Cell Therapy News Volume 15.20 | May 26 2014

Towards a Clinical Use of Human Embryonic Stem Cell-Derived Cardiac Progenitors: A Translational Experience
Data collected by researchers has led to approval by the French regulatory authorities for a first-in-man clinical trial of transplantation of stage-specific embryonic antigen-1⁺ progenitors in patients with severely impaired cardiac function. Although several facets of the manufacturing process still need to be improved, these data may yet provide a useful platform for the production of human embryonic stem cell-derived cardiac progenitor cells under safe and cost-effective good manufacturing practice conditions. [Eur Heart J] Abstract

Etanercept Blocks Inflammatory Responses Orchestrated by TNF-α to Promote Transplanted Cell Engraftment and Proliferation in Rat Liver
To define whether TNF-α served roles in cell-transplantation-induced hepatic inflammation, researchers used TNF-α antagonist, etanercept, for studies in syngeneic rat hepatocyte transplantation systems. [Hepatology] Abstract

RNAi-Mediated Silencing of Hepatic Alas1 Effectively Prevents and Treats the Induced Acute Attacks in Acute Intermittent Porphyria Mice
Scientists describe preclinical studies of liver-directed small interfering RNAs targeting 5-aminolevulinic acid synthase 1 (Alas1) in a mouse model of acute intermittent porphyria, the most common acute hepatic porphyria. [Proc Natl Acad Sci USA] Abstract

Therapeutic Efficacy of an Fc-Enhanced TCR-Like Antibody to the Intracellular WT1 Oncoprotein
Investigators engineered a second-generation monoclonal antibody, ESKM, to have enhanced antibody dependent cell-mediated cytotoxicity (ADCC) function due to altered Fc glycosylation. ESKM was compared to native ESK1 in binding assays, in vitro ADCC assays, and mesothelioma and leukemia therapeutic models and pharmacokinetic studies in mice. [Clin Cancer Res] Abstract

Indirect CD4⁺ T Cell-Mediated Elimination of MHC II^NEG Tumor Cells Is Spatially Restricted and Fails to Prevent Escape of Antigen-Negative Cells
Activated M1-like macrophages lack antigen (Ag) receptors and are presumably unable to discriminate between and thus kill both Ag-positive (Ag^POS) and Ag^NEG tumor cells (bystander killing). The lack of specificity of macrophage-mediated cytotoxicity might be of clinical importance as it could provide a means of avoiding tumor escape. Researchers tested this idea using mixed populations of Ag^POS and Ag^NEG tumor cells in a TCR-transgenic model in which CD4⁺ T cells recognize a secreted tumor-specific antigen. [Eur J Immunol] Abstract

Antitumor Effects of Oncolytic Adenovirus Armed with PSA-IZ-CD40L Fusion Gene against Prostate Cancer
Scientists developed a prostate cancer-specific oncolytic adenovirus armed with fusion gene of prostate-specific antigen and CD40 ligand, and evaluated its therapeutic effect in vitro and in vivo. [Gene Ther] Abstract

Expression of IL-2 in β Cells by AAV8 Gene Transfer in Pre-Diabetic NOD Mice Prevents Diabetes through Activation of FoxP3-Positive Regulatory T Cells
Investigators evaluated the effects of double-stranded adeno-associated virus serotype 8 (AAV8)-mouse insulin promoter-mediated delivery of interleukin (IL)-2 to pancreatic β cells in non-obese diabetic (NOD) mice. Abstract

Combined Injection of Three Different Lineages of Early Differentiating Human Amniotic Fluid-Derived Cells Restores Urethral Sphincter Function in Urinary Incontinence
Researchers investigated whether a triple combination of early-differentiated cells derived from human amniotic fluid stem cells would show synergistic effects in urethral sphincter regeneration. [BJU Int] Abstract

Dental Pulp Stem Cells: Function, Isolation and Applications in Regenerative Medicine
Dental pulp stem cells (DPSCs) are derived from the neural crest and, therefore, have a different developmental origin to bone marrow stromal cells (BMSCs). These differences from BMSCs in origin and phenotype are being exploited in neurological and other applications. This review briefly highlights the source and functions of DPSCs and then focuses on in vivo applications across the breadth of regenerative medicine. [J Tissue Eng Regen Med] Full Article

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

Tekmira Presents Positive Interim Results on Phase I/II Clinical Programs
Tekmira Pharmaceuticals Corporation announced that recent advances in its clinical programs were presented. The presentation titled “Progress in the Development of Lipid Nanoparticle-RNA Therapeutics” shared new interim data from the ongoing TKM-Ebola Phase I Clinical Trial and the Phase I/II Clinical Trial with TKM-PLK1. [Press release from Tekmira Pharmaceuticals Corporation discussing research presented at the 17^th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), Washington] Press Release

EmailAddress&u=http://www.stemcell.com/en/Technical-Resources/228ae/29331-The-CFC-Assay-For-Cord-Blood.aspx?utm_source=ctn%26utm_medium=link%26utm_campaign=ON218-CordBloodTechBull_645x110&t=(Archive)CTN15.20iiitext” style=”text-decoration: none”>From our sponsor: Learn about assays for cord blood. Download the free technical bulletin.

Revised FIRST Bill Would Give Science Agencies One Year to Make Papers Free
Responding to concerns from advocates for public access to government-funded research, the House of Representatives science committee has agreed to shorten the deadline within a draft bill requiring federal agencies to provide free access to scientific papers that they supported. [ScienceInsider] Editorial

UCL, Imperial & Cell Therapy Catapult Collaborate to Advance Leukemia Cell Therapy
A novel cell therapy approach to the treatment of acute myeloid leukemia is to be developed further via an innovative partnership between the Cell Therapy Catapult, UCL Business and Imperial Innovations. [Cell Therapy Catapult] Press Release

Gene Therapy Section Formed within the Alliance for Regenerative Medicine
The Alliance for Regenerative Medicine (ARM) and the American Society of Gene & Cell Therapy (ASGCT) announced their partnership. ARM’s new Gene and Gene-Modified Cell Therapy Section brings together the leading gene therapy companies and organizations in the U.S. and Europe to advocate for policies and programs to accelerate the development of new therapeutics to treat and cure a range of diseases for which no effective treatment options are available. [Alliance for Regenerative Medicine] Press Release

SKC Partners with UK Charities to Launch International Neuroblastoma Clinical Trial
Solving Kids’ Cancer (SKC), the Neuroblastoma Children’s Cancer Alliance, and Joining Against Cancer in Kids awarded its first grant through their INBRACED (International Neuroblastoma Research and Collaboration for Effective Delivery) initiative, which aims to improve access of promising clinical trials for children with high-risk neuroblastoma in North America, the UK and in Europe. The grant amount of $400,000 will create a new immunotherapy treatment option in the U.S., UK, and in Spain, for children with relapsed or refractory disease. [Solving Kids’ Cancer] Press Release

Stem Cell Research on Campus Grows after $3 Million Boost
Unlocking knowledge about how organisms develop and repair, stem cell research holds great promise for future therapies for injuries and conditions, from infertility and Alzheimer’s to heart failure and cancer. As part of its mission to promote cross-campus interactions and enhance training in stem cell biology at Cornell, the Cornell Stem Cell Program held its second Stem Cell Retreat on campus. [Cornell University] Press Release

SLU Virologists Harness Adenovirus to Kill Breast Cancer Cells
With a $40,250 grant from the Lottie Caroline Hardy Charitable Trust Fund, researchers at Saint Louis University (SLU)’s Institute for Molecular Virology will study a small fragment of adenovirus as a possible future therapy for HER2 breast cancer. [Saint Louis University] Press Release

Ground Breaking Hip and Stem Cell Surgery in Southampton
Doctors and scientists in Southampton have completed their first hip surgery with a 3D printed implant and bone stem cell graft. The 3D printed hip, made from titanium, was designed using the patient’s CT scan and CAD CAM (computer aided design and computer aided manufacturing) technology, meaning it was designed to the patient’s exact specifications and measurements. [University of Southampton] Press Release

TxCell’s Col-Treg Receives Advanced Therapy Medicinal Product Classification from EMA
TxCell SA announced that Col-Treg, its second therapeutic candidate from its ASTrIA platform, has been granted Advanced Therapy Medicinal Product classification by the Committee for Advanced Therapies of the European Medicines Agency (EMA). [TxCell SA] Press Release

National Institute of General Medical Sciences; Notice of Closed Meetings (FR Doc. No: 2014-11593)

Determination of Regulatory Review Period for Purposes of Patent Extension; INLYTA (FR Doc. No: 2014-11925)

Guidance for Institutional Review Boards, Clinical Investigators, and Sponsors: Considerations when Transferring Clinical Investigation Oversight to Another Institutional Review Board; Availability (FR Doc. No: 2014-11923)

Determination of Regulatory Review Period for Purposes of Patent Extension; PERJETA (FR Doc. No: 2014-11515)

NEW Cell Therapy Bioprocessing
September 15-16, 2014
Arlington, United States

NEW BioProcess International
October 20-23, 2014
Boston, United States

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

NEW Research/Review Scientist – Cell Therapy and Regenerative Medicine (Center for Biologics Evaluation and Research)

NEW Scientist – Immunology/Cell Separation (STEMCELL Technologies Inc.)

Postdoctoral Position – Cell and Extracellular Matrix Biology (Johns Hopkins University)

Postdoctoral Position – Cell Death and Inflammation (Inflammation Research Center – VIB-UGent)

Postdoctoral Position – Role of Calreticulin in TGF-Beta Signaling in the Kidney Proximal Tubule under Diabetic Conditions (University of Alabama)

Postdoctoral Fellow – Transgene Immunity in AAV Vector-Based Antiviral and/or Gene Therapeutics (University of Massachusetts)

Postdoctoral Fellow – Neurophysiology of Addiction (Medical University of South Carolina)

Principal Investigator – Molecular Mechanisms of Inflammation (Inflammation Research Center – VIB-UGent)

Postdoctoral Associate – Hematopoietic Differentiation (Northwestern University Feinberg School of Medicine)

Developmental-Stem Cell Biologist (Mayo Clinic – Rochester)

Postdoctoral Position – Functional Characterization of Neurons Derived from Human Pluripotent Stem Cells (Radboud University Nijmegen)

Postdoctoral Fellowship – Hematology, Immunology, Cancer Therapeutics (Nationwide Children’s Hospital)

Business Development Executive (Research Instruments Ltd)

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

Research Technologist – hPSC (STEMCELL Technologies Inc.)

Research Associate – Cell Separation (STEMCELL Technologies Inc.)

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

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

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