causing heart failure. We are studying a form of amyloid cardiomyopathy called immunoglobulin light chain (AL) amyloidosis, caused by the accumulation of light chain proteins. AL amyloidosis can also affect organs other than the heart and cause life-threatening multi-organ failure. We have designed a drug to potentially treat AL amyloidosis. The drug works by tagging these amyloid and harness the power of the immune system to recruit the body's own immune cells to clear these amyloid from the organs, restoring organ function. This project involves screening our drugs for safety and effectiveness in mice. as well as drug characterization before it can be used in humans.