Cell Therapy News Volume 13.21 | Jun 4 2012

Induction Therapy with Autologous Mesenchymal Stem Cells in Living-Related Kidney Transplants
Among patients undergoing renal transplant, the use of autologous mesenchymal stem cells compared with anti-IL-2 receptor antibody induction therapy resulted in lower incidence of acute rejection, decreased risk of opportunistic infection, and better estimated renal function at one year. [JAMA] Abstract | Press Release

Micro-RNA-34a Contributes to the Impaired Function of Bone Marrow-Derived Mononuclear Cells from Patients with Cardiovascular Disease
Researchers found that reducing the expression of the pro-apoptotic micro-RNA-34a improves the survival of bone marrow-mononuclear cells in vitro and enhances the therapeutic benefit of cell therapy in mice after acute myocardial infarction. [J Am Coll Cardiol] Abstract

In Vitro Microvessels for the Study of Angiogenesis and Thrombosis
Researchers engineered living microvascular networks in three-dimensional tissue scaffolds and demonstrated their biofunctionality in vitro. [Proc Natl Acad Sci USA] Abstract | Press Release

Lysine-Specific Demethylase 2A (KDM2A) Normalizes Human Embryonic Stem Cell Derived Keratinocytes
While attempting to improve proliferation of hES-cell-derived Nod keratinocytes, which grow poorly and have a short life span, scientists found that high expression of the KDM2A gene improves the poor proliferation of the cells. [Proc Natl Acad Sci USA] Abstract

Amniotic Fluid Stem Cells Restore the Muscle Cell Niche in a HSA–Cre, Smn^F7/F7 Mouse Model
Mice carrying a homozygous deletion of the survival of motor neuron gene (Smn) exon 7 directed to skeletal muscle (HSA–Cre, Smn^F7/F7 mice) present clinical features of human muscular dystrophies for which new therapeutic approaches are highly warranted. In this study, researchers demonstrated that tail vein transplantation of mouse amniotic fluid stem cells enhances the muscle strength and improves the survival rate of the affected animals. [Stem Cells] Abstract | Press Release

MSC Anti-Diabetic Effect Is Unrelated to Their Trans-Differentiation Potential but to Their Capability to Restore Th1/Th2 Balance and to Modify the Pancreatic Microenvironment
In type 1 diabetes mellitus (T1DM) animal models, it has been shown that the systemic administration of multipotent mesenchymal stromal cells (MSCs) results in the regeneration of pancreatic islets. Researchers assessed whether donor MSCs differentiate into pancreatic beta-cells and modify systemic and pancreatic pathophysiologic markers of T1DM. [Stem Cells] Abstract

Localization of Mesenchymal Stromal Cells Dictates Their Immune or Proinflammatory Effects in Kidney Transplantation
Researchers used a murine kidney transplant model with the aim to define the best timing of MSC infusion capable of promoting immune tolerance without negative effects on early graft function. [Am J Transplant] Abstract

Chimeric Antigen Receptor/FoxP3-Engineered T Regulatory Cells Target the CNS and Suppress EAE Upon Intranasal Delivery
Researchers examined gene engineering to achieve central nervous system (CNS)-targeting T regulatory cells from naive CD4 cells and demonstrated their efficacy in the experimental autoimmune encephalomyelitis (EAE) model. [J Neuroinflammation] Abstract | Full Article

Correction of Brain Oligodendrocytes by AAVrh.10 Intracerebral Gene Therapy in Metachromatic Leukodystrophy Mice
Investigators evaluated the short-term effects of AAVrh.10 driving the expression of human arylsulfatase A cDNA under the cytomegalovirus/β-actin hybrid promoter in eight-month-old metachromatic leukodystrophy mice that already showed marked sulfatide accumulation and brain pathology. [Hum Gene Ther] Abstract

Transient B Cell Depletion or Improved Transgene Expression by Codon Optimization Promote Tolerance to Factor VIII in Gene Therapy
Scientists investigated the use of adeno-associated viral (serotype 8) gene transfer to induce tolerance to human B domain deleted factor VIII in hemophilia A mice. [PLoS One] Full Article

NewLink Genetics Reports Two- and Three-Year Overall Survival Data from Its Phase-2 HyperAcute® Pancreas (algenpantucel-L) Immunotherapy Trial
NewLink Genetics Corporation announced that its HyperAcute® Pancreas (algenpantucel-L) Immunotherapy will be featured in a poster presentation. The study results show 37%, 59% and 121% improvement in one-, two- and three-year survival, respectively, as compared to standard-of-care. [Press release from NewLink Genetics Corporation discussing research presented at the American Society of Clinical Oncology (ASCO) 2012 Annual Meeting, Chicago] Press Release

NewLink Genetics HyperAcute® Lung (tergenpumatucel-L) Immunotherapy Demonstrates a Correlation between Immune Response and Survival in Patients With Non-Small Cell Lung Cancer
NewLink Genetics Corporation announced its HyperAcute® Lung (tergenpantucel-L) immunotherapy will be featured in a poster presentation. The study demonstrated a direct correlation between immune response and survival in non-small cell lung cancer patients. [Press release from NewLink Genetics Corporation discussing research presented at the American Society of Clinical Oncology (ASCO) 2012 Annual Meeting, Chicago]
Press Release

Top-Line Results of Phase IIb Clinical Trial of ALN-RSV01, an Inhaled RNAi Therapeutic for the Treatment of Respiratory Syncytial Virus (RSV) Infection
Alnylam Pharmaceuticals, Inc. announced top-line results from a Phase IIb trial with ALN-RSV01 for the treatment of RSV infection in lung transplant patients. [Alnylam Pharmaceuticals, Inc.] Press Release

Europe Science Ministers Approve 2014-20 Funding Framework
After long and hard negotiations, European Union (EU) science ministers have given their backing to a general structure for Horizon 2020, the bloc’s 2014-20 research-funding program. [EU Council of Ministers, European Union] Press Release

Scientists: Your Number Is Up
Just as barcodes at the supermarket allow the till to distinguish a tomato from a turnip, Open Researcher and Contributor ID (ORCID) aims to reliably attribute research outputs to their true author by assigning every scientist on the planet a machine-readable, 16-digit unique digital identifier. [ORCID Committee] Press Release

ICMS and AABB Collaborate to Help Improve Safety for Patients Receiving Autologous Adult Cell-Based Therapies
The International Cellular Medicine Society (ICMS) and AABB (formerly the American Association of Blood Banks) announced that AABB will be assisting ICMS in the development of quality-based, voluntary standards for the collection, processing and administration of autologous adult cells. [AABB] Press Release

Actium Research and McMaster University Collaborate to Commercialize Stem Cell Technologies
Actium Research Inc. and McMaster University have entered into a landmark collaboration covering McMaster’s proprietary adult human stem cell lines, cancer stem cells and the directed differentiation platform developed by Dr. Mick Bhatia and his team at the McMaster Stem Cell and Cancer Research Institute. [McMaster University] Press Release

Baxter Announces Collaboration with Chatham Therapeutics for Factor IX Hemophilia B Gene Therapy Treatment
Baxter International Inc. announced that it has entered into an exclusive global agreement with Chatham Therapeutics, LLC for the development and commercialization of potential treatments for hemophilia B utilizing Chatham’s gene therapy technology. [Baxter International Inc.] Press Release

Bristol-Myers Squibb Announces Global Collaboration with Leading Academic Institutions to Advance Science of Immuno-Oncology
Bristol-Myers Squibb Company announced the formation of the International Immuno-Oncology Network, a global collaboration between industry and academia that aims to further the scientific understanding of immuno-oncology. [Bristol-Myers Squibb Company] Press Release

City of Hope Receives $5 Million Grant to Develop T Cell Treatment Targeting Brain Tumor Stem Cells
City of Hope was granted a $5,217,004 early translational research award by the California Institute for Regenerative Medicine to support the development of a T cell-based immunotherapy that re-directs a patient’s own immune response against glioma stem cells. [City of Hope] Press Release

CHOC Children’s Research Project Awarded $5.5 Million Grant from the California Institute for Regenerative Medicine
A Children’s Hospital of Orange County (CHOC) Children’s research project has been awarded a $5.5 million grant from the California Institute for Regenerative Medicine. The grant will be used to develop a stem cell-based therapy for the treatment of mucopolysaccharidosis. [Children’s Hospital of Orange County] Press Release

RXi Pharmaceuticals Receives FDA Clearance to Begin Clinical Trial with RXI-109
RXi Pharmaceuticals Corporation announced that it has received clearance for its investigational new drug application from the U.S. Food and Drug Administration (FDA) to initiate clinical trials with RXI-109, a self-delivering RNAi compound that selectively targets connective tissue growth factor. [RXi Pharmaceuticals Corporation] Press Release

Kane Biotech Files PCT Patent on Bone Tissue Regeneration Technology
Kane Biotech Inc. is pleased to announce that the Company has filed the PCT (Patent Cooperation Treaty) on its bone tissue regeneration technology with the World Intellectual Property Organization. [Kane Biotech Inc.]
Press Release

Cytune Pharma Is Granted an EU Patent for Its Potent Immunotherapy CYP0150: An Optimized Recombinant Il-15 Fusion Protein and Will Attend BIO 2012 after a Year of Achievements
Cytune Pharma announced that it will attend the BIO2012 to present its major achievements over the last 12 months and to foster partnering discussions. Cytune also announced that the European Patent and Trademark Office issued patent EP06842352.4 for its recombinant Interleukin-15-based fusion protein CYP0150. [PR Newswire] Press Release

Cytori Obtains Celution(R) Approval in Russia; To Launch Product Line for Plastic Surgery and Select Soft Tissue Therapies
Cytori Therapeutics has obtained approval to sell the Celution® 800 System in Russia for various medical uses. These clinical uses are based on those in the current European CE Mark, and include the use of the Celution® system and its output of adipose-derived stem and regenerative cells for plastic surgery and select soft tissue therapies. [Cytori Therapeutics, Inc.] Press Release

Marina Biotech Ceases Most Day-to-Day Operations
Marina Biotech, Inc. announced that it does not currently have adequate funding to continue implementing its business plan. To conserve available cash, management has implemented a furlough of approximately 90% of its employees and ceased most day-to-day operations, including the ongoing FAP clinical trial. [Marina Biotech, Inc.] Press Release

Clarification of Position Statements on Implementation of the Guide for the Care and Use of Laboratory Animals: Eighth Edition (NOT-OD-12-112)

Request for Information (RFI): Input on Proposed Modifications of the Biographical Sketch Used in NIH Grant Applications (NOT-OD-12-115)

AHRQ National Research Service Awards (NRSA) Institutional Research Training Grants (T32) (RFA-HS-12-008)

AHRQ Patient Centered Outcomes Research (PCOR) Pathway to Independence Award (K99/R00) (RFA-HS-12-007)

Government-Owned Inventions; Availability for Licensing (FR Doc No: 2012-13007)

National Institute of General Medical Sciences; Notice of Closed Meeting (FR Doc No: 2012-13123)

National Institute of Diabetes and Digestive and Kidney Diseases; Notice of Closed Meetings (FR Doc No: 2012-12844)

May 24, 2012 Approval Letter – HPC, Cord Blood

Tissue Reference Group

European Medicines Agency (European Union)

European Medicines Agency Boosts European Union Transparency with Online Publication of Suspected Side Effect Reports
The European Medicines Agency has begun publishing suspected side effect reports for medicines authorized in the European Economic Area on a new public website: www.adrreports.eu. Press Release

Therapeutic Goods Administration (Australia)

Therapeutic Goods Administration Regulatory Framework

NEW Cell Symposia Human Immunity
August 19-21, 2012
Lisbon, Portugal

NEW ESGCT 20th Anniversary Congress in Collaboration with the SFTCG
October 25-29, 2012
Versailles, France

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

Scientist – Induced Pluripotent Stem Cells (STEMCELL Technologies, Inc.)

Scientist – Epithelial Cell Research (STEMCELL Technologies, Inc.)

Scientist – hPSC Bioengineering (STEMCELL Technologies, Inc.)

Scientist – Mesenchymal Cell Research (STEMCELL Technologies, Inc.)

Research Associate – Chemistry (STEMCELL Technologies, Inc.)

Research Associate – Particle Chemistry (STEMCELL Technologies, Inc.)

Research Technologist – Contract Assay Services (STEMCELL Technologies, Inc.)

Research Technologist – Media Development (STEMCELL Technologies, Inc.)

Business Analyst – Product Management (STEMSOFT Software, Inc.)

iPSC Scientist Position – Laboratory of Translational Cell Biology (The Bassell Lab, Emory University)

Business Development Manager – Stem Cell (SynGen Inc.)

Controller (SynGen Inc.)

Director: Regulatory Affairs (SynGen Inc.)

Manufacturing Manager (SynGen Inc.)

Chief Scientific Officer (SynGen Inc.)

Postdoctoral Fellow – Department of Bioengineering and Barbara Davis Center (University of Colorado at Denver)

Postdoctoral Position – GIGA-Neurosciences (University of Liège)

Postdoctoral Position – Spinal Cord Neural Stem Cells (Institute for Neurosciences of Montpellier)

Cellular Therapy Lab Specialist (The University of Texas Medical School at Houston)

Postdoctoral Research Position for Minority Candidate (New York Obesity Research Center)


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