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 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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