Volume 15.03 | Jan 27

Issue 15.03 January 27, 2014
Cell Therapy News - Your Industry in an Instant
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Gene Therapy Leads to Robust Improvements in Animal Model of Fatal Muscle Disease
Preclinical studies showed that gene therapy can improve muscle strength in small- and large-animal models of a fatal congenital childhood disease known as X-linked myotubular myopathy. [Press release from the University of Washington discussing online prepublication in Science Translational Medicine] Press Release | Video | Abstract
Free Sample: Untouched Mouse CD4 T Cells In Just 40 Minutes (NEW!)
PUBLICATIONS (Ranked by impact factor of the journal)
Does Transendocardial Injection of Mesenchymal Stem Cells Improve Myocardial Function Locally or Globally? An Analysis from the POSEIDON Randomized Trial
To address whether transendocardial stem cell injection exerts its effects at the site of injection only or also in remote areas, researchers hypothesized that segmental myocardial scar and segmental ejection fraction improve to a greater extent in injected than in non-injected segments. [Circulation] Abstract

Generation of a Vascularized and Functional Human Liver from an iPSC-Derived Organ Bud Transplant
Scientists describe a protocol for the in vitro generation of a 3D liver bud from human induced pluripotent stem cell (iPSC) cultures and the monitoring of further hepatic maturation after transplantation at various ectopic sites. Functional analyses show the appearance of multiple hepatic functions in a chronological manner in vivo. [Nat Protoc] Abstract

Macrophage-Inducing FasL on Chondrocytes Forms Immune Privilege in Cartilage Tissue Engineering, Enhancing In Vivo Regeneration
Since elucidation of mechanism on immune privilege formed in cartilage regeneration may contribute to suppression of excessive inflammation, scientists investigated the function of Fas ligand (FasL) and induction of immune privilege in tissue-engineered cartilage using a mouse subcutaneous model. [Stem Cells] Abstract

Gene Therapy Using a Liver-Targeted AAV Vector Restores Nucleoside and Nucleotide Homeostasis in a Murine Model of MNGIE
Researchers report that AAV2/8-mediated transfer of the human TYMP coding sequence under the control of a liver-specific promoter prevents the biochemical imbalances in a murine model of mitochondrial neurogastrointestinal encephalomyopathy (MNGIE). [Mol Ther] Abstract

The Peripheral Chimerism of Bone Marrow-Derived Stem Cells after Transplantation: Regeneration of Gastrointestinal Tissues in Lethally Irradiated Mice
Investigators examined a model using whole-body irradiation and the transplantation of bone marrow or hematopoietic stem cells to study the repair of hematopoiesis, extramedullary hematopoiesis and the migration of green fluorescent protein transplanted cells into non-hematopoietic tissues. [J Cell Mol Med] Full Article

TRAIL-Secreting Mesenchymal Stem Cells Promote Apoptosis in Heat-Shock-Treated Liver Cancer Cells and Inhibit Tumor Growth in Nude Mice
The secretable form of trimeric human tumor necrosis factor-related apoptosis-inducing ligand (stTRAIL) induces apoptosis in a variety of human cancers but not in normal tissues. It has shown potent cancer-selective killing activity and has drawn considerable attention as a possible cancer therapy. The therapeutic potential of this stTRAIL-based gene therapy was evaluated in hepatocellular carcinoma subjected to radiofrequency ablation. [Gene Ther] Abstract

Effects of Intravenous Administration of Umbilical Cord Blood CD34+ Cells in a Mouse Model of Neonatal Stroke
The authors examined the effects of human umbilical cord blood CD34+ cells (hematopoietic stem cell/endothelial progenitor cells) in a mouse model of neonatal stroke, which they recently developed. [Neuroscience] Abstract

Intra-Articular Injection of Human Meniscus Stem/Progenitor Cells Promotes Meniscus Regeneration and Ameliorates Osteoarthritis through Stromal Cell-Derived Factor-1/CXCR4-Mediated Homing
Scientists found that human meniscus stem/progenitor cells (hMeSPCs) displayed both mesenchymal stem cell characteristics and high expression levels of collagen II. In the rat meniscus injury model, hMeSPC transplantation not only led to more neo-tissue formation and better-defined shape but also resulted in more rounded cells and matured extracellular matrix. [Stem Cell Transl Med] Abstract

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Mitigating the Toxic Effects of Anticancer Immunotherapy
This review describes the toxicity profiles for various anticancer therapies based on the use of agents that block immune checkpoints, immunostimulatory agents, and adoptive T-cell therapy (that is, infusion of modified autologous T cells). [Nat Rev Clin Oncol] Abstract

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

A New System for High-Throughput Cell Isolation Directly from Whole Blood
BioLeonhardt Unveils Stem Cell Pump at Cell Therapy Meeting
BioLeonhardt as developed a programmable implantable stem cell infusion pump and electrical stimulator combination for treating advanced heart failure. The stem cell pump is implanted in the patient’s abdomen and has a silicone septum which allows re-loading via a micro needle syringe of stem cells, genes, growth factors and nutrient hydrogels. [Press release from PR Newswire Association LLC discussing research presented at the 9th Annual Cell Therapy for Cardiovascular Disease Meeting, New York] Press Release

Pluristem’s Phase I/II Muscle Injury Trial Successfully Meets Primary Safety & Efficacy Endpoints
Pluristem Therapeutics Inc. announced top-line results from its Phase I/II clinical trial testing the safety and efficacy of placental expanded cells in the treatment of muscle injury. [Pluristem Therapeutics Inc.]
Press Release

Strategies to Inhibit GvHD while Sparing the Graft-versus-Tumor Response: Finding the Sweet Spot
Putin Decree Shakes Up Russian Science Funding
President Vladimir Putin signed a clutch of decrees that could have a profound effect on science in Russia. One stipulates that all state research funding should be distributed via a competitive grants system. Previously, research institutes received government support to cover things such as upkeep of buildings and utility bills, but that could now stop, as will the government’s so-called state targeted programs, which single out certain areas for direct financial support. [Nature News] Editorial

Push for Regenerative Medicine R&D
An expert panel has proposed that the Japanese government focus the nation’s medical research and development on such cutting-edge fields as regenerative medicine and genomic medicine. Based on a report compiled by the panel, the Abe administration will soon adopt a comprehensive strategy on a future vision for Japan’s medical research at a meeting of its task force for health care strategy. [The Japanese Times] Editorial

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The HUB Foundation for Organoid Technology and STEMCELL Technologies Sign Agreement on the Manufacturing of Cell Culture Media for Organoids
The HUB Foundation for Organoid Technology announced that it has signed a licensing agreement with STEMCELL Technologies Inc. for the manufacturing and worldwide distribution of cell culture media for growing Organoids. Organoids are mini-organs grown in tissue culture from small pieces of tissue derived from patients with cancer and other diseases, and represent an exciting new tool for scientific research and drug discovery and validation. [STEMCELL Technologies Inc.] Press Release

Biocon Collaborates with Advaxis for ‘ADXS-HPV’: A Novel Cancer Immunotherapy
Biocon Ltd. and Advaxis, Inc. announced that they have entered into an exclusive licensing agreement for co-development and commercialization of ADXS-HPV, a novel cancer immunotherapy for the treatment of human papillomavirus-associated cervical cancer in women, for India and key emerging markets. [Biocon Ltd.]
Press Release

MD Anderson Announces Collaboration with Johnson & Johnson Innovation on Cancer Immunotherapy
The University of Texas MD Anderson Cancer Center will collaborate with Johnson & Johnson Innovation, LLC, and its affiliate Janssen Biotech, Inc., to develop immunology-based cancer treatments through the MD Anderson Moon Shots Program immunotherapy platform. [University of Texas M. D. Anderson Cancer Center] Press Release

Regeneus Partners with Lonza for Manufacturing CryoShot Canine Cell Therapy Product for US FDA Trial
Regeneus, announced that Lonza will manufacture Regeneus’ off-the-shelf cell therapy, CryoShot® Canine, for registration in the US. The cells will be manufactured at the Lonza facility in Walkersville, Maryland and then distributed to the participating trial veterinarian clinics. [Regeneus Ltd] Press Release

Minister Sherlock Opens Ireland’s First Human Use Stem Cell Manufacturing Facility at NUI Galway
The Centre for Cell Manufacturing Ireland at NUI Galway is the first ever facility on the island of Ireland to receive a license from the Irish Medicines Board to manufacture culture-expanded stem cells for human use. [National University of Ireland, Galway] Press Release

California Stem Cell Submits Phase II Clinical Trial Protocol to FDA for Treating Ovarian Cancer
California Stem Cell, Inc. (CSC) announced it has submitted a Phase II protocol to the U.S. Food and Drug Administration for a randomized, double-blind study to evaluate CSC’s patient-specific cancer immunotherapy in women with Stage III or IV ovarian, fallopian tube or primary peritoneal cancer. [California Stem Cell, Inc.]
Press Release

TotipotentRX Corporation and ThermoGenesis Corp. Report Statistically Significant Phase Ib Clinical Trial Results in Critical Limb Ischemia
ThermoGenesis Corp. and TotipotentRX Corporation announced their co-sponsored Phase Ib clinical trial safety and efficacy results treating no-option patients suffering from critical limb ischemia with Totipotent’s CLIRST (Critical Limb Ischemia Rapid Stem cell Therapy) treatment. [ThermoGenesis Corp.] Press Release

BioLineRx Receives Orphan Drug Designation for Novel Stem Cell Mobilization Treatment
BioLineRx announced that it has received notice from the U.S. Food & Drug Administration confirming an Orphan Drug Designation of BL-8040 as a treatment for stem cell mobilization, in addition to the Orphan Drug Designation previously granted to BL-8040 as a treatment for acute myeloid leukemia. [BioLineRx] Press Release

apceth’s Ground-Breaking First-in-Man, First-in-Class Clinical Trial in Oncology with Genetically Modified Mesenchymal Stromal Cells Is Now Enrolling
apceth announced that the TREAT ME Phase I/II clinical trial for patients suffering from advanced adenocarcinoma of the gastro-intestinal tract has commenced. This is the first clinical trial world-wide with genetically-modified mesenchymal stromal cells. [B3C Group GmbH] Press Release

Pluristem’s Cell Manufacturing Facility Marks Major Milestone with German Regulatory Approval of New Scaled-Up 3D Manufacturing Process
Pluristem Therapeutics, Inc. announced it has received approval from the Paul-Ehrlich-Institute, Germany’s health authority, to supply cells from its advanced, fully automated, and proprietary 3D cell expansion manufacturing platform implemented at its new state-of-the-art GMP facility. [Pluristem Therapeutics Inc.] Press Release

Bioheart Announces Exclusive License in Veterinary Medicine for Patents for Cancer-Related Immunotherapy Products
Bioheart, Inc. announced that it has entered into an agreement to license three different cellular based immunotherapy patents from inventor Allan Wu, M.D. [Bioheart, Inc.] Press Release
Government-Owned Inventions; Availability for Licensing (FR Doc No. 2014-01186)
Food and Drug Administration (United States)

Agency Information Collection Activities; Submission for Office of Management and Budget Review; Comment Request; Human Cells, Tissues, and Cellular and Tissue-Based Products: Establishment Registration and Listing; Form FDA 3356; Eligibility Determination for Donors; and Current Good Tissue Practice (FR Doc No. 2014-01223)
NEW Challenges toward Sound Scientific Regulation of Cell Therapy Products
March 7-8, 2014
Kyoto, Japan

NEW Annual Conference of the American Society for Neural Therapy and Repair
April 24-26, 2014
Clearwater Beach, United States

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Project Manager – Cell Therapy (University of California – Irvine)

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

Senior Scientist – Stem Cells and Regenerative Medicine (Universidad de Los Andes)

Postdoctoral Position – Tissue Engineering and Regenerative Medicine (RUNMC)

Postdoctoral Fellow – Morphogenetic Signaling Pathways in Development and Disease (University of Rochester Medical Center)

Scientific Affairs Coordinator (American Society of Hematology)

Postdoctoral Research Fellow (Brigham & Women’s Hospital and Harvard Medical School)

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

Stem Cell Manufacturing Scientist (Paramount Recruitment)

Postdoctoral Position – Stem Cells and Bioengineering (Queensland University of Technology/Translational Research Institute)

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