SLOW INTRAVENTRICULAR DELIVERY

• US 20190328682A1
• Filed: 02/21/2019
• Published: 10/31/2019
• Est. Priority Date: 02/09/2006
• Status: Active Grant

• Current Assignee: Genzyme Corporation (Sanofi )
• Original Assignee: Genzyme Corporation (Sanofi )
• Inventors: DODGE, James C., PASSINI, Marco A., SHIHABUDDIN, Lamya S., CHENG, Seng H.
• Granted Patent: US 11,253,485 B2
• Time in Patent Office: Days
• CPC Class Codes: A61K 31/00 Medicinal preparations containing organic active ingredients
• CPC Class Codes: A61K 9/0024 Solid, semi-solid or solidifying implants, which are implanted or injected in body tissue compositions for intravenous administration, normal injectable solutions or dispersions for, e.g. subcutaneous administration A61K9/0019; brain implants A61K9/0085; (coated) prostheses, catheters or stents A61L
• CPC Class Codes: A61K 9/0085 Brain, e.g. brain implants; Spinal cord
• CPC Class Codes: A61P 1/16 for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
• CPC Class Codes: A61P 11/00 Drugs for disorders of the respiratory system
• CPC Class Codes: A61P 13/12 of the kidneys
• CPC Class Codes: A61P 23/00 Anaesthetics
• CPC Class Codes: A61P 25/00 Drugs for disorders of the nervous system
• CPC Class Codes: A61P 25/08 Antiepileptics; Anticonvulsants
• CPC Class Codes: A61P 25/14 for treating abnormal movements, e.g. chorea, dyskinesia
• CPC Class Codes: A61P 25/16 Anti-Parkinson drugs
• CPC Class Codes: A61P 25/28 for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
• 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 3/08 for glucose homeostasis pancreatic hormones A61P5/48
• CPC Class Codes: A61P 3/10 for hyperglycaemia, e.g. antidiabetics
• CPC Class Codes: A61P 43/00 Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
• CPC Class Codes: C12N 15/86 Viral vectors
• CPC Class Codes: C12N 2750/14143 viral genome or elements thereof as genetic vector
• CPC Class Codes: C12N 9/16 acting on ester bonds (3.1)

Abstract

Neurological diseases, including lysosomal storage diseases, can be successfully treated using intraventricular delivery of the therapeutic agents to bypass the blood-brain barrier. Similarly, diagnostic agents and anesthetic agents can be delivered to the brain in this manner. The administration can be performed slowly to achieve maximum effect. Such administration permits greater penetration of distal portions of the brain.

Claims

1-2. -2. (canceled)

3. A method for delivering an agent to a patient'"'"'s brain, the method comprising:

estimating turn-over time of cerebrospinal fluid of the patient;

selecting a rate and a total delivery time for an agent via a lateral ventricle of the brain based on the turn-over time;

setting a pump to deliver the agent at said selected rate for said total delivery time, wherein the rate delivers a single dose of the agent for a time greater than or equal to 50% of the turn-over time,

wherein the agent is an enzyme that is deficient in a lysosomal storage disease.

8. The method of claim 3 wherein the rate delivers a single dose of the agent for a time greater than or equal to 100% of the estimated turn-over time.

10. The method of claim 3 wherein the rate delivers a single dose of the agent for a time greater than or equal to 150% of the estimated turn-over time.

23. The method of claim 3, wherein the agent accesses the serum.

32. The method of claim 3, wherein the agent is sphingomyelinase.

33. The method of claim 3, wherein the lysosomal storage disease is Niemann-Pick B disease.

57. The method of claim 33, wherein the agent is acid sphingomyelinase.

4. A method for delivering an agent to a patient'"'"'s brain, the method comprising:

estimating turn-over time of cerebrospinal fluid of the patient;

selecting a rate and a total delivery time for an agent via a lateral ventricle of the brain based on the turn-over time; and

delivering the agent to the patient at said selected rate for said total delivery time, wherein the rate delivers a single dose of the agent for a time greater than or equal to 50% of the turn-over time,

wherein the agent is an enzyme that is deficient in a lysosomal storage disease.

9. The method of claim 4 wherein the rate delivers a single dose of the agent for a time greater than or equal to 100% of the estimated turn-over time.

11. The method of claim 4 wherein the rate delivers a single dose of the agent for a time greater than or equal to 150% of the estimated turn-over time.

52. The method of claim 4, wherein the agent is delivered using an implantable pump.

54. The method of claim 4, wherein the agent accesses the serum.

55. The method of claim 4, wherein the agent is sphingomyelinase.

56. The method of claim 4, wherein the lysosomal storage disease is Niemann-Pick B disease.

58. The method of claim 56, wherein the agent is acid sphingomyelinase.

5-7. -7. (canceled)

12-22. -22. (canceled)

24-31. -31. (canceled)

34-51. -51. (canceled)

53. (canceled)