WO2019173394A1 – September 12, 2019 – COMPOSITIONS AND METHODS FOR ACTIVATING SIGNALING THROUGH THE CB1 CANNABINOID RECEPTOR FOR TREATING AND PREVENTING DISEASES AND DISORDERS CHARACTERIZED BY ABNORMAL CELLULAR ACCUMULATION OF SPHINGOLIPIDS SUCH AS SPHINGOMYELIN

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Inventors :

LEDESMA, Maria Dolores - c/o Wylder Nation Foundation, 8924 E. Pinnacle Peak Road G5-574, Scottsdale, Arizona 85255; BARTOLL, Adrian - c/o Wylder Nation Foundation, 8924 E. Pinnacle Peak Road G5-574, Scottsdale, Arizona 85255; SCHUCHMAN, Edward H. - c/o Wylder Nation Foundation, 8924 E. Pinnacle Peak Road G5-574, Scottsdale, Arizona 85255

Owner :

WYLDER NATION FOUNDATION

Application Number :

WOUS19020828

Document Number :

WO2019173394A1

Priority Date :

March 5, 2018

Filing Date :

March 5, 2019

Date of Grant/ Publication :

September 12, 2019

Class :

A61K45 / 06; A61K31 / 27; A61P43 / 00; A61K31 / 185; A61K31 / 454; A61K31 / 4545; A61K31 / 505; A61K31 / 4709

Abstract

The present invention provides, inter alia , compositions and methods for using CB1 cannabinoid receptor agonists, or other compounds capable of increasing endocannabinoids or endocannabinoid signaling, for treating and preventing lysosomal storage disorders in which lipid storage occurs (including, e.g., disorders associated with sphingomyelin accumulation). In particular embodiments, the present invention provides compositions and methods for treating such lysosomal storage disorders with one or more fatty acid amide hydrolase inhibitor alone or in combination with one or more additional agent.

Claim(s)

1. A method comprising modulating cellular sphingomyelin (SM) levels in a subject that has a lysosomal storage disease or disorder in which sphingomyelin (SM) accumulates comprising, activating the CB1 cannabinoid receptor.;
21. A method of inhibiting the cellular accumulation of sphingomyelin (SM) in a subject that has Acid Sphingmyelinase Deficiency (ASMD) comprising, administering to the subject a fatty acid amide hydrolase (FAAHi) inhibitor.;
22. A method of inhibiting a loss of motor skills, cognitive decline, and/or systemic organ pathology associated with ASMD comprising, administering to a subject a fatty acid amide hydrolase (FAAHi) inhibitor.;
23. A method of inhibiting the cellular accumulation of at least one sphingolipid in a subject that has a disease or disorder characterized by aberrant cellular sphingolipid concentrations comprising, administering to a subject a fatty acid amide hydrolase (FAAHi) inhibitor.;
71. A method of inhibiting the accumulation of sphingomyelin (SM) in the brain of a subject that has Niemann Pick disease type A (???) comprising, activating neutral sphingomyelinase.;
72. A method of decreasing sphingomyelin (SM) levels in the brain of a subject that has Niemann Pick disease type A (???) comprising, activating neutral sphingomyelinase.;
109. A method of inhibiting the cellular accumulation of sphingomyelin (SM) in a subject that has lysosomal storage disease or other disorder in which sphingomyelin (SM) accumulates comprising, activating the CB1 cannabinoid receptor.;
119. A method of inhibiting the accumulation of sphingomyelin (SM) in the brain of a subject that has Niemann Pick disease type A (???) comprising, administering to a subject a fatty acid amide hydrolase (FAAHi) inhibitor.;
120. A method of inhibiting the cellular accumulation of sphingomyelin (SM) in a subject that has a lysosomal storage disorder or other disorder in which sphingomyelin (SM) accumulates comprising, administering to a subject a fatty acid amide hydrolase (FAAHi) inhibitor.;
121. A combination therapy for treating a lipid storage disorder comprising a first agent that is a FAAHi and a second agent.;
125. A method comprising of modulating cellular sphingomyelin (SM) levels in a subject that has a lysosomal storage disease or disorder in which sphingomyelin (SM) accumulates comprising, increasing the levels of one or more cannabinoid in the subject.;
126. A method of inhibiting a loss of motor skills, cognitive decline, and/or systemic organ pathology associated with a disease or disorder in which sphingomyelin (SM) accumulates comprising, increasing the levels of one or more cannabinoid in the subject.;
127. A method of treating loss of motor skills, cognitive decline, and/or systemic organ pathology associated with a disease or disorder in which sphingomyelin (SM) accumulates comprising, increasing the levels of one or more cannabinoid in the subject.;
128. A method of treating a disease or disorder in which sphingomyelin (SM) accumulates comprising, increasing the levels of one or more cannabinoid in the subject.;
142. A composition comprising an agent capable of activating the CB1 cannabinoid receptor, for use in a method of treating a disease or disorder in which sphingomyelin (SM) accumulates.

Summary

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