Hideki Garren, MD, PhD, Chief Medical Officer for Prothena, discusses the pathophysiology of AL amyloidosis and the mechanism of action of investigational drug birtamimab.
Amyloid light chain (AL) amyloidosis is a progressive plasma cell disorder in which amyloid builds up in tissues and organs. The deposition of amyloid is caused by the misfolding of pro-amyloidogenic light chain protein produced by clonal plasma cells. The signs and symptoms of AL amyloidosis vary among patients because the build up may occur in various organs. However, the heart and kidneys are the most commonly affected.
Current treatment includes therapies that target clonal plasma cells and prevent further amyloid deposition. These include autologous stem cell transplants and plasma cell therapies. However, these treatment options do little to improve on survival rates in those with more advanced disease. A new class of therapies called amyloid depleters are currently in development to address the pathophysiology of the disease.
Birtamimab is an investigational humanized monoclonal antibody that binds misfolded kappa and lambda light chains, neutralizing soluble toxic light chain aggregates. The compound also promotes phagocytic clearance of deposited amyloid, improving organ function. Analyses of clinical trials for this treatment observe the standard of care plus birtamimab have survival benefits in patients with advanced AL amyloidosis.
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For more information on AL amyloidosis and other rare hematologic diseases, visit https://checkrare.com/diseases/hematologic-disorders/
