Volume 17.14 | Apr 11

Issue 17.14 April 11, 2016
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Ixmyelocel-T for Patients with Ischemic Heart Failure: A Prospective Randomized Double-Blind Trial
Scientists aimed to assess the safety and efficacy of catheter-based transendocardial injection of ixmyelocel-T cell therapy in patients with heart failure and reduced ejection fractions. [Press release from the University of Utah discussing online publication in the Lancet]
Abstract | Press Release (University of Utah) | Press Release (Cedars-Sinai)
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PUBLICATIONS (Ranked by impact factor of the journal)
Chimeric 2C10R4 Anti-CD40 Antibody Therapy Is Critical for Long-Term Survival of GTKO.hCD46.hTBM Pig-To-Primate Cardiac Xenograft
Investigators described a reproducible, long-term survival of cardiac xenografts from alpha 1-3 galactosyltransferase gene knockout pigs, which express human complement regulatory protein CD46 and human thrombomodulin (GTKO.hCD46.hTBM), that were transplanted into baboons. [Nat Commun]
Full Article | Press Release

PDGF-AB and 5-Azacytidine Induce Conversion of Somatic Cells into Tissue-Regenerative Multipotent Stem Cells
Investigators report that transient treatment with 5-azacytidine in combination with platelet-derived growth factor–AB (PDGF-AB) converts primary somatic cells into tissue-regenerative multipotent stem (iMS) cells. Importantly, unlike mesenchymal stem cells, iMS cells contribute directly to in vivo tissue regeneration in a context-dependent manner and, unlike embryonic or pluripotent stem cells, do not form teratomas. [Proc Natl Acad Sci USA] Abstract | Press Release | Video

Cranial Grafting of Stem Cell-Derived Microvesicles Improves Cognition and Reduces Neuropathology in the Irradiated Brain
The authors provide the first evidence that cranial grafting of microvesicles secreted from human neural stem cell affords similar neuroprotective phenotypes after head-only irradiation. Cortical- and hippocampal-based deficits found one month after irradiation were completely resolved in animals cranially grafted with microvesicles. [Proc Natl Acad Sci USA] Full Article | Press Release

Paracrine Engineering of Human Explant-Derived Cardiac Stem Cells to Over-Express Stromal-Cell Derived Factor 1α Enhances Myocardial Repair
As compared to injection with unmodified cardiac stem cells, transplant of stromal-cell derived factor 1α enhanced cells into immunodeficient mice improved myocardial function and angiogenesis while reducing scarring. [Stem Cells] Abstract

Direct Bone Marrow HSC Transplantation Enhances Local Engraftment at the Expense of Systemic Engraftment in NSG Mice
Anchoring donor cells to 3-dimensional multicellular spheroids, formed from mesenchymal stem/stromal cells might improve direct bone marrow (BM) transplantation. Relevant combinations of human umbilical cord blood-derived CD34+ cells and BM-derived mesenchymal stem/stromal cells were transplanted into NOD/SCID gamma mice using either intravenous or intrafemoral routes. [Sci Rep] Full Article

Carboxyl-Terminal Fusion of E7 into Flagellin Shifts TLR5 Activation to NLRC4/NAIP5 Activation and Induces TLR5-Independent Anti-Tumor Immunity
Researchers demonstrated that rFliCE7m induced higher levels of E7-specific IFN-gamma-secreting cells and cytotoxic T lymphocytes than rE7mFliC, and a single injection with rFliCE7m but not rE7mFliC inhibited E7-expressing tumor growth in vivo. [Sci Rep] Full Article

Gene Editing for the Efficient Correction of a Recurrent COL7A1 Mutation in Recessive Dystrophic Epidermolysis Bullosa Keratinocytes
Scientists combined adeno-associated virus-mediated delivery of donor template DNA with transcription activator-like nucleases expressed by adenoviral vectors to address the correction of the c.6527insC mutation in the COL7A1 gene, causing recessive dystrophic epidermolysis bullosa in a high percentage of Spanish patients. [Mol Ther Nucleic Acids] Full Article

Differences in Phenotype, Homing Properties and Suppressive Activities of Regulatory T Cells Induced by Epicutaneous, Oral or Sublingual Immunotherapy in Mice Sensitized to Peanut
Researchers aimed to compare the phenotype and function of Tregs induced in peanut-sensitized BALB/c mice using three routes of treatment. They show that although epicutaneous, oral and sublingual immunotherapy were all able to effectively desensitize peanut-sensitized mice, they induced different subsets of Tregs. [Cell Mol Immunol] Full Article

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Quality Cell Therapy Manufacturing by Design
The biological complexity of cells has hampered the translation of laboratory-scale experiments into industrial processes for reliable, cost-effective manufacturing of cell-based therapies. The authors argue that a solution to this challenge is to design cell manufacturing processes according to quality-by-design principles. [Nat Biotechnol] Abstract

Virus-Specific T-Cell Banks for ‘Off the Shelf’ Adoptive Therapy of Refractory Infections
The authors examine the reported experience with transplant donor and third party donor-derived virus-specific T cells, identifying characteristics of the viral pathogen, the T cells administered and the diseased host that contribute to treatment response or failure. [Bone Marrow Transplant] Abstract

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Gamida Cell Presents Additional Positive Data of NiCord® in Blood Cancer Patients at EBMT 2016
Gamida Cell announced additional positive results with the use of NiCord®, an experimental treatment for patients with high-risk blood cancers. [Press release from Gamida Cell discussing research presented at the 42nd Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT), Valencia] Press Release

Scientists Find Safer Way to Make Common Blood Thinner Heparin
Scientists have discovered a way to make the common blood-thinning medication heparin using human cells in the laboratory. The new method could offer a safer alternative to current heparin production methods. [Press release from Federation of American Societies for Experimental Biology (Newswise, Inc) discussing research presented at the Experimental Biology 2016, San Diego] Press Release

Stem Cell Therapy Improves Outcomes in Severe Heart Failure
A new stem cell therapy significantly improved long-term health outcomes in patients with severe and end-stage heart failure in a study presented at the American College of Cardiology’s 65th Annual Scientific Session. [Press release from the American College of Cardiology discussing research presented at the American College of Cardiology’s 65th Annual Scientific Session, Chicago] Press Release

ASGCT 2016 Annual Meeting
Funding Shift for Zika Helps NIH, but More Research Money Requested
The White House’s decision this week to shift $589 million in unspent Ebola response funding to fighting Zika won’t require cutting any Ebola research supported by the National Institutes of Health (NIH). But the Obama administration still is requesting more help from Congress to both fund Zika efforts and replenish money shifted away from Ebola [ScienceInsider] Editorial

Second Chinese Team Reports Gene Editing in Human Embryos
Researchers in China have reported editing the genes of human embryos to try to make them resistant to HIV infection. Their paper — which used CRISPR-editing tools in non-viable embryos that were destroyed after three days — is only the second published claim of gene editing in human embryos. [Nature News] Editorial

How One Lab Challenged a Grant Rejection and Won €5 Million
Faced with a rejected grant application, scientists experience a range of emotions — shock, sadness, anger — before usually accepting the verdict and moving on. But when the European Commission rejected a €5-million (US$5.7-million) grant application from computational scientist Peter Coveney, he hired a lawyer and challenged the decision. [Nature News] Editorial

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Juno Therapeutics and WuXi AppTec Announce New Company to Develop Novel Cell-Based Cancer Immunotherapies in China
Juno Therapeutics and WuXi AppTec announced that they have started a new company in China. JW Biotechnology Co., Ltd’s mission is to build China’s leading cell therapy company by leveraging Juno’s world-class chimeric antigen receptor and T cell receptor technologies together with WuXi AppTec’s R&D and manufacturing platform and local expertise to develop novel cell-based immunotherapies for patients with hematologic and solidorgan cancers. [WuXi AppTec] Press Release

Celgene and Juno Announce Celgene Exercised Option to Develop and Commercialize CD19-Directed Product Candidates
Celgene Corporation and Juno Therapeutics, Inc. announced that Celgene exercised its option to develop and commercialize the Juno CD19 program outside North America and China. [Juno Therapeutics, Inc.] Press Release

ERC Advanced Grant for Cancer Researcher from Vetmeduni Vienna
Veronika Sexl of the Institute for Pharmacology and Toxicology at Vetmeduni Vienna has been awarded a coveted ERC Advanced Grant from the European Research Council. The grant will provide funding of about € 2.5 million over the next five years for research into new possibilities of cancer therapy using the enzyme CDK6. [Vetmeduni Vienna] Press Release

Helmsley Charitable Trust Awards $2.1 Million to Penn, Stanford and Seattle Children’s Research Institute to Explore Cell-Based Therapies for Type 1 Diabetes
The Type 1 Diabetes (T1D) Program of The Leona M. and Harry B. Helmsley Charitable Trust has announced $2.1 million in grants to support research toward the creation of new T1D-specific cell therapies that could help delay or halt the progression of the disease. [The Leona M. and Harry B. Helmsley Charitable Trust] Press Release

SELLAS Life Sciences Group Receives a Favorable Opinion for European Orphan Drug Designations for WT1 Cancer Vaccine (Galinpepimut-S)
SELLAS Life Sciences Group announced that the Committee for Orphan Medicinal Products of the European Medicines Agency has rendered a favorable opinion as regards Orphan Drug Designation for the company’s WT1 cancer vaccine (galinpepimut-S) for the treatment of patients with acute myeloid leukemia and patients with malignant pleural mesothelioma. [SELLAS Life Sciences Group] Press Release

BioLineRx Reports Successful Top-Line Results in Phase II Trial for AML
BioLineRx announced positive top-line results from BL-8040’s Phase II clinical trial in relapsed or refractory acute myeloid leukemia (AML). The BL-8040 oncology platform is a short cyclic peptide that functions as a high-affinity antagonist for CXCR4, a chemokine receptor that is directly involved in tumor progression, angiogenesis, metastasis and cell survival. [BioLineRx] Press Release

Type 1 Diabetes Cell Therapy Trial Enrolls First Patient at Sanford
A clinical trial studying the body’s ability to type fight 1 diabetes using cell therapy has used the method on its first participant. “The Sanford Project: T-Rex Study” is a collaborative study between Sanford Health and Caladrius Biosciences, Inc., for adolescents with newly onset type 1 diabetes. [Sanford Health] Press Release

Cynata Reports Compelling Results from Pre-Clinical Stem Cell Study in Graft-versus-Host Disease
Australian stem cell and regenerative medicine company, Cynata Therapeutics Limited, has received extremely positive interim data from a proof of concept study of its lead Cymerus mesenchymal stem cell product, CYP-001. [Cynata Therapeutics Limited] Press Release

California Cryobank Stem Cell Services Acquires Southern Cord
California Cryobank Stem Cell Services LLC. announced the recent acquisition of Southern Cord Inc., a cord blood bank primarily servicing the Southeastern region of the US. [California Cryobank Stem Cell Services LLC. (PR Newswire Association LLC.)] Press Release
Cooperative Research and Development Agreement (CRADA) Opportunity for Development of an Assay To Detect Genetic Markers Related to Elevated Serum Tryptase in Familial Tryptasemia and Mast Cell Activation Disorders (FR Doc. No: 2016-08100)

Prospective Grant of Start-up Exclusive License: Therapeutics and PMA-Approved Diagnostics for Alzheimer’s Disease (intranasal delivery), Parkinson’s Disease, Neuropathy,Neuropathic Pain, Peripheral Neuropathy, Diabetic Neuropathy, Neurapraxia, Axonotmesis and Neurotmesis (FR Doc. No: 2016-08097)
Food and Drug Administration (United States)

Advisory Committee; Bone, Reproductive and Urologic Drugs Advisory Committee, Renewal (FR Doc. No: 2016-07908)

Agency Information Collection Activities; Proposed Collection; Comment Request; Guidance for Industry on How To Submit Information in Electronic Format to the Center for Veterinary Medicine Using the Food and Drug Administration Electronic Submission Gateway (FR Doc. No: 2016-08075)
NEW Bioprocessing of Advanced Cellular Therapies Congress
June 2-3, 2016
London, United Kingdom

NEW Hematopoietic Stem Cells: From the Embryo to the Aging Organism
June 3-5, 2016
Heidelberg, Germany

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Scientist – Bioengineering (STEMCELL Technologies Inc.)

NEW Project Lead – Engineering (Cytonome)

Scientist – Human Pluripotent Stem Cell Neural Differentiation (STEMCELL Technologies Inc.)

Faculty Position – Tumor Immunology (John Wayne Cancer Institute at Providence Saint John’s Health Center)

PhD Studentship – Shuttling of Proteins in Stem Cells (St. Anne’s University Hospital)

Postdoctoral Fellow – Non-Coding RNAs in Malignant Progression (The University of Texas MD Anderson Cancer Center)

Postdoctoral Associate – Stem Cell Biology (University of Illinois Chicago College of Medicine)

Postdoctoral Associate – Genetic Medicine (Cornell University)

Postdoctoral Fellow – Retinal Disease Genomics and Gene Therapy (Ocular Genomics Institute, Mass Eye and Ear and Harvard Medical School)

Postdoctoral Fellow – T Cell Reconstitution (Fred Hutchinson Cancer Research Center)

Postdoctoral Fellow – Self Organization of the Pluripotent Lineage (Max-Planck-Institute for Molecular Biomedicine)

Associate Scientist – Hematopoietic Stem Cell Biology (Editas Medicine)

Research Associate – Hematopoietic Stem Cell Biology (Editas Medicine)

Postdoctoral Fellowship – Adeno-Associated Virus Gene Therapy (Icahn School of Medicine at Mount Sinai)

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

Postdoctoral Fellowship – Stem Cells, Oligodendrogenesis and Remyelination (UC Davis)

PhD Positions – Cancer, Microbiology and Stem Cell Engineering (Virginia Commonwealth University)

PhD Positions – Tendon Repair and Regeneration (NUI Galway)

Postdoctoral Position – Hematology/Oncology (Penn State College of Medicine)

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

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

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