Volume 16.39 | Oct 26

Issue 16.39 October 26, 2015
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Research Explains Limits of Cancer Immunotherapy Drugs
Researchers revealed molecular changes within tumors that are preventing the immunotherapy drugs from killing off the cancer. Clinical trials with PD-L1 and PD-1 blockade suggested that tumors with a high number of inflammation-causing T cells were more responsive to the immunotherapy-based PD-L1 and PD-1 inhibitors. [Press release from the University of Michigan discussing online publication in Nature] Press Release | Abstract
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PUBLICATIONS (Ranked by impact factor of the journal)
Regeneration of Thyroid Function by Transplantation of Differentiated Pluripotent Stem Cells
Investigators used mouse pluripotent stem cells (PSCs) to identify key conserved roles for BMP and FGF signaling in regulating thyroid lineage specification from foregut endoderm in mouse and Xenopus. Thyroid progenitors derived from mouse PSCs can be matured into thyroid follicular organoids that provide functional secretion of thyroid hormones in vivo and rescue hypothyroid mice after transplantation [Cell Stem Cell] Full Article | Graphical Abstract | Press Release

Chromosome Transplantation as a Novel Approach for Correcting Complex Genomic Disorders
Researchers report the first successful case of chromosome transplantation by replacement of an endogenous X chromosome carrying a mutation in the Hprt gene with a normal one in mouse embryonic stem cells, correcting the genetic defect. Chromosome transplanted clones maintained in vitro and in vivo features of stemness and contributed to chimera formation. [Oncotarget] Full Article

Targeted Inhibition of Leucine-Rich Repeat and Immunoglobulin Domain-Containing Protein 1 in Transplanted Neural Stem Cells Promotes Neuronal Differentiation and Functional Recovery in Rats Subjected to Spinal Cord Injury
Leucine-rich repeat and immunoglobulin domain-containing protein (LINGO)-1 is expressed in neural stem cells, and its neutralization results in sustained neuronal immaturity. Scientists aimed to determine if targeted inhibition of LINGO-1 via RNA interference enhances transplanted neural stem cell survival and neuronal differentiation in vivo. [Crit Care Med] Abstract

T Cell Exhaustion in Multiple Myeloma Relapse after Autotransplant: Optimal Timing of Immunotherapy
Investigators evaluated lymphocyte composition and function after autologous stem cell transplantation to guide optimal timing of immunotherapy and to identify potential markers of relapse. [Cancer Immunol Res] Abstract

Hypothalamic Leptin Gene Therapy Reduces Body Weight without Accelerating Age-Related Bone Loss
Scientists investigated the efficacy of using hypothalamic leptin gene therapy as an alternative method for reducing weight in skeletally-mature female rats and determined the impact of leptin-induced weight loss on bone mass, density, and microarchitecture, and serum biomarkers of bone turnover. [J Endocrinol]
Abstract | Press Release

RNA Interference Targeting PSCA Suppresses Primary Tumor Growth and Metastasis Formation of Human Prostate Cancer Xenografts in SCID Mice
The authors previously reported that silencing prostate stem cell antigen (PSCA) by small interfering RNA (siRNA) targeting human PSCA inhibited biological activity of PSCA-positive prostate cancer cells leading to reduced proliferation, motility and invasion in vitro. They extended these in vitro findings to in vivo settings in order to investigate the effects of this specific siRNA on the tumor growth and metastasis development of prostate cancer in vivo. [Prostate] Abstract

Xeno-Immunosuppressive Properties of Human Decidual Stromal Cells in Mouse Models of Alloreactivity In Vitro and In Vivo
Human decidual stromal cells (hDSCs) may cure acute graft-versus-host disease in humans. Investigators evaluated immunosuppression by xenogenic hDSCs in mice, both in vitro and in vivo. [Cytotherapy] Abstract

Human Adipose-Derived Mesenchymal Stem Cells in Cell Therapy: Safety and Feasibility in Different “Hospital Exemption” Clinical Applications
Researchers demonstrated that autologous adipose-derived stem cells (ASC) transplantation can be considered a safe and feasible therapy tool for extreme clinical indications of ASC properties and physiopathology of disease. [PLoS One] Full Article

Natural Killer Cells Improve Hematopoietic Stem Cell Engraftment by Increasing Stem Cell Clonogenicity In Vitro and in a Humanized Mouse Model
The effects of accessory cells, particularly natural killer cells, on human cord blood hematopoietic stem cells functions were assessed in vitro and in vivo. [PLoS One] Full Article

Three-Dimensional Printing of Complex Biological Structures by Freeform Reversible Embedding of Suspended Hydrogels
Scientists demonstrated the additive manufacturing of complex three-dimensional biological structures using soft protein and polysaccharide hydrogels that are challenging or impossible to create using traditional fabrication approaches. [Sci Adv] Full Article | Press Release | Video

Learn more about STEMvision automated mouse hematopoietic colony-forming unit (CFU) assay imaging and counting.
CRISPR/Cas9: Molecular Tool for Gene Therapy to Target Genome and Epigenome in the Treatment of Lung Cancer
The authors frame utilization of CRISPR/Cas9 for molecular targeted gene therapy leading to long-term repression and activation or inhibition of molecular targets linked to lung cancer, avoiding the cycles of therapy. [Cancer Gene Ther] Abstract

Visit our reviews page to see a complete list of reviews in the cell, gene and immunotherapy research fields.

NEW: Animal Component-Free MSC Chondrogenic Differentiation. Learn More.
bluebird bio Announces Presentation
bluebird bio, Inc. announced that Marina Cavazzana, M.D., Ph.D. presented a review of the HGB-205 clinical study experience with lentiviral-based gene therapies for beta-thalassemia. [Press release from bluebird bio, Inc. discussing research presented at 10th Annual Cooley’s Anemia Foundation Symposium, Chicago] Press Release

Alnylam Demonstrates Continued Commitment to Transthyretin-Mediated Amyloidosis Patients with Advancement of ALN-TTRsc02, an Investigational RNAi Therapeutic with Potential for Low Volume, Once Quarterly, Subcutaneous Dose Regimen
Alnylam Pharmaceuticals, Inc. announced that it presented key pre-clinical data and provided program guidance for ALN-TTRsc02, an investigational RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis. [Press release from Alnylam Pharmaceuticals, Inc. discussing research presented at 11th Annual Meeting of the Oligonucleotide Therapeutics Society (OTS), Leiden] Press Release

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Canada’s New Government May Be a New Beginning for Science
Canadian scientists have been making do for nearly a decade, in the face of funding cuts, federal lab closures, and a blanket gag order on government researchers. For them, the fall of Prime Minister Stephen Harper’s Conservative government in the national election wasn’t just a relief—it was the rough emotional equivalent of blowing up the Death Star. [Wired] Editorial

Neuroscientist Team Calls for a National Brain Observatory
Roughly three years ago, six well-known neuroscientists drafted an ambitious proposal for a large U.S. neuroscience project to map activity in the living brain. Now, the same team has crafted another bold proposal aimed at accelerating Brain Research through Advancing Innovative Neurotechnologies (BRAIN)’s success: the creation of a National Brain Observatory, a network of neurotechnology centers tied to the Department of Energy’s National Labs. [ScienceInsider] Editorial
Lilly and AstraZeneca Expand Immuno-Oncology Research Collaboration with New Combinations
Eli Lilly and Company and AstraZeneca announced an extension to their existing immuno-oncology collaboration exploring novel combination therapies for the treatment of patients with solid tumors. Under the terms of the expanded agreement, Lilly and AstraZeneca will evaluate the safety and efficacy of a range of additional combinations across the companies’ complementary portfolios. [Eli Lilly and Company] Press Release

Novartis Broadens Immuno-Oncology Pipeline with Acquisition of Admune Therapeutics and Licensing Agreements with XOMA and Palobiofarma
Novartis announced that it is broadening its portfolio of cancer immunotherapies with the acquisition of Admune Therapeutics and licensing agreements with Palobiofarma and XOMA Corporation. [Novartis] Press Release

Joint Establishment of Strategic Platform for Industrialization of Biomedical Research
China Regenerative Medicine International Limited announced that the company entered into a MOU with CCBI and the University of Oxford in respect of the joint establishment of strategic platform for industrialization of biomedical research. [China Regenerative Medicine International Limited] Press Release

Athersys Updates on Development Strategy in Japan
Athersys, Inc. announced that Athersys and Chugai Pharmaceutical Co., Ltd. have ended the license agreement between them for the exclusive development and commercialization of MultiStem® cell therapy for ischemic stroke in Japan. [Athersys, Inc.] Press Release

University of Louisville Researchers Awarded Patents for Innovations to Improve Cancer Treatments, Bone Grafts and Therapies for Spinal Cord Damage
Technologies to improve bone grafts and cancer immunotherapies, protect lung tissue during radiation treatment, reverse neurological damage and improve recovery from spinal cord injury are among the patents issued to the University of Louisville Research Foundation recently. [University of Louisville School of Medicine] Press Release

RXi Pharmaceuticals Announces Compound Selection for Cosmetic Product Development with Its Novel Self-Delivering RNAi Platform
RXi Pharmaceuticals Corporation announced that it has selected two compounds for cosmetic product development based on its proprietary self-delivering RNAi platform. [RXi Pharmaceuticals Corporation] Press Release

Amgen Receives CHMP Positive Opinion for IMLYGIC™ (Talimogene Laherparepvec)
Amgen announced that the Committee for Medicinal Products for Human Use (CHMP), the scientific committee of the European Medicines Agency, has adopted a positive opinion recommending that IMLYGIC™ be granted approval for the treatment of adults with unresectable melanoma that is regionally or distantly metastatic with no bone, brain, lung or other visceral disease. [Amgen] Press Release

Bogus Cell Therapy Market Worth Up to $2.4 Billion
The global market for unproven cell therapies may be worth as much as $2.4 billion, according to a report published by the International Society for Cellular Therapy. [Thomson Reuters] Press Release
Center for Scientific Review; Notice of Closed Meetings (FR Doc. No: 2015-26502)
2015 Biological Device Application Approvals
Food and Drug Administration (United States)

Selection of the Appropriate Package Type Terms and Recommendations for Labeling Injectable Medical Products Packaged in Multiple-Dose, Single-Dose, and Single-Patient-Use Containers for Human Use; Draft Guidance for Industry; Availability (FR Doc. No: 2015-26849)

Agency Information Collection Activities; Proposed Collection; Comment Request; Medical Device User Fee Cover Sheet, Form FDA 3601 (FR Doc. No: 2015-26640)
NEW Keystone Symposium – Molecular and Cellular Basis of Growth and Regeneration
January 10-14, 2016
Breckenridge, United States

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Group Leader – Translational Research (Cancer Research Center of Toulouse)

NEW Group Leader Position – Cancer Research (Oslo University Hospital)

NEW PhD Studentships – Cancer Research (Cancer Research UK Manchester Institute)

Postdoctoral Research Fellow/Research Scientist – Biotechnology (Qu Biologics Inc.)

Project Manager – Manufacturing (Qu Biologics Inc.)

Faculty Positions – Stem Cell Biology (University of Notre Dame)

Research Technician – TCR Gene Therapy (Fred Hutchinson Cancer Research Center)

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

Assistant/Associate/Full Professor – Biomedical Engineering (Worcester Polytechnic Institute)

Assistant Professor – Stem Cell Biology (University of South Carolina)

Postdoctoral Researcher – Epigenetic Modifiers (Van Andel Research Institute)

Postdoctoral Fellow – Immunotherapy/T Cell Biology (California Institute for Biomedical Research)

Postdoctoral Position – Spinal Cord Injury Research (Indiana University-Purdue University)

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