Treatment for Wolfram syndrome and other endoplasmic reticulum stress disorders

Patent metadata

Field	Value
Publication number	US-11426415-B2
Application number	US-201816491826-A
Country	US
Kind code	B2
Filing date	Mar 6, 2018
Priority date	Mar 6, 2017
Publication date	Aug 30, 2022
Grant date	Aug 30, 2022

Abstract

The present invention generally relates to compositions and methods for treating or preventing an endoplasmic reticulum stress disorder in subjects, including compositions and methods for treating or preventing Wolfram syndrome. The present invention also relates to methods for treating symptoms of neurodegenerative diseases.

First claim

What is claimed is:

1. A method for treating or preventing Wolfram syndrome in a subject in need thereof comprising administering to the subject a pharmaceutical composition comprising a therapeutically effective amount of at least one compound selected from the group consisting of JTV519, oxidized JTV519, salts and esters thereof, and mixtures thereof.

2. The method of claim 1 , wherein the compound is administered in an amount of from about 0.05 mg to about 100 mg per kg of body weight.

3. The method of claim 1 , wherein the pharmaceutical composition is administered parenterally to the subject.

4. The method of claim 1 , wherein the pharmaceutical composition is administered orally to the subject.

5. The method of claim 1 , further comprising administering at least one additional active agent to the subject.

6. The method of claim 5 , wherein the additional active agent comprises dantrolene, azumolene, or pharmaceutically acceptable salts or hydrates thereof.

7. The method of claim 5 wherein the additional active agent comprises a glitazone, GLP-1, sitagliptin, and/or metformin.

8. The method of claim 1 , wherein the subject is a human.

9. The method of claim 1 , wherein the method treats clinical symptoms of Wolfram syndrome.

10. The method of claim 9 , wherein the clinical symptoms comprise β cell and neuronal cell death.

11. The method of claim 5 wherein the additional active agent comprises sitagliptin and/or metformin.

Assignees

• Univ Washington
• The Us Secretary Department Of Health And Human Services Office Of Technology Transfer National Inst
• Us Health

Inventors

• Urano Fumihiko
• Mahadevan Jana
• Clark Amy
• Jadhav Ajit
• Maloney David
• Harvey Brandon
• Yang Shyh-Ming
• Henderson Mark

Classifications

• A61K31/554 Primary

  having at least one nitrogen and one sulfur as ring hetero atoms, e.g. clothiapine, diltiazem · CPC title

• A61K31/4985

  Pyrazines or piperazines ortho- or peri-condensed with heterocyclic ring systems · CPC title

• A61K31/155

  Amidines ([IMAGE cpc-sch-A61K-1029.gif]), e.g. guanidine (H2N—C(=NH)—NH2), isourea (N=C(OH)—NH2), isothiourea (—N=C(SH)—NH2) · CPC title

• A61K31/4178

  not condensed 1,3-diazoles and containing further heterocyclic rings, e.g. pilocarpine, nitrofurantoin · CPC title

• C07K14/605

  Glucagons · CPC title