Dr. Wong on Anti-Amyloid Fibril Treatments in AL Amyloidosis

Sandy Wong, MD, assistant professor, University of California, San Francisco School of Medicine, discusses anti-amyloid fibril treatments, a class of agents that have yet to receive FDA approval in the treatment of patients with immunoglobulin light chain amyloidosis (AL amyloidosis).

The NEOD001 agent has received a lot of attention over the past couple years. It is an anti-amyloid fibril monoclonal antibody that works against the amyloid fibrils to clean up the amyloid deposits in the body, allowing for organ recovery.

Early-phase studies reveal that it was well-tolerated in a first-in-human study of 27 patients with AL amyloidosis. The phase I trial, published in the Journal of Clinical Oncology, detailed a promising safety profile. Preliminary data also showed cardiac, renal, and neurologic responses.

The other anti-amyloid fibril therapy is CAEL-101. Data presented at the 2017 ASH Annual Meeting suggested that this drug looks similar to NEOD001 with regard to efficacy and safety.