Cell Therapy News Volume 16.21 | Jun 1 2015

Polymeric Nanovehicle Regulated Spatiotemporal Real-Time Imaging of the Differentiation Dynamics of Transplanted Neural Stem Cells after Traumatic Brain Injury
Scientists developed a polymer nanovehicle for morphogen sustained release to overcome the drawbacks of conventional methods to realize the long-term directed neural stem cell differentiation in vivo. [ACS Nano] Abstract

RNAi Mediated Silencing of hnRNP-A2/B1 Inhibits Inflammation and Bone Erosion in Two Experimental Models of Rheumatoid Arthritis
The nuclear protein hnRNP-A2/B1 is involved in post-transcriptional regulation of gene expression. It is constitutively expressed in lymphoid organs and highly up-regulated in synovial tissue of patients with rheumatoid arthritis (RA) who may also generate autoantibodies to this protein. To investigate its potential involvement in the pathogenesis of autoimmune arthritis, expression of hnRNP-A2/B1 was silenced in two animal models of RA. [Arthritis Rheumatol] Abstract

Long-Term Outcome of Extranodal NK/T Cell Lymphoma Patients Treated with Post-Remission Therapy Using EBV LMP1 and LMP2a-Specific CTLs
Investigators evaluated the efficacy and safety of Epstein-Barr virus (EBV) latent membrane protein (LMP)-1 and LMP-2a specific cytotoxic T cells (CTLs) stimulated with LMP1/2a RNA-transferred dendritic cells, they treated ten extranodal NK/T-cell lymphoma patients who showed complete response to induction therapy. [Mol Ther] Abstract

Pilot Study Evaluating Regulatory T Cell-Promoting Immunosuppression and Nonimmunogenic Donor Antigen Delivery in a Nonhuman Primate Islet Allotransplantation Model
Researchers report an immunotherapy regimen that combines 1-ethyl-3-(3′-dimethylaminopropyl)-carbodiimide-treated donor lymphoid cell infusion with thymoglobulin, anti-interleukin-6 receptor antibody and rapamycin to achieve prolonged allogeneic islet graft survival in a nonhuman primate model. [Am J Transplant] Abstract

Fabrication of Three-Dimensional Bioplotted Hydrogel Scaffolds for Islets of Langerhans Transplantation
Investigators used three-dimensional (3D) bioplotting for the fabrication of a 3D alginate-based porous scaffold as an extra-hepatic islet delivery system. In 3D-plotted alginate scaffolds the surface to volume ratio, and thus oxygen and nutrient transport, is increased compared to conventional bulk hydrogels. [Biofabrication]
Abstract | Full Article

Lentivirus-Induced ‘Smart’ Dendritic Cells: Pharmacodynamics and GMP-Compliant Production for Immunotherapy against TRP2-Positive Melanoma
Monocyte-derived conventional dendritic cells (ConvDCs) loaded with melanoma antigens showed modest responses in clinical trials. Efficacy studies were hampered by difficulties in ConvDC manufacturing and low potency. Overcoming these issues, scientists demonstrated higher potency of lentiviral vector-programmed DCs. [Gene Ther] Full Article

Intradiscal Application of rhBMP-7 Does Not Induce Regeneration in a Canine Model of Spontaneous Intervertebral Disc Degeneration
Investigators aimed to determine the most effective dose of an intradiscal injection of recombinant human bone morphogenetic protein 7 (rhBMP-7) in a spontaneous canine intervertebral disc degeneration model for translation into clinical application for patients with low back pain. [Arthritis Res Ther] Full Article

Immunological Basis for Rapid Progression of Diabetes in Older NOD Mouse Recipients Post BM-HSC Transplantation
Older non-obese diabetic (NOD) mice recipients receiving syngeneic bone marrow-derived hematopoietic stem cell (BM-HSC) progressed more rapidly to end-stage diabetes post-transplantation than younger recipients. [PLoS One] Full Article

Autologous iPSC-Derived Dopamine Neuron Transplantation in a Nonhuman Primate Parkinson’s Disease Model
Investigators tested the safety and efficacy of autologous induced pluripotent stem cell (iPSC)-derived autologous dopamine cells for treatment of a cynomolgus monkey Parkinson’s disease model. [Cell Discov] Full Article

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

Ludwig Scientists to Report Advances in Treatment of Brain, Skin, Gastrointestinal Cancers
Ludwig researchers will present recent data from early and late stage clinical trials and participate in a number of plenary and educational sessions. [Press release from Ludwig Cancer Research discussing research to be presented at the 2015 American Society for Clinical Oncology (ASCO) Annual Meeting, Chicago] Press Release

International Stem Cell Corporation to Present at International Society for Cellular Therapy Annual Meeting
International Stem Cell Corporation announced that the company has been selected to give an oral presentation on its preclinical studies in Parkinson’s. [Press release from International Stem Cell Corporation discussing research to be presented at the 21^st Annual Meeting of the International Society for Cellular Therapy, Las Vegas] Press Release

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A Note on Genome Editing
The National Academy of Sciences and its National Academy of Medicine announced that they will convene an international meeting this fall at which researchers, ethicists, and other experts will discuss the implications of human germline gene-editing technologies in both research and clinical applications. [Office of Science and Technology Policy] Press Release

Amgen and Merck Announce Expansion of Collaboration to Support Studies of Talimogene Laherparepvec in Combination with KEYTRUDA^® (Pembrolizumab) in Patients with Head and Neck Cancer
Amgen and Merck announced an expanded collaboration to evaluate the efficacy and safety of talimogene laherparepvec, Amgen’s investigational oncolytic immunotherapy, in combination with KEYTRUDA^® (pembrolizumab), Merck’s anti-PD-1 therapy, in a Phase I, open-label trial of patients with recurrent or metastatic squamous cell carcinoma of the head and neck. [Amgen Inc.] Press Release

Peregrine Pharmaceuticals Enters into Research Collaboration to Investigate Novel PS-Targeting Immunotherapy Combinations
Peregrine Pharmaceuticals, Inc., a biopharmaceutical company focused on advancing bavituximab, a novel immuno-oncology agent in Phase III development for the treatment of lung cancer, announced that the company has entered into a sponsored research agreement with Memorial Sloan Kettering Cancer Center to explore the potential of Peregrine’s proprietary phosphatidylserine (PS)-targeting antibody platform. [Peregrine Pharmaceuticals, Inc.] Press Release

bluebird bio and Five Prime Therapeutics Enter into License Agreement for Novel Antibodies to Develop Chimeric Antigen Receptor T Cell Therapy
bluebird bio, Inc. and Five Prime Therapeutics, Inc. announced that they have entered into an exclusive license agreement to research, develop and commercialize chimeric antigen receptor T cell therapies using Five Prime’s proprietary human antibodies to an undisclosed cancer target for hematologic malignancies and solid tumors. [bluebird bio, Inc.] Press Release

Juno Therapeutics and Editas Medicine Announce Exclusive Collaboration to Create Next-Generation CAR T and TCR Cell Therapies
Juno Therapeutics, Inc. and Editas Medicine announced an exclusive collaboration focused on creating chimeric antigen receptor (CAR T) and high-affinity T cell receptor (TCR) therapies to treat cancer. [Juno Therapeutics, Inc.] Press Release

Cancer Genetics Selected to Power Next-Generation Sequencing Services for ReproCELL, a Global Regenerative Medicine Market Leader, in a Multi-Year Agreement
Cancer Genetics, Inc. announced that it was selected by ReproCELL, Inc. to provide next-generation sequencing based services and molecular information to enhance ReproCELL’s translational medicine initiatives. [Cancer Genetics, Inc.] Press Release

Alnylam Initiates Phase I Clinical Trial for ALN-AS1, an Investigational RNAi Therapeutic Targeting Aminolevulinic Acid Synthase 1 (ALAS1) for the Treatment of Acute Hepatic Porphyrias, Including Acute Intermittent Porphyria (AIP)
Alnylam Pharmaceuticals, Inc. announced that it has initiated a Phase I clinical trial with ALN-AS1, a subcutaneously administered investigational RNAi therapeutic targeting ALAS1 for the treatment of acute hepatic porphyrias, including AIP. [Alnylam Pharmaceuticals, Inc.] Press Release

Regeneus Receives Approval for Personalized Cancer Immunotherapy Trial
Regeneus has received ethics approval to commence its first-in-human trial for a personalized therapeutic cancer vaccine that is aimed at harnessing the body’s own immune system to fight cancer cells. [Regeneus Ltd.] Press Release

MCW Researcher to Study Gene Therapies for Hemophilia
A researcher at the Medical College of Wisconsin (MCW) has received a four-year, $1.5 million dollar grant from the National Institutes of Health’s National Heart, Lung, and Blood Institute to continue her study of blood platelet gene therapies for hemophilia A, a genetic bleeding disorder. [Medical College of Wisconsin] Press Release

Center for Scientific Review; Notice of Closed Meetings (FR Doc. No: 2015-12546)

Agency Information Collection Activities; Submission for Office of Management and Budget Review; Comment Request; Requirements on Content and Format of Labeling for Human Prescription Drug and Biological Products (FR Doc. No: 2015-12957)

Agency Information Collection Activities; Proposed Collection; Comment Request; Prescription Drug Product Labeling; Medication Guide Requirements (FR Doc. No: 2015-12976)

Postmarketing Safety Reports for Human Drug and Biological Products; Electronic Submission Requirements; Delay of Compliance Date; Safety Reporting Portal of Electronic Submission of Postmarketing Safety Reports for Human Drugs and Nonvaccine Biological Products (FR Doc. No: 2015-12753)

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

Scientist – Reprogramming and Pluripotent Stem Cell Biology (STEMCELL Technologies Inc.)

Head of Stem Cells Immunotherapies Service Lab (NHS Blood and Transplant)

Postdoctoral Fellow – Immune Reconstitution in Hematopoietic Stem Cell Transplantation (Stanford University)

Head of Cellular Therapies Laboratory (NHS Blood and Transplant)

Postdoctoral Fellow – Immune Tolerance (Stanford University)

Postdoctoral Fellow Positions – iPSC Research and Banking (University of Magna Graecia)

In Vivo Biologist – Novel Cell and Gene Therapies (GlaxoSmithKline)

Process Facilitator (Opexa Therapeutics)

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