Methods for diagnosing, prognosing and treating muscular dystrophy

• US 9,867,866 B2
• Filed: 08/09/2013
• Issued: 01/16/2018
• 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, Wuebbles, Ryan, Van Ry, Pam
• Primary Examiner(s): Xie, Xiaozhen
• Application Number:
  US14/419,063
• Publication Number:
  US 20150196618A1
• Time in Patent Office: 1,621 Days
• Field of Search: None
• 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 drug screening with animal cells G01N33/5008
• 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. Also disclosed are methods of determining the effectiveness of an agent for the treatment of muscular dystrophy. 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. Also disclosed are methods of increasing or maintaining muscle strength and/or bone density in a subject by administering an effective amount of a Galectin-1 composition, Galectin-3 composition or a combination thereof to the subject in need thereof. Methods of preventing, inhibiting and/or reducing muscle loss and/or bone loss in a subject by administering an effective amount of a Galectin-1 composition, Galectin-3 composition or a combination thereof to the subject in need thereof are disclosed.

Claims

1. A method of increasing sarcolemmal stabilizing dystroglycan complex for increasing or maintaining muscle strength, muscle density, and/or bone density in a subject suffering from Duchenne muscular dystrophy (DMD), merosin deficient congenital muscular dystrophy Type 1A (MDC1A), limb-girdle muscular dystrophy (LGMD), facioscapulohumeral muscular dystrophy (FHMD), or Beckers muscular dystrophy (BMD), the method comprising systemically administering an effective amount of a Galectin-1 composition comprising a native mammalian Galectin-1 protein or a recombinant Galectin-1 protein having a full-length wild-type amino acid sequence, to the subject with DMD, MDC1A, LGMD, FHMD, or BMD and in need thereof, wherein the effective amount increases sarcolemmal stabilizing dystroglycan complex, thereby increasing or maintaining muscle strength, muscle density, and/or bone density.

2. The method of claim 1, wherein the subject has Duchenne muscular dystrophy.

3. The method of claim 1, wherein the Galectin-1 composition comprises a native mammalian Galectin-1 protein or a recombinant Galectin-1 protein having a full-length wild-type amino acid sequence and a carrier.

4. The method of claim 3, wherein the Galectin-1 composition is administered intramuscularly.

5. The method of claim 3, wherein the Galectin-1 composition is administered intraperitoneally.

6. The method of claim 3, wherein systemic administration is intravenous.

7. The method of claim 1, wherein the effective amount is systemically administered at least once daily.

8. The method of claim 7, wherein at least 1 mg of the Galectin-1 composition is administered systemically at least once daily.

9. A method of increasing sarcolemmal stabilizing dystroglycan complex for preventing, inhibiting and/or reducing muscle loss and/or bone loss in a subject suffering from Duchenne muscular dystrophy (DMD), merosin deficient congenital muscular dystrophy Type 1A (MDC1 A), limb-girdle muscular dystrophy (LGMD), facioscapulohumeral muscular dystrophy (FHMD), or Beckers muscular dystrophy (BMD), the method comprising systemically administering an effective amount of a Galectin-1 composition comprising a native mammalian Galectin-1 protein or a recombinant Galectin-1 protein having a full-length wild-type amino acid sequence, to the subject with DMD, MDC1A, LGMD, FHMD, or BMD and in need thereof, wherein the effective amount increases sarcolemmal stabilizing dystroglycan complex, thereby preventing, inhibiting and/or reducing muscle loss and/or bone loss.

10. The method of claim 9, wherein the subject has Duchenne muscular dystrophy.

11. The method of claim 9, wherein the Galectin-1 composition comprises a native mammalian Galectin-1 protein or a recombinant Galectin-1 protein having a full-length wild-type amino acid sequence and a carrier.

12. The method of claim 11, wherein the Galectin-1 composition is administered by intramuscular or intraperitoneal administration.

13. The method of claim 11, wherein systemic administration is intravenous.

14. The method of claim 9, wherein the effective amount is systemically administered at least once daily.

15. The method of claim 14, wherein at least 1 mg of the Galectin-1 composition is administered systemically at least once daily.