申请人:Szarek Walter
公开号:US20110281951A1
公开(公告)日:2011-11-17
Therapeutic compounds and methods for modulating amyloid aggregation in a subject, whatever its clinical setting, are described. Amyloid aggregation is modulated by the administration to a subject of an effective amount of a therapeutic compound of the formula
or a pharmaceutically acceptable salt or ester, such that modulation of amyloid aggregation occurs. R
1
and R
2
are each independently a hydrogen atom or a substituted or unsubstituted aliphatic or aryl 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, provided when k is 1, R
1
is not a hydrogen atom, and when m is 1, R
2
is not a hydrogen atom. In an embodiment, at least one of k or m must equal 1. “p” and “s” are each independently positive integers selected such that the biodistribution of the therapeutic compound for an intended target site is not prevented while maintaining activity of the therapeutic compound. T is a linking group and Y is a group of the formula -A X wherein A is an anionic group at physiological pH, and X is a cationic group.
本文描述了用于调节主体中淀粉样聚集的治疗化合物和方法,无论其临床设置如何。通过向主体施用公式中的治疗化合物或药物可接受的盐或酯的有效量,从而调节淀粉样聚集。其中R1和R2分别是氢原子或取代或未取代的脂肪族或芳香族基团。Z和Q分别是羰基(C═O)、硫代羰基(C═S)、磺酰基(SO2)或亚磺酰基(S═O)基团。k和m为0或1,当k为1时,R1不是氢原子,当m为1时,R2不是氢原子。在一种实施方式中,k或m中至少有一个必须等于1。“p”和“s”分别是正整数,被选为使治疗化合物的生物分布不受阻碍,同时保持治疗化合物的活性。T是连接基团,Y是公式-A X的基团,其中A是生理pH下的负离子基团,X是阳离子基团。