Methods for modulating the aggregation of amyloids in an individual, for the treatment of a disease condition associated with amyloidosis, composition for modulating amyloidosis, and, methods for inhibiting the amyloid deposits associated with iapp in an individual, to inhibit iapp fibrillogenesis in an individual , to reduce aggregates of amyloids associated with iapp in an individual, to modulate damage associated with amyloids in cells, and to modulate amyloidosis in vivo or ex vivo

摘要

"METHODS FOR MODULATING THE AGGREGATION OF AMYLOIDES IN AN INDIVIDUAL, FOR THE TREATMENT OF A SICK STATE ASSOCIATED WITH AMYLOIDOSIS, COMPOSITION FOR MODULAR AMYLOIDOSIS, AND, METHODS FOR INHIBITING EMITORIOUS EMYZOID EMYPOCOPPED EMYPHOPID EMYPOP EMITTED EMYPOP EMITTED EMAPPIRATED EMIPAPE INDIVIDUAL, TO REDUCE IAPP-ASSOCIATED AMYLOID AGGREGATES IN ONE INDIVIDUAL, TO MODULAR DAMAGE ASSOCIATED WITH AMYLOIDS IN CELLS, AND TO MODULAR AMYLOIDOSIS IN VIVO OU.EX LIVE ". Therapeutic compounds and methods for modulating amyloid aggregation in an individual, whatever their clinical status, are described. The aggregation of amyloids is modulated by administering to an individual an effective amount of a therapeutic compound of formula (1): or a pharmaceutically acceptable salt or ester, so that modulation of the aggregation of amyloids occurs. R ^ 1 ^ and R ^ 2 ^ are each independently a hydrogen atom or a substituted or unsubstituted aryl or aliphatic group. Z and Q are each independently a carbonyl (C = O), thiocarbonyl (C = S), sulfonyl (SO ~ 2 ~), or sulfoxide (S = O) group. "k" and "m" are 0 or 1, since when k is 1, R ^ 1 ^ is not a hydrogen atom, and when m is 1, R ^ 2 ^ is not a hydrogen atom. In one embodiment, at least one of k or m must equal 1. "p" and "s" are each independently selected positive integers so that the biodistribution of the therapeutic compound to a desired targeting site is not prevented while maintaining activity of the therapeutic compound. T is a linking group and Y is a group of formula: -AX, where A is an anionic group at physiological pH, and X is a cationic group.