US2009123451A1PendingUtilityA1

Slow intraventricular delivery

62
Assignee: GENZYME CORPPriority: Feb 9, 2006Filed: Aug 7, 2008Published: May 14, 2009
Est. expiryFeb 9, 2026(expired)· nominal 20-yr term from priority
Inventors:James DodgeMarco A. PassiniLamya ShihabuddinSeng H. Cheng
A61P 3/08A61P 3/10A61P 43/00A61P 25/08A61P 25/00A61P 25/14A61P 3/00A61P 25/16A61P 25/28A61P 11/00A61P 23/00A61P 1/16A61P 13/12A61K 9/0024A61K 31/00A61K 9/0085C12N 2750/14143C12N 15/86C12N 9/16
62
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Claims

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

exact text as granted — not AI-modified
1 . A method of delivering an agent to a patient's brain, the method comprising:
 administering the agent to the patient via a lateral ventricle of the brain at a rate such that the administration of a single dose consumes more than two hours.   
   
   
       2 . A method of delivering an agent to a patient's brain, the method comprising:
 administering the agent to the patient via a lateral ventricle of the brain at a rate such that the administration of a single dose consumes at least 50% of the turn-over time of the cerebrospinal fluid in the patient.   
   
   
       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.   
   
   
       4 . A method of 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;   delivering the agent to the patient at said selected rate for said total delivery time.   
   
   
       5 . A method of delivering an agent to a patient's brain, the method comprising:
 administering the agent to the patient via a lateral ventricle of the brain at a rate such that the administration of a single dose continues at least until the agent is detectable in serum of the patient.   
   
   
       6 . The method of  claim 3  wherein the rate delivers a single dose of the agent for a time greater than or equal to 50% of the estimated turn-over time. 
   
   
       7 . The method of  claim 4  wherein the rate delivers a single dose of the agent for a time greater than or equal to 50% of the estimated turn-over time. 
   
   
       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. 
   
   
       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. 
   
   
       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. 
   
   
       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. 
   
   
       12 . The method of  claim 2  wherein the administration consumes at least 100% of the turn-over time. 
   
   
       13 . The method of  claim 2  wherein the administration consumes at least 150% of the turn-over time. 
   
   
       14 . The method of  claim 2  wherein the administration consumes at least 200% of the turn-over time. 
   
   
       15 . The method of  claim 2  wherein the administration consumes at least 250% of the turn-over time. 
   
   
       16 . The method of  claim 2  wherein the agent accesses the third ventricle. 
   
   
       17 . The method of  claim 2  wherein the agent accesses the Aqueduct of Sylvius. 
   
   
       18 . The method of  claim 2  wherein the agent accesses the fourth ventricle. 
   
   
       19 . The method of  claim 2  wherein the agent accesses Foramina of Lushka. 
   
   
       20 . The method of  claim 2  wherein the agent accesses the Foramina of Magendie. 
   
   
       21 . The method of  claim 2  wherein the agent accesses the spinal cord. 
   
   
       22 . The method of  claim 2 , wherein the agent accesses the subarachnoid space. 
   
   
       23 . The method of  claim 2  wherein the agent accesses the serum. 
   
   
       24 - 26 . (canceled) 
   
   
       27 . The method of  claim 1 ,  2 ,  3 ,  4 , or  5  wherein the agent is an enzyme. 
   
   
       28 . The method of  claim 1 ,  2 ,  3 ,  4 , or  5  wherein the agent is an enzyme that is deficient in a lysosomal storage disease. 
   
   
       29 - 31 . (canceled) 
   
   
       32 . The method of  claim 2  wherein the agent is sphingomyelinase. 
   
   
       33 . The method of  claim 2  wherein the patient has Niemann-Pick B disease. 
   
   
       34 . The method of  claim 2  wherein the agent is alpha-L-iduronidase. 
   
   
       35 . The method of  claim 2  wherein the patient has Hurler syndrome. 
   
   
       36 . The method of  claim 2  wherein the patient has Gaucher's disease. 
   
   
       37 . The method of  claim 2  wherein the agent is glucocerebrosidase. 
   
   
       38 . The method of  claim 2 , wherein the patient has Fabry disease 
   
   
       39 . The method of  claim 2 , wherein the agent is alpha-galactosidase A. 
   
   
       40 . The method of  claim 2  wherein the patient has Pompe disease. 
   
   
       41 . The method of  claim 2  wherein the agent is acid maltase. 
   
   
       42 . The method of  claim 2 , wherein the patient has Tay-Sachs disease. 
   
   
       43 . The method of  claim 2  wherein the agent is hexosaminidase. 
   
   
       44 . The method of  claim 2  wherein the patient has Glycogen storage disease type II. 
   
   
       45 . The method of  claim 2  wherein the agent is alpha-glucosidase. 
   
   
       46 . The method of  claim 1  wherein the rate is such that the administration of a single dose consumes more than four hours. 
   
   
       47 . The method of  claim 1  wherein the rate is such that the administration of a single dose consumes more than six hours. 
   
   
       48 . The method of  claim 1  wherein the rate is such that the administration of a single dose consumes more than eight hours. 
   
   
       49 . The method of  claim 1  wherein the rate is such that the administration of a single dose consumes more than ten hours. 
   
   
       50 . The method of  claim 2  wherein the agent is delivered using a catheter. 
   
   
       51 . The method of  claim 2  wherein the agent is delivered using a pump. 
   
   
       52 . The method of  claim 2  wherein the agent is delivered using an implantable pump. 
   
   
       53 . (canceled)

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