Volume 15.15 | Apr 21

Issue 15.15 April 21, 2014
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TOP STORY
How a Silly Putty Ingredient Could Advance Stem Cell Therapies
The sponginess of the environment where human embryonic stem cells are growing affects the type of specialized cells they eventually become, a new study shows. Researchers coaxed human embryonic stem cells to turn into working spinal cord cells more efficiently by growing the cells on a soft, ultrafine carpet made of a key ingredient in Silly Putty. [Press release from the University of Michigan discussing online prepublication in Nature Materials] Press Release | Abstract
Webinar: Modeling Human Neurological Disease with iPS Cells
 
PUBLICATIONS (Ranked by impact factor of the journal)
Induction of Antigen-Specific Immunity with a Vaccine Targeting NY-ESO-1 to the Dendritic Cell Receptor DEC-205
Researchers aimed to enhance anticancer immunity by exploiting the capacity of dendritic cells (DCs) to initiate T cell immunity by efficient uptake and presentation of endocytosed material. Delivery of tumor-associated antigens to DCs using receptor-specific monoclonal antibodies in the presence of DC-activating agents elicits robust antigen-specific immune responses in preclinical models. [Sci Transl Med] Abstract | Press Release

Experimental Orthotopic Transplantation of a Tissue-Engineered Esophagus in Rats
Scientists decellularized rat esophagi inside a perfusion bioreactor to create biocompatible biological rat scaffolds that mimic native architecture, resist mechanical stress and induce angiogenesis. Seeded allogeneic mesenchymal stromal cells spontaneously differentiate into epithelial- and muscle-like cells. The reseeded scaffolds were used to orthotopically replace the entire cervical esophagus in immunocompetent rats. [Nat Commun] Abstract | Press Release

Parthenogenetic Embryonic Stem Cells Are an Effective Cell Source for Therapeutic Liver Repopulation
Investigators determined whether parthenogenetic embryonic stem cell derivatives are effective in rescuing mice with lethal liver failure due to deficiency of fumarylacetoacetate hydrolase. [Stem Cells] Abstract

Dendritic Cell-Mediated Immune Humanization of Mice: Implications for Allogeneic and Xenogeneic Stem Cell Transplantation
Researchers demonstrated that adoptive transfer of long-lived human dendritic cells coexpressing high levels of human IFN-α, human GM-CSF, and a clinically relevant Ag (CMV pp65 protein) promoted human lymphatic remodeling in immune-deficient NOD.Rag1-/-.IL-2rγ-/- mice transplanted with human CD34+ cells. [J Immunol] Abstract

Safety and Efficacy of Embryonic Stem Cell Microenvironment in a Leukemia Mouse Model
Investigators injected embryonic stem cells modified with a suicide gene into a leukemia mouse model through peripheral blood to observe the treatment effectiveness. [Stem Cells Dev] Abstract

Human Wharton’s Jelly Mesenchymal Stem Cells Plasticity Augments Scar-Free Skin Wound Healing with Hair Growth
Wharton’s jelly mesenchymal stem cells (WJ-MSCs) did not present any aberrant cell state upon in vivo transplantation in SCID mice and in vitro soft agar assays. The immunomodulatory potential assessed by gene expression levels of immunomodulatory factors upon exposure to inflammatory cytokines in the fetal WJ-MSCs was relatively higher compared to adult bone marrow-derived MSCs. [PLoS One] Full Article

Neural Stem Cells Genetically-Modified to Express Neprilysin Reduce Pathology in Alzheimer Transgenic Models
Scientists studied the effects of neprilysin (sNEP) expression in vitro by quantifying beta-amyloid (Aβ)-degrading activity, neural stem cell (NSC) multipotency markers, and Aβ-induced toxicity. To determine whether sNEP-expressing NSCs can also modulate Alzheimer’s disease (AD)-pathogenesis in vivo, control-modified and sNEP-NSCs were transplanted unilaterally into the hippocampus of two independent and well characterized transgenic models of AD: 3xTg-AD and Thy1-APP mice. [Stem Cell Res Ther] Full Article | Press Release

Human Neural Stem Cells Survive Long Term in the Midbrain of Dopamine-Depleted Monkeys after GDNF Overexpression and Project Neurites Toward an Appropriate Target
Researchers determined whether manipulation of the neural microenvironment by overexpression of a developmentally critical molecule, glial cell-derived neurotrophic factor (GDNF), in the host striatum could enhance dopamine differentiation of fetal neural stem cells injected into the substantia nigra and elicit growth of their axons to the GDNF-expressing target. [Stem Cells Transl Med] Abstract

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REVIEWS
Stem Cell Imaging: From Bench to Bedside
The authors discuss how in vivo molecular imaging has helped identify barriers to clinical translation and potential strategies that may contribute to successful transplantation and improved outcomes, with a focus on cardiovascular and neurological diseases. [Cell Stem Cell] Abstract

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

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SCIENCE NEWS
Baxter Meets Primary Efficacy Endpoint in Phase III Trial of BAX 111, Investigational Stand-Alone Recombinant Treatment for Von Willebrand Disease
Baxter International Inc. announced topline results from a Phase III clinical trial evaluating the safety, efficacy and pharmacokinetics of BAX 111. BAX 111 is a recombinant von Willebrand factor under investigation for the treatment of bleeding episodes in patients with von Willebrand disease, the most common type of inherited bleeding disorder. [Baxter International Inc.] Press Release

Dicerna Pharmaceuticals Initiates Phase I Study of DCR-MYC in Patients with Solid Tumors and Hematological Malignancies
Dicerna Pharmaceuticals, Inc. announced the initiation of a Phase I dose-escalating clinical study of DCR-MYC, (also known as DCR-M1711), in patients with solid tumors, multiple myeloma, or lymphoma. DCR-MYC is a Dicer Substrate siRNA that targets the driver oncogene MYC, which is central to the growth of many hematologic and solid tumor malignancies. [Dicerna Pharmaceuticals, Inc.] Press Release

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POLICY
NIH to Relax ‘Two Strikes’ Grant Submission Policy
The National Institutes of Health (NIH) is dropping a policy that gave researchers only one chance to revise a rejected grant application before having to start over with a new idea – a rule that was especially hard on young investigators. Instead, the agency will allow an applicant to resubmit the identical proposal as many times as they like as a new submission. [ScienceInsider] Editorial

U.S. Biomedical Research ‘Unsustainable,’ Prominent Researchers Warn
The U.S. biomedical science system “is on an unsustainable path” and needs major reform, four prominent researchers write in an opinion piece published by the Proceedings of the National Academy of Sciences. [ScienceInsider] Editorial | Full Article

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BUSINESS
Aastrom Announces Definitive Agreement to Acquire Sanofi’s Cell Therapy and Regenerative Medicine Business
Aastrom Biosciences, Inc. announced that it has entered into a definitive agreement to acquire Sanofi’s Cell Therapy and Regenerative Medicine business for a purchase price of $6.5 million, with $4 million payable in cash at closing and $2.5 million payable in the form of a promissory note. [Aastrom Biosciences, Inc.] Press Release

BioCision’s Proprietary Temperature Standardization Technology to be used in TxCell Phase IIb Cell Therapy Clinical Trial in the Treatment of Crohn’s Disease
BioCision, LLC announced that several of the company’s products used to support temperature control, as well as processing and handling of cell therapy products, will be used in a Phase IIb clinical trial conducted by TxCell. The trial will investigate the use of Ovasave®, an antigen-specific regulatory T autologous cell-based immunotherapy for the treatment of patients with moderate to severe refractory Crohn’s disease. [BioCision, LLC] Press Release

ReNeuron Signs Agreement to Lease a New R&D, Manufacturing and Headquarters Facility in South Wales
ReNeuron Group plc, the UK-based stem cell therapy company, announced that it has signed an agreement with the Welsh Government to lease a landmark building at Pencoed Technology Park, near Cardiff, South Wales. [ReNeuron Group plc] Press Release

inVentiv Health Clinical Engaged by IntelliCell Biosciences, a Regenerative Medicine Company, as Clinical Research Organization for Osteoarthritis IND
inVentiv Health Clinical announced that it has been selected for an osteoarthritis Investigational New Drug Application (IND) by IntelliCell BioSciences, Inc. IntelliCell will be studying the use of adult autologous vascular cells from blood vessels in adipose tissue to treat osteoarthritis of the knee. [inVentiv Health Clinical]
Press Release

First Volunteers to Receive Blood Cultured from Stem Cells in 2016
Red blood cells cultured in a laboratory will be trialed in human volunteers for the first time within the next three years, as part of a long-term research program funded by the Wellcome Trust. The £5 million Strategic Award was granted to a consortium led by the Scottish National Blood Transfusion Service, and will follow on from previous research that proved that red blood cells could be generated from stem cells. [Wellcome Trust] Press Release

Neuralstem Spinal Cord Injury Stem Cell Trial Approved to Commence at University of California, San Diego
Neuralstem, Inc. announced that the Institutional Review Board of the University of California, San Diego, School of Medicine has approved the Phase I safety trial to treat chronic spinal cord injury with its NSI-566 stem cells. [Neuralstem, Inc.] Press Release

Clinical Trial Investigates Cord Blood Stem Cells to Treat Cerebral Palsy in Children
Cord Blood Registry® announced the launch of an FDA-regulated clinical trial being conducted at The University of Texas Health Science Center at Houston to investigate two forms of stem cell therapy in children diagnosed with cerebral palsy. The randomized, placebo-controlled study aims to compare the safety and efficacy of an intravenous infusion of cord blood stem cells to bone marrow stem cells. [Cord Blood Registry®] Press Release

AAVLife Raises $12 Million in Series A Financing to Advance Gene Therapy for Friedreich’s Ataxia
AAVLife, a gene-therapy company focusing on rare diseases, announced that it has raised $12 million in Series A financing to advance into clinical studies the promising work on Friedreich’s ataxia recently reported in Nature Medicine. [Business Wire] Press Release
 
NIH
Government-Owned Inventions; Availability for Licensing (FR Doc. No: 2014-08881)

Prospective Grant of an Exclusive Option License: Immunotherapy Vaccine for Treating Lymphoma and Leukemia (FR Doc. No: 2014-08387)
 
CBER
Clinical Investigator Status (Biologics)
 
REGULATORY
Food and Drug Administration (United States)

Live Case Presentations During Investigational Device Exemption Clinical Trials; Draft Guidance for Institutional Review Boards, Industry, Investigators, and Food and Drug Administration Staff; Availability (FR Doc. No: 2014-08710)
 
EVENTS
NEW 10th Swiss Stem Cells Network (SSCN) Annual Meeting
June 4, 2014
Geneva, Switzerland


Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
 
JOB OPPORTUNITIES
NEW Postdoctoral Fellowship – Hematology, Immunology, Cancer Therapeutics (Nationwide Children’s Hospital)

Business Development Executive (Research Instruments Ltd)

MSc & PhD Positions – Cell Biology, Neurobiology, Neuroimmunology, Cancer Biology, Computational Biology (Hanyang University)

Postdoctoral Fellow – Therapeutic Corneal Endothelial Cell Culture (Lions VisionGift)

Postdoctoral Research Fellow / Research Officer – Stem Cell Research (Bioprocessing Technology Institute)

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)

Postdoctoral Position – Tissue Engineering and Regenerative Medicine (RUNMC)

Research Associate – Cell Separation (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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