Methods and pharmaceutical compositions for treating peripheral demyelinating diseases

Patent metadata

Field	Value
Publication number	US-10758548-B2
Application number	US-201615572001-A
Country	US
Kind code	B2
Filing date	May 19, 2016
Priority date	May 20, 2015
Publication date	Sep 1, 2020
Grant date	Sep 1, 2020

Abstract

The present invention relates to methods and pharmaceutical compositions for treating peripheral demyelinating diseases. In particular the present invention relates to a method of treating a peripheral demyelinating disease in a subject in need thereof comprising administering to the subject a therapeutically effective amount of an inhibitor of VDAC1 activity or expression.

First claim

The invention claimed is:

1. A method of treating a peripheral demyelinating disease in a subject in need thereof comprising administering to the subject a therapeutically effective amount of an inhibitor of VDAC1 activity or expression in Schwann cells,
wherein said therapeutically effective amount is sufficient to inhibit VDAC1 activity or expression in said Schwann cells, and
wherein said peripheral demyelinating disease is selected from the group consisting of Refsum's disease, Abetalipoproteinemia, Tangier disease and Dejerine-Sottas syndrome.

2. The method of claim 1 wherein the peripheral demyelinating disease is hereditary.

3. The method of claim 1 wherein the inhibitor of VDAC1 activity is olesoxime.

4. The method of claim 1 wherein the inhibitor of VDAC1 expression is a siRNA.

5. The method of claim 1 wherein the inhibitor of VDAC1 activity is a small organic molecule.

Assignees

• Inserm Institut National De La Rech Medicale
• Univ Montpellier
• Inst Nat Sante Rech Med

Inventors

• Tricaud Nicolas
• Gonzalez Sergio

Classifications

• A61K31/713

  Double-stranded nucleic acids or oligonucleotides · CPC title

• A61K31/575 Primary

  substituted in position 17 beta by a chain of three or more carbon atoms, e.g. cholane, cholestane, ergosterol, sitosterol · CPC title

• A61P25/02

  for peripheral neuropathies · CPC title

• G01N33/5008

  for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics · CPC title