Methods for treating muscular dystrophy

• US 9,694,049 B2
• Filed: 08/10/2012
• Issued: 07/04/2017
• Est. Priority Date: 08/11/2011
• Status: Active Grant

• Current Assignee: Board of Regents Nevada System of Higher Education (State of Nevada)
• Original Assignee: Board of Regents Nevada System of Higher Education (State of Nevada)
• Inventors: Burkin, Dean, Van Ry, Pam, Wuebbles, Ryan
• Primary Examiner(s): Landsman, Robert
• Assistant Examiner(s): Hissong, Bruce D
• Application Number:
  US13/572,508
• Publication Number:
  US 20130065242A1
• Time in Patent Office: 1,789 Days
• CPC Class Codes: A61K 38/1709 from mammals
• CPC Class Codes: A61K 38/1732 Lectins
• CPC Class Codes: A61P 19/00 Drugs for skeletal disorders
• CPC Class Codes: A61P 19/02 for joint disorders, e.g. arthritis, arthrosis
• CPC Class Codes: A61P 19/08 for bone diseases, e.g. rachitism, Paget's disease
• CPC Class Codes: A61P 19/10 for osteoporosis
• CPC Class Codes: A61P 21/00 Drugs for disorders of the muscular or neuromuscular system
• CPC Class Codes: A61P 25/00 Drugs for disorders of the nervous system
• CPC Class Codes: A61P 29/00 Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
• CPC Class Codes: C07K 1/22 Affinity chromatography or related techniques based upon selective absorption processes
• CPC Class Codes: C12N 15/09 Recombinant DNA-technology
• CPC Class Codes: C12N 15/70 Vectors or expression systems specially adapted for E. coli
• CPC Class Codes: C12Q 1/6883 for diseases caused by alterations of genetic material
• CPC Class Codes: C12Q 2600/118 Prognosis of disease development
• CPC Class Codes: C12Q 2600/136 Screening for pharmacological compounds
• CPC Class Codes: C12Q 2600/158 Expression markers
• CPC Class Codes: G01N 2333/42 Lectins, e.g. concanavalin, phytohaemagglutinin
• CPC Class Codes: G01N 2333/4724 Lectins
• CPC Class Codes: G01N 2500/00 Screening for compounds of potential therapeutic value
• CPC Class Codes: G01N 2800/2878 Muscular dystrophy
• CPC Class Codes: G01N 2800/2885 : Duchenne dystrophy
• CPC Class Codes: G01N 2800/52 : Predicting or monitoring the response to treatment, e.g. for selection of therapy based on assay results in personalised medicine; Prognosis
• CPC Class Codes: G01N 33/15 : Medicinal preparations ; Physical properties thereof, e.g. dissolubility
• CPC Class Codes: G01N 33/6896 : Neurological disorders, e.g. Alzheimer's disease

Abstract

Disclosed herein are methods for diagnosing, prognosing and treating muscular dystrophy. The disclosed methods can be used to diagnosis, prognosis or treat a subject with merosin-deficient congenital muscular dystrophy Type 1A (MDC1A), limb-girdle muscular dystrophy (LGMD), facioscapulohumeral (FHMD), Beckers muscular dystrophy (BMD) or Duchenne muscular dystrophy (DMD). Also disclosed are methods of determining the effectiveness of an agent for the treatment of muscular dystrophy. In an example, a method of diagnosing or prognosing a subject with muscular dystrophy includes detecting expression of Galectin-1 or Galectin-3 in a sample obtained from the subject at risk of having or having one or more signs or symptoms associated with muscular dystrophy, thereby diagnosing or prognosing the subject with muscular dystrophy. Also provided are methods of enhancing muscle regeneration, repair, or maintenance in a subject by administering galectin, such as Galectin-1 and/or Galectin-3 to a subject in need thereof.

Claims

1. A method of decreasing muscle damage, increasing muscle repair, and/or increasing muscle function in a subject with a muscular dystrophy comprising administering systemically 100-1000 μ

g of Galectin-1, Galectin-3 or a combination thereof to the subject, thereby improving muscle function, decreasing muscle damage and/or increasing muscle repair in the subject with the muscular dystrophy.

2. The method of claim 1, wherein the method includes administering a therapeutically effective amount of Galectin-1.

3. The method of claim 1, wherein the muscular dystrophy is merosin deficient congenital muscular dystrophy Type 1A (MDC1A), limb-girdle muscular dystrophy (LGMD), facioscapulohumeral (FSHD), Beckers muscular dystrophy (BMD) or Duchenne muscular dystrophy (DMD).

4. The method of claim 3, wherein the muscular dystrophy is DMD.

5. The method of claim 1, wherein systemic administration is intravenous, intramuscular or intraperitoneal.

6. A method of decreasing muscle damage, increasing muscle repair, and/or increasing muscle function in a subject with a muscular dystrophy comprising administering systemically at least 1 mg of Galectin-1, Galectin-3 or a combination thereof to the subject, thereby improving muscle function, decreasing muscle damage and/or increasing muscle repair in the subject with the muscular dystrophy.

7. The method of claim 6, wherein the muscular dystrophy is merosin deficient congenital muscular dystrophy Type 1A (MDC1A), limb-girdle muscular dystrophy (LGMD), facioscapulohumeral (FSHD), Beckers muscular dystrophy (BMD) or Duchenne muscular dystrophy (DMD).

8. The method of claim 6, wherein the muscular dystrophy is DMD.

9. The method of claim 6, wherein systemic administration is intravenous, intramuscular or intraperitoneal.

10. A method of decreasing muscle damage, increasing muscle repair, and/or increasing muscle function in a subject with a muscular dystrophy comprising administering systemically a therapeutically effective amount of recombinant Galectin-1 protein, Galectin-3 protein or a combination thereof to the subject with muscular dystrophy thereby decreasing muscle damage, increasing muscle repair, and/or increasing muscle function in the subject with a muscular dystrophy.

11. The method of claim 10, wherein the method includes administering a therapeutically effective amount of recombinant Galectin-1 protein.

12. The method of claim 10, wherein the muscular dystrophy is merosin deficient congenital muscular dystrophy Type 1A (MDC1A), limb-girdle muscular dystrophy (LGMD), facioscapulohumeral (FSHD), Beckers muscular dystrophy (BMD) or Duchenne muscular dystrophy (DMD).

13. The method of claim 12, wherein the muscular dystrophy is DMD.

14. The method of claim 10, wherein administering comprises systemic administration of at least 100 μ

g of recombinant Galectin-1 protein, Galectin-3 protein or a combination thereof.

15. The method of claim 10, wherein the method includes administering a therapeutically effective amount of recombinant Galectin-1 protein in which the recombinant Galectin-1 protein has an amino acid sequence that shares 98% sequence identity to human Galectin-1 wild-type amino acid sequence or mouse Galectin-1 wild-type amino acid sequence.

16. The method of claim 10, wherein the method includes administering a therapeutically effective amount of recombinant Galectin-1 protein in which the recombinant Galectin-1 protein has an amino acid sequence that shares 99% sequence identity to human Galectin-1 wild-type amino acid sequence or mouse Galectin-1 wild-type amino acid sequence.

17. The method of claim 10, wherein the method includes administering a therapeutically effective amount of recombinant Galectin-1 protein in which the recombinant Galectin-1 protein has the amino acid sequence that shares 100% sequence identity to human Galectin-1 wild-type amino acid sequence or mouse Galectin-1 wild-type amino acid sequence.