Cell Therapy News Volume 17.28 | Jul 25 2016

TOP STORY

Age-Dependent Niche Signals from the Choroid Plexus Regulate Adult Neural Stem Cells

Researchers showed that the lateral ventricle choroid plexus, in addition to performing important homeostatic support functions, secretes factors that promote colony formation and proliferation of purified quiescent and activated ventricular-subventricular zone stem cells and transit-amplifying cells. [Cell Stem Cell] Abstract | Press Release | Graphical Abstract

(Ranked by impact factor of the journal)

Tumor Regression and Delayed Onset Toxicity following B7-H4 CAR T Cell Therapy

Researchers engineered T cells with novel B7-H4-specific chimeric antigen receptors (CAR) that recognized both human and murine B7-H4 to test the hypothesis that B7-H4 CAR T cell therapy can be applied safely in preclinical models. [Mol Ther] Abstract

In Vivo Therapeutic Potential of Mesenchymal Stem Cell-Derived Extracellular Vesicles with Optical Imaging Reporter in Tumor Mice Model

Non-invasive methods of monitoring extracellular vesicles (EVs) in animal models are crucial for developing EV-based cancer therapies. Scientists developed bioluminescent EVs using Renilla luciferase-expressing mesenchymal stem cells. [Sci Rep] Full Article

The Myocardial Regenerative Potential of Three-Dimensional Engineered Cardiac Tissues Composed of Multiple Human iPS Cell-Derived Cardiovascular Cell Lineages

Researchers generated engineered cardiac tissues from three cellular compositions of cardiomyocytes, endothelial cells, and vascular mural cells differentiated from human induced pluripotent stem cells. They then determined the impact of cell composition on engineered cardiac tissues structural and functional properties. [Sci Rep] Full Article

MiR-34a Promotes Osteogenic Differentiation of Human Adipose-Derived Stem Cells via the RBP2/NOTCH1/CYCLIN D1 Coregulatory Network

MiR-34a was demonstrated to be upregulated during the osteogenic differentiation of human adipose-derived stem cells (hASCs). Overexpression of miR-34a significantly increased alkaline phosphatase activity, mineralization capacity, and the expression of osteogenesis-associated genes in hASCs in vitro. Enhanced heterotopic bone formation in vivo was also observed upon overexpression of miR-34a in hASCs. [Stem Cell Reports] Full Article | Graphical Abstract

Liver Extracellular Matrix Promotes BM-MSCs Hepatic Differentiation and Reversal of Liver Fibrosis through Activation of Integrin Pathway

Scientists aimed to investigate the hepatogenicity and underlying mechanism of liver-specific ECM as well as its potential regulative role in the mesenchymal stem cell (MSC)-based liver recovery. [J Tissue Eng Regen Med] Abstract

Effective Adoptive Immunotherapy of Triple-Negative Breast Cancer by Folate Receptor-Alpha Redirected CAR T Cells Is Influenced by Surface Antigen Expression Level

The aim of the present study was to apply a chimeric antigen receptor (CAR) approach for the targeting of FRα expressed on triple-negative breast cancer cells and evaluate the antitumor activity of CAR-engineered T cells in vitro and in vivo. [J Hematol Oncol] Full Article

Intratracheal Administration of Mesenchymal Stem Cells Modulates Tachykinin System, Suppresses Airway Remodeling and Reduces Airway Hyperresponsiveness in an Animal Model

GFP-tagged mouse mesenchymal stem cells (MSCs) were intratracheally delivered in the ovalbumin mouse model with subsequent functional tests, the analysis of cytokine levels, neuropeptide expression and histological evaluation of MSCs fate and airway pathology. Additionally, MSCs were exposed to pro-inflammatory factors in vitro. [PLoS One] Full Article

An Acellular Biologic Scaffold Treatment for Volumetric Muscle Loss: Results of a 13-Patient Cohort Study

The results of a 13-patient cohort study were described and involved a regenerative medicine approach for volumetric muscle loss treatment. Acellular bioscaffolds composed of mammalian extracellular matrix were implanted and combined with aggressive and early physical therapy following treatment. [npj Regenerative Medicine] Full Article

Halfway There: The Past, Present and Future of Haploidentical Transplantation

The reviewers summarize the current and future need for haploidentical donors and detail the rise of post-transplant cyclophosphamide as the dominant haploidentical approach. Further, they examined ongoing controversies in the field of haploidentical transplant, including conditioning regimens and graft source. [Bone Marrow Transplant] Abstract

Generating Kidney Tissue from Pluripotent Stem Cells

The authors review recent progress towards the recreation of not only specific kidney cell types but complex kidney organoids, models of the developing human organ, in vitro. They will also discuss potential short and long term applications of these approaches. [Cell Death Discov] Full Article

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

bluebird bio Presents megaTAL Genome Editing Data at American Society of Hematology (ASH) Workshop on Genome Editing

bluebird bio, Inc. announced the presentation of pre-clinical data from its megaTAL genome editing platform. In an invited presentation entitled “Gene Editing with megaTALs: Advantages, Challenges and Future Prospects,” Jordan Jarjour, Ph.D., director of nuclease research and development, bluebird bio, detailed the anatomy and physiology of megaTALs and discussed how to expand their DNA targeting capacity and refine their specificity. [Press release from bluebird bio, Inc. discussing research presented at the American Society of Hematology (ASH) Workshop on Genome Editing, Washington, DC] Press Release

Hard to Treat Chest Pain May Be Improved with a Patient’s Own Stem Cells

A non-surgical treatment that uses a patient’s own bone marrow stem cells to treat chest pain or angina improved both symptoms and the length of time treated patients could be physically active, according to preliminary research presented. [Press release from the American Heart Association discussing research presented at Basic Cardiovascular Sciences 2016 Scientific Sessions, Phoenix] Press Release

AbbVie and Bristol-Myers Squibb Announce Oncology Clinical Collaboration to Evaluate the Combination of Rova-T plus Opdivo and Opdivo + Yervoy Regimen

AbbVie and Bristol-Myers Squibb Company announced a clinical trial collaboration to evaluate the safety, tolerability and efficacy of AbbVie’s investigational biomarker-specific antibody drug conjugate Rova-T in combination with Bristol-Myers Squibb’s Opdivoand Opdivo + Yervoy regimen as a treatment for relapsed extensive-stage small cell lung cancer. [AbbVie] Press Release

Deal Including Multi-Year Services Agreement for Long Term Discovery Service Collaboration Made Final

Icagen, Inc. announced that the acquisition of Sanofi’s ultra high-throughput biology, screening and chemistry capabilities and research facility in Oro Valley, Arizona is officially complete. The acquisition includes an agreement between Icagen and Sanofi to collaborate in a multi-year services contract for long term discovery services. [Icagen, Inc.] Press Release

ViaCyte Awarded New CIRM Grant to Develop PEC-Direct for High-Risk Type 1 Diabetes

ViaCyte, Inc. announced that the California Institute for Regenerative Medicine (CIRM) approved a grant of $3.9 million to support pre-clinical development of ViaCyte’s PEC-Direct cell therapy product candidate which is being developed for a subset of type 1 diabetes patients. [ViaCyte, Inc.] Press Release

TSRI Scientists Receive Funding to Advance Stem Cell-Based Parkinson’s Therapy

Scientists at The Scripps Research Institute (TSRI) and Scripps Clinic have received a grant of nearly $2.4 million from the California Institute for Regenerative Medicine to support safety and quality tests of a potential stem cell therapy for Parkinson’s disease. [The Scripps Research Institute] Press Release

£1.2 Million for Injectable Stem-Cell Carrying Microspheres to Regenerate Bones

The University of Nottingham has secured £1.2 million to develop injectable stem cell-carrying materials to treat and prevent fractures caused by osteoporosis and other bone-thinning diseases. [University of Nottingham] Press Release

BioLife Solutions Products Embedded in Cook MyoSite^® Phase III Cell Therapy Clinical Trial for Stress Urinary Incontinence

BioLife Solutions, Inc. announced that its media products are embedded into the autologous cell therapy being developed by Pittsburgh-based Cook MyoSite, a subsidiary of the Cook Group, for treatment of female stress urinary incontinence. [BioLife Solutions, Inc.] Press Release

BrainStorm Announces Positive Top Line Results from the U.S. Phase II Study of NurOwn^® in Patients with Amyotrophic Lateral Sclerosis (ALS)

BrainStorm Cell Therapeutics Inc. announced results from the recently completed U.S. randomized, double-blind, placebo-controlled Phase II Study of NurOwn^® in Patients with ALS. The study achieved its primary objective, demonstrating that NurOwn was safe and well tolerated. [BrainStorm Cell Therapeutics Inc.] Press Release

Targazyme Enrolls First Patient in Phase I/II Study of TZ101 Fucosylated Regulatory T Cells to Treat and Prevent Graft vs Host Disease in the Transplant Setting

Targazyme, Inc. announced that it has enrolled the first patient in a Phase 1/2 TZ 101-treated regulatory T cell clinical study. This Phase I/II clinical study will evaluate the efficacy and safety of TZ101-treated regulatory T cells in the prevention and treatment of acute and chronic Graft-Versus-Host-Disease in stem cell transplants. [Targazyme, Inc.] Press Release

Alnylam Announces New Positive Interim Phase I Study Results for Fitusiran

Alnylam Pharmaceuticals, Inc. announced new positive results from its ongoing Phase I study with fitusiran, an investigational RNAi therapeutic targeting antithrombin for the treatment of hemophilia A and B and rare bleeding disorders. [Alnylam Pharmaceuticals, Inc.] Press Release

Bellicum Pharmaceuticals Announces Patent on CaspaCIDe Method for Selectively Eliminating Therapeutic Cells

Bellicum Pharmaceuticals, Inc. announced that U.S. patent 9,393,292 has issued to Baylor College of Medicine for a method of cell therapy that enables the selective elimination of administered cells that have been modified to express an inducible caspase-9 protein. [Bellicum Pharmaceuticals, Inc.] Press Release

Europe Overhauls Rules for ‘First-In-Human’ Trials in Wake of French Disaster

The European Union is beefing up protections for volunteers in Phase I clinical trials in the wake of a disastrous clinical study in Rennes, France, that resulted in the death of one volunteer and the hospitalization of five others. The European Medicines Agency (EMA) in London announced in a “concept paper” that it wants to improve strategies to identify and reduce risks in “first-in-human” studies on healthy volunteers. EMA is asking for input from stakeholders. [ScienceInsider] Editorial | Article

Chinese Scientists to Pioneer First Human CRISPR Trial

Chinese scientists are on the verge of being first in the world to inject people with cells modified using the CRISPR–Cas9 gene-editing technique. A team led by Lu You, an oncologist at Sichuan University’s West China Hospital in Chengdu, plans to start testing such cells in people with lung cancer next month. [Nature News] Editorial

Scientific Literature: Information Overload

Recent bibliometrics show that the number of published scientific papers has climbed by 8–9% each year over the past several decades. In the biomedical field alone, more than 1 million papers pour into the PubMed database each year — about two papers per minute. [Nature Careers] Editorial

Science Advice for Europe

Exactly 1 month after Britain’s momentous decision to leave the European Union, around 4500 scientists and friends of science will assemble in Manchester, UK, for the opening of the EuroScience Open Forum, Europe’s largest interdisciplinary research conference. [Science] Editorial

FDA

Agency Information Collection Activities; Submission for Office of Management and Budget Review; Comment Request; Evaluation of the Program for Enhanced Review Transparency and Communication for New Molecular Entity New Drug Applications and Original Biologics License Applications in Prescription Drug User Fee Acts (FR Doc. No:2016-17185) Notice

Determination of Regulatory Review Period for Purposes of Patent Extension; QUTENZA (FR Doc. No:2016-17187) Notice

Pre-Clinical Evaluation of Red Blood Cells for Transfusion; Public Workshop (FR Doc. No:2016-17008) Notice

NEW Symposium on Regenerative Rehabilitation
October 14-16, 2016
Atlanta, United States

NEW 9^th Stem Cell Clonality Meeting
October 17-18, 2016
Florence, Italy

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

NEW Research Fellow – Hematology and Epigenetics (University of Edinburgh)

NEW Research Fellow – Hematology (Mayo Clinic)

Scientist – Bioengineering (STEMCELL Technologies Inc.)

Center Manager – Bioengineering and Nanotechnology (The University of Queensland)

Research Assistants – Pluripotent Stem Cell Biology (The University of Queensland)

Postdoctoral Research Fellows – Stem Cell, Niche and Systemic Ageing (The University of Queensland)

Postdoctoral Research Scientist – Cancer Immunotherapy (Children’s Hospital of Eastern Ontario Research Institute)

Resource Technologist B – Cellular Manufacturing Activities for Cell and Gene Therapy (University of Pennsylvania)

Postdoctoral Fellow – Pluripotent Stem Cell Technology (University of California Davis)

Staff Scientist – Cell Therapy Process Development (Fred Hutchinson Cancer Research Center)

Associate Member – Stem Cell and Gene Therapy (Fred Hutchinson Cancer Research Center)

Faculty Position – Cancer Immunology (University of New Mexico)

Postdoctoral Fellow – Pathogenic Mechanisms of Rare Bone Disorders (University of Connecticut Health)

Postdoctoral Fellow – Oxidative Lung Injury (Temple University)

Postdoctoral Fellow – Lentivirus-Mediated FOXP3 Gene Transfer (Stanford University School of Medicine)

Postdoctoral Fellowship – Deciphering the Link between Adhesion and Pluripotency (The Danish Stem Cell Center at Faculty of Health & Medical Sciences at the University of Copenhagen)

Scientist – Target Validation (Innate Pharma)

Postdoctoral Fellow – Data Analysis and Stem Cell Research (Nestle Institute of Health Sciences)

Postdoctoral Fellow – Hematology/Oncology (Boston Children’s Hospital)

Postdoctoral Research Fellow – Cell Cycle Control and Tumorigenesis (Fred Hutchinson Cancer Research Center)

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