Volume 15.41 | Oct 20

Issue 15.41 October 20, 2014
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Personalized Cellular Therapy Achieves Complete Remission in 90 Percent of Acute Lymphoblastic Leukemia Patients Studied
Ninety percent of children and adults with acute lymphoblastic leukemia who had relapsed multiple times or failed to respond to standard therapies went into remission after receiving an investigational personalized cellular therapy, CTL019. [Press release from Penn Medicine discussing publication in The New England Journal of Medicine] Press Release | Abstract
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PUBLICATIONS (Ranked by impact factor of the journal)
Human Embryonic Stem Cell-Derived Retinal Pigment Epithelium in Patients with Age-Related Macular Degeneration and Stargardt’s Macular Dystrophy: Follow-Up of Two Open-Label Phase I/II Studies
Two prospective Phase I/II studies were done to assess the primary endpoints safety and tolerability of subretinal transplantation of human embryonic stem cells-derived retinal pigment epithelium in nine patients with Stargardt’s macular dystrophy and nine with atrophic age-related macular degeneration. [Lancet] Abstract | Press Release

T Cells Expressing CD19 Chimeric Antigen Receptors for Acute Lymphoblastic Leukemia in Children and Young Adults: A Phase I Dose-Escalation Trial
Researchers aimed to define feasibility, toxicity, maximum tolerated dose, response rate, and biological correlates of response in children and young adults with refractory B-cell malignancies treated with CD19-chimeric antigen receptor T cells. [Lancet] Abstract | Press Release

Myeloproliferative Neoplasms Can Be Initiated from a Single Hematopoietic Stem Cell Expressing JAK2-V617F
Researchers demonstrate by limiting dilution and single-cell transplantations that myeloproliferative neoplasm (MPN) disease, manifesting either as erythrocytosis or thrombocytosis, can be initiated clonally from a single cell carrying JAK2-V617F. However, only a subset of mice reconstituted from single hematopoietic stem cells displayed MPN phenotype. [J Exp Med] Abstract

A Phase I/IIa Follistatin Gene Therapy Trial for Becker Muscular Dystrophy
In this Becker muscular dystrophy proof-of-principle clinical trial, a potent myostatin antagonist, follistatin, was used to inhibit the myostatin pathway. Extensive pre-clinical studies, using adeno-associated virus to deliver follistatin, demonstrated an increase in strength. [Mol Ther] Abstract | Press Release

Murine and Human Tissue-Engineered Esophagus Form from Sufficient Stem/Progenitor Cells and Do Not Require Microdesigned Biomaterials
Esophageal organoid units were isolated from murine or human esophagi and implanted on a polyglycolic acid/poly-L-lactic acid collagen-coated scaffold in adult allogeneic or immune-deficient mice. [Tissue Eng Part A] Abstract | Press Release

Targeted Transplantation of Human Umbilical Cord Blood Endothelial Progenitor Cells with Immunomagnetic Nanoparticles to Repair Corneal Endothelium Defect
A novel method of targeted cellular transplantation without permanent residence of cell carriers in the host was proposed. Human umbilical cord blood endothelial progenitor cells (UCB EPCs) were labeled with CD34 immunomagnetic nanoparticles. The UCB EPCs labeled with nanoparticles were transplanted into the anterior chamber of rabbits with magnet attraction. [Stem Cells Dev] Abstract

Mesenchymal Stromal Cells Engineered to Produce IGF-I by Recombinant Adenovirus Ameliorate Liver Fibrosis in Mice
Scientists analyzed the effect of applying insulin growth factor like-I (IGF-I) overexpressing mouse bone marrow-derived mesenchymal stromal cells (MSCs) on hepatic fibrosis. Liver fibrosis was ameliorated after single dose application of MSCs engineered to produce IGF-I when compared to MSCs engineered to produce GFP and/or recombinant IGF-I treatments. [Stem Cells Dev] Abstract

Multipotent Mesenchymal Stromal Cells Synergize with Costimulation Blockade in the Inhibition of Immune Responses and the Induction of Foxp3+ Regulatory T Cells
Mesenchymal stromal cell (MSCs) from C57BL/6 (H2b) mice were infused together with fully major histocompatibility complex-mismatched Balb/c allogeneic islets into the portal vein of diabetic H2b mice, which were subsequently treated with costimulation blockade for the first 10 days after transplantation. Mice receiving both recipient-type MSCs, CTLA4Ig, and anti-CD40L demonstrated indefinite graft acceptance, just as did most of the recipients receiving MSCs and CTLA4Ig. [Stem Cells Transl Med] Abstract

Testing Stem Cell Therapy in a Rat Model of Inflammatory Bowel Disease: Role of Bone Marrow Stem Cells and Stem Cell Factor in Mucosal Regeneration
Researchers explored the role of bone marrow mesenchymal stromal cells and soluble stem cell factor in gastrointestinal mucosa regeneration in a rat model of inflammatory bowel diseases. [PLoS One] Full Article

Salvage Therapy with ARA-C and Gemtuzumab Ozogamicin in AML Patients Relapsing after Stem Cell Transplantation
Researchers report their experience with ARA-C and gemtuzumab ozogamicin (GO) combination in relapsed post-stem cell transplantation acute myeloid leukemia (AML) patients. Therapy consisted of ARA-C for four days followed by one dose of GO 9 mg/m2 on day five and was supported by donor stem cells when possible. [Ann Hematol] Abstract

View Data: UM729, a Novel Small Molecule for Ex Vivo Expansion of Human hSCs
Bone Marrow-Derived Stem Cell Therapy for Metastatic Brain Cancers
The authors discuss the potential repair of the blood brain barrier and attenuation of brain metastatic through transplantation of bone marrow-derived mesenchymal stem cells with the endothelial progenitor cell phenotype. [Cell Transplant] Full Article

Gene Therapy for Inherited Muscle Diseases: Where Genetics Meets Rehabilitation Medicine
Recent advances in the application of gene therapies to treat inherited muscle disorders are presented, including Duchenne muscular dystrophy and x-linked myotubular myopathy. Potential areas for therapeutic synergies between rehabilitation medicine and genetics are also discussed. [Am J Phys Med Rehabil] Abstract

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

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MOLOGEN AG Presents EnanDIM: A New Generation of Immunomodulators
MOLOGEN has presented preclinical data on its EnanDIM technology. EnanDIM represents a new generation of the company’s immunoactivating TLR-9 agonists that is expected to trigger a broad immune activation while being well tolerated. [Press release from MOLOGEN AG discussing research presented at the 10th Annual Meeting of the Oligonucleotide Therapeutics Society (OTS), San Diego] Press Release

Provectus’ Intralesional PV-10 Clinical Data to Be Presented
Provectus Biopharmaceuticals, Inc. announced that Sanjiv S. Agarwala, MD, of St. Luke’s University Hospital and Professor of Medicine at Temple University will present data on its investigational new drug PV-10 for the treatment of melanoma. [Press release from Provectus Biopharmaceuticals, Inc. discussing research to be presented at the 11th International Congress of the Society for Melanoma Research, Zurich] Press Release

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Updated: U.S. Halts Funding for New Risky Virus Studies, Calls for Voluntary Moratorium
The White House stepped into an ongoing debate about controversial virus experiments with a startling announcement: It is halting all federal funding for so-called gain-of-function studies that alter a pathogen to make it more transmissible or deadly so that experts can work out a U.S. government-wide policy for weighing the risks. [ScienceInsider] Editorial

Issues Continue to Dog the Testing of Ebola Drugs and Vaccines
At a U.S. congressional hearing that examined the country’s public health response to Ebola, an official from the Food and Drug Administration said it’s working to develop “a flexible and innovative protocol” to evaluate experimental treatments for the disease. [ScienceInsider] Editorial

Australia’s New Innovation Agenda Leaves Little Room for Science
Australia’s scientific leaders are cautiously hopeful that the government’s new innovation policy marks a more positive stance on research. [ScienceInsider] Editorial
Aduro Expands Collaboration with Johnson & Johnson Innovation and Janssen for Lung Cancer Immunotherapies
Aduro BioTech, Inc. announced that it has entered into its second agreement with Janssen Biotech, Inc. granting an exclusive, worldwide license to certain product candidates engineered for the treatment of lung cancer and certain other cancers based on its novel LADD immunotherapy platform. [Aduro BioTech, Inc. (Business Wire)] Press Release

Batu Biologics Awarded Exclusive License from University of California San Diego for Drug that Stimulates Immune System to Kill Cancer
Batu Biologics, an immunotherapy company developing an anti-angiogenic cancer vaccine, announced the signing of a license agreement with the Regents of the University of California, granting exclusive rights to novel peptide drugs developed at UC San Diego that have been proven to stimulate cancer-killing immune responses. [Batu Biologics (Business Wire)] Press Release

Selecta, JDRF and Sanofi Extend Collaboration to Develop a Synthetic Vaccine Particle Immunotherapy for Type 1 Diabetes
Selecta Biosciences, Inc. and JDRF announced that they have extended their joint research collaboration and attracted Sanofi as a partner in support of their program to develop a synthetic vaccine particle immunotherapy with the long-term goal to treat and potentially prevent the underlying cause of type 1 diabetes. [Selecta Biosciences, Inc.] Press Release

The New York Stem Cell Foundation Announces $9 Million to Six New NYSCF – Robertson Investigators
The New York Stem Cell Foundation (NYSCF) named six of the most promising scientists as its 2014 NYSCF – Robertson Investigators. Each Investigator will receive a $1.5 million award, which will be disbursed over the next five years, which will enable them to expand their laboratories and train other scientists. [The New York Stem Cell Foundation] Press Release

Asterias Biotherapeutics Announces Notice of Grant Award with CIRM for Phase I/IIa Clinical Trial of AST-OPC1 in Complete Cervical Spinal Cord Injury
Asterias Biotherapeutics, Inc. announced that the company has signed a notice of grant award (NGA) with the California Institute of Regenerative Medicine (CIRM), effective October 1, 2014. The NGA provides for the immediate release of clinical development payments and the release of additional grant funds pursuant to the previously announced $14.3 million CIRM grant award for clinical development of Asterias’ product, AST-OPC1. [Asterias Biotherapeutics, Inc.] Press Release

FDA Approves Expanded Use of Navidea’s Lymphoseek® for Lymphatic Mapping in Solid Tumors
Navidea Biopharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has approved the supplemental new drug application for the expanded use of Lymphoseek® injection for lymphatic mapping in solid tumors and adding sentinel lymph node detection for breast cancer and melanoma to the approved indications. [Navidea Biopharmaceuticals, Inc.] Press Release

bluebird bio Announces First Patient with Sickle Cell Disease Transplanted with LentiGlobin Gene Therapy
bluebird bio, Inc. announced that the first subject with severe sickle cell disease has undergone infusion with bluebird bio’s LentiGlobin BB305 drug product in an autologous hematopoietic stem cell transplantation. [bluebird bio, Inc.] Press Release

BioLife Solutions Biopreservation Media Products Used at NeoStem Subsidiary Progenitor Cell Therapy
BioLife Solutions, Inc. provided an update on use of the company’s HypoThermosol storage/shipping media and CryoStor cryopreservation freeze media by Progenitor Cell Therapy, LLC, a wholly owned subsidiary of NeoStem, Inc. [BioLife Solutions, Inc.] Press Release
Prospective Grant of Exclusive License: Development of T Cell Receptors for Adoptive Transfer in Humans To Treat Cancer (FR Doc. No: 2014-24502)

Prospective Grant of Exclusive Option License: The Development of a Single Domain Human Anti-Mesothelin Monoclonal Antibody for the Treatment of Human Cancers (FR Doc. No: 2014-24501)
Food and Drug Administration (United States)

Society of Clinical Research Associates-Food and Drug Administration: Food and Drug Administration Clinical Trial Requirements, Regulations, Compliance, and Good Clinical Practice; Public Workshop (FR Doc. No: 2014-24307)

Medicines and Healthcare Products Regulatory Agency

‘One Stop Shop’ for Advice on Regenerative Medicine Opening for Business
NEW The Australasian Society for Stem Cell Research (ASSCR) Meeting 2014
November 9-11, 2014
Lorne, Australia

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Postdoctoral Position – Muscle Cell Therapy (I-Stem)

NEW Graduate Student – Pediatric Cancers (University of Calgary)

NEW Research Assistant – Cell Therapy (Institute of Biochemistry of the Romanian Academy)

Quality Improvement Coordinator – Regenerative Medicine (The University of Texas Health Science Center at Houston)

Postdoctoral Position – Leukemic Stem Cells (BC Cancer Agency)

Postdoctoral Position – Hepatic Regenerative Medicine (INSERM)

Postdoctoral Fellowship – AAV Gene Therapy (Icahn School of Medicine at Mount Sinai)

Postdoctoral Position – Novel Mediators of Cardiovascular Disease and Novel Therapeutic Strategies (Monzino Cardiology Center)

Postdoctoral Position – Transplantation Aspects of Hepatocytes Derived from Human Pluripotent Stem Cells (INSERM UMR 1064)

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

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

Scientist – Liver Cell Biology (STEMCELL Technologies Inc.)

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

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