Volume 15.38 | Sep 29

Issue 15.38 September 29, 2014
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Protein that Causes Frontotemporal Dementia also Implicated in Alzheimer’s Disease
Researchers have shown that low levels of the protein progranulin in the brain can increase the formation of amyloid-beta plaques (a hallmark of Alzheimer’s disease), cause neuroinflammation, and worsen memory deficits in a mouse model of this condition. Conversely, by using a gene therapy approach to elevate progranulin levels, they were able to prevent these abnormalities and block cell death in this model. [Press release from the Gladstone Institutes discussing online prepublication in Nature Medicine] Press Release | Abstract
SmartDish™ with STEMgrid™-6 for Accurate Counting of Hematopoietic CFU Assays
PUBLICATIONS (Ranked by impact factor of the journal)
The Immune Microenvironment Confers Resistance to MAPK Pathway Inhibitors through Macrophage-Derived TNFα
Researchers identified macrophage-derived TNFα as a crucial melanoma growth factor that provides resistance to MAPK pathway inhibitors through the lineage transcription factor MITF (microphthalmia transcription factor). In BRAF-mutant melanomas of patients and BRAFV600E melanoma allografts, MAPK pathway inhibitors increased the number of tumor-associated macrophages, and TNFα and MITF expression. [Cancer Discov]
Abstract | Full Article | Press Release

A Double-Negative Feedback Loop between MicroRNA-422a and FOXG1/Q1/E1 Regulates Hepatocellular Carcinoma Tumor Growth and Metastasis
A mechanistic study identified three genes, forkhead box G1 (FOXG1), FOXQ1, and FOXE1, as microRNA-422a targets in the regulation of human hepatocellular carcinoma (HCC) development. Scientists investigated the function of the three targets themselves in HCC tumorigenesis using RNAi manipulation and demonstrated that the knockdown of these targets led to significant inhibition of tumor cell proliferation and migration both in vitro and in vivo. [Hepatology] Abstract

Enhanced Bone Tissue Regeneration by Antibacterial and Osteoinductive Silica-HACC-Zein Composite Scaffolds Loaded with rhBMP-2
Biodegradable rhBMP-2 loaded zein-based scaffolds with a macroporous structure were synthesized, and SBA-15 nanoparticles and hydroxypropyltrimethyl ammonium chloride chitosan (HACC) were incorporated into the scaffolds to produce an anti-infective composite scaffold for delivery of osteogenic factors that facilitate the functional repair of bone defects. [Biomaterials] Abstract

Persistence of Virus Reservoirs in ART-Treated SHIV-Infected Rhesus Macaques after Autologous Hematopoietic Stem Cell Transplant
Researchers performed autologous hematopoietic stem cell transplantation (HSCT) in three Simian/Human Immunodeficiency Virus (SHIV)-infected, antiretroviral therapy (ART)-treated rhesus macaques using HSCs collected prior to infection and compared them to three SHIV-infected, ART-treated, untransplanted control animals to assess the effect of conditioning and autologous HSCT on viral persistence. [PLoS Pathog]
Full Article | Press Release

Immunogenicity of Allogeneic Mesenchymal Stem Cells Transplanted via Different Routes in Diabetic Rats
Scientists evaluated the immunogenicity of allogeneic mesenchymal stem cells (MSCs) in vivo by transplanting MSCs into normal and diabetic rats via the tail vein or pancreas and found that MSCs exhibited low immunogenicity in normal recipients and even exerted some immunosuppressive effects in diabetic rats during the initial phase. [Mucosal Immunol] Abstract

Combining Autologous Dendritic Cell Therapy with CD3 Antibodies Promotes Regulatory T Cells and Permanent Islet Allograft Acceptance
Scientists investigated a novel approach combining administration of autologous tolerogenic dendritic cells with short-term treatment with CD3-specific Abs. Permanent acceptance of pancreatic islet allografts was achieved in mice treated with the combination therapy the day before transplantation but not in recipients treated with either therapy alone. [J Immunol] Abstract

Cell-Based Therapy for Acute and Chronic Liver Failures: Distinct Diseases, Different Choices
Which cell type is the most suitable source of cell-based therapies (CBTs) for acute liver failure (ALF) or chronic liver failure (CLF) remains unclear. To investigate this, mature hepatocytes in adult liver, fetal liver cells, induced hepatic stem cells and bone marrow derived mesenchymal stromal cells were used as CBTs for ConA-induced ALF and Fah-deficient induced CLF in mice. [Sci Rep] Full Article

Aromatic-Turmerone Induces Neural Stem Cell Proliferation In Vitro and In Vivo
It has been suggested that aromatic (ar-) turmerone inhibits microglia activation, a property that may be useful in treating neurodegenerative disease. In vitro as well as in vivo, ar-turmerone promoted neuronal differentiation of neural stem cells. [Stem Cell Res Ther] Full Article | Press Release

Mesenchymal Stem Cells with Increased SDF-1 Expression Enhanced Fracture Healing
To enhance stem cell involvement, researchers investigated over-expression of stem cells with SDF-1 using an adenovirus. In a rat defect bone model, bone marrow mesenchymal stem cells over-expressing SDF-1 showed significantly more new bone formation within the gap and less bone mineral loss at the area adjacent to the defect site during the early bone healing stage. [Tissue Eng Part A] Abstract

Transplantation of SHED Prevents Bone Loss in the Early Phase of Ovariectomy-Induced Osteoporosis
The mechanism by which stem cells from human exfoliated deciduous teeth (SHED) treat immune diseases is not fully understood. Investigators show that systemic transplantation of SHED via the tail vein ameliorates ovariectomy (OVX)-induced osteopenia by reducing T-helper 1 and T-helper 17 cell numbers in the recipient OVX mice. [J Dent Res] Abstract

In Vivo Evaluation of Adeno-Associated Virus Gene Transfer in Airways of Mice with Acute or Chronic Respiratory Infection
While gene therapy development for cystic fibrosis often involves viral-based vectors, little is known about gene transfer in the context of an infected airway. In this study, three mouse models were established to evaluate adeno-associated virus gene transfer in such an environment. [Hum Gene Ther] Abstract

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Biomimetic Nanoparticles for siRNA Delivery in the Treatment of Leukemia
Identification of a number of unique fusion genes has facilitated the development of highly specific small interfering RNAs (siRNA). The authors describe current treatments for leukemia and highlight the potential of leukemic fusion genes as therapeutic targets for RNA interference. [Biotech Adv] Abstract

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

View Data: StemSpan™ SFEM II Serum-Free Medium
Results of a Phase III Randomized Study of Nivolumab in Patients with Advanced Melanoma after Prior Anti-CTLA4 Therapy
In patients with metastatic melanoma who progressed on or after anti-CTLA-4 therapy and treatment with BRAF inhibitors in the case of BRAF mutation positive disease, nivolumab was well tolerated and showed a higher overall response rate when compared with investigator’s choice chemotherapy. [Press release from the European Society for Medical Oncology (ESMO) discussing research presented at the ESMO 2014 Congress, Madrid] Press Release

Provectus Biopharmaceuticals’ PV-10 Clinical Data on Melanoma
Provectus Biopharmaceuticals, Inc. announced that the abstract to be presented entitled, “Subgroup efficacy in patients receiving intralesional rose bengal to all existing melanoma in Phase II study PV-10-MM-02,” is available online. [Press release from Provectus Biopharmaceuticals, Inc. discussing research presented at the European Society for Medical Oncology (ESMO) 2014 Congress, Madrid] Press Release | Abstract

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New Washington Player Joins Push for NIH Funding
Scores of patient groups, scientific societies, and university coalitions devote much of their time to lobbying the U.S. Congress for more funding for biomedical research and the National Institutes of Health (NIH). This week another group, ACT for NIH: Advancing Cures Today, joined their ranks. [ScienceInsider] Editorial

Nature Communications to Become Open Access Only
Nature Communications is to become the first Nature-branded open access only journal. Nature Communications will only accept open access research submissions from 20th October 2014. [Nature] Editorial
Genzyme Collaborates on Gene Therapy for Rare Disease that Causes Childhood Blindness
Genzyme announced the establishment of a research collaboration with the University of Florida and the University of Pennsylvania to develop a gene therapy for the treatment of a rare genetic disease that causes childhood blindness. [Genzyme Corporation] Press Release

ImmunoCellular Therapeutics Expands Cancer Immunotherapy Platform with Antigen-Specific T-Cell Technology Licensed from Caltech
ImmunoCellular Therapeutics, Ltd. announced that it has established a licensing agreement with the California Institute of Technology (Caltech) for exclusive rights to novel technology for the development of certain antigen specific T-cell immunotherapies for the treatment of cancer. [ImmunoCellular Therapeutics, Ltd.] Press Release

Sumitomo Dainippon Pharma and SanBio Conclude Joint Development and License Agreement for North America over SB623, Therapy for Stroke
Sumitomo Dainippon Pharma Co., Ltd. and SanBio Co., Ltd. announced that the two companies have entered into a joint development and license agreement for exclusive marketing rights in the U.S. and Canada for SB623, a cell therapy for the treatment of patients with chronic stroke. [Sumitomo Dainippon Pharma Co., Ltd.] Press Release

Novartis AIN457 (Secukinumab) Is the First Ever IL-17A Inhibitor to Meet Primary Endpoint in Two Phase III Studies in Psoriatic Arthritis
Novartis announced that two pivotal Phase III studies of secukinumab in psoriatic arthritis met primary and key secondary endpoints. Secukinumab is an investigational medicine that works by stopping the action of interleukin-17A, a protein that is central to the development of inflammatory diseases. [Novartis AG] Press Release

Advanced Cell Technology Announces Final Patient Treated in Stargardt’s Macular Degeneration Phase I Trial in the United Kingdom
Advanced Cell Technology, Inc. announced that it has completed treatment of the final patient in its U.K. based Phase I clinical trial for Stargardt’s macular degeneration. The successful transplantation of the company’s proprietary retinal pigment epithelium cells in the last patient of twelve represents the completion of the enrollment stage of this trial. [Advanced Cell Technology, Inc.] Press Release

AbbVie’s HUMIRA® (Adalimumab) Receives U.S. FDA Approval for the Treatment of Pediatric Patients with Moderately to Severely Active Crohn’s Disease
AbbVie announced that the U.S. Food and Drug Administration (FDA) has approved HUMIRA® for reducing signs and symptoms, and achieving and maintaining clinical remission, in pediatric Crohn’s disease patients six years of age and older when certain other treatments have not worked well enough. [AbbVie Inc.] Press Release

Fibrocell Science and Intrexon Advance GM-HDF-COL7 toward Clinical Development for the Treatment of Recessive Dystrophic Epidermolysis Bullosa
Fibrocell Science, Inc., and Intrexon Corporation announced plans to file an investigational new drug application with the U.S. Food and Drug Administration for their drug candidate, GM-HDF-COL7 (genetically-modified human dermal fibroblast, collagen VII). [Fibrocell Science, Inc.] Press Release

Bioheart Announces Successful Grand Opening of Facility in South Africa
Bioheart, Inc. announced a successful grand opening of the laboratory and clinic in South Africa. The new facility, named South African Stem Cell Institute, immediately began treating patients. [Bioheart, Inc.] Press Release

Regeneus’ Osteoarthritis Cell Therapy, HiQCell, to Launch in Singapore
Regeneus Ltd. announced plans to open the HiQCell Regenerative Medicine Clinic in Singapore, and the appointment of leading specialist sports physician, Dr. Patrick Goh, as Medical Director. Through the clinic, Goh will provide patients in Singapore with access to Regeneus’ HiQCell, an innovative cell therapy for the treatment of osteoarthritis, with first treatments expected to commence in late 2014. [Regeneus Ltd.] Press Release
National Cancer Institute; Notice of Closed Meetings (FR Doc. No: 2014-22892)

Government-Owned Inventions; Availability for Licensing (FR Doc. No: 2014-22763)
Termination of Order to Cease Manufacturing of HCT/Ps – New York Fertility Institute
Food and Drug Administration (United States)

Agency Information Collection Activities; Submission for Office of Management and Budget Review; Comment Request; Good Laboratory Practice Regulations for Nonclinical Studies (FR Doc. No: 2014-22986)
NEW 2014 Till & McCulloch Meetings
October 27-29, 2014
Ottawa, Canada

NEW CellTech India 2015
March 2-3, 2015
Bangalore, India

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Postdoctoral Fellowship – AAV Gene Therapy (Icahn School of Medicine at Mount Sinai)

Head of Cellular and Molecular Therapies Laboratory (NHS Blood & Transplant – Oxford)

Head of Cellular and Molecular Therapies Laboratory (NHS Blood & Transplant – Leeds)

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

Research Technician – Stem Cells and Regenerative Medicine (King’s College London)

Postdoctoral Fellow – Cardiac Differentiation of Pluripotent Stem Cells (Stanford University)

Postdoctoral Fellow – Cardiac Animal Surgery (Stanford University)

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

Research Associate – Gene Therapy Center (University of Massachusetts)

Postdoctoral Fellow – New Combination Immune Therapies for Cancer (Massachusetts General Hospital)

Postdoctoral Position – Scaffolding and Drug Delivery for Regenerative Medicine Applications (Ospedale Niguarda)

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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