CA2278507C - Method for treatment of cns-involved lysosomal storage diseases - Google Patents

Method for treatment of cns-involved lysosomal storage diseases Download PDF

Info

Publication number
CA2278507C
CA2278507C CA002278507A CA2278507A CA2278507C CA 2278507 C CA2278507 C CA 2278507C CA 002278507 A CA002278507 A CA 002278507A CA 2278507 A CA2278507 A CA 2278507A CA 2278507 C CA2278507 C CA 2278507C
Authority
CA
Canada
Prior art keywords
dideoxy
storage
glucitol
dnj
mice
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Expired - Fee Related
Application number
CA002278507A
Other languages
English (en)
French (fr)
Other versions
CA2278507A1 (en
Inventor
Frances M. Platt
Gabrielle R. Neises
Raymond A. Dwek
Terry D. Butters
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Monsanto Co
Original Assignee
Monsanto Co
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Monsanto Co filed Critical Monsanto Co
Publication of CA2278507A1 publication Critical patent/CA2278507A1/en
Application granted granted Critical
Publication of CA2278507C publication Critical patent/CA2278507C/en
Anticipated expiration legal-status Critical
Expired - Fee Related legal-status Critical Current

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7004—Monosaccharides having only carbon, hydrogen and oxygen atoms
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445—Non condensed piperidines, e.g. piperocaine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7012—Compounds having a free or esterified carboxyl group attached, directly or through a carbon chain, to a carbon atom of the saccharide radical, e.g. glucuronic acid, neuraminic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Biomedical Technology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Peptides Or Proteins (AREA)
CA002278507A 1997-01-13 1998-01-13 Method for treatment of cns-involved lysosomal storage diseases Expired - Fee Related CA2278507C (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US08/782,321 1997-01-13
US08/782,321 US5798366A (en) 1993-05-13 1997-01-13 Method for treatment of CNS-involved lysosomal storage diseases
PCT/US1998/000031 WO1998030219A1 (en) 1997-01-13 1998-01-13 Method for treatment of cns-involved lysosomal storage diseases

Publications (2)

Publication Number Publication Date
CA2278507A1 CA2278507A1 (en) 1998-07-16
CA2278507C true CA2278507C (en) 2007-08-28

Family

ID=25125672

Family Applications (1)

Application Number Title Priority Date Filing Date
CA002278507A Expired - Fee Related CA2278507C (en) 1997-01-13 1998-01-13 Method for treatment of cns-involved lysosomal storage diseases

Country Status (11)

Country Link
US (1) US5798366A (https=)
EP (2) EP1491196A3 (https=)
JP (1) JP2001508064A (https=)
AT (1) ATE268598T1 (https=)
AU (1) AU5813898A (https=)
CA (1) CA2278507C (https=)
DE (1) DE69824421T2 (https=)
DK (1) DK1007043T3 (https=)
ES (1) ES2223118T3 (https=)
PT (1) PT1007043E (https=)
WO (1) WO1998030219A1 (https=)

Families Citing this family (37)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6465488B1 (en) 1997-12-11 2002-10-15 Chancellor, Masters & Scholars Of The University Of Oxford Inhibition of glycolipid biosynthesis
US6274597B1 (en) * 1998-06-01 2001-08-14 Mount Sinai School Of Medicine Of New York University Method of enhancing lysosomal α-Galactosidase A
US6610703B1 (en) 1998-12-10 2003-08-26 G.D. Searle & Co. Method for treatment of glycolipid storage diseases
GB9909064D0 (en) 1999-04-20 1999-06-16 Oxford Glycosciences Uk Ltd Therapies
GB0100889D0 (en) 2001-01-12 2001-02-21 Oxford Glycosciences Uk Ltd Compounds
WO2000066105A2 (en) * 1999-04-30 2000-11-09 The Trustees Of Boston College Treatment of epilepsy with imino sugars
EP1196190B1 (en) 1999-07-26 2003-03-19 G.D. SEARLE & CO. Use of long-chain n-alkyl derivatives of deoxynojirimycin and a glucocerebrosidase enzyme for the manufacture of a medicament for the treatment of glycolipid storage diseases
US6770468B1 (en) 1999-09-14 2004-08-03 Genzyme Glycobiology Research Institute, Inc. Phosphodiester-α-GlcNAcase of the lysosomal targeting pathway
US6534300B1 (en) 1999-09-14 2003-03-18 Genzyme Glycobiology Research Institute, Inc. Methods for producing highly phosphorylated lysosomal hydrolases
US7629309B2 (en) 2002-05-29 2009-12-08 Zystor Therapeutics, Inc. Targeted therapeutic proteins
WO2002087510A2 (en) * 2001-04-30 2002-11-07 Symbiontics, Inc. Subcellular targeting of therapeutic proteins
US7560424B2 (en) 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted therapeutic proteins
US20040005309A1 (en) * 2002-05-29 2004-01-08 Symbiontics, Inc. Targeted therapeutic proteins
US20030072761A1 (en) * 2001-10-16 2003-04-17 Lebowitz Jonathan Methods and compositions for targeting proteins across the blood brain barrier
US20030124652A1 (en) * 2001-12-21 2003-07-03 Novazyme Pharmaceuticals, Inc. Methods of producing high mannose glycoproteins in complex carbohydrate deficient cells
US6905856B2 (en) * 2001-12-21 2005-06-14 Genzyme Glycobiology Research Institute, Inc. Soluble GlcNAc phosphotransferase
US6800472B2 (en) 2001-12-21 2004-10-05 Genzyme Glycobiology Research Institute, Inc. Expression of lysosomal hydrolase in cells expressing pro-N-acetylglucosamine-1-phosphodiester α-N-acetyl glucosimanidase
AU2003246942B2 (en) * 2002-07-17 2009-06-25 Idorsia Pharmaceuticals Ltd Piperidinetriol derivatives as inhibitors of glycosylceramide synthase
US7994198B2 (en) * 2002-07-17 2011-08-09 Actelion Pharmaceuticals Ltd. Piperidinetriol derivatives as inhibitors of glycosylceramidsynthase
AU2003302724A1 (en) * 2002-08-13 2004-07-09 Mcivor, Scott, R. Methods of using vectors to treat metabolic disorders
EP2857036B1 (en) 2003-01-31 2016-12-21 Mount Sinai School of Medicine of New York University Combination therapy for treating protein deficiency disorders
GB0313677D0 (en) * 2003-06-13 2003-07-16 Oxford Glycosciences Uk Ltd Novel compound
GB0313678D0 (en) * 2003-06-13 2003-07-16 Oxford Glycosciences Uk Ltd Novel compounds
CA2553955C (en) * 2004-02-10 2012-08-28 Zystor Therapeutics, Inc. Acid alpha-glucosidase and fragments thereof
US20050208090A1 (en) * 2004-03-18 2005-09-22 Medtronic, Inc. Methods and systems for treatment of neurological diseases of the central nervous system
DE102004029310A1 (de) * 2004-06-17 2005-12-29 Clariant Gmbh Hochkonzentrierte, wässrige Formulierungen von Oligo-und Polyestern
JP2008503590A (ja) * 2004-06-21 2008-02-07 メドトロニック・インコーポレーテッド 組成物を細胞にデリバリーするための医学用システム及び方法
US7884115B2 (en) * 2004-09-28 2011-02-08 Allergan, Inc. Methods and compositions for the treatment of pain and other neurological conditions
US8500720B2 (en) 2005-05-09 2013-08-06 Medtronic, Inc Method and apparatus for treatment of cardiac disorders
EP3782655A1 (en) 2005-05-17 2021-02-24 Amicus Therapeutics, Inc. A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives
EP2099523A2 (en) * 2006-11-13 2009-09-16 ZyStor Therapeutics , Inc. Methods for treating pompe disease
WO2008070769A1 (en) * 2006-12-06 2008-06-12 Medtronic, Inc. Intrathecal catheter
JP5627571B2 (ja) 2008-05-07 2014-11-19 バイオマリン ファーマシューティカル インコーポレイテッド リソソーム標的化ペプチドおよびその使用
WO2010056746A1 (en) * 2008-11-11 2010-05-20 Amicus Therapeutics, Inc. Therapy regimens, dosing regimens and stable medicaments for the treatment of pompe disease
EP3679942A1 (en) 2009-06-17 2020-07-15 BioMarin Pharmaceutical Inc. Formulations for lysosomal enzymes
CA2731346A1 (en) * 2010-02-09 2011-08-09 The Hospital For Sick Children Compounds for the treatment of lysosomal storage diseases
EP3318277A1 (en) * 2016-11-04 2018-05-09 Institut du Cerveau et de la Moelle Epiniere-ICM Inhibitors of glucosylceramide synthase for the treatment of motor neuron diseases

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB1555654A (en) * 1977-06-25 1979-11-14 Exxon Research Engineering Co Agricultural burner apparatus
US4065562A (en) * 1975-12-29 1977-12-27 Nippon Shinyaku Co., Ltd. Method and composition for reducing blood glucose levels
NO154918C (no) * 1977-08-27 1987-01-14 Bayer Ag Analogifremgangsmaate til fremstilling av terapeutisk aktive derivater av 3,4,5-trihydroksypiperidin.
GB2020278B (en) * 1978-05-03 1983-02-23 Nippon Shinyaku Co Ltd Moranoline dervitives
US4849430A (en) * 1988-03-09 1989-07-18 Monsanto Company Method of inhibiting virus
US5011829A (en) * 1989-06-02 1991-04-30 G. D. Searle & Co. Pharmaceutical composition and method of inhibiting virus
US5030638A (en) * 1990-02-26 1991-07-09 G. D. Searle & Co. Method of antiviral enhancement
US6291657B1 (en) * 1993-05-13 2001-09-18 Monsanto Company Deoxygalactonojirimycin derivatives

Also Published As

Publication number Publication date
EP1007043B9 (en) 2004-12-22
ATE268598T1 (de) 2004-06-15
DE69824421D1 (de) 2004-07-15
WO1998030219A1 (en) 1998-07-16
EP1491196A3 (en) 2005-03-16
DK1007043T3 (da) 2004-10-11
JP2001508064A (ja) 2001-06-19
CA2278507A1 (en) 1998-07-16
EP1007043A1 (en) 2000-06-14
AU5813898A (en) 1998-08-03
EP1007043B1 (en) 2004-06-09
PT1007043E (pt) 2004-10-29
EP1491196A2 (en) 2004-12-29
US5798366A (en) 1998-08-25
ES2223118T3 (es) 2005-02-16
DE69824421T2 (de) 2005-03-03

Similar Documents

Publication Publication Date Title
CA2278507C (en) Method for treatment of cns-involved lysosomal storage diseases
Jeyakumar et al. Glycosphingolipid lysosomal storage diseases: therapy and pathogenesis
Platt et al. Substrate reduction therapy
Schulze et al. Lysosomal lipid storage diseases
US8252789B2 (en) Compositions and methods for treating lysosomal disorders
US20150044193A1 (en) COMPOSITIONS AND METHODS FOR THE TREATMENT OF ALTERED a-SYNUCLEIN FUNCTION
Lin et al. N-Octyl-β-valienamine up-regulates activity of F213I mutant β-glucosidase in cultured cells: a potential chemical chaperone therapy for Gaucher disease
Lachmann et al. Substrate reduction therapy for glycosphingolipid storage disorders
Platt et al. Substrate reduction therapy in mouse models of the glycosphingolipidoses
JP3636363B2 (ja) 新規デオキシガラクトノジリマイシン誘導体
EP1753397A2 (en) Galantamine salts, method of producing it and nasal composition thereof
CA2482161A1 (en) Administration of acetylcholinesterase inhibitors to the cerebral spinal fluid
Bagetta et al. Tacrine-induced seizures and brain damage in LiCl-treated rats can be prevented by Nω-nitro-l-arginine methyl ester
Drenckhahn et al. Drug-induced experimental lipidosis in the nervous system
Eckhardt Pathology and current treatment of neurodegenerative sphingolipidoses
Drenckhahn et al. Experimental myopathy induced by amphiphilic cationic compounds including several psychotropic drugs
Giacobini Drugs that target cholinesterases
Kaidonis et al. N-butyldeoxynojirimycin treatment restores the innate fear response and improves learning in mucopolysaccharidosis IIIA mice
EP2311463A1 (en) Analogs of galanthamine and lycoramine as modulators of nicotinic receptors
US20190248790A1 (en) Deuterated compounds for treating fabry, gaucher, parkinson's and related diseases and conditions, and compositions and methods thereof
EP1699489B1 (en) Pharmaceutical composition of a sodium channel blocker and a selective serotonin uptake inhibitor
Walkley et al. Pathomechanisms in lysosomal storage disorders
LacCer Development of AL01211, an oral, non-brain penetrant glucosylceramide synthase inhibitor (GCSi), to treat Fabry disease
AU2014265137A1 (en) Compositions and methods for the treatment of altered alpha-synuclein function
Beck Use of Small Molecules for the Treatment of Lysosomal Storage Diseases.

Legal Events

Date Code Title Description
EEER Examination request
MKLA Lapsed
MKLA Lapsed

Effective date: 20100113