Advances in Treatment of Cardiac Amyloid

Cherie N. Dahm, R. Frank Cornell, Daniel J. Lenihan

Systemic amyloidosis represents a complex group of diseases with a common feature characterized by misfolded autologous proteins depositing into tissues or organs throughout the body. Light chain amyloidosis (AL) and transthyretin (TTR) amyloid are the two most prevalent forms of this disease that commonly results in cardiac amyloidosis. In both of these conditions, the myocardium is a frequent site of infiltration and end-organ involvement often with devastating consequences. With cardiac amyloidosis becoming an increasingly identified disease that has previously been under-recognized, the purpose of this comprehensive review is to focus on the diagnosis and treatment of these two types of cardiac amyloidosis including a contemporary update on currently available therapies being investigated in clinical trials. Subsequently, we will detail potential therapeutic efficacy and limitations of these regimens, and then complete the review by highlighting newer treatment modalities. A high-level overview of modern therapeutic approaches for AL amyloid includes targeted therapies directed at reducing the production of precursor proteins and inhibitors intended to limit the deposition of fibrils in tissues. In the case of TTR amyloid, current therapy is focused on stabilization of TTR proteins, suppression of protein formation, and blocking the deposition of amyloid fibrils in tissue. Novel therapies are focused on removing amyloid fibril deposition from affected tissues. In summary, cardiac amyloidosis is a progressively devastating disease requiring swift recognition and treatment now with groundbreaking therapies on the horizon.