SLOW INTRAVENTRICULAR DELIVERY

• Main Authors: CHENG SENG, MARCO PASSINI, LAMYA SHIHABUDDIN, DODGE JAMES
• Format: Patent
• Language: English; Japanese
• Published: 15.02.2018
• Subjects: HUMAN NECESSITIES; HYGIENE; MEDICAL OR VETERINARY SCIENCE; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES; SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS

PROBLEM TO BE SOLVED: To provide a method of delivering diagnostic agents and therapeutic agents which do not easily cross the blood brain barrier to the brain of patients in the diagnosis and treatment of lysosomal storage diseases (LSD) such as type B Niemann-Pick disease, Hurler syndrome, Gaucher disease, Fabry disease, Pompe disease and the like.SOLUTION: The method of delivering a pharmaceutical to the brain of a patient includes administering the pharmaceutical to the patient via the brain lateral ventricle at an administration speed taking more than two hours for a single dose. The pharmaceutical may be a diagnostic agent such as a contrast medium (e.g. X-ray impermeable, fluorescent, radioactive), or a therapeutic agent such as an enzyme (e.g. sphingomyelinase, alpha-L-iduronidase, glucocerebrosidase, alpha-galactosidase A, acid maltase, hexosaminidase, alpha-glucosidase) and a catheter or a pump such as an embeddable pump may be used to deliver the pharmaceutical.SELECTED DRAWING: None 【課題】Ｂ型ニーマン・ピック病、フルラー症候群、ゴーシェ病、ファブリー病、ポンペ病等のリソソーム蓄積症（ＬＳＤ）の診断及び治療において、血液脳関門を容易に越えない診断剤及び治療剤を患者の脳に送達する方法の提供。【解決手段】患者の脳に薬剤を送達する方法であって、単回投与が２時間を超える時間を費やすような投与の速度で、脳の側脳室を経由して患者へ薬剤を投与することを含む方法。前記薬剤は、造影剤（例：Ｘ線非透過性、蛍光性、放射性）等の診断薬であっても、酵素（例：スフィンゴミエリナーゼ、アルファ−Ｌ−イズロニダーゼ、グルコセレブロシダーゼ、アルファ−ガラクトシダーゼＡ、酸性マルターゼ、ヘキソサミニダーゼ、アルファ−グルコシダーゼ等）等の治療薬であってもよく、薬剤の送達のためにカテーテルや、埋め込み可能ポンプ等のポンプを用いてもよい。【選択図】なし