Cell Therapy News Volume 14.05 | Feb 11 2013

MRI-Detectable pH Nanosensors Incorporated into Hydrogels for In Vivo Sensing of Transplanted-Cell Viability
Although their potential benefit has been widely evaluated, at present it is not possible to determine, in vivo, if and how long cells remain viable following their administration without the use of a reporter gene. Here, researchers report a pH-nanosensor-based magnetic resonance imaging (MRI) technique that can monitor cell death in vivo non-invasively. [Nat Mater] Abstract | Press Release

Rescue of Hearing and Vestibular Function by Antisense Oligonucleotides in a Mouse Model of Human Deafness
Investigators showed that hearing and vestibular function can be rescued in a mouse model of human hereditary deafness. An antisense oligonucleotide was used to correct defective pre-mRNA splicing of transcripts from the USH1C gene with the c.216G>A mutation, which causes human Usher syndrome, the leading genetic cause of combined deafness and blindness. [Nat Med] Abstract | Press Release

Synergistic and Tunable Human Gene Activation by Combinations of Synthetic Transcription Factors
Mammalian genes are regulated by the cooperative and synergistic actions of many transcription factors. In this study researchers recapitulate this complex regulation in human cells by targeting endogenous gene promoters, including regions of closed chromatin upstream of silenced genes, with combinations of engineered transcription activator-like effectors. [Nat Methods] Abstract | Press Release

Amelioration of Arthritis through Mobilization of Peptide-Specific CD8+ Regulatory T Cells
Researchers found that peptide-induced expansion of tetramer-binding CD8+ Tregs that recognize Qa-1-Hsp60_p216 but not Qa-1-Qdm strongly inhibited collagen-induced arthritis, an animal model of human rheumatoid arthritis. Infusion of in vitro-expanded CD8+ Tregs increased the efficacy of methotrexate treatment and halted disease progression after clinical onset, suggesting an alternative approach to this first-line treatment. [J Clin Invest] Full Article | Press Release

Discovery and Evaluation of a Functional Ternary Polymer Blend for Bone Repair: Translation from a Microarray to a Clinical Model
Ternary mixtures of natural and synthetic polymers were solvent blended and evaluated as potential bone tissue engineering matrices for osteoregeneration by the assessment of growth and differentiation of STRO-1+ skeletal stem cells. Several of the blend materials were found to be excellent supports for human bone marrow-derived STRO-1+ skeletal cells and fetal skeletal cells, with the optimized blend exhibiting in vivo osteogenic potential, suggesting that these polymer blends could act as suitable matrices for bioengineering of hard tissues. [Adv Funct Mater] Abstract | Press Release

H-Y Antigen-Binding B Cells Develop in Male Recipients of Female Hematopoietic Cells and Associate with Chronic Graft vs. Host Disease
The authors previously showed that IgG allo-antibodies recognize Y chromosome-encoded proteins (H-Y) and a dominant H-Y epitope, DEAD box protein (DBY-2) detectable 6-12 months after transplant in male patients who receive grafts from female donors (F→M) in hematopoietic cell transplantation (HCT). Here they present FACS studies of peripheral blood mononuclear cells collected 6 months after transplant showing that 16 of 28 F→M HCT patients have circulating donor B cells that express B-cell receptor specific for DBY-2. The detection of these DBY-2 B cells 6 months after HCT are associated with chronic graft vs. host disease development. [Proc Natl Acad Sci USA] Abstract | Press Release

Prospective Isolation of Human Embryonic Stem Cell-Derived Cardiovascular Progenitors that Integrate into Human Fetal Heart Tissue
Researchers have prospectively identified a population of human ES cell (hESC)-derived ROR2⁺/CD13⁺/KDR⁺/PDGFRα⁺ cells that give rise to cardiomyocytes, endothelial cells, and vascular smooth muscle cells in vitro at a clonal level. They then developed an in vivo transplantation model by transplanting second-trimester human fetal heart tissues s.c. into the ear pinna of a SCID mouse. ROR2⁺/CD13⁺/KDR⁺/PDGFRα⁺ cells were delivered into these functioning fetal heart tissues: in contrast to traditional murine heart models for cell transplantation, the researchers show structural and functional integration of hESC-derived cardiovascular progenitors into human heart. [Proc Natl Acad Sci USA] Abstract

Treatment of Diabetes and Long-Term Survival following Insulin and Glucokinase Gene Therapy
A one-time intramuscular administration of adenoassociated viral vectors of serotype 1 (AAV1) encoding for glucokinase and insulin in diabetic dogs resulted in normalization of fasting glycemia, accelerated disposal of glucose after oral challenge, and no episodes of hypoglycemia during exercise for >4 years after gene transfer. [Diabetes] Abstract | Press Release

Repair or Replace? Exploiting Novel Gene and Cell Therapy Strategies for Muscular Dystrophies
In this review researchers highlight recent developments and key challenges for the strategies based upon gene replacement and gene/expression repair, including exon-skipping, vector-mediated gene therapy and cell therapy, just to mention a few. [FEBS J] Abstract | Full Article

Gene Therapy for Malignant Mesothelioma: Current Prospects and Challenges
In this review the authors summarize major preclinical and clinical gene therapy for mesothelioma, and discuss the advantages of gene therapy in the context of stimulating host immune systems. [Cancer Gene Ther] Abstract

Gamida Cell’s StemEx® Achieves Primary Endpoint in Phase II/III Clinical Study
Gamida Cell announced that its flagship product, StemEx, reached its primary endpoint of improving overall survival in a Phase II/III study which compared the use of StemEx as part of a transplantation regimen to historical controls in the treatment of patients with hematological malignancies such as leukemia and lymphoma. [Gamida Cell] Press Release

Northwestern Medicine Researchers Investigate Stem Cell Therapy for Stroke
Northwestern Medicine® researchers are investigating a novel stem cell therapy, known as SB623, that may hold the key to improving motor function following a disabling stroke. While the study’s primary purpose is to examine the safety of SB623 stem cells, researchers will also seek to determine if the cells are effective in improving stroke symptoms. [Northwestern Memorial Hospital] Press Release

Europe’s Leaders Slash Proposed Research Budget
In an acrimonious meeting of the European Council to decide the European Union’s general budget for 2014-2020, leaders of the Union’s 27 member states slashed the €77.6 billion research budget proposed by the European Commission back to €69.24 billion. [European Council, European Union] Press Release

Lofty Goals in Spanish Research Plan. But Very Little Money
The Spanish government has issued a new road map for science that sets high goals for the national research system, but researchers are disappointed that it has little new money to offer. Instead, the government hopes corporate funding will increase dramatically, which the scientific community says is not likely to happen. [Spanish Strategy of Science, Technology, and Innovation, Spain] Press Release

Progressive MS Therapy Sparks Deal Worth Up to $225 Million
Opexa Therapeutics said it granted Merck Serono an option for exclusive license to develop and commercialize Tcelna (imilecleucel-T), an investigational personalized T-cell therapy for patients with multiple sclerosis (MS), in a deal that could be worth as much as $225 million. [Genetic Engineering & Biotechnology News] Press Release

Aduro Announces Acquisition of GVAX Assets from BioSante
Aduro BioTech, Inc. announced the acquisition of all GVAX assets from BioSante Pharmaceuticals, Inc., including intellectual property and cell lines. This acquisition substantially expands Aduro’s immunotherapy portfolio and strengthens its competitive position in the field. [Business Wire] Press Release

Insception Biosciences Inc. Offers New Expertise to CCRM’s Industry Consortium
Canada’s largest and most established stem cell bank is the newest member of the Centre for Commercialization of Regenerative Medicine’s (CCRM) industry consortium. Insception Lifebank Cord Blood Program, a FACT‐accredited family cord blood bank, has extensive experience working with hematopoietic stem cells that CCRM will soon require to advance its therapeutic product pipeline. [Insception Biosciences Inc.] Press Release

Pioneering Research to Manufacture Cells from Umbilical Cord Blood
A new project to develop tools for the manufacture of large quantities of medically valuable cells from umbilical cord blood has been launched by Loughborough University. Dr. Rob Thomas from the University’s School of Mechanical and Manufacturing Engineering has been awarded a £1.3 million research fellowship by the Engineering and Physical Sciences Research Council for the study. [Loughborough University] Press Release

Shire Regenerative Medicine Initiates Phase III Study of ABH001 for Patients with Epidermolysis Bullosa
Shire plc announced the initiation of a Phase III study designed to evaluate the efficacy and safety of ABH001, its dermal substitute therapy, for the treatment of non-healing wounds in patients with epidermolysis bullosa. [Shire plc] Press Release

RNL BIO Filed Phase II/III Clinical Trial with KFDA to Treat Cerebral Palsy Using Autologous Fat Derived Stem Cells
RNL BIO CO LTD announced the filing of an Investigational New Drug application with the Korean Food and Drug Administration (KFDA) to initiate clinical trials phase II and III assessing the company’s RNL-Astrostem(TM) stem cell drug in patients with cerebral palsy. [PR Newswire Association LLC] Press Release

Regen BioPharma Files Investigational New Drug (IND) Application with FDA on HemaXellerate I™ Stem Cell Drug for Aplastic Anemia
Regen BioPharma a wholly-owned subsidiary of Bio-Matrix Scientific Group Inc., announced filing of an IND application with the FDA to initiate clinical trials assessing the company’s HemaXellerate I™ stem cell drug in patients with drug-refractory aplastic anemia. [Marketwire] Press Release

Key Mesenchymal Precursor Cell Patents Granted in United States and China
Regenerative medicine company Mesoblast Limited announced that it has been granted three key new patents by the United States Patent and Trade Mark Office and the State Intellectual Property Office of the People’s Republic of China. These patents deliver major commercial advantages and offer long term protection in these territories for the company’s products based on its proprietary mesenchymal precursor cell adult stem cell technology platform. [Mesoblast Limited] Press Release

Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2013-02943)

Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2013-02697)

National Cancer Institute; Notice of Closed Meetings (FR Doc No: 2013-02387)

National Institute of Biomedical Imaging and Bioengineering; Notice of Closed Meeting (FR Doc No: 2013-02696)

Team-Based Design in Biomedical Engineering Education (R25)(PAR-13-111)

NEW The Jackson Laboratory: Cutting Edge Human Disease Modeling – The NSG Mouse
February 14, 1:00PM (ET in US)
Webinar

NEW The Jackson Laboratory: Mouse TLC – Tips for Breeding and Housing Immunodeficient Mice
February 21, 1:00PM (ET in US)
Webinar

NEW Clinical Translation of Stem Cells 2013
April 22-23, 2013
Palm Springs, United States

NEW 2013 Society for Hematology and Stem Cells (ISEH) Annual Scientific Meeting
August 22-25, 2013
Vienna, Austria

NEW Stem Cells & Cell Therapy Summit
September 5-6, 2013
London, United Kingdom

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.

Senior QC Analyst/Assay Development Associate (StemCells, Inc.)

Postdoctoral Position (Sandra Rotman Centre for Global Health)

Faculty Position – Stem Cell, Vascular and Developmental Biology in Ophthalmology (Zhongshan Ophthalmic Center, Sun Yat-Sen University)

Postdoctoral Position – Cardiovascular Regenerative Therapies (University of California, Los Angeles)

Postdoctoral Researcher (The University of Texas Health Science Center at San Antonio)

Postdoctoral Fellow – Transcriptional Regulation in CD8 T Lymphocytes (Université Libre de Bruxelles – Institute for Medical Immunology)

Postdoctoral Researcher Position in Bioinformatics (University of Oulu)

Physician-Scientist – Cancer Immunotherapy Program (The University of Chicago)

Postdoctoral Position – Center for Cell Engineering (Memorial Sloan-Kettering Cancer Center)

Research Assistants (Plasticell Limited)


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