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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).
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.