Cell Therapy News Volume 21.15 | Apr 27 2020

TOP STORY

Locoregional Delivery of CAR T Cells to the Cerebrospinal Fluid for Treatment of Metastatic Medulloblastoma and Ependymoma

Investigators demonstrated that administration of CAR T cells into the cerebrospinal fluid, alone or in combination with azacytidine, was a highly effective therapy for multiple metastatic mouse models of group 3 medulloblastoma and posterior fossa group A ependymoma. [Nat Med] Abstract

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Locoregionally Administered B7-H3-Targeted CAR T Cells for Treatment of Atypical Teratoid/Rhabdoid Tumors

B7-H3.BB.z-CAR T cells administered intracerebroventricularly or intratumorally mediated potent antitumor effects against cerebral atypical teratoid/rhabdoid tumor xenografts in mice, with faster kinetics, greater potency and reduced systemic levels of inflammatory cytokines compared to CAR T cells administered intravenously. [Nat Med] Abstract

Gene-Edited Human Stem Cell-Derived β Cells from a Patient with Monogenic Diabetes Reverse Preexisting Diabetes in Mice

Scientists used CRISPR-Cas9 to correct a diabetes-causing pathogenic variant in Wolfram syndrome (WS) 1 in induced pluripotent stem cells (iPSCs) derived from a patient with WS. Corrected WS iPSC-derived β cells performed robust dynamic insulin secretion in vitro in response to glucose and reversed preexisting streptozocin-induced diabetes after transplantation into mice. [Sci Transl Med] Abstract | Press Release

A Vaccine-Based Nanosystem for Initiating Innate Immunity and Improving Tumor Immunotherapy

The authors generated a vaccine-based nanosystem by integrating siRNA for Cd274 into the commercial human papillomavirus L1 protein. This nanosystem had good biosafety and enhanced the therapeutic response rate of anti-tumor immunotherapy. [Nat Commun] Full Article

scAAV2-Mediated c3 Transferase Gene Therapy in a Rat Model with Retinal Ischemia/Reperfusion Injuries

scAAV2-C3 significantly reduced the number of apoptotic retinal ganglion cells and decreased the cells loss in ganglion cell layer after ischemia/reperfusion (I/R) injury, and the I/R injured retinas treated with scAAV2-C3 were the thickest in all I/R groups. [Mol Ther Methods Clin Dev] Abstract | Full Article | Graphical Abstract

Delta-1 Functionalized Hydrogel Promotes hESC-Cardiomyocyte Graft Proliferation and Maintains Heart Function Post-Injury

Investigators designed a hydrogel that activated notch signaling through 3D presentation of the notch ligand delta-1 to use as an injectate for transplanting human stem cell-derived cardiomyocytes (hESC-CMs) into the infarcted rat myocardium. After four weeks, hESC-CM proliferation increased two-fold and resulted in a three-fold increase in graft size with the delta-1 hydrogel compared to controls. [Mol Ther Methods Clin Dev] Abstract | Full Article | Graphical Abstract

Cell Therapy with Mesenchymal Stem Cells Induces an Innate Immune Memory Response that Attenuates Experimental Colitis in the Long-Term

Dextran sulfate sodium (DSS)-induced colitic mice treated with adipose-derived mesenchymal stem cells (aMSCs) showed significant reduction in their colitic disease activity index during the second DSS challenge when compared to non-aMSC-treated DSS-induced colitic mice. [J Crohns Colitis] Full Article

Human Umbilical Cord-Derived Mesenchymal Stem Cells Alleviate Schizophrenia-Relevant Behaviors in Amphetamine-Sensitized Mice by Inhibiting Neuroinflammation

Human umbilical cord-derived mesenchymal stem cells (hUC-MSCs) inhibited schizophrenia-relevant and the neuroinflammatory changes. The main mechanism of hUC-MSCs was associated with the induction of T_regs and production of the anti-inflammatory cytokine, IL-10, in periphery. [Transl Psychiatry] Full Article

Myogenic Progenitor Cell Lineage Specification by CRISPR/Cas9-Based Transcriptional Activators

Transplantation of human myogenic progenitors derived from endogenous activation of PAX7 into immunodeficient mice resulted in a greater number of human dystrophin⁺ myofibers compared with exogenous PAX7 overexpression. [Stem Cell Reports] Full Article | Graphical Abstract

Platelet-Rich Plasma Enhances the Repair Capacity of Muscle-Derived Mesenchymal Stem Cells to Large Humeral Bone Defect in Rabbits

Platelet-rich plasma application significantly enhanced the regeneration capacity of muscle-derived mesenchymal stem cells (M-MSCs) in a large bone defect via promoting the migration and proliferation of M-MSCs, and also inducing the osteogenic differentiation. [Sci Rep] Full Article

Intra-Striatal AAV2.Retro Administration Leads to Extensive Retrograde Transport in the Rhesus Macaque Brain: Implications for Disease Modeling and Therapeutic Development

Scientists evaluated the capability of AAV2.retro to deliver disease-related gene cargo to biologically-relevant nonhuman primate brain circuits by packaging a fragment of human mutant HTT, the causative gene mutation in Huntington’s disease. [Sci Rep] Full Article

AAV-Mediated Cardiac Gene Transfer of Wild-Type Desmin in Mouse Models for Recessive Desminopathies

Researchers investigated the effects of AAV-mediated cardiac wild-type desmin expression in homozygous desmin knockout and homozygous R349P desmin knockin mice. [Gene Ther] Full Article

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Mesenchymal Stem Cell Therapy for COVID-19: Present or Future

Preventing the severe acute respiratory infection form of COVID-19 as the most dangerous phase of this disease can be helpful for the treatment and reduction of the death rate. In this regard, mesenchymal stem cell-based immunomodulation treatment has been proposed as a suitable therapeutic approach and several clinical trials have begun. [Stem Cell Rev Rep] Abstract

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WindMIL Therapeutics Opens Combination Therapy Portion of Its Phase IIa Clinical Trial of Marrow-Infiltrating Lymphocytes (MILs^™) for Treatment of Patients with Non-Small Cell Lung Cancer

WindMIL Therapeutics announced it has begun enrollment in the combination therapy portion of its open-label, multi-center Phase IIa clinical trial to assess the safety and efficacy of MILs in patients with locally advanced unresectable or metastatic non-small cell lung cancer (NSCLC) who are refractory to, or have relapsed on, an anti-PD-1-containing regimen. [WindMIL Therapeutics (GlobeNewswire, Inc.)] Press Release

Novartis Kymriah^® Receives FDA Regenerative Medicine Advanced Therapy Designation in Follicular Lymphoma

Novartis announced that the FDA granted Regenerative Medicine Advanced Therapy designation to Kymriah^®, for an investigational new indication to treat patients with relapsed or refractory follicular lymphoma. Kymriah, which is designed to be a one-time treatment, is the first-ever FDA-approved CAR-T cell therapy. [Novartis] Press Release

CARsgen Therapeutics Receives Positive EMA Opinion on Orphan Drug Designation for Fully Human Anti-BCMA CAR-T Cells (CT053) for the Treatment of Multiple Myeloma

CARsgen Therapeutics Co., Ltd. announced the European Medicines Agency (EMA) Committee for Orphan Medicinal Products adopted a positive opinion on CARsgen’s application for orphan designation of its investigational CT053 CAR T-cell therapy, fully human anti-B cell maturation antigen (BCMA) autologous CAR T cells for the treatment of multiple myeloma. [CARsgen Therapeutics Co., Ltd. (PR Newswire Asia Ltd.)] Press Release

Mustang Bio Receives Advanced Therapy Medicinal Product Classification from European Medicines Agency for MB-107 Lentiviral Gene Therapy for X-Linked Severe Combined Immunodeficiency

Mustang Bio, Inc. announced that the European Medicines Agency has granted Advanced Therapy Medicinal Product classification to MB-107, Mustang’s lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency, also known as bubble boy disease. [Mustang Bio, Inc.] Press Release

Citius Announces Pre-IND Submission to FDA under the Coronavirus Treatment Acceleration Program for a Novel Stem Cell Therapy for Acute Respiratory Distress Syndrome (ARDS) in COVID-19

Citius Pharmaceuticals, Inc. announced that it submitted a pre-IND meeting request and supporting briefing documents to the Center for Biologics Evaluation and Research of the FDA under the Coronavirus Treatment Acceleration Program. [Citius Pharmaceuticals, Inc.] Press Release

Open Science Takes On the Coronavirus Pandemic

When reports emerged in late 2019 of an outbreak of a new coronavirus centred in Wuhan, China, researchers at the virological-analysis website Nextstrain were ready. The open-source project tracks the spread of viruses through genetic variations in the sequences that scientists find. [Nature News] Editorial

Untapped Potential: More US Labs Could Be Providing Tests for Coronavirus

A survey of more than 4,000 researchers in the United States suggests that better coordination at an institutional and national level could make hundreds of thousands more tests for coronavirus available. [Nature News] Editorial

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NEW Lab Director – Cancer Immunotherapy (Vrije Universiteit Brussel)

Postdoctoral Fellow – Cancer Drug Discovery (Uniformed Services University of Health Sciences)

Scientist – Cancer Immunotherapy (Vrije Universiteit Brussel)

Postdoctoral Associate – Diabetes (University of Miami Health System)

Associate/Senior Editor – Nature Biotechnology (Nature Research)

Senior Research Scientist – Orthopedic Surgery/Regenerative Medicine (NYU Grossman School of Medicine)

Team Leaders – Stem Cell Biology (RIKEN Center for Biosystems Dynamics Research)

Postdoctoral Research Associate – Induced Pluripotent Stem Cell Therapies (The University of Arizona College of Medicine)

Professorship – Biological Therapies of the Inner Ear (Hannover Medical School)

Group leaders – Cancer Biology (Institute of Pharmacology and Structural Biology)

Scientist – Cancer Immunotherapy (Lund University)

Postdoctoral Position – Cancer Immunotherapy (The University of Texas MD Anderson Cancer Center)

Sr Associate Scientist – Autoimmunity and Tumor Immunotherapy (Amgen)

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