Dichlorophenyl-ABA is an inhibitor of transthyretin (TTR) amyloid fibril formation, inhibiting aggregate formation in more than 80% in TTR L55P-expressing cells[1].
Dichlorophenyl-ABA (DCPA) is able to prevent L55P aggregate formation in the conditioned medium. With regard to the ultrastructural analysis, Dichlorophenyl-ABA does not show an inhibitory effect as high as DFPB and benzoxazole, indicating that the Y78F mutant may not be as sensitive to this drug as TTR L55P and V30M are[1]. Dichlorophenyl-ABA is the best stabilizers of V30M tetramers in plasma from carriers of this mutant, and clearly inhibit aggregation in the cellular system. Therefore Dichlorophenyl-ABA is promising for the treatment of valine at position 30 (V30M)-associated familial amyloidotic polyneuropathy (FAP) but need to undergo further stages of drug development to overcome their toxicity[1].
[1]. Cardoso I, et al. Comparative in vitro and ex vivo activities of selected inhibitors of transthyretin aggregation: relevance in drug design. Biochem J. 2007 Nov 15;408(1):131-8.