Cell Therapy News Volume 17.36 | Oct 3 2016

TOP STORY

ESC-Derived BDNF-Overexpressing Neural Progenitors Differentially Promote Recovery in Huntington’s Disease Models by Enhanced Striatal Differentiation

By transplanting embryonic stem cell (ESC)-derived neural progenitors overexpressing brain-derived neurotrophic factor (BDNF), the authors combined cell replacement and BDNF supply as a potential Huntington’s disease therapy approach. [Stem Cell Reports] Full Article | Graphical Abstract

(Ranked by impact factor of the journal)

Restoration of Corneal Transparency by Mesenchymal Stem Cells

Using an in vivo mouse model of ocular injury, investigators report that mesenchymal stem cells have the capacity to restore corneal transparency by secreting high levels of hepatocyte growth factor. [Stem Cell Reports] Full Article | Graphical Abstract

Baboon Envelope Pseudotyped Lentiviral Vectors Transduce Efficiently Human B Cells and Allow Active Factor IX B Cell Secretion In Vivo in NOD/SCID Mice

Researchers tested for the first time a newly developed baboon envelope pseudotyped lentiviral vectors for human B-cell transduction followed their adaptive transfer into NOD/SCID/γ^-/- mouse. [J Thromb Haemost] Abstract

Mesenchymal Stem Cells Stimulate Intestinal Stem Cells to Repair Radiation-Induced Intestinal Injury

Scientists found that, following exposure to radiation, transplantation of mesenchymal stem cells improved the survival of the mice, ameliorated intestinal injury and increased the number of regenerating crypts. [Cell Death Dis] Full Article

Neural Stem Cell Tumorigenicity and Biodistribution Assessment for Phase I Clinical Trial in Parkinson’s Disease

Researchers describe preclinical tumorigenicity and biodistribution safety studies that were required by the US Food and Drug Administration and Australian Therapeutic Goods Administration prior to conducting a Phase I clinical trial evaluating the safety and tolerability of human parthenogenetic stem cell derived neural stem cells ISC-hpNSC for treating Parkinson’s disease. [Sci Rep] Full Article

Exosomes Derived from MSCs Ameliorate Retinal Laser Injury Partially by Inhibition of MCP-1

The authors intravitreally injected mesenchymal stem cells (MSCs) derived from either mouse adipose tissue or human umbilical cord, and their exosomes to observe and compare their functions in a mouse model of laser-induced retinal injury. They found that both MSCs and their exosomes reduced damage, inhibited apoptosis, and suppressed inflammatory responses to obtain better visual function to nearly the same extent in vivo. [Sci Rep] Full Article

Fiber-Modified Adenovirus-Mediated Suicide Gene Therapy Can Efficiently Eliminate Bladder Cancer Cells In Vitro and In Vivo

The investigators constructed an Arg-Gly-Asp (RGD)-modified adenovirus, RGDAd-UPII-TK, that carries a suicide gene called HSV-TK that is driven by a human UPII promoter. They tested the bladder cancer specificity of the UPII promotor and the expression of the HSV-TK protein. [Oncotarget] Full Article

Bio-Active Coating of Decellularized Vascular Grafts with a Temperature-Sensitive VEGF-Conjugated Hydrogel Accelerates Autologous Endothelialization In Vivo

Researchers used a functional in vivo rodent aortic transplantation model in order to evaluate the benefit of bio-active coating of decellularized vascular grafts with vascular endothelial growth factor (VEGF) conjugated to a temperature-sensitive aliphatic polyester hydrogel. [J Tissue Eng Regen Med] Abstract

Outcome of AL Amyloidosis after High-Dose Melphalan and Autologous Stem Cell Transplantation in Sweden, Long-Term Results from All Patients Treated in 1994–2009

High-dose melphalan and autologous stem cell transplantation (HDM/ASCT) is widely used in immunoglobulin light chain (AL) amyloidosis, but the benefit is debated mainly because of the high treatment-related mortality. The investigators report on the long-term outcome of all patients treated with HDM/ASCT for AL amyloidosis in Sweden between 1994 and 2009. [Bone Marrow Transplant] Abstract

Overexpression of Factor VIII after AAV Delivery is Transiently Associated with Cellular Stress in Hemophilia a Mice

Researchers sought to determine whether increased factor VIII expression in mice after adeno-associated viral liver gene transfer would affect the unfolded protein response and/or immune response to the transgene. [Mol Ther Methods Clin Dev] Full Article

Materials and Technologies for Soft Implantable Neuroprostheses

The authors emphasize the importance of minimizing the physical and mechanical mismatch between neural tissues and implantable interfaces. They explore possible materials solutions to design and manufacture neurointegrated prostheses, and outline their immense therapeutic potential. [Nature Reviews Materials] Abstract

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

The Nobel Assembly at Karolinska Institutet Has Decided to Award the 2016 Nobel Prize in Physiology or Medicine to Yoshinori Ohsumi

This year’s Nobel Laureate discovered and elucidated mechanisms underlying autophagy, a fundamental process for degrading and recycling cellular components. [Nobel Prize] Press Release | Editorial

A Research Consortium Under the Direction of Grünenthal Will Receive a Grant of €1.5 Million

The LifeSciences North Rhein-Westphalia called for consortia of small and medium-sized companies and academic groups within North Rhein-Westphalia to compete in making proposals for developing solutions to solve current challenges within the field of biomedical sciences. [Grünenthal Group] Press Release

AiVita Biomedical Awarded NIH R01 Grant for Development of 3D-Transplantable ‘Retinas’

AiVita Biomedical announced that the National Eye Institute of the National Institute of Health (NIH) has awarded the company a Research Project Grant (R01) for the development of stem cell-derived 3D-transplantable retinas to treat vision loss. AiVita CEO Dr. Hans S. Keirstead will serve as principal investigator on the project, to be conducted in collaboration with the University of California, Irvine. [AiVita Biomedical] Press Release

BioBridge Global-Led Biotech Team Awarded $7.8 Million Army Contract

A four-organization biotech group led by San Antonio-based nonprofit BioBridge Global has been awarded a $7.8 million contract from the Medical Technology Enterprise Consortium. The funds will enable the development of large-scale manufacturing capabilities for clinical-grade stem cells for research and therapeutic use. [BioBridge Global (PR Newswire Association LLC)] Press Release

Athersys Receives FDA Agreement Under Special Protocol Assessment for Phase III Study of MultiStem^® Treatment for Ischemic Stroke

Athersys, Inc. announced that it has received agreement from the U.S. Food and Drug Administration (FDA) under a Special Protocol Assessment for the design and planned analysis of a Phase III clinical trial of Athersys’ novel MultiStem^® cell therapy product for the treatment of ischemic stroke. [Athersys, Inc.] Press Release

New Quick-Tissue^™ Kits Revolutionize Skeletal Muscle and Neuron Differentiation

Elixirgen, LLC launched Quick-Muscle^™ 1.0, a groundbreaking biological reagent that develops skeletal muscle cells from human stem cells in just a few days — a cutting-edge advancement for a laboratory process that previously took a month or longer. [Elixirgen, LLC] Press Release

MatriTek Launches Commercial Operations at the Alexandria Center for Life Science in New York City

MatriTek, Inc. announced the launch of commercial production of novel extracellular matrix (ECM) biomaterials for biomedical research. MatriTek’s proprietary TissueSpec^™ platform technology enables scientists to culture cells in a physiologically relevant extracellular microenvironment and is coupled with the company’s core expertise in cell and tissue culture biomaterials and in vitro models for biomedical research. [MatriTek, Inc.] Press Release

Asterias Biotherapeutics Announces Dosing of First Patient in New SCiSTAR Clinical Trial Cohort Testing AST-OPC1 in an Expanded Cervical Spinal Cord Injury Patient Population

Asterias Biotherapeutics, Inc. announced that the first patient with incomplete AIS-B cervical spinal cord injury was successfully dosed with 10 million cells of AST-OPC1 (oligodendrocyte progenitor cells) in the SCiSTAR clinical trial at Shepherd Center in Atlanta. The company recently received FDA clearance to expand the study into this additional patient population based on the continued favorable safety profile observed in the ongoing clinical study. [Asterias Biotherapeutics, Inc.] Press Release

Fate Therapeutics Receives FDA Orphan Drug Designation for ProTmune™ in Allogeneic Hematopoietic Cell Transplantation

Fate Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for ProTmune^™, the company’s lead product candidate that is currently undergoing Phase I/II clinical investigation. The FDA designation is for “prevention of graft-versus-host disease in patients undergoing allogeneic hematopoietic cell transplantation” and broadly covers diseases, including blood cancers and genetic disorders, for which the procedure is performed. [Fate Therapeutics, Inc.] Press Release

Democratic Databases: Science on GitHub

Scientists are turning to a software–development site to share data and code. [Nature Toolbox] Editorial

UK Bioethicists Eye Designer Babies and CRISPR Cows

From designer babies to engineered mosquitoes, advances in genome-editing technologies such as CRISPR–Cas9 have raised the possibility of tremendous scientific advances — and serious ethical concerns. [Nature News] Editorial

Biologists Plan Scoring System for Antibodies

Biomedical experts plan to create a scoring system that will help researchers choose reliable antibodies for their experiments. The only problems: figuring out how such a ranking would work — and getting manufacturers to adopt the standard. [Nature News] Editorial

FDA

Agency Information Collection Activities; Proposed Collection; Comment Request; Donor Risk Assessment Questionnaire for the Food and Drug Administration/National Heart, Lung, and Blood Institute-Sponsored Transfusion-Transmissible Infections Monitoring System-Risk Factor Elicitation (FR Doc. No:2016-23622) Notice

A List of Biomarkers Used as Outcomes in Development of FDA-Approved New Molecular Entities and New Biological Therapeutics (October 2007 to December 2015); Establishment of a Public Docket; Correction (FR Doc. No:2016-23106) Notice

Fee for Using a Rare Pediatric Disease Priority Review Voucher in Fiscal Year 2017 (FR Doc. No:2016-23624) Notice

NEW Advances in Cell Engineering, Imaging and Screening
November 17-18, 2016
Leuven, Belgium

NEW European Society for Medical Oncology (ESMO) Asia 2016 Congress
December 16-19, 2016
Singapore, Singapore

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

NEW Postdoctoral Fellowship – Genetic Medicine (Weill Cornell Medical College)

NEW Research Fellow in Pluripotent Stem Cells Self-Renewal Program and Cell Cycle (KU Leuven)

Faculty Position – Stem Cell Biology and Regenerative Medicine (Stanford University)

Research Associate – Hematology (Editas Medicine)

Faculty Position – Stem Cell Research (Children’s Research Institute at UTSW)

Assistant Professorship – Stem Cells and Regenerative Medicine (University of Southern California)

Postdoctoral Positions – Stem Cell Research, Hematopoiesis, Cancer, Genome Editing (Icahn School of Medicine)

Faculty Position – Cancer Research (Children’s Research Institute at UTSW)

Assistant Professor – Molecular and Cellular Biology (Harvard University)

Postdoctoral Fellow – Novel Systems for Treatment of Type 1 Diabetes (Toronto General Research Institute & McEwen Centre for Regenerative Medicine)

Assistant Professor – Developmental Biology (Florida State University)

Research Associate – Cellular Therapy (California Institute for Biomedical Research)

Research Scientist – Downstream Bioprocess for Vaccine, Gene Therapy and Biosimilars (Univercells)

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

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

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

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

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