Cell Therapy News Volume 19.05 | Feb 19 2018

TOP STORY

Smart Bomb Virus Shows Promise as Brain Tumor Immunotherapy

A common cold virus engineered to attack the most common and deadly of brain tumors allowed 20 percent of patients with recurrent glioblastoma to live for three years or longer, researchers report on a Phase I clinical trial. [Press release from MD Anderson Cancer Center discussing online prepublication in the Journal of Clinical Oncology] Press Release | Abstract

(Ranked by impact factor of the journal)

Complete Remission with Reduction of High-Risk Clones following Haploidentical NK Cell Therapy against MDS and AML

Scientists evaluated the safety, efficacy and immunobiological correlates of allogeneic NK cell-based therapy in primary chemotherapy-refractory or relapsed high-risk myelodysplastic syndrome (MDS), secondary AML, and de novo AML patients. [Clin Cancer Res] Abstract | Full Article | Press Release

Generation of Functioning Nephrons by Implanting Human Pluripotent Stem Cell-Derived Kidney Progenitors

Kidney progenitors were subcutaneously implanted into immunodeficient mice. By 12 weeks, they formed organ-like masses detectable by bioluminescence imaging. Implants included perfused glomeruli containing human capillaries, podocytes with regions of mature basement membrane, and mesangial cells. [Stem Cell Reports] Full Article | Press Release

Neurons Generated by Mouse ESCs with Hippocampal or Cortical Identity Display Distinct Projection Patterns when Co-Transplanted in the Adult Brain

By manipulating Wnt and BMP signaling, investigators steered the differentiation of mouse embryonic stem cells (ESCs) toward isocortical or hippocampal molecular identity. These two types of cells showed different degrees of axonal outgrowth and targeted different regions when co-transplanted in healthy or lesioned isocortex or in hippocampus. [Stem Cell Reports] Full Article | Graphical Abstract

Human ISL1⁺ Ventricular Progenitors Self-Assemble into an In Vivo Functional Heart Patch and Preserve Cardiac Function after Infarction

The authors documented a specific temporal window in which human pluripotent stem cell-derived ventricular progenitors (HVPs) will engraft in vivo. On day six of differentiation, HVPs were MAC-sorted for pluripotency marker Tra-1-60, and three million sorted cells were sub-capsularly transplanted onto kidneys of NSG mice where, after two months, they formed a 7mm x 3mm x 4mm myocardial patch resembling the ventricular wall. [Mol Ther] Abstract | Full Article

Supplemental Treatment for Huntington’s Disease with miR-132 that Is Deficient in Huntington’s Disease Brain

Scientists showed amelioration in motor function and lifespan of Huntington’s disease (HD)-model mice, R6/2 mice, by supplying miR-132 to HD brains using a recombinant adeno-associated virus miRNA expression system. [Mol Ther Nucleic Acids] Full Article

Antitumor Activity of EGFR-Specific CAR T Cells against Non-Small-Cell Lung Cancer Cells In Vitro and in Mice

Modified CAR T cells exhibited expansion capability and anticancer efficacy in a time- and antigen-dependent manner in vitro as well as regression of epidermal growth factor receptor (EGFR)-positive human lung cancer xenografts in vivo. [Cell Death Dis] Full Article

OPA1 Gene Therapy Prevents Retinal Ganglion Cell Loss in a Dominant Optic Atrophy Mouse Model

Researchers tested the efficiency of gene therapy on a genetically-modified mouse model reproducing dominant optic atrophy vision loss. They performed intravitreal injections of an adeno-associated virus carrying the human OPA1 cDNA under the control of the cytomegalovirus promotor. [Sci Rep] Full Article

Protein-Engineered Hydrogels Enhance the Survival of Induced Pluripotent Stem Cell-Derived Endothelial Cells for Treatment of Peripheral Arterial Disease

Scientists demonstrated the improved acute viability of human induced pluripotent stem cell-derived endothelial cells following syringe injection delivery in Shear-thinning Hydrogel for Injectable Encapsulation and Long-term Delivery, compared to saline. [Biomater Sci] Abstract

Programming Gene and Engineered-Cell Therapies with Synthetic Biology

Gene and engineered-cell therapies promise to treat diseases by genetically modifying cells to carry out therapeutic tasks. Although the field has had some success in treating monogenic disorders and hematological malignancies, current approaches are limited to overexpression of one or a few transgenes, constraining the diseases that can be treated with this approach and leading to potential concerns over safety and efficacy. [Science] Abstract

Potentiating Prostate Cancer Immunotherapy with Oncolytic Viruses

Oncolytic-virus-induced subversion of tumor-associated immunosuppression can potentiate the effectiveness of current immunotherapeutics, including immune checkpoint inhibitors and chemotherapeutics that induce immunogenic cell death. [Nat Rev Urol] Abstract

Potential of Stem Cell-Based Therapy for Ischemic Stroke

Investigators provide a synopsis of different preclinical and clinical studies related to the use of stem cell-based stroke therapy, and explore possible beneficial/detrimental outcomes associated with the use of different types of stem cells. [Front Neurol] Full Article

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

Obsidian Therapeutics to Present Data Demonstrating Regulation of Protein Activity with Its Next-Generation CAR-T Platform

Obsidian Therapeutics, Inc. announced the presentation of preclinical data on its technology platform and applications. [Press release from Obsidian Therapeutics, Inc. (Business Wire Inc.) discussing research presented at the 2018 Keystone Symposium on Emerging Cellular Therapies: T Cells and Beyond, Keystone] Press Release

Poseida Therapeutics Presents Stem Cell Memory CAR-T Therapy for Prostate Cancer

Poseida Therapeutics Inc. presented preclinical data on P-PSMA-101. P-PSMA-101 is the company’s PSMA-specific stem cell memory CAR-T drug candidate for the treatment of prostate cancer, which is the most common cancer among men in the United States. [Press release from Poseida Therapeutics Inc. discussing research presented at the 2018 Keystone Symposium on Emerging Cellular Therapies: T Cells and Beyond, Keystone] Press Release

Sangamo Presents Oncology Genome Editing Capabilities

Sangamo Therapeutics presented preclinical data demonstrating the company’s engineering capabilities in T cell genome editing using zinc finger nucleases. Sangamo Scientist Sumiti Jain, Ph.D. delivered the presentation, “Dual Knock-Out of Endogenous T-Cell Receptor and Human Leukocyte Antigen and Site-Specific Insertion of a CD19-CAR: Implications for Allogeneic T Cell Therapy”. [Press release from Sangamo Therapeutics, Inc. discussing research presented at the 2018 Keystone Symposium on Emerging Cellular Therapies: T Cells and Beyond, Keystone] Press Release

Capricor to Give Update on HOPE-2 Clinical Trial for CAP-1002

Capricor Therapeutics provided an update on HOPE-2, its trial for CAP-1002, the company’s lead investigational therapy for the treatment of Duchenne muscular dystrophy. [Press release from Capricor Therapeutics discussing research presented at the 16^th International Conference on Duchenne and Becker Muscular Dystrophy, Rome] Press Release

Abeona Therapeutics Reports Top-Line Data from Phase I/II Gene Therapy Trial in MPS IIIA

Abeona Therapeutics Inc. announced updated clinical data from the ongoing Phase I/II trial for ABO-102, the company’s investigational gene therapy for the treatment of Sanfilippo syndrome Type A (MPS IIIA). [Press release from Abeona Therapeutics Inc. discussing research presented at the WORLDSymposium for Lysosomal Diseases, San Diego] Press Release

Oxford BioMedica Announces Collaboration and Licence Agreement with Bioverativ in the Field of Haemophilia Gene Therapy

Oxford BioMedica plc announced that it has completed a major new collaboration & licence agreement with Bioverativ Inc. for the development and manufacturing of lentiviral vectors to treat hemophilia. [Oxford BioMedica plc] Press Release

Pitt, UPMC Announce $200 Million Immunotherapy Investment, Creation of Innovation Facility

The University of Pittsburgh (Pitt) and University of Pittsburgh Medical Center (UPMC) announced plans to establish the new UPMC Immune Transplant and Therapy Center, with much of its activity planned within an eight-story innovation hub—all part of a concerted effort to harness the power of the human immune system to treat and cure a wide range of diseases. [The University of Pittsburgh Medical Center] Press Release

Texas A&M Institute for Regenerative Medicine and Celltex Therapeutics Enter Agreement for Alzheimer’s Research

Celltex Therapeutics Corporation and Texas A&M University Health Science Center College of Medicine Institute for Regenerative Medicine announced an intellectual property license acquisition and research agreement. The announcement signals the first year of a multi-year research study investigating potential therapies for Alzheimer’s disease using autologous mesenchymal stem cell-derived exosomes. [Texas A&M University] Press Release

US FDA Approves Imfinzi for Unresectable Stage III Non-Small Cell Lung Cancer

AstraZeneca and MedImmune announced that the FDA has approved Imfinzi for the treatment of patients with unresectable stage III non-small cell lung cancer whose disease has not progressed following concurrent platinum-based chemotherapy and radiation therapy. [AstraZeneca] Press Release

Abeona Therapeutics Receives FDA Orphan Drug Designation for ABO-202 Gene Therapy Program in Infantile Batten Disease

Abeona Therapeutics Inc. announced that the FDA has granted Orphan Drug Designation to its ABO-202 program, an AAV-based gene therapy for the treatment of infantile Batten disease. [Abeona Therapeutics Inc.] Press Release

Phase I/II Clinical Trial Results Positive for SpinalCyte’s CybroCell™ Dermal Fibroblasts

SpinalCyte has completed the first double blind, placebo-controlled human trial using human dermal fibroblast injections to treat sufferers of degenerative disc disease. [SpinalCyte] Press Release

Bone Therapeutics Completes Patient Recruitment for Phase IIa Spinal Fusion Study with ALLOB^®

Bone Therapeutics announced that it has completed the recruitment of its Phase IIa lumbar spinal fusion study with its allogeneic bone-cell therapy product, ALLOB^®. [Bone Therapeutics S.A.] Press Release

Abeona Therapeutics Reports on Initial Safety and Biopotency Signals in MPS IIIB Gene Therapy Clinical Trial

Abeona Therapeutics Inc. reported preliminary 30-Day safety and biopotency signals from the first patient dosed in the company’s ongoing Phase I/II trial for ABO-101, a gene therapy treatment for patients with MPS IIIB. [Abeona Therapeutics Inc.] Press Release

Astellas Acquires Universal Cells, Inc.

Astellas Pharma Inc. and Universal Cells, Inc. announced that Astellas has acquired Universal Cells. [Astellas Pharma Inc.] Press Release

Can Civil Lawsuits Stem the Tide of Direct-to-Consumer Marketing of Unproven Stem Cell Interventions

The authors explore the value of a public health litigation strategy as a countermeasure against the exploitative practices of the unproven stem cell intervention industry by analyzing stem cell lawsuits and comparing them with other major public health litigation efforts. [npj Regen Med] Full Article

Biden Blasts Science Denialists, Calls to Dramatically Speed Up Fight against Cancer

Fresh off a plane from the NATO meeting in Munich, where he’d conferred with world leaders on global defense, former U.S. Vice President Joe Biden issued a forceful call here last night to build on the “Cancer Moonshot” he started while in the White House and transform the way the world fights cancer. [ScienceInsider] Editorial

Trump’s Science Advisor, Age 31, Has a Political Science Degree

A job that’s been held by some of the nation’s top scientists is now occupied by a 31-year-old politics major from Princeton University. And it’s unlikely to change soon, observers say, leaving President Trump without a science advisor as the administration wrestles with a severe outbreak of the flu, lead-poisoned drinking water and record-breaking disasters that many scientists say are sharpened by rising temperatures. [Scientific American] Editorial

NEW Cell and Gene Therapy Manufacturing – Next Generation Manufacturing
March 14-15, 2018
London, United Kingdom

NEW The New York Stem Cell Foundation (NYSCF) Conference 2018
October 23-24, 2018
New York, New York, United States

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

NEW Research Assistant – Cancer Therapy (Philipps-Universität Marburg)

NEW Postdoctoral Fellow – Cancer Research (Indiana University School of Medicine)

NEW Postdoctoral Associate – Cardiovascular Science (Duke University)

Postdoctoral Position – Pancreas Development and Stem Cells (Helmholtz Zentrum München)

Postdoctoral Research Scientist – Autophagy and Cell Death in Cancer (Beatson Institute for Cancer Research)

Postdoctoral Fellowships – Cancer Research (University of Miami)

Tenure-Track Group Leader Position – Pancreatic Cancer Biology (University of Copenhagen)

Research Associate – Immunology and Gene Therapy (Fred Hutchinson Cancer Research Center)

Postdoctoral Associate – Gene Therapy (University of Massachusetts)

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

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