Cell Therapy News Volume 18.34 | Oct 16 2017

TOP STORY

Gene Therapy Halts Progression of Cerebral Adrenoleukodystrophy in Clinical Trial

In a clinical trial, a gene therapy to treat cerebral adrenoleukodystrophy — a neurodegenerative disease that typically claims young boys’ lives within ten years of diagnosis — effectively stabilized the disease’s progression in 88 percent of patients. [Press release from the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center discussing online prepublication in The New England Journal of Medicine] Press Release | Press Release 2 | Press Release 3 | Full Article | Editorial

(Ranked by impact factor of the journal)

Endothelial Activation and Blood–Brain Barrier Disruption in Neurotoxicity after Adoptive Immunotherapy with CD19 CAR-T Cells

In 133 adults treated with CD19 CAR-T cells, the authors found that acute lymphoblastic leukemia, high CD19⁺ cells in bone marrow, high CAR-T cell dose, cytokine release syndrome, and preexisting neurologic comorbidities were associated with increased risk of neurologic adverse events. [Cancer Discov] Abstract | Press Release

CAR T Therapy Targeting ICAM-1 Eliminates Advanced Human Thyroid Tumors

In mice with systemic anaplastic thyroid cancer, a single administration of ICAM-1 CAR-T cells mediated profound tumor killing that resulted in long term remission and significantly improved survival. [Clin Cancer Res] Abstract

Mobilan: A Recombinant Adenovirus Carrying Toll-Like Receptor 5 Self-Activating Cassette for Cancer Immunotherapy

Injection of Mobilan into primary tumors of the prostate cancer-prone transgenic adenocarcinoma of the mouse prostate mice resulted in a strong induction of multiple genes involved in inflammatory responses and mobilization of innate immune cells into the tumors, including neutrophils and NK cells and suppressed tumor progression. [Oncogene] Full Article

Optimization of IL13Rα2-Targeted Chimeric Antigen Receptor T Cells for Improved Persistence and Antitumor Efficacy against Glioblastoma

Investigating the impact of corticosteroids, given their frequent use in the clinical management of glioblastoma, researchers demonstrated that low-dose dexamethasone does not diminish CAR T cell antitumor activity in vivo. [Mol Ther] Abstract

A Rabbit Model for Testing Helper-Dependent Adenovirus-Mediated Gene Therapy for Vein Graft Atherosclerosis

Investigators developed a model of vein graft atherosclerosis in fat-fed rabbits and demonstrated long-term persistence of helper-dependent adenovirus genomes after graft transduction. [Mol Ther Methods Clin Dev] Full Article

Mixed Cell Therapy of Bone Marrow-Derived Mesenchymal Stem Cells and Articular Cartilage Chondrocytes Ameliorates Osteoarthritis Development

Scientists report of a series of experiments in which bone marrow-derived stem cells and articular cartilage chondrocytes were mixed in a 1:1 ratio and tested for their ability to enhance cartilage regeneration in three different conditions. [Lab Invest] Abstract

A Novel Mouse Model of iNKT Cell-Deficiency Generated by CRISPR/Cas9 Reveals a Pathogenic Role of iNKT Cells in Metabolic Disease

Investigators generated a new strain of invariant natural killer T (iNKT) cell-deficient mice by deleting the Traj18 locus using CRISPR/Cas9 technology, and these animals contained an unbiased T cell receptor repertoire. They employed these mice to investigate the contribution of iNKT cells to metabolic disease and found a pathogenic role of these cells in obesity-associated insulin-resistance. [Sci Rep] Full Article

Autologous Mesenchymal Stem Cells or Meniscal Cells: What Is the Best Cell Source for Regenerative Meniscus Treatment in an Early Osteoarthritis Situation?

Scientists evaluated the appropriate cell source for autologous meniscal repair in a demanding setting of early osteoarthritis. A rabbit model was used to test autologous meniscal repair. [Stem Cell Res Ther] Full Article

Effects of Intra-Arterial Transplantation of Adipose-Derived Stem Cells on the Expression of Netrin-1 and Its Receptor DCC in the Peri-Infarct Cortex after Experimental Stroke

As netrin-1 and its receptor deleted in colorectal cancer (DCC) are important regulators in neuronal and vascular activities, researchers explored whether netrin-1 and DCC are involved in the neuroprotection of stem cell-based therapies in a rat ischemic stroke model. [Stem Cell Res Ther] Full Article

Challenges and Strategies for Improving the Regenerative Effects of Mesenchymal Stromal Cell-Based Therapies

The authors review the major factors that hamper successful cell engraftment and the strategies that have been studied to enhance the beneficial effects of cell therapy. [Int J Mol Sci] Full Article

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

GenSight Biologics to Present Data on GS010 and GS030

GenSight Biologics, a biopharma company that discovers and develops innovative gene therapies for neurodegenerative retinal diseases and diseases of the central nervous system, announced multiple data presentations. [Press release from GenSight Biologics S.A. discussing research to be presented at the 2017 Congress of the European Society of Gene and Cell Therapy (ESGCT), Berlin] Press Release

Cell and Gene Therapy Catapult Enters into Partnership with Forum for Innovative Regenerative Medicine

The Cell and Gene Therapy Catapult (CGT Catapult) announced it has signed a Memorandum of Understanding with the Forum for Innovative Regenerative Medicine (FIRM), Japan. This agreement demonstrates the shared ambition between the CGT Catapult and FIRM and aims to explore the possibility of a cooperative relationship to raise the international reach of the respective organizations. [The Cell and Gene Therapy Catapult] Press Release

Abeona Announces $13.85 Million Grant from Leading Sanfilippo Syndrome Foundations for Clinical Development of MPS III Gene Therapies

Abeona Therapeutics Inc. announced a collaborative agreement between nine Sanfilippo foundations to provide approximately $13.85 million of grants to Abeona in installments for the advancement of the company’s clinical stage gene therapies for Sanfilippo Syndrome Type A (MPS IIIA) and Sanfilippo Syndrome Type B (MPS IIIB). [Abeona Therapeutics Inc.] Press Release

SU2C Awards $5.5 Million to VARI for Cancer Clinical Trials to Enhance Immunotherapy

Van Andel Research Institute-Stand Up To Cancer (SU2C) Epigenetics Dream Team scientists have been awarded two grants totaling nearly $5.5 million to pursue clinical trials of epigenetic drugs to enhance tumor response to immunotherapy. [Stand Up To Cancer (EurekAlert!)] Press Release

BrainStorm Enrolls First Patients in Phase III Trial of NurOwn^® in ALS

BrainStorm Cell Therapeutics Inc. announced that the first patients have been enrolled in the Phase III clinical trial of NurOwn^® for the treatment of amyotrophic lateral sclerosis (ALS) at the Massachusetts General Hospital and University of California Irvine Medical Center. [BrainStorm Cell Therapeutics Inc.] Press Release

FDA Advisory Committee Unanimously Recommends Approval of Investigational LUXTURNA™ (Voretigene Neparvovec) for Patients with Biallelic RPE65-Mediated Inherited Retinal Disease

Spark Therapeutics announced that the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee has unanimously recommended approval of LUXTURNA™, an investigational, potential one-time gene therapy, for the treatment of patients with vision loss due to confirmed biallelic RPE65-mediated inherited retinal disease. [Spark Therapeutics, Inc.] Press Release

Agilis Biotherapeutics Updates on Progress in CNS Gene Therapy Programs

Agilis Biotherapeutics, Inc. announced that dosing of the last of six planned patients has been completed in the ongoing Phase IIb clinical study for the company’s gene therapy for the treatment of Aromatic L-amino acid decarboxylase (AADC) deficiency, the third prospective study conducted on the AADC gene therapy, termed AGIL-AADC. [Agilis Biotherapeutics, Inc.] Press Release

Be The Match BioTherapies Launches New Technology Platform for Cell Therapy Supply Chain Management

Be The Match BioTherapies^® announced the launch of a new, implementation-ready technology platform for cell and gene therapies in clinical development or poised for commercialization. [Be The Match BioTherapies^®] Press Release

Creative Medical Technology Holdings Expands Stem Cell Therapeutic Platform by Entry into Field of Stroke and Neurodegeneration

Creative Medical Technology Holdings announced filing of a patent application using a combination of an FDA approved drug together with the patient’s own bone marrow derived stem cells for the treatment of stroke. [Creative Medical Technology Holdings (PR Newswire Association LLC.)] Press Release

Celyad Reports a First Complete Response in a Relapsed Refractory AML Patient in the THINK Trial

Celyad announced early clinical results of the first dose-level in the hematological arm of its THINK trial (THerapeutic Immunotherapy with CAR-T NKG2D). [Celyad] Press Release

FDA Advisers Back Gene Therapy for Rare Form of Blindness

Advisers to the FDA have paved the way for the agency’s first approval of a gene therapy to treat a disease caused by a genetic mutation. A panel of external experts unanimously voted that the benefits of the therapy, which treats a form of hereditary blindness, outweigh its risks. [Nature News] Editorial

German Researchers Resign from Elsevier Journals in Push for Nationwide Open Access

Five leading German scientists have resigned from their editorial positions at journals published by Elsevier, the latest step in a battle over open-access and subscription policies between the Dutch publishing giant and a consortium of German libraries, universities, and research institutes. [ScienceInsider] Editorial

European Drug Regulation at Risk of Stalling as Agency Prepares to Leave London

Drug regulation in Europe could temporarily freeze if the European Medicines Agency loses staff during its post-Brexit move from London. Up to 70 per cent of its 900 staff have said they would quit if the agency relocated to some of the cities bidding to host the organization. [Nature News] Editorial

NIH

Prospective Grant of Exclusive Patent License: DNA-Based Vaccine for Prevention of Zika Virus Infection (FR Doc. No:2017-21986) Notice

NEW 51^st Miami Winter Symposium
January 28-31, 2018
Miami, Florida, United States

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

NEW Postdoctoral Fellowship – CART-Cell Research (National Institutes of Health)

NEW Postdoctoral Fellow – Cardiac Regeneration (Kyoto University)

NEW Postdoctorate Research Associate – Cell Therapy (Nova Southeastern University)

NEW Postdoctoral Position – Human PSC-Based Cell Therapy in Type 1 Diabetes (University of Copenhagen)

Research Technologist – Immunotherapy (STEMCELL Technologies Inc.)

Postdoctoral Position – Development of Gene Therapy (Vitalité Health Network, Université de Moncton)

Non-Clinical Lecturer – Respiratory Gene Therapy (Imperial College London)

Technologist – Cell Therapy (Yale New Haven Health)

Researcher – Skeletal Tissue Engineering (KU Leuven)

Postdoctoral Fellow – Cell Biology, Tumor Immunology and Targeted Therapy (University of Minnesota)

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)

Recruit Top Talent: Reach potential candidates by posting your organization’s career opportunities on the Connexon Creative Job Board at no cost.

Have we missed an important article or publication in Cell Therapy News? Click here to submit!

Comments or suggestions? Submit your feedback here.