Cell Therapy News Volume 17.27 | Jul 18 2016

TOP STORY

New Approach for Regenerative Therapy

The marker Flattop subdivides the insulin-producing beta cells of the pancreas into those that maintain glucose metabolism and into immature cells that divide more frequently and adapt to metabolic changes. This could provide a starting point for regenerative diabetes therapies, as scientists report. [Press release from the Helmholtz Zentrum München discussing online prepublication in Nature] Press Release | Abstract

(Ranked by impact factor of the journal)

An miRNA-Mediated Therapy for SCA6 Blocks IRES-Driven Translation of the CACNA1A Second Cistron

Researchers identified miR-3191-5p as a microRNA (miRNA) that targeted CACNA1A internal ribosomal entry site (IRES) and preferentially inhibited the CACNA1A IRES–driven translation of α1ACT in an Argonaute 4–dependent manner. They found that eukaryotic initiation factors (eIFs), eIF4AII and eIF4GII, interacted with the CACNA1A IRES to enhance α1ACT translation. [Sci Transl Med] Abstract

Neurons Differentiated from Transplanted Stem Cells Respond Functionally to Acoustic Stimuli in the Awake Monkey Brain

Researchers examined whether neurons differentiated from transplanted stem cells can integrate into the host neural network and function in awake animals, a goal of transplanted stem cell therapy in the brain. [Cell Rep] Full Article | Graphical Abstract

Cardiac Chemical Exchange Saturation Transfer MR Imaging Tracking of Cell Survival or Rejection in Mouse Models of Cell Therapy

One million C2C12 cells labeled with either europium 10-(2-hydroxypropyl)-1,4,7-tetraazacyclododecane-1,4,7-triacetic acid or saline via the hypotonic swelling technique were implanted into the anterior-lateral left ventricular wall in C57BL/6J and C3H mice. Imaging was performed 1, 10, and 20 days after implantation, with the asymmetrical magnetization transfer ratio calculated from image pairs. [Radiology] Abstract

Feasibility and Safety of RNA-Transfected CD20-Specific Chimeric Antigen Receptor T cells in Dogs with Spontaneous B cell lymphoma

Preclinical murine models of chimeric antigen receptor (CAR) T cell therapy are widely applied, but are greatly limited by their inability to model the complex human tumor microenvironment and adequately predict safety and efficacy in patients. Investigators therefore sought to develop a system that would enable us to evaluate CAR T cell therapies in dogs with spontaneous cancers. [Mol Ther] Abstract

Tumorigenicity and Immunogenicity of Induced Neural Stem Cell Grafts versus Induced Pluripotent Stem Cell Grafts in Syngeneic Mouse Brain

Along with the development of stem cell-based therapies for central nervous system (CNS) disease, the safety of stem cell grafts in the CNS, such as induced pluripotent stem cells and induced neural stem cells, should be of primary concern. To provide scientific basis for evaluating the safety of these stem cells, the authors determined their tumorigenicity and immunogenicity in syngeneic mouse brain. [Sci Rep] Full Article

Function of Human Pluripotent Stem Cell-Derived Photoreceptor Progenitors in Blind Mice

The study assessed the therapeutic potential of photoreceptor progenitors derived from human embryonic and induced pluripotent stem cells using a protocol that is suitable for future clinical trials. [Sci Rep] Full Article

Manufacture of GMP-Compliant Functional Adenovirus-Specific T-Cell Therapy for Treatment of Post-Transplant Infectious Complications

Scientists described the clinical scale Good Manufacturing Practice (GMP)-compliant manufacture and characterization of 40 adenovirus (ADV)-specific T-cell products, Cytovir ADV, which are currently being tested in a multi-center Phase I/IIa clinical trial. Their process requires minimal intervention, is high yield, and results in a pure T-cell product that is functional. [Cytotherapy] Abstract

Single Residue AAV Capsid Mutation Improves Transduction of Photoreceptors in the Abca4^-/- Mouse and Bipolar Cells in the rd1 Mouse and Human Retina Ex-Vivo

The authors assessed two vectors with single capsid mutations, rAAV2/2(Y444F) and rAAV2/8(Y733F) in their ability to transduce retina in the Abca4^-/- and rd1 mouse models of retinal degeneration. [Gene Ther] Full Article

Potent Spinal Parenchymal AAV9-Mediated Gene Delivery by Subpial Injection in Adult Rats and Pigs

Scientists demonstrated that the spinal pia membrane represents the primary barrier limiting effective adeno-associated virus (AAV) 9 penetration into the spinal parenchyma after intrathecal AAV9 delivery. They developed a novel subpial AAV9 delivery technique and AAV9-dextran formulation. [Mol Ther Methods Clin Dev] Full Article

Epigenetic Control of Adult Stem Cell Function

The authors explore the effects of epigenetic dysregulation on adult stem cell function. They conclude that, depending on the tissue type and the epigenetic regulator affected, the consequences range from negligible to stem cell malfunction and disruption of tissue homeostasis, which may predispose to diseases such as cancer. [Nat Rev Mol Cell Biol] Abstract

Cardiac Progenitor Cells for Heart Repair

The heart has been traditionally considered a post-mitotic organ, however, this view has recently changed with the identification of stem/progenitor cells residing within the adult heart. Given their cardiac developmental origins, these endogenous cardiac progenitor cells may represent better candidates for cardiac cell therapy compared with stem cells from other organs such as the bone marrow and adipose tissue. [Cell Death Disc] Full Article

Generating Kidney Tissue From Pluripotent Stem Cells

The authors review recent progress towards the recreation of not only specific kidney cell types but complex kidney organoids, models of the developing human organ, in vitro. They also discuss potential short and long term applications of these approaches. [Cell Death Disc] Full Article

Visit our reviews page to see a complete list of reviews in the cell therapy research field.

Vigilant Biosciences Announces Oral Presentation on CD44 and Associated Markers in Oral Rinses and Tissues from Oral Cancer Patients

Vigilant Biosciences, Inc. announced a presentation of data related to proof-of-concept studies by Sylvester Comprehensive Cancer Center at the University of Miami Miller School of Medicine, titled CD44 and Associated Markers in Oral Rinses and Tissues from Oral Cancer Patients. [Press release from Vigilant Biosciences, Inc. discussing research presented at American Head and Neck Society 9^th International Conference on Head and Neck Cancer, Seattle] Press Release

AiVita Biomedical Awarded CIRM Grant for Development of 3D-Transplantable ‘Retinas’

AiVita Biomedical announced that the California Institute for Regenerative Medicine (CIRM) has approved the company to collaborate on an early translational research awards grant for the development of stem cell-derived 3D-transplantable retinas to treat vision loss. [AiVita Biomedical] Press Release

First Patient is Randomized in STEMTRA Trial For Traumatic Brain Injury

SanBio, Inc. announced the randomization of the first patient in the STEMTRA Phase II clinical trial study for traumatic brain injury. The STEMTRA “Stem cell therapy for traumatic brain injury” trial will examine the effects of SB623 stem cell treatment in patients with chronic motor deficits resulting from traumatic brain injury. [SanBio, Inc.] Press Release

Kite Pharma Completes Enrollment of All Patients with Diffuse Large B-Cell Lymphoma (DLBCL) in Pivotal Multi-Center Phase II Study of KTE-C19 (ZUMA-1)

Kite Pharma, Inc. announced that it has completed enrollment of 72 patients in DLBCL cohort in the Phase II portion of ZUMA-1. KTE-C19 is an investigational therapy in which a patient’s T-cells are genetically modified to express a chimeric antigen receptor that is designed to target the antigen CD19, a protein expressed on the cell surface of B-cell lymphomas and leukemias. [Kite Pharma, Inc.] Press Release

Juno Therapeutics to Resume JCAR015 Phase II ROCKET Trial

Juno Therapeutics, Inc. announced that the U.S. Food and Drug Administration has removed the clinical hold on the Phase II clinical trial of JCAR015 in adult patients with relapsed or refractory B cell acute lymphoblastic leukemia. [Juno Therapeutics, Inc.] Press Release | Editorial

BrainStorm Announces Positive Top Line Results from the U.S. Phase II Study of NurOwn^® in Patients with Amyotrophic Lateral Sclerosis (ALS)

BrainStorm Cell Therapeutics Inc. announced results from the recently completed U.S. randomized, double-blind, placebo-controlled Phase II Study of NurOwn^® in Patients with ALS. [BrainStorm Cell Therapeutics Inc.] Press Release

Investing in Cures for Cancer: GE Poised to Lead Industrialization of Cell Therapy Industry with Acquisition of Biosafe Group SA

GE Healthcare has acquired Biosafe Group SA, a supplier of integrated cell bioprocessing systems for the rapidly growing cell therapy and regenerative medicine industry for an undisclosed sum. [GE Healthcare] Press Release

Asterias Biotherapeutics Completes Enrollment and Dosing for the First Efficacy Cohort of the AST-OPC1 SCiSTAR Phase I/IIa Clinical Trial in Complete Cervical Spinal Cord Injury

Asterias Biotherapeutics, Inc. announced completion of enrollment and dosing of five patients in the first efficacy cohort of the SCiSTAR study, a Phase I/IIa clinical trial evaluating activity of escalating doses of AST-OPC1 (oligodendrocyte progenitor cells) in newly injured patients with sensory and motor complete cervical spinal cord injury. [Asterias Biotherapeutics, Inc.] Press Release

Regeneron Announces 2016 Winners of the Regeneron Prize for Creative Innovation

Regeneron Pharmaceuticals, Inc. announced the winners of the fourth annual Regeneron Prize for Creative Innovation, an award designed to acknowledge, reward and foster talented early-career biomedical scientists. This year, Regeneron awarded $155,000 in prize money to 10 awardees and two institutions. [Regeneron Pharmaceuticals, Inc.] Press Release

Thrive Bioscience Announces Instruments and Services Agreement with the Harvard Stem Cell Institute

Thrive Bioscience and the Harvard Stem Cell Institute (HSCI) announced an agreement under which Thrive will develop instruments for the growth and maintenance of induced pluripotent stem cells. [Thrive Bioscience] Press Release

FASEB Welcomes New President and Officers

The Federation of American Societies for Experimental Biology (FASEB) welcomed its new President, Hudson H. Freeze, PhD. Dr. Freeze is Professor of Glycobiology and Director of the Human Genetics Program at the Sanford-Burnham-Prebys Medical Discovery Institute in La Jolla. [Federation of American Societies for Experimental Biology] Press Release

NIH Expands Investment in HIV Cure Research

The National Institutes of Health has awarded approximately $30 million in annual funding over the next five years to six research collaborations working to advance basic medical science toward an HIV cure. [National Institutes of Health] Editorial

Drugs Watchdog Thwarting UK Progress in Stem Cell Therapy, Report Says

Britain’s hopes of leading the world in revolutionary therapies that regenerate damaged body parts are under threat because the NHS drugs watchdog cannot properly evaluate them, a report says. [The Guardian] Editorial | Report

Science’s Status Shifts in New Brexit Government

Three weeks after UK voters chose to leave the European Union, the country has a new prime minister, Theresa May — and a revamped administration that is poised to change science’s place in government. [Nature News] Editorial

Science Under Siege: How Venezuela’s Economic Crisis Is Affecting Researchers

The political and economic crisis in Venezuela continues to worsen. As the price of oil — the country’s major export — has fallen, Venezuela has struggled to pay for imported goods while maintaining socialist economic policies put in place by former president Hugo Chávez. [Nature News] Editorial

Research Charities Help Marry Two Major South African HIV/Tuberculosis Institutes

As the International AIDS Conference kicked off in Durban, South Africa, two of the nation’s most prominent biomedical research institutions announced that they will marry and combine resources to attack the raging coepidemic of tuberculosis and HIV in the region. [ScienceInsider] Editorial

FDA

Principles for Codevelopment of an In Vitro Companion Diagnostic Device with a Therapeutic Product; Draft Guidance for Industry and Food and Drug Administration Staff; Availability (FR Doc. No:2016-16735) Notice

Agency Information Collection Activities; Submission for Office of Management and Budget Review; Comment Request; Clinical Laboratory Improvement Amendments Act of 1988 Waiver Applications (FR Doc. No:2016-16886) Notice

Refurbishing, Reconditioning, Rebuilding, Remarketing, Remanufacturing, and Servicing of Medical Devices Performed by Third-Party Entities and Original Equipment Manufacturers; Public Workshop (FR Doc. No:2016-16887) Notice

NEW 4^th International Oncology Conference
September 29-30, 2016
Abu Dhabi, United Arab Emirates

NEW International Workshop on Waldenstrom’s Macroglobulinemia & Symposium on Multiple Myeloma
October 5-8, 2016
Amsterdam, Netherlands

Visit our events page to see a complete list of events in the cell therapy community.

NEW Center Manager – Bioengineering and Nanotechnology (The University of Queensland)

NEW Research Assistants – Pluripotent Stem Cell Biology (The University of Queensland)

NEW Postdoctoral Research Fellows – Stem Cell, Niche and Systemic Ageing (The University of Queensland)

Scientist – Bioengineering (STEMCELL Technologies Inc.)

Postdoctoral Research Scientist – Cancer Immunotherapy (Children’s Hospital of Eastern Ontario Research Institute)

Resource Technologist B – Cellular Manufacturing Activities for Cell and Gene Therapy (University of Pennsylvania)

Postdoctoral Fellow – Pluripotent Stem Cell Technology (University of California Davis)

Staff Scientist – Cell Therapy Process Development (Fred Hutchinson Cancer Research Center)

Associate Member – Stem Cell and Gene Therapy (Fred Hutchinson Cancer Research Center)

Faculty Position – Cancer Immunology (University of New Mexico)

Postdoctoral Fellow – Pathogenic Mechanisms of Rare Bone Disorders (University of Connecticut Health)

Postdoctoral Fellow – Oxidative Lung Injury (Temple University)

Postdoctoral Fellow – Lentivirus-Mediated FOXP3 Gene Transfer (Stanford University School of Medicine)

Postdoctoral Fellowship – Deciphering the Link between Adhesion and Pluripotency (The Danish Stem Cell Center at Faculty of Health & Medical Sciences at the University of Copenhagen)

Scientist – Target Validation (Innate Pharma)

Postdoctoral Fellow – Data Analysis and Stem Cell Research (Nestle Institute of Health Sciences)

Postdoctoral Fellow – Stem Cells to Study Disease (Hamad bin Khalifa University)

Postdoctoral Fellow – Protein and Antibody Engineering (Hamad bin Khalifa University)

Postdoctoral Fellow – Cancer Immunology and Immunotherapy (Hamad bin Khalifa University)

Postdoctoral Fellow – Hematology/Oncology (Boston Children’s Hospital)

Postdoctoral Research Fellow – Cell Cycle Control and Tumorigenesis (Fred Hutchinson Cancer Research Center)

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