Cell Therapy News Volume 17.11 | Mar 21 2016

Generation and Transplantation of Reprogrammed Human Neurons in the Brain Using 3D Microtopographic Scaffolds
Researchers present the design of three-dimensional (3D) microtopographic scaffolds, using tunable electrospun microfibrous polymeric substrates that promote in situ stem cell neuronal reprogramming, neural network establishment and support neuronal engraftment into the brain. [Nat Commun] Full Article | Press Release

Antigen-Encoding Bone Marrow Terminates Islet-Directed Memory CD8⁺ T-Cell Responses to Alleviate Islet Transplant Rejection
Scientists showed that transfer of bone marrow encoding cognate antigen directed to dendritic cells, under mild, immune-preserving conditions inactivates established memory CD8⁺ T-cell populations and generates a long-lived, antigen-specific tolerogenic environment. [Diabetes] Abstract

Comparative Study of Human Embryonic Stem Cells (hESC) and Human Induced Pluripotent Stem Cells (hiPSC) as a Treatment for Retinal Dystrophies
Scientists evaluated and compared the therapeutic effects of two pluripotent stem cell-based therapies. They differentiated retinal pigment epithelium from hESC or hiPSC and transplanted them into the subretinal space of the Royal College of Surgeons rat. [Mol Ther] Full Article

Expanding Transplant Options to Patients over 50 Years- Improved Outcome after Reduced Intensity Conditioning Mismatched-Unrelated Donor Transplantation for Patients with Acute Myeloid Leukemia: A Report from the Acute Leukemia Working Party of the EBMT
Eight hundred eighty three patients receiving reduced-intensity conditioning were compared with 1041 myeloablative conditioning regimen recipients in the setting of mismatched-unrelated donor transplantation. [Haematologica] Abstract | Full Article

Systemic Mesenchymal Stromal Cell Transplantation Prevents Functional Bone Loss in a Mouse Model of Age-Related Osteoporosis
Using minimally expanded exogenous mesenchymal stromal cells injected systemically into a mouse model of human age-related osteoporosis, scientists show long-term engraftment and markedly increased bone formation. [Stem Cells Transl Med] Abstract | Full Article | Press Release

Minicircle DNA Provides Enhanced and Prolonged Transgene Expression following Airway Gene Transfer
The authors compared minicircle DNA with plasmid DNA in transfections of airway epithelial cells. Administration of equimolar amounts of DNA to mouse lungs resulted in a reduced inflammatory response and more persistent transgene expression, with luciferase activity persisting for two weeks from minicircle DNA compared to plasmid formulations. [Sci Rep] Full Article

Guiding Migration of Transplanted Glial Progenitor Cells in the Injured Spinal Cord
Researchers explored the possibility of producing a migratory stream of glial-restricted progenitors via directional cues to create a supportive pathway for axon regeneration. [Sci Rep] Full Article

β1-Na⁺,K⁺-ATPase Gene Therapy Upregulates Tight Junctions to Rescue Lipopolysaccharide-Induced Acute Lung Injury
Investigators showed that delivery of a plasmid expressing β1-subunit of the Na⁺,K⁺-ATPase (β1-Na⁺,K⁺-ATPase) alone or in combination with epithelial sodium channel α1-subunit using electroporation not only protected from subsequent lipopolysaccharide-mediated lung injury, but also treated injured lungs. [Gene Ther] Abstract

Adenoviral Vector Encoding Soluble Flt-1 Engineered Human Endometrial Mesenchymal Stem Cells Effectively Regress Endometriotic Lesions in NOD/SCID Mice
The authors studied the efficiency of Adsflt-1 engineered human eutopic mesenchymal stem cells secreting anti-angiogenic sFlt-1 as a targeted cell-based therapy for endometriosis. [Gene Ther] Abstract

Silencing of S100A4, a Metastasis-Associated Protein, Inhibits Retinal Neovascularization via the Downregulation of BDNF in Oxygen-Induced Ischemic Retinopathy
Researchers investigated the underlying mechanism of S100A4 function and whether it has a role in retinal neovascularization in a mouse model of oxygen-induced retinopathy. [Eye (Lond)] Abstract

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Kite Pharma Announces Clinical and Manufacturing Updates on KTE-C19 and MAGE-A3 Product Candidates
Kite Pharma, Inc. announced two oral presentations and two poster presentations to be delivered. The oral presentations will address KTE-C19, Kite’s lead chimeric antigen receptor product candidate, and, separately, an engineered T cell receptor product candidate targeting the cancer testis antigen MAGE-A3. [Press release from Kite Pharma, Inc. discussing research to be presented at the American Association for Cancer Research (AACR) Annual Meeting 2016, New Orleans] Press Release

Celsion Announces Presentation of Preclinical Data for GEN-1 IL-12 Immunotherapy in Combination with Avastin^® and Doxil^®
Celsion Corporation announced that its preclinical data for GEN-1 in combination with Avastin^® and Doxil^® for the treatment of ovarian cancer will be presented. [Press release from Celsion Corporation discussing research to be presented at the American Association for Cancer Research (AACR) Annual Meeting 2016, New Orleans] Press Release

MacroGenics Announces Five Posters at AACR Annual Meeting 2016
MacroGenics, Inc. announced that it will present five posters. Four of the five posters refer to programs that are based on MacroGenics’ Dual-Affinity Re-Targeting bispecific technology. [Press release from MacroGenics, Inc. discussing research to be presented at the American Association for Cancer Research (AACR) Annual Meeting 2016, New Orleans] Press Release

What China’s Latest Five-Year Plan Means for Science
In the wake of the five-year plan, China will gain a new funding initiative called ‘Stem Cell and Translational Research.’ The grants will be awarded under a new competitive review and evaluation process, replacing a system that critics said rewarded scientific and political connections rather than merit. [Nature News] Editorial

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Profectus and Vyriad Form Global Collaboration to Develop Oncolytic Recombinant VSV (rVSV) Vaccines for Cancer Immunotherapy
Profectus BioSciences, Inc. and Vyriad will combine and leverage their respective recombinant vesicular stomatitis virus (rVSV)-derived oncolytic and vaccine platform technologies to develop novel cancer drugs that selectively destroy tumor cells while simultaneously boosting patients’ immune systems to continue killing any residual cancer cells that may survive the initial virus attack. [Vyriad] Press Release

Blueprint Medicines Announces Worldwide Collaboration to Accelerate and Expand Its Development of Novel Medicines in the Field of Cancer Immunotherapy
Blueprint Medicines Corporation announced that it has entered into a worldwide collaboration and exclusive license agreement with F. Hoffmann-La Roche Ltd and Hoffmann-La Roche Inc. for the discovery, development and commercialization of up to five small molecule therapeutics targeting kinases believed to be important in cancer immunotherapy. [Blueprint Medicines Corporation] Press Release

Caladrius Biosciences Subsidiary PCT, LLC Enters into Global Collaboration and License Agreement with Hitachi Chemical
PCT has licensed its cell therapy technology and know-how to Hitachi Chemical for cell therapy manufacturing in certain Asian territories, including Japan, where the Pharmaceuticals and Medical Devices Agency has introduced more favorable legislation to stimulate the growth of regenerative medicine development in Japan. [Caladrius Biosciences, Inc.] Press Release

SRI International Receives $19.8 Million National Cancer Institute Contract for PREVENT Cancer Program
SRI International has been awarded a contract of up to $19.8 million from the National Cancer Institute PREVENT Cancer Preclinical Drug Development Program to support the development of potential cancer preventive agents or vaccines. [SRI International] Press Release

CIRM Grant to Fund Proposed Stem Cell Trials for ALS
The Independent Citizens Oversight Committee of the California Institute for Regenerative Medicine approved yesterday a $6.3 million grant to a research team from the University of California, San Diego School of Medicine and University of California, Davis to pursue a novel human embryonic stem cell-based therapy to rescue and restore neurons devastated by amyotrophic lateral sclerosis or ALS. [University of California, San Diego] Press Release

Children’s Hospital Los Angeles Awarded $7.1 Million by CIRM
Tracy C. Grikscheit, MD, of The Saban Research Institute of Children’s Hospital Los Angeles has received a $7.1 million grant from the California Institute of Regenerative Medicine Translational Research program to develop a cellular therapy for the treatment of nerve disorders of the digestive system. [Children’s Hospital Los Angeles] Press Release

FDA Grants Roche’s Cancer Immunotherapy Atezolizumab Priority Review for Advanced Bladder Cancer
Roche Group announced that the U.S. Food and Drug Administration has accepted the company’s Biologics License Application and granted Priority Review for atezolizumab for the treatment of people with locally advanced or metastatic urothelial carcinoma. [Roche Group] Press Release

Agency Information Collection Activities; Proposed Collection; Comment Request; Investigational New Drug Safety Reporting Requirements for Human Drug and Biological Products and Safety Reporting Requirements for Bioavailability and Bioequivalence Studies in Humans (FR Doc. No: 2016-06128)

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

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

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

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)

PhD Position – Leukemogenesis and Treatment Resistance (Institute for Tumor Biology and Experimental Therapy)

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

Translational Research Scientist – Hematology/Oncology (University of Chicago)

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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