Cell Therapy News Volume 12.34 | Aug 29 2011

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    Cell Therapy News 12.34, August 29, 2011

         In this issue: Science | Policy | Business | NIH | CBER | Regulatory | Events
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    TOP STORY
    Gene-Therapy Successes Spur Hope for Embattled Field
    Two studies show that 13 of 16 children treated with gene therapy for severe combined immune deficiency have had their immune systems restored, and one is in remission for leukemia that developed due to the gene therapy treatment. [Press release from NatureNews discussing online prepublications in Science Translational Medicine]

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    SCIENCE NEWS

    Study Sheds Light on Stem Cell Role in Regenerating Fingers, Toes
    Tissue-specific adult stem cells are responsible for the ability of mammals to re-grow the tips of fingers or toes lost to trauma or surgery, say researchers. [Press release from Stanford University School of Medicine discussing online prepublication in Nature]

    Synthetic Self-Assembling Collagen for Tissue Engineering
    A team has tailored chains of amino acids so that they assemble themselves first into nanoscale fibrils, then entangle into larger fibers which in turn trap water to form a hydrogel – which mirrors the sequence of self-organization undertaken by natural collagen. [Press release from the Royal Society of Chemistry discussing online prepublication in Nature Chemistry]

    Mural Cells from Saphenous Vein Could Have Long-Term Benefits in Heart Attacks
    New research has found the transplantation of stem cells that reside in human veins can help in the recovery of a heart attack. [Press release from the University of Bristol discussing online prepublication in Circulation Research]

    Cardiac Researchers Discover Predictor of Stem Cell Efficacy in Treating Heart Failure
    Stem cell researchers discovered a factor that can serve as a predictor of stem cell development into blood vessels. [Press release from the University of Miami Miller School of Medicine discussing online prepublication in PLoS One]

    Study Will Aid Parkinson’s Research
    Scientists have for the first time generated stem cells from one of the most rapidly progressing forms of Parkinson’s disease. [Press release from the University of Edinburgh discussing online prepublication in Nature Communications]

    Controlling Cells’ Environments: A Step Toward Building Much-Needed Tissues and Organs
    With stem cells so fickle and indecisive that they make Shakespeare’s Hamlet pale by comparison, scientists described an advance in encouraging stem cells to make decisions about their fate. [Press release from the American Chemical Society discussing research presented at the 242nd National Meeting & Exposition for the American Chemical Society, Denver]

    Hair Cell-Derived Patient-Specific Heart Cells for Disease Modeling and Drug Screening 
    Hair follicle keratinocytes offer a simple and accessible route to generate patient-specific induced pluripotent stem cells with minimum inconvenience for the patients, shows study. [Press release from the European Society of Cardiology (ESC) discussing research presented at the ESC Congress 2011, Paris]

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    CURRENT PUBLICATIONS
    (Ranked by Impact Factor of the Journal)

    Germ-Layer and Lineage-Restricted Stem/Progenitors Regenerate the Mouse Digit Tip
    Here scientists show that a wide range of tissue stem/progenitor cells contribute towards the restoration of the mouse distal digit. [Nature]

    Conversion of Mouse and Human Fibroblasts into Functional Spinal Motor Neurons
    Here researchers report that the forced expression of select transcription factors is sufficient to convert mouse and human fibroblasts into induced motor neurons. [Cell Stem Cell]

    Transplantation of Human Pericyte Progenitor Cells Improves the Repair of Infarcted Heart Through Activation of an Angiogenic Program Involving MicroRNA-132
    Scientists investigated the therapeutic activity and mechanistic targets of saphenous vein-derived pericyte progenitor cells in a mouse myocardial infarction model. [Circ Res]

    Heterogeneity in Stromal Cell-Derived Factor-1 Expression Defines the Vasculogenic Potential of Adult Cardiac Progenitor Cells
    The objective of this study is to characterize and understand vasculogenic heterogeneity within c-kit+ presumptive cardiac progenitor cell populations. [PLoS One]

    Long-Term Persistence of a Polyclonal T Cell Repertoire After Gene Therapy for X-Linked Severe Combined Immunodeficiency
    Gene therapy for X-linked severe combined immunodeficiency without myelosuppressive conditioning effectively restored T cell immunity and was associated with high survival rates for up to 9 years. [Sci Transl Med]

    Hematopoietic Stem Cell Gene Therapy for Adenosine Deaminase–Deficient Severe Combined Immunodeficiency Leads to Long-Term Immunological Recovery and Metabolic Correction
    Here, six children were treated with autologous CD34-positive hematopoietic bone marrow stem and progenitor cells transduced with a conventional gamma retroviral vector encoding the human adenosine deaminase gene. [Sci Transl Med]

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    POLICY

    Government of Canada Puts Patients First with New Research Strategy
    The Honorable Leona Aglukkaq, Minister of Health announced Canada’s Strategy for Patient-Oriented Research, a transformative research initiative that places patients at the centre of health care. [Canadian Institutes of Health Research, Canada]

    HHS Tightens Financial Conflict of Interest Rules for Researchers
    The U.S. Department of Health and Human Services (HHS) issued an updated Final Rule on conflict of interest, providing a framework for identifying, managing, and ultimately avoiding investigators’ financial conflicts of interest. [U.S. Department of Health and Human Services, United States]

    Cutting Back in Academia
    Upon the request of Congress, a US National Academy of Sciences panel is drafting a plan to help state universities deal with continuing budget cuts. [US National Academy of Sciences, United States]

    Texas Med Board to Restrict Stem Cell Use
    The Texas Medical Board is seeking to put restrictions on stem cell transplants, a board member says. [Texas Medical Board, United States]

    Scientists Promised ‘One Voice’ in European Policy
    A new Brussels-based group, ScienceEurope, is now positioning itself as the scientists’ champion in the fight to sway decision-makers. [ScienceEurope, Belgium]

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    BUSINESS

    Cleveland Researchers Collaborate to Launch Phase I Clinical Trial for New Multiple Sclerosis Treatment
    A team of researchers at three landmark Cleveland institutions have come together to launch a new clinical trial of an experimental treatment for multiple sclerosis. [Cleveland Clinic Press Release]

    Grabel Receives New Human Stem Cell Research Grant
    Professor Laura Grabel has received a $750,000 grant for from The State of Connecticut Stem Cell Research Advisory Committee for her study titled “Angiogenesis of Embryonic Stem Cell Derived Hippocampus Transplants.” [Wesleyan University Press Release]

    UC Davis Awarded Five Therapy Development Stem Cell Grants
    The California Institute for Regenerative Medicine approved research planning grants for five University of California, Davis (UC Davis) Health System teams that are working to develop human clinical trials to treat illnesses such as Huntington’s disease, vascular disease, osteoporosis, HIV/AIDS and airway disease in children. [University of California, Davis School of Medicine Press Release]

    apceth Starts First Phase I/II Clinical Study on Somatic Cell Therapy for Peripheral Arterial Occlusive Disease
    apceth started its first Phase I/II clinical study on somatic cell therapy for advanced peripheral arterial occlusive disease after angioplasty. [B3C Newswire]

    Top-Line Results from Pivotal Trial of Dermagraft® for Venous Leg Ulcers
    Shire plc announced that Advanced BioHealing has concluded its Phase III pivotal trial of Dermagraft® in subjects with venous leg ulcers. Following preliminary analysis of the top-line results, the decision has been made not to pursue the venous leg ulcer indication. [Shire plc Press Release]

    Tekmira Acquires Worldwide Exclusive License to Novel RNAi Platform
    Tekmira Pharmaceuticals Corporation announced that it has obtained an exclusive, worldwide license to a novel and proprietary RNAi technology called MV-RNA (multivalent RNA) from Halo-Bio RNAi Therapeutics, Inc. [Tekmira Pharmaceuticals Corporation Press Release]

    Pluristem’s PLX Cells Receive Orphan Drug Status for Treatment of Buerger’s Disease
    Pluristem Therapeutics, Inc. announced that on August 22, 2011 the U.S. Food and Drug Administration designated Pluristem’s PLX cells orphan status for the treatment of thromboangiitis obliterans (Buerger’s disease). [Pluristem Therapeutics, Inc. Press Release]


    NIH

    NIH Releases Best Practices for Combining Qualitative and Quantitative Research
    The National Institutes of Health released recommendations or best practices for scientists conducting mixed methods health research.

    Issuance of the Final Rule – Responsibility of Applicants for Promoting Objectivity in Research for which Public Health Service Funding is Sought and Responsible Prospective Contractors (NOT-OD-11-109)

    National Cancer Institute Notice of Meeting (FR Doc No: 2011-21679)

    National Institute of Biomedical Imaging and Bioengineering; Notice of Closed Meeting (FR Doc No: 2011-21978)


    CBER

    September 20, 2011: Vaccines and Related Biological Products Advisory Committee Meeting Waivers for Conflicts of Interest

    September 20, 2011: Vaccines and Related Biological Products Advisory Committee Meeting Announcement


    REGULATORY

    FOOD AND DRUG ADMINISTRATION (United States)

    Vaccines and Related Biological Products Advisory Committee; Amendment of Notice [Docket No. FDA-2011-N-0002]

    Draft Guidance for Industry on Oversight of Clinical Investigations: A Risk-Based Approach to Monitoring; Availability [FR Doc No: 2011-21972]


    EVENTS

    NEW Infantile Neuroaxonal Dystrophy: Pathophysiology of Phospholipase Mutations in Humans and Mice
    September 1, 2011
    Webinar

    NEW Regenerative Medicine: New Approaches to Healthcare
    September 28, 2011
    New York, United States

    NEW Italian Society for Immunology and German Society of Immunology
    September 28-October 1, 2011
    Riccione, Italy


    Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.


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    Postdoctoral Scientists (North Carolina Central University)

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