WO2009111030A1 - Use of mesenchymal stem cells for treating genetic diseases and disorders - Google Patents

Use of mesenchymal stem cells for treating genetic diseases and disorders Download PDF

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Publication number
WO2009111030A1
WO2009111030A1 PCT/US2009/001390 US2009001390W WO2009111030A1 WO 2009111030 A1 WO2009111030 A1 WO 2009111030A1 US 2009001390 W US2009001390 W US 2009001390W WO 2009111030 A1 WO2009111030 A1 WO 2009111030A1
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WO
WIPO (PCT)
Prior art keywords
gene
mesenchymal stem
stem cells
cells
mscs
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.)
Ceased
Application number
PCT/US2009/001390
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English (en)
French (fr)
Inventor
Timothy R. Varney
Charles R. Mills
Alla Danilkovitch
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.)
Osiris Therapeutics Inc
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Osiris Therapeutics Inc
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
Priority to JP2010549663A priority Critical patent/JP6037597B2/ja
Priority to CA2717498A priority patent/CA2717498A1/en
Priority to CN2009801155331A priority patent/CN102014936A/zh
Priority to MX2010009767A priority patent/MX2010009767A/es
Priority to NZ587809A priority patent/NZ587809A/xx
Priority to AU2009220137A priority patent/AU2009220137A1/en
Priority to BRPI0909817A priority patent/BRPI0909817A2/pt
Priority to EP09717978A priority patent/EP2262513A1/en
Application filed by Osiris Therapeutics Inc filed Critical Osiris Therapeutics Inc
Publication of WO2009111030A1 publication Critical patent/WO2009111030A1/en
Priority to US12/874,796 priority patent/US20100330052A1/en
Anticipated expiration legal-status Critical
Priority to US13/733,550 priority patent/US20130121975A1/en
Priority to US14/330,084 priority patent/US20140322180A1/en
Ceased legal-status Critical Current

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0652—Cells of skeletal and connective tissues; Mesenchyme
    • C12N5/0662—Stem cells
    • C12N5/0663—Bone marrow mesenchymal stem cells (BM-MSC)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
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    • 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
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • A—HUMAN NECESSITIES
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    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P35/00—Antineoplastic agents
    • A61P35/02—Antineoplastic agents specific for leukemia
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K2035/124—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells the cells being hematopoietic, bone marrow derived or blood cells

Definitions

  • the present technology generally relates to mesenchymal stem cells. More particularly, the presently described technology relates to the use of mesenchymal stem cells for treating genetic diseases and disorders. Still more particularly, the present technology relates to the use of mesenchymal stem cells for treating genetic diseases or disorders that are characterized by inflammation of at least one tissue and/or at least one organ.
  • Amyotrophic lateral sclerosis is a neurological disorder characterized by progressive degeneration of motor neuron cells in the spinal cord and brain, which results ultimately in paralysis and death.
  • the SOD1 gene (or ALS1 gene) is associated with many cases of familial ALS (See, e.g., Nature, vol. 362:59-62). Again not wanting to be bound by any particular theory, it is believed that the enzyme coded for by SOD1 removes superoxide radicals by converting them into non-harmful substances. Defects in the action of SOD1 result in -cell death due to excess levels of superoxide radicals.
  • the host mesenchymal stem cell population is reduced by any of a variety of means known to those skilled in the art, including, but not limited to those recited herein above.
  • Host tissue then may be repopulated by administration of the donor MSCs. Following administration of the donor MSCs, the host tissue MSC population may comprise greater than 50% donor or exogenously-derived cells. Alternatively, the host tissue MSC population may comprise greater than 80% donor or exogenously-derived cells. Alternatively, substantially all of the repopulated host tissue MSCs may be of donor origin or exogenously-derived.
  • the donor MSCs may be allogeneic to the host.
  • the donor MSCs may be human leukocyte antigen (HLA) matched or mismatched to the host.
  • HLA human leukocyte antigen
  • the donor MSCs may be partially HLA-mismatched to the host.
  • the donor and host may be non-identical siblings.
  • allogeneic donor MSCs including donor MSCs that are partially HLA-mismatched to the host, may increase the engraftment rate and persistence of donor MSCs under certain circumstances where donor hematopoietic stem cells are co-administered with MSCs to the patient.
  • Example 1 Mesenchymal Stem Cells for Treatment of Cystic Fibrosis
  • a rat model of bone marrow transplant following irradiation is being used to test the hypothesis that either intravenous (IV) or intraosseous (IO) MSC delivery, concurrently with a bone marrow transplant, will result in engraftment following ablative procedures.
  • the protocol also was designed to gain a preliminary comparative measure of the relative success of the two MSC delivery procedures.
  • MSC population replacement as a treatment for Wilson's disease can be evaluated in human patients in the following manner.
  • the patient is given an intravenous infusion or an intraosseous injection of MSCs (2.5x10 6 cells/ml,) in Plasma LyteA saline solution (Baxter) to which has been added DMSO at 3.75% vol. /vol. and human serum albumin at 1.875% wt./vol.
  • the infusion is continued until the patient receives 2 million MSCs per kilogram of body weight.

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  • Health & Medical Sciences (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Neurology (AREA)
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  • Developmental Biology & Embryology (AREA)
  • Immunology (AREA)
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  • Wood Science & Technology (AREA)
  • Cell Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Rheumatology (AREA)
  • Diabetes (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Psychology (AREA)
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  • Biophysics (AREA)
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PCT/US2009/001390 2006-01-12 2009-03-04 Use of mesenchymal stem cells for treating genetic diseases and disorders Ceased WO2009111030A1 (en)

Priority Applications (11)

Application Number Priority Date Filing Date Title
BRPI0909817A BRPI0909817A2 (pt) 2008-03-05 2009-03-04 uso de células-tronco mesenquimais para tratamento de doenças e distúrbios genéticos
CN2009801155331A CN102014936A (zh) 2008-03-05 2009-03-04 间充质干细胞用于治疗遗传疾病和病症的用途
MX2010009767A MX2010009767A (es) 2008-03-05 2009-03-04 Uso de celulas madre mesenquimales para tratar trastornos y enfermedades geneticas.
NZ587809A NZ587809A (en) 2008-03-05 2009-03-04 Use of mesenchymal stem cells expressing cd73 and/or cd105 for treating genetic diseases and disorders
AU2009220137A AU2009220137A1 (en) 2008-03-05 2009-03-04 Use of mesenchymal stem cells for treating genetic diseases and disorders
JP2010549663A JP6037597B2 (ja) 2008-03-05 2009-03-04 遺伝子疾患および障害を治療するための間葉系幹細胞の使用
CA2717498A CA2717498A1 (en) 2008-03-05 2009-03-04 Use of mesenchymal stem cells for treating genetic diseases and disorders
EP09717978A EP2262513A1 (en) 2008-03-05 2009-03-04 Use of mesenchymal stem cells for treating genetic diseases and disorders
US12/874,796 US20100330052A1 (en) 2006-01-12 2010-09-02 Use of Mesenchymal Stem Cells for Treating Genetic Diseases and Disorders
US13/733,550 US20130121975A1 (en) 2006-01-12 2013-01-03 Use of mesenchymal stem cells for completely repopulating host tissue
US14/330,084 US20140322180A1 (en) 2006-01-12 2014-07-14 Use of mesenchymal stem cells for completely repopulating host tissue

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US12/042,487 US20080286249A1 (en) 2006-01-12 2008-03-05 Use of mesenchymal stem cells for treating genetic diseases and disorders
US12/042,487 2008-03-05

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
US12/042,487 Continuation US20080286249A1 (en) 2006-01-12 2008-03-05 Use of mesenchymal stem cells for treating genetic diseases and disorders

Related Child Applications (1)

Application Number Title Priority Date Filing Date
US12/874,796 Continuation US20100330052A1 (en) 2006-01-12 2010-09-02 Use of Mesenchymal Stem Cells for Treating Genetic Diseases and Disorders

Publications (1)

Publication Number Publication Date
WO2009111030A1 true WO2009111030A1 (en) 2009-09-11

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PCT/US2009/001390 Ceased WO2009111030A1 (en) 2006-01-12 2009-03-04 Use of mesenchymal stem cells for treating genetic diseases and disorders

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US (8) US20080286249A1 (https=)
EP (1) EP2262513A1 (https=)
JP (1) JP6037597B2 (https=)
CN (1) CN102014936A (https=)
AU (1) AU2009220137A1 (https=)
BR (1) BRPI0909817A2 (https=)
CA (1) CA2717498A1 (https=)
MX (1) MX2010009767A (https=)
NZ (1) NZ587809A (https=)
WO (1) WO2009111030A1 (https=)

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US11013716B2 (en) 2013-03-12 2021-05-25 Hawking Biological Technology Co., Ltd. Method for providing an increased expression of telomerase, brain-derived neurotrophic factor, stromal cell-derived factor-1, CXC chemokine receptor 4, and/or immune regulatory factor of stem cell
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WO2013055476A1 (en) * 2011-09-09 2013-04-18 Anthrogenesis Corporation Treatment of amyotrophic lateral sclerosis using placental stem cells
CN103183736A (zh) * 2011-12-27 2013-07-03 北京和信非凡生物技术有限公司 一种抗clcn7蛋白的单克隆抗体及其应用
CN104042606B (zh) * 2013-03-12 2017-05-24 国钦生物科技股份有限公司 苯酞化合物的应用
EP3151847B1 (en) * 2014-06-04 2020-07-29 Cedars-Sinai Medical Center Human mesenchymal stem cells and pth for use in a method for non surgical repair of vertebral compression fractures
CN104726496B (zh) * 2015-03-27 2017-07-07 中国科学院生物物理研究所 携带人类成年早衰症基因突变的多能干细胞及制备方法
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US10463767B2 (en) 2016-04-22 2019-11-05 Vivex Biologics Group, Inc. Moldable bone composition
US9788950B1 (en) 2016-04-22 2017-10-17 Vivex Biomedical, Inc. Cohesive bone composition
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WO2018092769A1 (ja) 2016-11-15 2018-05-24 株式会社カネカ 胎児付属物に由来する間葉系幹細胞を含む細胞集団とその製造方法、及び医薬組成物
CN111406106A (zh) * 2017-11-28 2020-07-10 伊利诺伊大学理事会 多嵌合细胞以及移植疗法与免疫缺陷和遗传病症的治疗
WO2019132026A1 (ja) 2017-12-28 2019-07-04 株式会社カネカ 接着性幹細胞を含む細胞集団とその製造方法、及び医薬組成物
WO2020251020A1 (ja) 2019-06-14 2020-12-17 株式会社カネカ 間葉系細胞を含む細胞集団、それを含む医薬組成物、及び、その製造方法

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