Use of delta tocopherol for the treatment of lysosomal storage disorders

US9044451B2 · kind B2 · utility

Assignee

• The United States of America, as represented by the Secretary, Department of Health and Human Services · US

Inventors

• Wei Zheng · Rockville, US
• Juan Jose Marugan · Gaithersburg, US
• Ke Liu · Hanover, US
• Noel Terrence Southall · Bethesda, US
• Christopher P. Austin · Potomac, US

Key dates

• Filing date: Jul 19, 2011
• Grant date: Jun 2, 2015
• Expiry date: Sep 26, 2031

Classification

• Technology area (CPC A): Human Necessities
• CPC primary: A61P43/00
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

This disclosure relates generally to the treatment of lysosomal storage disorders. Specifically, the disclosure relates to a novel use of delta tocopherol in the treatment of diseases and conditions related to lysosomal storage disorders. Included in the present disclosure is a method for the modulation of cholesterol recycling. Further, the disclosure relates to conditions such as Niemann-Pick type C disease, Farber disease, Niemann-Pick type A disease, Wolman disease and Tay Sachs disease. Further included in the present disclosure is a method for treating lysosomal storage disorders comprising the administration of delta tocopherol. Further included in the present disclosure is a method for treating lysosomal storage disorders comprising the administration of delta tocopherol in combination with cyclodextrin to a patient in need thereof.