Volume 15.24 | Jun 23

Issue 15.24 June 23, 2014
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Gut Bacteria Predict Survival after Stem Cell Transplant, Study Shows
New research published online suggests that the diversity of bacteria in the gastrointestinal tract of patients receiving stem cell transplants may be an important predictor of their post-transplant survival. To better understand the association between post-transplant microbiota and patient outcomes, researchers collected fecal specimens from 80 patients undergoing allogeneic stem cell transplant and sequenced each sample’s bacterial DNA. [Press release from the American Society of Hematology discussing online prepublication in Blood]
Press Release | Abstract
mTeSR™1: Most Published Feeder-Free hPSC Maintenance Medium. Click to View Top Applications
PUBLICATIONS (Ranked by impact factor of the journal)
Remote Control of Induced Dopaminergic Neurons in Parkinsonian Rats
Scientists determined that induced dopaminergic (iDA) neurons retain a transgene-independent stable phenotype in culture and in animal models. Furthermore, transplanted iDA neurons functionally integrated into host neuronal tissue, exhibiting electrically excitable membranes, synaptic currents, dopamine release, and substantial reduction of motor symptoms in a Parkinson’s disease animal model. [J Clin Invest] Full Article | Press Release | Editorial

Murine Spleen Tissue Regeneration from Neonatal Spleen Capsule Requires Lymphotoxin Priming of Stromal Cells
Researchers reveal in a murine model that transplant of neonatal spleen capsule alone leads to the regeneration of full spleen tissue. This finding indicates that graft-derived spleen stromal cells, but not lymphocytes, are essential components of tissue neogenesis, a finding verified by transplant and regeneration of Rag1KO spleen capsules. [J Immunol] Abstract | Full Article

Humanized Mice as a Model for Aberrant Responses in Human T Cell Immunotherapy
Investigators found that reconstituted “humanized” mice treated with anti-CTLA-4 Ab (ipilimumab) develop autoimmune disease characterized by hepatitis, adrenalitis, sialitis, anti-nuclear Abs, and weight loss. [J Immunol] Abstract

iRGD Tumor-Penetrating Peptide-Modified Oncolytic Adenovirus Shows Enhanced Tumor Transduction, Intratumoral Dissemination and Antitumor Efficacy
Scientists genetically inserted iRGD peptide in the fiber C terminus of ICOVIR15K, an oncolytic tumor-retargeted adenovirus to increase its tumor penetration. In vivo, iRGD did not change the normal organ transduction pattern, with liver and spleen as main targeted organs. In tumors, however, iRGD enhanced transduction and early adenovirus dissemination through the tumor mass leading to an improved antitumor efficacy. [Gene Ther] Abstract

α-Synuclein Immunotherapy Blocks Uptake and Templated Propagation of Misfolded α-Synuclein and Neurodegeneration
Scientists demonstrate that alpha-synuclein (α-syn) monoclonal antibodies reduce α-syn preformed fibril (pff)-induced Lewy body/Lewy neurite formation and rescue synapse/neuron loss in primary neuronal cultures by preventing both pff uptake and subsequent cell-to-cell transmission of pathology. [Cell Rep]
Full Article | Graphical Abstract | Press Release

Immunotherapy Converts Nonimmunogenic Pancreatic Tumors into Immunogenic Foci of Immune Regulation
Researchers designed a neoadjuvant and adjuvant clinical trial comparing an irradiated, granulocyte-macrophage colony-stimulating factor-secreting, allogeneic pancreatic ductal adenocarcinoma vaccine given as a single agent or in combination with low-dose cyclophosphamide to deplete regulatory T cells as a means to study how the tumor microenvironment is altered by immunotherapy. [Cancer Immunol Res] Abstract | Press Release

A Phase II, Single-Arm Study of an Autologous Dendritic Cell Treatment against Mucin 1 in Patients with Advanced Epithelial Ovarian Cancer
A previous successful Phase I trial was conducted in patients with adenocarcinoma who were injected with autologous monocyte-derived dendritic cells incubated with mannanosylated mucin 1 protein. The present study was a Phase II trial of Cvac in patients with advanced epithelial ovarian cancer. [J Immunother Cancer]
Abstract | Full Article | Press Release

Targeting 4-1BB Costimulation to the Tumor Stroma with Bispecific Aptamer Conjugates Enhances the Therapeutic Index of Tumor Immunotherapy
In preclinical murine tumor models systemic administration of vascular endothelial growth factor (VEGF) VEGF-targeted 4-1BB aptamer conjugates engendered potent antitumor immunity against multiple unrelated tumors in subcutaneous, post post-surgical lung metastasis, methylcholantrene-induced fibrosarcoma, and oncogene-induced autochthonous glioma models, and exhibited a superior therapeutic index compared to non-targeted administration of an agonistic 4-1BB antibody or 4-1BB aptamer. [Cancer Immunol Res] Abstract

Take the "if" out of "diff": Reduce variability when differentiating to hematopoietic cells
Progress and Prospects for Engineered T Cell Therapies
There have been notable successes recently using viral vector-mediated transfer of chimeric antigen receptors specific for B cell antigens, but also reports of anticipated and unanticipated complications in these and other studies. The authors review progress in this promising area of cellular therapy, and consider developments in antigen receptor therapies including the application of emerging gene-editing technologies. [Br J Haematol]
Full Article

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

Free Wallchart: Small Molecules, Big Impact in PSC Research
NeoStem Announces University of California, San Francisco Presentation of Phase I Results for T Regulatory Cell Immunotherapy for Type 1 Diabetes
NeoStem, Inc. announced the presentation of the results of an open-label dose escalation Phase I clinical study of autologous T regulatory cell immunotherapy for type 1 diabetes mellitus indicating safety and tolerability following administration. [Press release from NeoStem, Inc. discussing research presented at the American Diabetes Association 74th Scientific Sessions, San Francisco] Press Release

Neuralstem NSI-189 Select Data to Be Presented
Neuralstem, Inc. announced that select data from the NSI-189 Phase Ib trial in patients with major depressive disorder (MDD) will be presented. During the course of the NSI-189/MDD Phase Ib trial, patients underwent quantitative electroencephalography brain measurements, in addition to the clinical tests. [Press release from Neuralstem, Inc. discussing research to be at the International College of Neuropyschopharmacology (CINP) 2014 World Congress, Vancouver] Press Release

BioProcess International Conference & Exhibition - Boston MA
USC Considers Acquiring Scripps Research Institute
In an unexpected announcement, the University of Southern California in Los Angeles revealed it may be acquiring or merging with the Scripps Research Institute. Scripps, a major nonprofit biomedical research hub based in San Diego, California, issued a joint statement with the university, telling that they were “discussing the possibility of a relationship that would enhance the missions of both institutions.” [ScienceInsider] Editorial

Stem Cells: Taking a Stand against Pseudoscience
Elena Cattaneo and Gilberto Corbellini are among the academics working to protect patients from questionable stem-cell therapies. They share their experiences and opinions of the long, hard fight for evidence to prevail. [Nature News] Editorial

From our sponsor: Learn how ex vivo models drive progress in HIV research. Read the research profiles.
Dimension Therapeutics and Bayer HealthCare Enter Collaboration to Develop Novel Gene Therapy for Hemophilia A
Dimension Therapeutics announced it has entered into a collaboration with Bayer HealthCare for the development and commercialization of a novel gene therapy for the treatment of hemophilia A. [Dimension Therapeutics]
Press Release

Pfizer and Cellectis Enter into Global Strategic Cancer Immunotherapy Collaboration
Pfizer Inc. and Cellectis announced that they have entered into a global strategic collaboration to develop chimeric antigen receptor T-cell immunotherapies in the field of oncology directed at select targets. [Pfizer Inc.]
Press Release

Hyperion Therapeutics Completes Acquisition of Andromeda Biotech, Ltd.
Hyperion Therapeutics, Inc. announced that it has completed its acquisition of Andromeda Biotech Ltd. The acquisition broadens Hyperion’s pipeline to include DiaPep277®, a potentially first-in-class immunotherapy for new onset Type 1 diabetes. [Hyperion Therapeutics, Inc.] Press Release

$15 Million Award to Go toward Exploring New Treatments for Autism, Other Brain Disorders
Duke Medicine has been awarded $15 million to support an innovative research program that explores the use of umbilical cord blood cells to treat autism, stroke, cerebral palsy and related brain disorders. [Duke University Health System] Press Release

Western Oncolytics Receives $275,000 Seed Funding for Development of Cancer Immunotherapy
Western Oncolytics Ltd. has closed $275,000 in seed funding. The proceeds are planned to set a path toward clinical trials and execute initial manufacturing for its novel virus-based cancer therapy, which delivers genes to guide the immune system to kill cancer cells. [PR Newswire Association LLC] Press Release

Response Genetics Launches New Test for Cancer Immunotherapy Drug Development
Response Genetics, Inc. announced the availability of new testing capabilities to advance cancer immunotherapy clinical development. The Immuno-Oncology assay, which runs on HTG Molecular’s Edge System, is designed to measure the RNA expression of 26 commonly investigated immunotherapy related genes enabling screening for response to immunoregulatory pathways. [Response Genetics, Inc.] Press Release

StemCells, Inc. Announces Closure of Enrollment in Phase I/II Dry Age-Related Macular Degeneration Trial
StemCells, Inc. announced that based on positive interim results, it has closed enrollment in its Phase I/II clinical trial for dry age-related macular degeneration in order to focus its efforts on a follow-on Phase II randomized, controlled proof-of-concept study. [StemCells, Inc.] Press Release

Pluristem Granted Patent in Russia for Treatment of Inflammatory Bowel Disease Using Placenta-Derived Cells
Pluristem Therapeutics Inc. announced that Russia’s Federal Service for Intellectual Property has granted to Pluristem Patent No. 2515156 titled, “Methods of Treating Inflammatory Colon Diseases”. The patent covers methods for treating ulcerative colitis or Crohn’s disease using placenta-derived cells. This patent has already been issued to Pluristem in South Africa and is currently pending in several other jurisdictions. [Pluristem Therapeutics Inc.] Press Release

Pluristem Receives Patent in India for Cell Therapy Production Methods and Compositions
Pluristem Therapeutics Inc. announced it has been granted Patent No. 261087 from India’s Office of the Controller General of Patents, Designs & Trade Marks for a patent titled, “Methods for Cell Expansion and Conditioned Media Produced Thereby for Therapy.” [Pluristem Therapeutics Inc.] Press Release
Center for Scientific Review; Notice of Closed Meetings (FR Doc. No: 2014-14296)
Food and Drug Administration (United States)

Draft Guidance for Industry: Determining the Need for and Content of Environmental Assessments for Gene Therapies, Vectored Vaccines, and Related Recombinant Viral or Microbial Products; Availability (FR Doc. No: 2014-14470)

Methodological Considerations in Evaluation of Cancer as an Adverse Outcome Associated With Use of Non-Oncological Drugs and Biological Products in the Postapproval Setting; Public Meeting; Request for Comments (FR Doc. No: 2014-13907)
NEW Translational Regenerative Medicine Congress 2014
October 21-22, 2014
Leipzig, Germany

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Postdoctoral Fellow – AAV Vectors for Gene Therapy of Neurological and Other Disorders (University of Massachusetts Medical School)

NEW Scientist – Pluripotent Stem Cell Media Development, High Throughput Screening (STEMCELL Technologies Inc.)

NEW Scientist – Cell Culture Support Products (STEMCELL Technologies Inc.)

NEW Scientist – Liver Cell Biology (STEMCELL Technologies Inc.)

Postdoctoral Scientist – Immune Focused In Vitro Analytics (Celentyx Ltd.)

Assistant or Associate Professor – Stem Cell Transplantation Program (Dana-Farber Cancer Institute)

PhD Studentship – Endogenous Retroviruses as Immunotherapy Targets (University of Plymouth)

Postdoctoral Fellow – Prostate Cancer Research (Henry Ford Hospital)

Postdoctoral Position – Neurotoxicity and Epigenetics (Thomas Jefferson University)

Research/Review Scientist – Cell Therapy and Regenerative Medicine (Center for Biologics Evaluation and Research)

Postdoctoral Position – Cell Death and Inflammation (Inflammation Research Center – VIB-UGent)

Postdoctoral Position – Role of Calreticulin in TGF-Beta Signaling in the Kidney Proximal Tubule under Diabetic Conditions (University of Alabama)

Postdoctoral Fellow – Transgene Immunity in AAV Vector-Based Antiviral and/or Gene Therapeutics (University of Massachusetts)

Postdoctoral Fellow – Neurophysiology of Addiction (Medical University of South Carolina)

Principal Investigator – Molecular Mechanisms of Inflammation (Inflammation Research Center – VIB-UGent)

Postdoctoral Associate – Hematopoietic Differentiation (Northwestern University Feinberg School of Medicine)

Scientist – Immunology/Cell Separation (STEMCELL Technologies Inc.)

Research Associate – Cell Separation (STEMCELL Technologies Inc.)

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