Method for treating amyloidosis

• US 20070265334A1
• Filed: 02/16/2007
• Published: 11/15/2007
• Est. Priority Date: 03/29/1993
• Status: Active Grant

• Current Assignee: BHI Limited Partnership
• Original Assignee: Neurochem (International) Limited
• Inventors: Szarek, Walter, Kisilevsky, Robert, Weaver, Donald
• Granted Patent: US 7,754,761 B2
• Time in Patent Office: Days
• US Class Current: 514/465
• CPC Class Codes: A61K 31/095 Sulfur, selenium, or tellurium compounds, e.g. thiols
• CPC Class Codes: A61K 31/185 Acids; Anhydrides, halides or salts thereof, e.g. sulfur acids, imidic, hydrazonic or hydroximic acids hydroxamic acids A61K31/16; peroxy acids A61K31/327
• CPC Class Codes: A61K 31/255 of sulfoxy acids or sulfur analogues thereof
• CPC Class Codes: A61K 31/34 having five-membered rings with one oxygen as the only ring hetero atom, e.g. isosorbide
• CPC Class Codes: A61K 31/41 having five-membered rings with two or more ring hetero atoms, at least one of which being nitrogen, e.g. tetrazole
• CPC Class Codes: A61K 31/70 Carbohydrates; Sugars; Derivatives thereof sorbitol A61K31/047
• CPC Class Codes: A61K 47/51 the non-active ingredient being a modifying agent
• CPC Class Codes: A61K 47/549 Sugars, nucleosides, nucleotides or nucleic acids
• CPC Class Codes: A61K 47/55 the modifying agent being also a pharmacologically or therapeutically active agent, i.e. the entire conjugate being a codrug, i.e. a dimer, oligomer or polymer of pharmacologically or therapeutically active compounds
• CPC Class Codes: A61K 47/555 pre-targeting systems involving an organic compound, other than a peptide, protein or antibody, for targeting specific cells
• CPC Class Codes: A61K 47/58 obtained by reactions only involving carbon-to-carbon unsaturated bonds, e.g. poly[meth]acrylate, polyacrylamide, polystyrene, polyvinylpyrrolidone, polyvinylalcohol or polystyrene sulfonic acid resin
• CPC Class Codes: A61P 3/00 Drugs for disorders of the metabolism of the blood or the extracellular fluid A61P7/00
• CPC Class Codes: A61P 43/00 Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
• CPC Class Codes: C07C 305/10 being further substituted by singly-bound oxygen atoms
• CPC Class Codes: C07C 309/05 containing at least two sulfo groups bound to the carbon skeleton
• CPC Class Codes: C07D 491/04 Ortho-condensed systems
• CPC Class Codes: C07H 11/00 Compounds containing saccharide radicals esterified by inorganic acids; Metal salts thereof halo-sugars C07H5/02; thio-, seleno-, or telluro-sugars C07H5/08
• CPC Class Codes: C07H 15/04 attached to an oxygen atom of the saccharide radical

Abstract

Therapeutic compounds and methods for inhibiting amyloid deposition in a subject, whatever its clinical setting, are described. Amyloid deposition is inhibited by the administration to a subject of an effective amount of a therapeutic compound comprising an anionic group and a carrier molecule, or a pharmaceutically acceptable salt thereof, such that an interaction between an amyloidogenic protein and a basement membrane constituent is inhibited. Preferred anionic groups are sulfonates and sulfates. Preferred carrier molecules include carbohydrates, polymers, peptides, peptide derivatives, aliphatic groups, alicyclic groups, heterocyclic groups, aromatic groups and combinations thereof.

Claims

1. A method for treating amyloidosis in a subject comprising orally administering to the subject an effective amount of a therapeutic compound, the therapeutic compound comprising at least one sulfonate group covalently attached to a carrier molecule, or a pharmaceutically acceptable salt thereof.

3. The compound of claim 1, wherein the pharmaceutically acceptable salt is a sodium salt.

4. A pharmaceutical composition comprising an effective amount of the compound of claim 1, and a pharmaceutically acceptable vehicle.

5. The pharmaceutical composition of claim 4, wherein said composition is formulated for oral administration.

6. The pharmaceutical composition of claim 4, wherein said composition is formulated for the treatment of amyloidosis.

2. A compound selected from the group consisting of:

7. A method for the prevention or treatment of an amyloid-related disease, comprising administering the compound of claim 2 to a subject.

8. The method of claim 7, wherein the subject is a human.

9. The method of claim 7, wherein the compound is administered orally.

10. A method for inhibiting amyloid deposition in a subject, comprising administering to the subject an effective amount of the compound of claim 2.