Method and composition for treating hunter syndrome through cerebral lateral ventricle administration
AssigneesMedigenebio Corporation, The Green Cross Corporation
InventorJae Yeon YEO
Life of this patent
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Abstract
A pharmaceutical composition including an iduronate-2-sulfatase beta (IDS-β) may be administered into the cerebral lateral ventricle once every four weeks to treat Hunter syndrome in a subject. Compared to a single administration of the same dose of an active substance, due to repeated administrations over a long period of time, the administration exhibits superior effects in treating Hunter syndrome, and further has the following effects which cannot be anticipated from the result of a single administration: treating or restoring a damaged brain structure; and substantially treating or improving brain functions, particularly, improving memory and learning.
Claims20
-18. (canceled)
A method for treating a subject with Hunter syndrome, comprising:
- a step of administering to the subject a pharmaceutical composition comprising an iduronate-2-sulfatase beta (IDS-β),
- wherein the pharmaceutical composition is administered at an interval of 4 weeks into a cerebral lateral ventricle of the subject,
- the administering does not cause a severe adverse effect in the subject, and
- the administering decreases an accumulation of heparan sulfate (HS) in brain cells or cerebrospinal fluid of the subject decreases cellular vacuolization in brain cells of the subject;
- or inhibits expression of lysosome-associated membrane protein 2 (LAMP2) in brain cells of the subject.
-21. (canceled)
A method for treating a subject with Hunter syndrome, comprising:
- a step of administering to the subject a pharmaceutical composition comprising an iduronate-2-sulfatase beta (IDS-β),
- wherein the pharmaceutical composition is administered three times or more at an interval of 4 weeks into a cerebral lateral ventricle of the subject,
- wherein the pharmaceutical composition comprises
- about 150 mM sodium chloride;
- about 0.05 mg/ml of polysorbate 20;
- and
- about 6 mg/ml of iduronate-2-sulfatase beta (IDS-β),
- wherein the pharmaceutical composition has a pH of about 6;
- and
- wherein the administering treats or inhibits ventriculomegaly, or improves brain function in the subject.
Dependencies are inferred from the claim text.