Friday, July 17, 2015

In the NEWS: Amyloidosis and potential novel treatment

Systemic amyloidosis contains non fibrillar protein called serum amyloid P ( SAP).   Novel agent that is called CPHPC depletes SAP from the plasma but leaves some SAP in the amyloid deposits.  Following giving CPHPC, if given anti SAP antibodies, it can clear the remainder of the free SAP. A recent study in NEJM did an open label single dose phase 1 trial to look at 15 patients with systemic amyloidosis using the above strategy and there was clearance of amyloid deposits from liver and other tissues.

Few interesting and key points
What type of amylodosis: the patients included had AA, AFib, AL, AApoA1 type of amyloidosis.
What tissues involved: Liver, spleen, kidney, bone marrow, ( no patients with cardiac or severe renal involvement)
Any renal adverse events:- None in liver or kidney

This is a fascinating new pathway of treating patients with fibril based disease like amyloidosis. Wonder if this similar approach might be useful in other renal fibril forming disease such as fibrillary GN or immunotactoid GN. 

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