IP Library Granted Patent US 12678423
Granted Patent B2
US 12678423 · App. 16/328,636 · Granted Jul 14, 2026

Therapeutic agent for amyotrophic lateral sclerosis and composition for treatment

Inventors: Hideyuki Okano (Tokyo, JP); Koki Fujimori (Tokyo, JP); Hironobu Okuno (Tokyo, JP)
Assignee: KEIO UNIVERSITY
A61K31/404A61P25/00
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Quick Facts
Patent No.
US 12678423
App. No.
16/328,636
Granted
Jul 14, 2026
Kind
B2
Abstract

A therapeutic agent for amyotrophic lateral sclerosis including a compound represented by Formula (1) [in Formula (1), R 1 each independently represents an alkyl group having 1 to 6 carbon atoms or 4-hydroxyphenethyl group, and n represents an integer of 1 to 3], a pharmaceutically acceptable salt thereof, or a solvate thereof, as an effective ingredient.

Claims (13)

1 . A method of treating sporadic amyotrophic lateral sclerosis (ALS), or familial ALS having a mutation in the FUS gene and/or TAR DNA-binding protein 43 kDa (TDP-43) gene, after onset of the ALS phenotype, comprising:

administering an effective amount of a compound represented by Formula (1), a pharmaceutically acceptable salt thereof, or a solvate thereof to a patient in need thereof

in Formula (1), R 1 each independently represents an alkyl group having 1 to 6 carbon atoms or a 4-hydroxyphenethyl group, and n represents an integer of 1 to 3.

2 . The method according to claim 1 , wherein n in Formula (1) is 2.

3 . The method according to claim 1 ,

wherein R 1 in Formula (1) is an n-propyl group.

4 . The method according to claim 1 ,

wherein the compound represented by Formula (1) is 4-(2-di-n-propylaminoethyl)-2 (3H)-indolone.

5 . The method according to claim 1 ,

wherein the pharmaceutically acceptable salt of the compound represented by Formula (1) is 4-(2-di-n-propylaminoethyl)-2 (3H)-indolone hydrochloride.

6 . A method of treating sporadic ALS, or familial ALS having a mutation in the FUS gene and/or TDP-43 gene, after onset of the ALS phenotype, comprising:

administering an effective amount of a composition to a patient in need thereof, wherein the composition includes a compound represented by Formula (1), a pharmaceutically acceptable salt thereof, or a solvate thereof and a pharmaceutically acceptable carrier

in Formula (1), R 1 each independently represents an alkyl group having 1 to 6 carbon atoms or a 4-hydroxyphenethyl group, and n represents an integer of 1 to 3.