Method for treatment of cardiac disorders

• US 9,314,511 B2
• Filed: 07/12/2013
• Issued: 04/19/2016
• Est. Priority Date: 05/09/2005
• Status: Active Grant

• Current Assignee: Medtronic Incorporated (Medtronic Plc)
• Original Assignee: Medtronic Incorporated (Medtronic Plc)
• Inventors: Keimel, John G.
• Primary Examiner(s): GILBERT, ANDREW M
• Application Number:
  US13/940,453
• Publication Number:
  US 20140012230A1
• Time in Patent Office: 1,012 Days
• US Class Current: 1/1
• CPC Class Codes: A61K 31/713 Double-stranded nucleic acids or oligonucleotides
• CPC Class Codes: A61K 38/00 Medicinal preparations containing peptides peptides containing beta-lactam rings A61K31/00; cyclic dipeptides not having in their molecule any other peptide link than those which form their ring, e.g. piperazine-2,5-diones, A61K31/00; ergot alkaloids of the cyclic peptide type A61K31/48; containing macromolecular compounds having statistically distributed amino acid units A61K31/74; medicinal preparations containing antigens or antibodies A61K39/00; medicinal preparations characterised by the non-active ingredients, e.g. peptides as drug carriers, A61K47/00
• CPC Class Codes: A61K 38/47 acting on glycosyl compounds (3.2), e.g. cellulases, lactases
• CPC Class Codes: A61M 2210/125 Heart
• CPC Class Codes: A61M 39/0208 Subcutaneous access sites for injecting or removing fluids transcutaneous access sites A61M39/0247; implantable infusion devices A61M5/14276
• CPC Class Codes: A61M 39/04 having pierceable self-sealing members
• CPC Class Codes: A61M 5/14276 specially adapted for implantation
• CPC Class Codes: A61M 5/172 electrical or electronic A61M5/16804, A61M5/16831 take precedence

Abstract

The present invention is directed to systems and methods for delivering therapy for a cardiac disorder, wherein the system comprises a source for supplying a protein formulation containing a protein that is otherwise deficient in cardiac cells in a patient with a cardiac disorder, and a catheter having a proximal end and a distal end for delivering the protein formulation to the pericardial sac region of a human heart.

Claims

1. A method comprising:

delivering a therapeutic protein formulation from an implantable source through an implantable catheter directly to a pericardial sac region of a heart of a patient,

wherein the patient has a cardiac disorder caused by a protein deficiency due to a gene mutation,

wherein the therapeutic protein formulation comprises a protein in a form that is deficient in cardiac cells of the patient due to the gene mutation,

wherein the therapeutic protein formulation is delivered at a rate based on the sequence of the patient'"'"'s gene encoding the deficient protein; and

wherein the cardiac disorder and the protein deficiency are selected from;

Mucopolvsaccharidoese Hurler (MPS IH) and α

-L-iduronidase;

Schie (MPS IS) and α

-L-iduronidase;

Hunter (MPS II) and Sulfoiduranate sulfate;

Marquio syndrome (Mucopolysaccharidosis II) and β

-Galactosidase;

Maroteaux-Lamy syndrome (MPS VI) and Aryl sulfatase B;

Mucolipidoses I cell disease (Mucolipidosis II) and N-acetylglucosamine-I-phosphotransferase;

PseudoHurler polydystrophy (Mucolipidoses III) and N-acetylglucosamine-I-phosphotransferase;

Sphingolipidoses Gaucher disease Type I and Glucocerebrosidase;

Type III Glycogenosis and Amlo-1,6-glucosidase;

Acyl-CoA Dehydrogenase Deficiencies LCAD Deficiency and Long-chain acylCoA dehydrogenase;

Primary Hyperoxaluria I and Alumine;

glyoxylate amino transferase;

Primary Hyperoxaluria II and D-glyceric acid dehydrogenase;

Homocystinuria and Cystathionine;

Triose-phosphate isomerase deficiency and Triose-phosphate isomerase;

Hereditary Orotic Aciduria and Uridine-5′

-monophosphate synthase;

Amyloidosis and Prealbumin; and

Refsum'"'"'s disease and Phytanic acid α

hydroxylase.

2. The method of claim 1, wherein the protein formulation comprises at least one stabilizing agent or anti-degradation agent operable for maintaining a desired pH.

3. The method of claim 1, wherein the implantable source comprises a refillable reservoir to store the protein formulation.

4. The method of claim 1, further comprising administering an RNA interference therapy to the patient to inhibit production of a mutated protein resulting from the gene mutation.

5. The method of claim 1, wherein the rate of delivery of the therapeutic protein formulation is further based on assessment of cardiac tissue enzyme level in the patient.

6. A method comprising:

determining a rate of delivery of a therapeutic protein formulation from an implantable source through an implantable catheter directly to a pericardial sac region of a heart of a patient,

wherein the patient has a cardiac disorder caused by a protein deficiency due to a gene mutation,

wherein the therapeutic protein formulation comprises a protein in a form that is deficient in cardiac cells of the patient due to the gene mutation, and

wherein the therapeutic protein formulation is delivered at a rate based on the the sequence of the patent'"'"'s gene encoding the deficient protein;

delivering the therapeutic protein formulation from the implantable source through the implantable catheter directly to the pericardial sac region of the heart of the patient; and

wherein the cardiac disorder and the protein deficiency are selected from;

Mucopolysaccharidoese Hurler (MPS IH) and α

-L-iduronidase;

Schie (MPS IS) and α

-L-iduronidase;

Hunter (MPS II) and Sulfoiduranate sulfate;

Marquio syndrome (Mucopolysaccharidosis II) and β

-Galactosidase;

Maroteaux-Lamy syndrome (MPS VI) and Aryl sulfatase B;

Mucolipidoses I cell disease (Mucolipidosis II) and N-acetylglucosamine-I-phosphotransferase;

PseudoHurler polydystrophy (Mucolipidoses III) and N-acetylglucosamine-I-phosphotransferase;

Sphingolipidoses Gaucher disease Type I and Glucocerebrosidase;

Type III Glycogenosis and Amlo-1,6-glucosidase;

Acyl-CoA Dehydrogenase Deficiencies LCAD Deficiency and Long-chain acylCoA dehydrogenase;

Primar Hyperoxaluria I and glyoxylate amino transferase;

Primary Hyperoxaluria II and D-glyceric acid dehydrogenase;

Homocystinuria and Cystathionine;

Triose-phosphate isomerase deficiency and Triose-phosphate isomerase;

Hereditary Orotic Aciduria and Uridine-5′

-monophosphate synthase;

Amyloidosis and Prealbumin; and

Refsum'"'"'s disease and Phytanic acid α

hydroxylase.

7. The method of claim 6, wherein determining the rate of delivery of the therapeutic protein formulation is further based on assessment of cardiac tissue enzyme level in the patient.

8. A method comprising:

injecting a therapeutic protein formulation into an implantable source; and

delivering the therapeutic protein formulation from an implantable source through an implantable catheter directly to a pericardial sac region of a heart of a patient,

wherein the patient has a cardiac disorder caused by a protein deficiency due to a gene mutation,

wherein the therapeutic protein formulation comprises a protein in a form that is deficient in cardiac cells of the patient due to the gene mutation, and

wherein the therapeutic protein formulation is delivered at a rate based on the sequence of the patient'"'"'s gene encoding the deficient protein;

wherein the cardiac disorder and the protein deficiency are selected from;

Mucopolvsaccharidoese Hurler (MPS IH) and α

-L-iduronidase;

Schie (MPS IS) and α

-L-iduronidase;

Hunter (MPS II) and Sulfoiduranate sulfate;

Marquio syndrome (Mucopolysaccharidosis II) and β

-Galactosidase;

Maroteaux-Lamy syndrome (MPS VI) and Aryl sulfatase B;

Mucolipidoses I cell disease (Mucolipidosis II) and N-acetylglucosamine-I-phosphotransferase;

PseudoHurler polydystrophy (Mucolipidoses III) and N-acetylglucosamine-I-phosphotransferase;

Sphingolipidoses Gaucher disease Type I and Glucocerebrosidase;

Type III Glycogenosis and Amlo-1,6-glucosidase;

Acyl-CoA Dehydrogenase Deficiencies LCAD Deficiency and Long-chain acylCoA dehydrogenase;

Primary Hyperoxaluria I and Alumine;

glyoxylate amino transferase;

Primary Hyperoxaluria II and D-glyceric acid dehydrogenase;

Homocystinuria and Cystathionine;

Triose-phosphate isomerase deficiency and Triose-phosphate isomerase;

Hereditary Orotic Aciduria and Uridine-5′

-monophosphate synthase;

Amyloidosis and Prealbumin; and

Refsum'"'"'s disease and Phytanic acid α

hydroxylase.

9. The method of claim 8, wherein the rate of delivery of the therapeutic protein formulation is further based on assessment of cardiac tissue enzyme level in the patient.