Cell Therapy News Volume 18.20 | Jun 12 2017

TOP STORY

Matrix Production Affects MRI Outcomes after Matrix-Associated Autologous Chondrocyte Transplantation in the Knee

The authors investigated the influence of gene expression of chondrocytes at the time of transplantation on MRI outcomes up to two years after matrix-associated autologous chondrocyte transplantation. [Am J Sports Med] Abstract

(Ranked by impact factor of the journal)

Human Liver Mesenchymal Stem/Progenitor Cells Inhibit Hepatic Stellate Cell Activation: In Vitro and In Vivo Evaluation

Scientists evaluated the influence of adult-derived human liver mesenchymal stem/progenitor cells (ADHLSCs) on hepatic stellate cells, both in vitro and in vivo. ADHLSCs were transplanted into a juvenile rat model of fibrosis established after co-administration of phenobarbital and CCl₄. [Stem Cell Res Ther] Full Article

Hematopoietic Cell Transplantation in Myelodysplastic Syndromes after Treatment with Hypomethylating Agents

Researchers identified 125 myelodysplastic syndrome (MDS) patients who had been treated with hypomethylating agent (HMA) and underwent subsequent hematopoietic cell transplantation. Among these, 68 were considered HMA failures, and 57 responders. Failure was defined as progression to higher grade MDS or acute myeloid leukemia, lack of hematological improvement after at least four HMA cycles, or loss of response after initial improvement. [Biol Blood Marrow Transplant] Abstract

In Vitro Maturation of Human iPS-Derived Neuroepithelial Cells Influences Transplant Survival in the Stroke-Injured Rat Brain

Cells were transplanted into an endothelin-1 stroke-injured rat brain and their survival, and fate were investigated one week later. Significantly more cells were found in the brain after transplanting early or mid- differentiated cortically-specified neuroepithelial stem cells compared to the late-differentiated population. [Tissue Eng Part A] Abstract

RNAi Targeting STMN Alleviates the Resistance to Taxol and Collectively Contributes to Down Regulate the Malignancy of NSCLC Cells In Vitro and In Vivo

In vivo animal experiments proved that stathmin (STMN) RNA interference (RNAi) and taxol cooperatively inhibited the tumorigenicity of NCI-H1299 cells and histological atypia and Ki-67 proliferative index of xenografted tumors and promoted cell differentiation to a higher grade with well-differentiated indicators of glandular lumen-like structure and proliferative fibroblasts. [Cell Biol Toxicol] Abstract

Transplantation of Allogeneic Adipose-Derived Mesenchymal Stem Cells in a Cerebral Palsy Patient

Investigators studied the safety and efficacy of allogeneic human adipose-derived mesenchymal cell (hAMSC) transplantation in a patient with cerebral palsy. The patient received six infusions of allogeneic hAMSCs intravenously, without or with intramuscular or local injections and was followed up for 12 months. [Regen Med] Abstract

Delivered Adipose-Derived Stromal Cells Improve Host-Derived Adipose Tissue Formation in Composite Constructs In Vivo

The authors used a mouse model for the development of volume-stable adipose tissue using polyurethane scaffolds combined with a long-term stable fibrin gel and adipose-derived stromal cells to investigate the influence of cell delivery on tissue development. [Laryngoscope] Abstract

Single Cell-Based Vector Tracing in Patients with ADA-SCID Treated with Stem Cell Gene Therapy

Scientists analyzed the engraftment of vector-transduced cells in two patients with severe combined immunodeficiency (SCID) who were treated with stem cell gene therapy. Sufficient engraftment of the transduced cells was limited to the T cell lineage in peripheral blood (PB), and a small percentage of CD34⁺ cells exhibited vector integration in bone marrow, indicating that the transgene-positive cells in PB might have differentiated from a small population of stem cells or lineage-restricted precursor cells. [Mol Ther Methods Clin Dev] Full Article | Graphical Abstract

Engineered CRISPR Systems for Next Generation Gene Therapies

The authors focus on evaluating CRISPR potential as a next generation in vivo gene therapy platform and discuss bioengineering advancements that can address challenges associated with clinical translation of this emerging technology. [ACS Synth Biol] Abstract | Graphical Abstract

Progress and Application of CRISPR/Cas Technology in Biological and Biomedical Investigation

Researchers discuss the recent development of the CRISPR/Cas gene-editing technology and how the extensive applications of the CRISPR platforms have improved strategies for biological and biomedical investigation, including disease modeling in animal and human cell line, development of gene therapy, as well as high-throughput genetic screening. [J Cell Biochem] Abstract

Gene Therapy for Pancreatic Cancer: Specificity, Issues and Hopes

Scientists give an overview of the general strategy of gene therapy as well as the limitations and stakes of different experimental in vivo models, expression vectors, molecular tools and therapeutic genes. [Int J Mol Sci] Full Article

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

Novartis Interim Results from Global, Pivotal CTL019 Trial Show Durable Complete Responses in Adults with r/r DLBCL

Novartis announced findings from an interim analysis of its multi-center Phase II JULIET study of CTL019 in adult patients with relapsed or refractory (r/r) diffuse large B-cell lymphoma (DLBCL). [Press release from Novartis AG discussing research presented at the 2017 International Conference on Malignant Lymphoma (ICML) Meeting, Lugano] Press Release

Asterias Biotherapeutics to Present New Nine-Month Efficacy Data from AST-OPC1 SCiStar Study

Asterias Biotherapeutics, Inc. announced that it will present new nine-month efficacy and safety data on those AIS-A patients treated with ten million cells of AST-OPC1 in the company’s ongoing SCiStar Phase I/IIa study in complete cervical spinal cord injury. [Press release from Asterias Biotherapeutics, Inc. discussing research to be presented at the 2017 International Society for Stem Cell Research (ISSCR) Annual Meeting, Boston] Press Release

Intellia Therapeutics and San Raffaele University and Research Hospital to Combine CRISPR/Cas9 Genome Editing with Enhanced Cell Therapies to Fight Cancer

Intellia Therapeutics and San Raffaele University and Research Hospital have entered into a three-year research collaboration, option and license agreement to engineer optimized T cell cancer therapies. [Intellia Therapeutics, Inc.] Press Release

Dragonfly Therapeutics Announces Strategic Collaboration with Celgene to Discover and Develop Novel Natural Killer (NK) Cell-Based Immunotherapies Using Dragonfly’s TriNKET™ Technology Platform

Dragonfly Therapeutics, Inc. announced a global strategic collaboration with Celgene Corporation and its affiliates to discover, develop and commercialize innovative immuno-oncology treatment options for patients with hematological malignancies based on Dragonfly’s NK cell based TriNKET™ technology platform. [Dragonfly Therapeutics, Inc. (PR Newswire Association LLC.)] Press Release

Myonexus Therapeutics, Inc. Launches to Develop Pioneering, Clinical-Stage Gene Therapies Targeting Limb-Girdle Muscular Dystrophies

Myonexus Therapeutics, Inc. announced its launch plans. The company is committed to accelerating development of five experimental gene therapies pioneered within the laboratories of Louise Rodino-Klapac, Ph.D. and Jerry Mendell, M.D., Principal Investigators at the Research Institute at Nationwide Children’s Hospital Center for Gene Therapy in Columbus, Ohio, USA. [Myonexus Therapeutics, Inc.] Press Release

Lonza Acquires Cell and Gene Contract Manufacturer PharmaCell

Lonza successfully completed the acquisition of PharmaCell B.V. PharmaCell was primarily selected due to its core expertise in autologous cell and gene therapy manufacturing, which includes experience with two commercial advanced therapy medicinal products-licensed products. [Lonza] Press Release

VBL Therapeutics Awarded $2.5 Million Grant by the Israel Innovation Authority

VBL Therapeutics announced that it has been awarded a grant of 8.75 million New Israeli Shekels by the Israel Innovation Authority. The grant will support clinical trials and development activities for calendar year 2017. [VBL Therapeutics] Press Release

Cell and Gene Therapy Catapult Licenses Cell Culture Data to Ajinomoto Co., Inc.

The Cell and Gene Therapy Catapult (CGT Catapult) announced it has licensed data from its pluripotent stem cell development program ‘Cell Plasticity’ to Ajinomoto Co., Inc., further advancing CGT Catapult’s place as one of the global leaders in the development of solutions for the industrialization of cell therapies. [The Cell and Gene Therapy Catapult] Press Release

NantKwest Expands NANT Cancer Vaccine Program with Additional Clinical Trials Announced Addressing Multiple Cancer Types across All Stages of Disease

NantKwest and Nantworks announced the expansion of the company’s existing NANT Cancer Vaccine program in pancreatic cancer to target a number of additional tumor types. [NantKwest] Press Release

CBSET Expands Its Preclinical Research Services by Adding GLP-Compliant Cell-Based-Therapies Support Capability

CBSET, Inc. announced that it has expanded its preclinical cell therapy offerings. CBSET recognizes the importance of preclinical partners capable of supporting the needs of the rapidly advancing cell therapy field by providing technical and regulatory support services in the cell therapy and regenerative medicine sectors. [CBSET, Inc.] Press Release

Curiosity and Irritation Meet Macron’s Effort to Lure Foreign Scientists to France

The French government unveiled a website aimed at attracting foreign scientists with four-year grants worth up to €1.5 million each. [ScienceInsider] Editorial

Trump Names Sharpless to Lead U.S. Cancer Institute

President Donald Trump announced his intention to appoint Norman “Ned” Sharpless to be the next director of the National Cancer Institute in Bethesda, Maryland. Sharpless, who is 50 years old, is a physician and currently director of the Lineberger Comprehensive Cancer Center at the University of North Carolina in Chapel Hill. He also holds an academic appointment at the university’s school of medicine. [ScienceInsider] Editorial

UK Scientists Hope for Softened Brexit after Shock Election Result

The United Kingdom (UK)’s general election produced no clear outcome — but scientists trying to divine meaning from the chaos say they hope the result will ultimately benefit their country’s research ties with the European Union. [Nature News] Editorial

New Clues to Why a French Drug Trial Went Horribly Wrong

Scientists are one step closer to understanding how a clinical trial in France killed one volunteer and led to the hospitalization of five others. A new study shows that the compound tested in the study, BIA 10-2474, has effects on many other enzymes in addition to the one it was supposed to inhibit. These “off-target” effects might explain why the drug caused side effects ranging from headaches to irreversible brain damage. [ScienceInsider] Editorial

FDA

Bacteriophage Therapy: Scientific and Regulatory Issues; Public Workshop (FR Doc. No:2017-11862) Notice

NEW The 5^th Annual Next Gen Stem Cell Conference
August 3-4, 2017
Hartford, Connecticut, United States

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

NEW Quality Improvement Coordinator – Cellular Therapy Core (University of Texas Health Science Center at Houston)

Tenure-Track Professorship – Experimental Cell Therapy (University of Bonn)

Postdoctoral Position – Tumor Immunology and Cancer Immunotherapy (VIB)

Professor – Stem Cell Biology (University College London)

Postdoctoral Researcher – Cellular Therapy for Degenerative Disc Disease (NUI Galway)

Specialist – Cell Procurement (Celgene Corporation)

Head – CAR-T Operations and Technology (Celgene Corporation)

Scientist – Analytical Development (KBI Biopharma)

PhD Fellow – Experimental Genetic Cardiology (Oslo University Hospital)

GMP Manufacturing Director – Cell and Gene Therapy (Fred Hutchinson Cancer Research Center)

Research Fellow – Functional Roles of Stem Cell Derived Extracellular Vesicles (Mayo Clinic)

Professor and Faculty Positions – Various Projects (Academy of Medical Sciences, Zhengzhou University)

Postdoctoral Fellow – Various Projects (University of Oklahoma)

GMP Manufacturing Director – Cell and Gene Therapy (Fred Hutchinson Cancer Research Center)

Assistant or Associate Member – Stem CellGene Therapy (Fred Hutchinson Cancer Research Center)

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