Cell Therapy News Volume 21.02 | Jan 20 2020

TOP STORY

Safety and Feasibility of Anti-CD19 CAR T Cells with Fully Human Binding Domains in Patients With B-Cell Lymphoma

Researchers treated 20 patients with B-cell lymphoma on a phase I, first-in-human clinical trial of T cells expressing the new anti-CD19 CAR Hu19-CD828Z. Their primary objective was to assess safety and feasibility of Hu19-CD828Z T-cell therapy. Secondary objectives included assessments of blood levels of CAR T cells, anti-lymphoma activity, second infusions and immunogenicity. [Nat Med] Abstract

(Ranked by impact factor of the journal)

Human Umbilical Cord Perivascular Cells Improve Human Pancreatic Islet Transplant Function by Increasing Vascularization

Transplanting an islet equivalent human umbilical cord perivascular mesenchymal stromal cells ratio of 1:30 under the kidney capsule in diabetic NSG mice demonstrated the fastest return to normoglycemia by three days after transplant: Superior glycemic control was seen at both early and later stages versus ratios of 1:0, 1:10, and 1:50, respectively. [Sci Transl Med] Abstract

Glypican-3-Specific CAR T Cells Co-Expressing IL15 and IL21 Have Superior Expansion and Antitumor Activity against Hepatocellular Carcinoma

The authors generated constructs encoding IL15, IL21, or both with GPC3-CAR incorporating a 4-1BB costimulatory endodomain and examined the ability of transduced T cells to kill, produce effector cytokines, and expand in an antigen-dependent manner. They performed gene expression and phenotypic analyses of GPC3-CAR T cells and CRISPR-Cas9 knock-out of the TCF7 gene. [Cancer Immunol Res] Abstract

HDAC10 Deletion Promotes Foxp3⁺ T-Regulatory Cell Function

Scientists investigated the effect of histone/protein deacetylases (HDAC)10 in murine Tregs. HDAC10 deletion had no adverse effect on the health of mice, which retained normal CD4⁺ and CD8⁺ T cell function. However, HDAC10^−/− Treg exhibited increased suppressive function in vitro and in vivo. [Sci Rep] Full Article

Efficient Generation of Thymic Epithelium from Induced Pluripotent Stem Cells That Prolongs Allograft Survival

Induced pluripotent stem cells (iPSC)-derived thymic epithelial cells) (TECs) generated T cells in nude recipient mice after renal subcapsular transplantation. Moreover, iPSC-TEC transplantation to immuno-competent recipients significantly prolonged the survival of allogeneic skin. [Sci Rep] Full Article

Isolation Methods for Human CD34 Subsets Using Fluorescent and Magnetic Activated Cell Sorting: an In Vivo Comparative Study

Mobilized human mononuclear peripheral blood cells (mhPBMNCs) were processed following acquisition using fluorescence activated cell sorting (FACS) or magnetic beads cell (MACS) sorting according to clinically established protocols. For in vivo characterization of cardiac repair, the authors conducted LAD ligation surgery on eight-ten weeks old female NOD/SCID mice followed by intramyocardial transplantation of unselected mhPBMNCs, FACS or MACS enriched CD34⁺ cells. [Stem Cell Rev Rep] Abstract

Rapamycin-Preactivated Autophagy Enhances Survival and Differentiation of Mesenchymal Stem Cells after Transplantation into Infarcted Myocardium

Mesenchymal stem cells isolated from rat bone marrow were treated with 50 nmol/L rapamycin for two hours, and then the cytoprotective effect of rapamycin was examined. After intramyocardial transplantation in rat ischemia/reperfusion models, the survival and differentiation of the rapamycin-pretreated calls were (assessed?. [Stem Cell Rev Rep] Full Article

Sonic Hedgehog Upregulation Does Not Enhance the Survival and Engraftment of Stem Cell-Derived Cardiomyocytes in Infarcted Hearts

An adenovirus expressing the vascular growth factor Sonic Hedgehog (Shh) was injected into the infarcted myocardium of rats immediately after ischemia/reperfusion, four days prior to human stem cell-derived cardiomyocytes injection. By two weeks post-cell injection, Shh treatment had successfully increased capillary density outside the scar, but not within the scar. [PLoS One] Full Article

Human Adipose-Derived Mesenchymal Stem Cells, Low-Intensity Pulsed Ultrasound, or Their Combination for the Treatment of Knee Osteoarthritis: Study Protocol for a First-in-Man Randomized Controlled Trial

This study involves a prospective, randomized, placebo-controlled, and single-blind trial based on the SPIRIT guidelines, and aimed to recruit 96 patients initially diagnosed with knee osteoarthritis, following American College of Rheumatology criteria. Patients were randomized in a 1:1:1 ratio to receive Intraarticular human adipose-derived mesenchymal stem cells (HADMSCs) injection with low-intensity pulsed ultrasound (LIPUS), Intraarticular HADMSCs injection with shame LIPUS, or Normal saline with LIPUS. [BMC Musculoskelet Disord] Full Article

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Calcium Phosphate Nanoparticles-Based Systems for RNAi Delivery: Applications in Bone Tissue Regeneration

Gene therapy is an emerging approach for effective bone repair and regeneration, with notable interest in the use of RNA interference (RNAi) systems to regulate gene expression in the bone microenvironment. Calcium phosphate nanoparticles represent promising materials for use as non-viral vectors for gene therapy in bone tissue engineering applications due to their many favorable properties, including biocompatibility, osteoinductivity, osteoconductivity, and strong affinity for binding to nucleic acids. [Nanomaterials (Basel)] Full Article

Autologous CAR T-Cell Therapies Supply Chain: Challenges and Opportunities?

The authors present an overview of the current state of the art in the CAR T cell market and present novel concepts that can debottleneck key elements of the current supply chain model and, they believe, help this technology achieve its long-term potential. [Cancer Gene Ther] Abstract

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

Iovance Biotherapeutics and Cellectis Enter into a Research Collaboration and Exclusive Worldwide License Agreement

Iovance Biotherapeutics, Inc. announced that the companies have entered into a research collaboration and exclusive worldwide license agreement whereby Iovance will license certain TALEN^® technology from Cellectis in order to develop tumor infiltrating lymphocytes that have been genetically edited to create more potent cancer therapeutics. [Iovance Biotherapeutics] Press Release

Abeona Therapeutics Initiates Pivotal Phase III Clinical Trial Evaluating EB-101 Gene Therapy for Recessive Dystrophic Epidermolysis Bullosa

Abeona Therapeutics Inc. announced that it has received Institutional Review Board approval from Stanford University to commence the VIITAL™ study, the company’s pivotal Phase III clinical trial evaluating EB-101 for the treatment of recessive dystrophic epidermolysis bullosa. [Abeona Therapeutics Inc.] Press Release

Axovant Announces Positive 12-Month Data on AXO-Lenti-PD and Provides Updates across Gene Therapy Pipeline Programs

Axovant Gene Therapies Ltd. announced recent progress in its gene therapy programs, including a 12-month update on its Parkinson’s program. At 12 months post-dosing, patients in the first dose cohort demonstrated an average 22-point change from baseline in motor function as assessed by the UPDRS Part III “OFF” score, which represents a 37% improvement. [Axovant Gene Therapies Ltd] Press Release

Precision BioSciences Announces FDA Acceptance of IND for PBCAR269A, a BCMA Targeted Genome Edited Allogeneic CAR T Therapy Candidate for Multiple Myeloma

Precision BioSciences, Inc. announced that the FDA has accepted its Investigational New Drug (IND) application for PBCAR269A, the company’s third allogeneic chimeric antigen receptor (CAR) T cell therapy candidate. The FDA has also granted Orphan Drug Designation to PBCAR269A for the treatment of multiple myeloma. [Precision BioSciences] Press Release

ISSCR Announces 2020 Award Winners

Stem cell scientists and leaders earn 2020 International Society for Stem Cell Research (ISSCR) Awards for their significant contributions to the field of stem cell research. Honorees include Fred H. Gage, PhD, Salk Institute for Biological Studies and Mitinori Saitou, MD, PhD, Kyoto University. [The International Society for Stem Cell Research] Press Release

MDA Awards Venture Philanthropy Funding of More Than $1M to AavantiBio to Develop Gene-Targeting Therapy for Friedreich’s Ataxia

The Muscular Dystrophy Association (MDA) and AavantiBio announced the award of MDA Venture Philanthropy funding totaling $1,076,232 to advance AavantiBio’s Phase II clinical trial of a gene-replacement therapy for the disease. [The Muscular Dystrophy Association] Press Release

Research on Embryo-Like Structures Struggles to Win US Government Funding

Scientists can now create clumps of cells that resemble human embryos, raising hopes that they could study the elusive first stages of human development while avoiding the ethical concerns that make it difficult to study actual human embryos. But as these embryo models grow in popularity, US researchers say they are finding it increasingly difficult to obtain federal funding for such work. [Nature News] Editorial

Campus Attacks by Nationalists and Police Alarm India’s Scientific Community

The attack on one of India’s most prestigious universities sent shock waves around the country and is the latest sign that the political forces tearing apart Indian society are also affecting the country’s academic community. Students at JNU, a liberal bastion, had been on strike for months against both a major hike in student fees and the government’s controversial Citizenship Amendment Act, widely decried as discriminatory against Muslims. [ScienceInsider] Editorial

Moffitt Cancer Center Details Links of Fired Scientists to Chinese Talent Programs

Six Florida cancer researchers who were dismissed last month for hiding their ties to a Chinese medical university appear to have been motivated by simple greed and a disregard for both institutional and federal rules. [ScienceInsider] Editorial

NEW 4^th DNA Repair/Replication Structures and Cancer Conference
February 16-20, 2020
Nassau, Bahamas

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NEW Research Assistant – Organoid Modeling for Stem Cell Therapies (Boston Children’s Hospital)

Research Fellow – Regenerative Medicine (University of Edinburgh)

Postdoctoral Researcher – Personalized Cancer Treatments (National University of Ireland Galway)

Postdoctoral Researcher – Drug Delivery (University of Minnesota Twin Cities)

Postdoctoral Researchers – Cell Biology (Medicinal Bioconvergence Research Center)

Postdoctoral Scientist – Functional Pharmacology (Genentech, Inc.)

Postdoctoral Researcher – Ciliopathies and Neurogenetics (University of Alberta)

Junior Research Fellow – Synthetic Biology (Dresden University of Technology)

Postdoctoral Fellow – Cancer Research (BC Cancer)

Research Assistant – Cancer Research (BC Cancer)

Postdoctoral Fellow – Single Cell Genomics (BC Cancer)

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