ReportsSYSTEMIC AMYLOIDOSIS

Repeat doses of antibody to serum amyloid P component clear amyloid deposits in patients with systemic amyloidosis

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Science Translational Medicine  03 Jan 2018:
Vol. 10, Issue 422, eaan3128
DOI: 10.1126/scitranslmed.aan3128

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Making amyloid vanish

Fatal systemic amyloidosis is caused by extracellular amyloid deposition that disrupts tissue structure and function. The normal plasma protein, serum amyloid P component (SAP), is always present within amyloid deposits. Richards et al. now show that previous depletion of circulating SAP by the drug, miridesap, uniquely enables subsequent administration of the humanized anti-SAP antibody, dezamizumab, to patients with systemic amyloidosis. Dezamizumab bound to residual SAP in the amyloid deposits and triggered their removal. Repeat cycles of miridesap followed by dezamizumab progressively removed amyloid from the liver, spleen, and kidneys of the patients. Evidence of clinical benefit suggests that this new approach has potential to improve management and outcome for patients with systemic amyloidosis.