Volume 15.11 | Mar 24

Issue 15.11 March 24, 2014
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Publications | Reviews | Science | Policy | Business | NIH | CBER | Regulatory | Events | Jobs
Team Implants Human Innate Immune Cells in Mice
Overcoming a major limitation to the study of the origins and progress of human disease, researchers report that they have transplanted human innate immune cells into mouse models, which resulted in human immune responses. This study has reproduced human immune function at a level not seen previously, and could significantly improve the translation of knowledge gained from mouse studies into humans. [Press release from Yale University discussing online prepublication in Nature Biotechnology] Press Release | Abstract
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PUBLICATIONS (Ranked by impact factor of the journal)
Human Muscle-Derived Stem/Progenitor Cells Promote Functional Murine Peripheral Nerve Regeneration
Investigators demonstrated that muscle-derived stem/progenitor cells isolated from adult human skeletal muscle can adopt neuronal and glial phenotypes in vitro and ameliorate a critical-sized sciatic nerve injury and its associated defects in a murine model. [J Clin Invest] Full Article | Press Release

Image-Guided Intrathymic Injection of Multipotent Stem Cells Supports Life-Long T Cell Immunity and Facilitates Targeted Immunotherapy
Scientists showed that intrathymic injection of multipotent hematopoietic stem/progenitor cells into irradiated syngeneic or allogeneic young or aged recipients resulted in efficient and long-lasting generation of functional donor T cells. [Blood] Abstract

Intravenous Injection of a Foamy Virus Vector to Correct Canine SCID-X1
Investigators evaluated the safety and efficacy of in vivo gene therapy using a foamy virus vector for the correction of canine SCID-X1. In newborn SCID-X1 dogs, injection of a foamy virus vector expressing the human IL2RG gene resulted in an expansion of lymphocytes expressing the common gamma chain and the development of CD3+ T lymphocytes. [Blood] Abstract

Parathyroid Hormone Enhances Hematopoietic Expansion via Upregulation of Cadherin-11 in Bone Marrow Mesenchymal Stromal Cells
When parathyroid hormone-treated bone marrow mesenchymal stromal cells were subcutaneously implanted into NOD/SCID mice, the induction of hematopoietic cells was enhanced. [Stem Cells] Abstract

Mesenchymal Stem Cell Population Derived from Human Pluripotent Stem Cells Displays Potent Immunomodulatory and Therapeutic Properties
Researchers report that hESC-mesenchymal stem cells can inhibit CD83 upregulation and IL-12p70 secretion from dendritic cells and enhance regulatory T cell populations induced by IL-2. [Stem Cell Dev] Abstract

Bone Marrow Mesenchymal Stromal Cells and Olfactory Ensheathing Cells Transplantation after Spinal Cord Injury – A Morphological and Functional Comparison in Rats
Investigators compared the effects of mesenchymal stromal cell and olfactory ensheathing cell transplants at early or delayed time after a spinal cord contusion injury in the rat. Functional outcomes for locomotion, sensory perception and electrophysiological responses were assessed. [Eur J Neuro] Abstract

Combination Therapy of Human Umbilical Cord Blood Cells and Granulocyte Colony Stimulating Factor Reduces Histopathological and Motor Impairments in an Experimental Model of Chronic Traumatic Brain Injury
Researchers tested the potential of a combined therapy of human umbilical cord blood cells and granulocyte colony stimulating factor at the acute stage of traumatic brain injury (TBI) to counteract the progressive secondary effects of chronic TBI using the controlled cortical impact model. [PLoS One] Full Article | Press Release

Maximum Efficacy of Mesenchymal Stem Cells in Rat Model of Renal Ischemia-Reperfusion Injury: Renal Artery Administration with Optimal Numbers
Scientists investigated whether locally administration through renal artery can enhance the migration and therapeutic potential of mesenchymal stem cells in ischemic kidney. [PLoS One] Full Article

Allotransplantation for Patients Age 40 Years and Greater with Non-Hodgkin Lymphoma (NHL): Encouraging Progression-Free Survival
Investigators analyzed CIBMTR data on 1248 patients ≥40 years receiving reduced-intensity conditioning or non-myeloablative allogeneic hematopoietic cell transplantation for aggressive and indolent NHL. [Biol Blood Marrow Transplant] Abstract

Webinar: Brent Reynolds on Identification and Characterization of Neural Stem Cells
Stem Cells for Investigation and Treatment of Inherited Retinal Disease
The authors discuss the current literature pertaining to retinal transplantation. They focus on the use of induced pluripotent stem cells for interrogation of disease pathophysiology, analysis of drug and gene therapeutics and as a source of autologous cells for cell replacement. [Hum Mol Genet] Abstract | Full Article

Concise Review: Pluripotent Stem Cell-Based Regenerative Applications for Failing β-Cell Function
In light of emerging clinical experiences with human embryonic stem cells and approval of the first in-human trial with induced pluripotent stem cells, the authors highlight advances in β-cell regeneration strategies with a focus on pluripotent stem cell platforms in the context of translational applications. [Stem Cells Transl Med] Abstract

Visit our reviews page to see a complete list of reviews in the cell, gene and immunotherapy research field.

STEMvision(TM) Instrument
Independent Sports Medicine Study Using Cytori Cell Therapy to Treat ACL Injury Underway in Spain
Cytori Therapeutics announced the first investigator-initiated study evaluating Cytori Cell Therapy as a potential concomitant treatment for anterior-cruciate ligament (ACL) injury. [Press release from Cytori Therapeutics Inc. discussing research presented at the Football Medicine Strategies for Joint & Ligament Injuries, 23rd International Conference on Sports Rehabilitation and Traumatology, Milan] Press Release

Arrowhead Begins Dosing in Phase IIa Trial of RNAi Therapeutic ARC-520 in Chronic Hepatitis B Patients
Arrowhead Research Corporation announced that patient dosing has begun in a Phase IIa clinical trial of ARC-520, its RNAi therapeutic for the treatment of chronic hepatitis B virus infection. [Arrowhead Research Corporation] Press Release

Cell Line Development Asia 2014
UK Budget Sees Boosts for Data Science, Graphene and Cell Therapy
British scientists already know that their public funding for the next two years is frozen at £4.6 billion annually (as it has been since 2010, which for the nation’s seven research-grants agencies has meant a 10% cut in real terms over the past three years), so they did not expect anything transformative from budget. George Osborne announced £55 million over five years for a center aimed at large-scale manufacturing of cell therapies for late-stage clinical trials. [Nature News] Editorial

Irreproducibility Dogs New Reprogramming Method
Since January, scientists around the world have unsuccessfully attempted to reproduce a surprising stem cell finding that claimed simply stressing adult cells could turn them into powerful stem cells that resemble those found in early embryos. Now, ScienceInsider has learned that some of the labs involved in producing the two papers describing the work had not attempted to reproduce the technique before the papers were published. [Nature News] Editorial

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Cerebral Palsy and the Potential of Cord Blood Stem Cells
Cord Blood Registry® is partnering with two preeminent institutions, the University of Texas Health Science Center at Houston and Georgia Regents University, to establish FDA-regulated clinical trials investigating whether an intravenous infusion of a child’s own cord blood cells, banked at the time of their birth, will lessen the symptoms of cerebral palsy. [PR Newswire Association LLC] Press Release

Bioheart Announces Joint Venture with Magnum Cell Therapies
Bioheart, Inc. announced that it has entered into a joint venture with Magnum Cell Therapies. Magnum Cell Therapies’ mission is to provide the latest regenerative medicine therapies to patients suffering from degenerative diseases. [Bioheart, Inc.] Press Release

Austrianova Contracted by Nuvilex Inc. for cGMP-Compliant Cell-in-a-Box® Encapsulation of Cells
Austrianova announced that it has been contracted by Nuvilex, Inc. to produce living encapsulated cells to cGMP (Good Manufacturing Practices) standard. The two companies signed a Manufacturing Framework Agreement that covers all aspects of the production of the encapsulated live cells. [Austrianova] Press Release

Budget Unveils £55 Million Large-Scale Cell Therapy Manufacturing Center for the UK
The Cell Therapy Catapult, which is focused on the development of the UK cell therapy industry to increase the nation’s health and wealth, is to manage the new UK Cell Therapy Manufacturing Center announced in the budget. The first such UK facility, it is a recognition of the quality and potential of the UK cell therapy community, and will have £55 million of funding from 2014/15. [Cell Therapy Catapult] Press Release

$4.25 Million Grant Awarded to Rutgers Cancer Institute of New Jersey and University of Wisconsin Carbone Cancer Center to Develop Innovative Clinical Trials
Dr. DiPaola, who was just awarded a $4.25 million grant (UM1CA186716) by the National Cancer Institute of the National Institutes of Health to support a precision experimental therapeutics endeavor, will collaborate with investigators from the University of Wisconsin Carbone Cancer Center on the research that will have a nationwide reach. [Rutgers Cancer Institute of New Jersey] Press Release

UC Merced Earns Grant from California’s Stem Cell Push
Univeristy of California (UC), Merced Professor Kara McCloskey was awarded a $500,000 grant to continue tackling significant, unresolved issues in human stem cell biology, as part of an effort to enhance stem-cell research in California. [UC Merced] Press Release

New DNA-Editing Technology Spawns Bold UC Initiative
The University of California (UC), Berkeley, and UC San Francisco are launching the Innovative Genomics Initiative to lead a revolution in genetic engineering based on a new technology already generating novel strategies for gene therapy and the genetic study of disease. The Li Ka Shing Foundation has provided a $10 million gift to support the initiative, establishing the Li Ka Shing Center for Genomic Engineering and an affiliated faculty chair at UC Berkeley. [UC Health] Press Release

Pioneering Stem Cell Therapies to Be Trialed in Birmingham
University of Birmingham researchers are to lead a worldwide collaboration of scientists looking at the possibility of transplanting stem cells from one person to another to reduce inflammation in the liver. [University of Birmingham] Press Release

Cellular Biomedicine Group Launches Development Program for Asthma Stem Cell Therapy
Cellular Biomedicine Group Inc. announced the launch of their pre-clinical study on human adipose derived mesenchymal progenitor cell (haMPC) therapy for asthma. The study will evaluate the safety and efficacy of haMPCs to treat severe asthma. [GlobeNewswire, Inc.] Press Release

Regenerative Sciences Announces Knee Osteoarthritis Clinical Research Study in Chicago, Illinois
Regenerative Sciences, Inc. announced the start of a new clinical research study on the use of autologous stem cells for treating knee osteoarthritis. The study is being conducted in Chicago, Illinois in collaboration with Regenexx Physician Network Provider, Dr. Mitchell Sheinkop. [PR Newswire Association LLC] Press Release

ATL1102 for Stem Cell Mobilization Trial Commencement
Antisense Therapeutics advised that it has commenced dosing in its Phase I Stem Cell Mobilization Human Proof of Concept trial of ATL1102, the company’s second generation antisense drug targeting the VLA-4 receptor. The trial will assess the safety, tolerability and effect of ATL1102 on the release of hematopoietic stem cells into the blood when dosed alone and in combination with an existing therapy (Granulocyte Colony Stimulating Factor). [PR Newswire Association LLC] Press Release
Prospective Grant of an Exclusive Evaluation License Agreement: Pre-Clinical Evaluation and Commercial Development of Anti-Tyrosine Kinase-Like Orphan Receptor 1 Antibody-Drug Conjugates for the Treatment of Human Cancers (FR Doc No. 2014-05678)

Proposed Collection; 60-Day Comment Request; Generic Clearance for the Collection of Qualitative Feedback on Agency Service Delivery (NCI) (FR Doc No. 2014-05962)
Humanitarian Device Exemption (HDE): Questions and Answers – Draft Guidance for HDE Holders, Institutional Review Boards, Clinical Investigators, and Food and Drug Administration Staff
Food and Drug Administration (United States)

Request for Nominations for Voting and/or Nonvoting Consumer Representatives on Public Advisory Committees or Panels and Request for Notification from Consumer Organizations Interested in Participating in the Selection Process for Nominations for Voting and/or Nonvoting Consumer Representatives on Public Advisory Committees or Panels (FR Doc No. 2014-06326)
NEW NYSTEM 2014 Meeting
May 29-30, 2014
New York City, United States

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Postdoctoral Position – Gene Therapy of Lysosomal Storage Diseases (Cincinnati Children’s Hospital Medical Center)

Cell Therapy Lab Manager (Fred Hutchinson Cancer Research Center)

Stem Cell Research Technician (Van Andel Research Institute)

Postdoctoral Position – Human Stem Cell Biology and Hematopoiesis (Johns Hopkins University School of Medicine)

Director of GMP/GLP Quality Operations (University of Pennsylvania, Perelman School of Medicine)

Cell Culture Specialist – Manufacturing of Cell Therapy Products (Tisch MS Research Center of New York)

Translational Research Scientist – Hematopoietic Cellular Therapy Program (University of Chicago)

Postdoctoral Position – Cardiovascular Electrophysiology Research Involving Human ESC or iPSC-Derived Cardiomyocytes (Stanford University)

Postdoctoral Fellow – Hemostasis Gene Therapy (Children’s Hospital of Philadelphia)

Postdoctoral Fellowships – Tissue Regeneration and Molecular Cell Engineering (MD Anderson Cancer Center)

Postdoctoral Position – Epigenetic Enzymes in Breast Cancer (University of Wisconsin – Madison)

Postdoctoral Position – Tissue Engineering and Regenerative Medicine (RUNMC)

Scientific Communications & Publishing Coordinator (STEMCELL Technologies, Inc.)

Research Technologist – Pluripotent Stem Cells (STEMCELL Technologies Inc.)

Research Technologist – Particle Chemistry (STEMCELL Technologies Inc.)

Research Technologist – PSC Biology and Bioengineering (STEMCELL Technologies Inc.)

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