Volume 15.39 | Oct 6

Issue 15.39 October 6, 2014
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Researchers Develop Novel Gene/Cell Therapy Approach for Lung Disease
Researchers developed a new type of cell transplantation to treat mice mimicking a rare lung disease that one day could be used to treat this and other human lung diseases caused by dysfunctional immune cells. They transplanted either normal or gene-corrected macrophages into the respiratory tracts of mice. Treatment with both normal and gene-corrected macrophages corrected the disease in the mice. [Press release from Cincinnati Children’s Hospital Medical Center discussing online prepublication in Nature] Press Release | Abstract
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PUBLICATIONS (Ranked by impact factor of the journal)
Oligopeptide Complex for Targeted Non-Viral Gene Delivery to Adipocytes
Scientists show that an adipocyte-targeting fusion-oligopeptide gene carrier consisting of an adipocyte-targeting sequence and 9-arginine (ATS-9R) selectively transfects mature adipocytes by binding to ​prohibitin. Injection of ATS-9R into obese mice confirmed specific binding of ATS-9R to fat vasculature, internalization and gene expression in adipocytes. [Nat Mater] Abstract | Editorial

Liver-Directed Gene Therapy Corrects Cardiovascular Lesions in Feline Mucopolysaccharidosis Type I
Using the naturally occurring feline model of mucopolysaccharidosis type I, scientists tested liver-directed gene therapy as a means of achieving long-term systemic α-l-iduronidase reconstitution. [Proc Natl Acad Sci USA] Abstract | Press Release

SDF-1 Inhibition Targets the Bone Marrow Niche for Cancer Therapy
Using in vivo murine and xenograft mouse models, investigators documented that in vivo stromal cell-derived factor-1 (SDF-1) neutralization within bone marrow niches leads to a microenvironment that is less receptive for multiple myeloma (MM) cells and reduces MM cell homing and growth, thereby inhibiting MM disease progression. [Cell Rep] Full Article | Graphical Abstract | Press Release

Blockade of mTOR Signaling via Rapamycin Combined with Immunotherapy Augments Anti-Glioma Cytotoxic and Memory T Cells’ Functions
Using the refractory rat RG2 glioma model, investigators demonstrate that administration of Rapamycin with Fms-like tyrosine kinase 3 ligand and Thymidine kinase/Ganciclovir immunotherapy enhanced the cytotoxic activity of anti-tumor CD8+ T cells. [Mol Cancer Ther] Abstract | Press Release

Injectable In Situ Self-Crosslinking Hydrogels Based on Poly(L-Glutamic Acid) and Alginate for Cartilage Tissue Engineering
A series of injectable poly(L-glutamic acid)/alginate hydrogels were fabricated by self-crosslinking of hydrazide-modified poly(L-glutamic acid) and aldehyde-modified alginate. A preliminary study exhibited injectability and rapid in vivo gel formation, as well as mechanical stability, cell ingrowth and ectopic cartilage formation. [Biomacromolecules] Abstract

Intramuscular Delivery of Heterodimeric IL-15 DNA in Macaques Produces Systemic Levels of Bioactive Cytokine Inducing Proliferation of NK and T Cells
Investigators have generated DNA vectors expressing the heterodimeric interleukin (IL)-15 by optimizing mRNA expression and protein trafficking. Administration of DNAs expressing heterodimeric IL-15 resulted in an increased frequency of NK and T cells undergoing proliferation in peripheral blood. [Gene Ther] Full Article

Gene Therapy Approach to FAP: In Vivo Influence of T119M in TTR Deposition in a Transgenic V30M Mouse Model
The authors propose to establish a new gene therapy approach using adeno-associated virus vectors to deliver the trans-suppressor transthyretin (TTR) T119M variant to the liver of transgenic TTR V30M mice at different ages. [Gene Ther] Full Article

Combining Enriched Environment, Progesterone, and Embryonic Neural Stem Cell Therapy Improves Recovery following Brain Injury
Scientists determined whether varying combinations of post-injury enriched environments, progesterone therapy, or embryonic neural stem cell transplantation would improve functional outcomes over just a single therapy. [J Neurotrauma] Abstract

MSC Therapy Attenuates Obliterative Bronchiolitis after Murine Bone Marrow Transplant
Administration of mesenchymal stromal cells (MSCs) was evaluated for the ability to ameliorate obliterative bronchiolitis in mice using an established allogeneic bone marrow transplant model. [PLoS One] Full Article

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Mechanism-Based Cancer Therapy: Resistance to Therapy, Therapy for Resistance
The authors review the most generalized mechanisms of resistance to targeted therapies, together with some experimental strategies employed to identify such mechanisms. [Oncogene] Abstract

Gene Therapy for Autoimmune Disease
The authors summarize the recent progress in identifying genes responsible for autoimmune diseases and present examples where gene therapy has been applied as treatment or prevention in autoimmune diseases both in animal models and clinical trials. [Clin Rev Allergy Immunol] Abstract

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

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Compugen Announces Presentations of Results for CGEN-15001 Supporting Immune Tolerance Induction
Compugen Ltd. announced that experimental results supporting CGEN-15001’s induction of long-term autoimmune disease remission in disease animal models, potentially through a novel mechanism of action, were presented. [Press release from Compugen Ltd. discussing research presented at the 3rd International Conference on Immune Tolerance, Amsterdam] Press Release

Roche’s Investigational Combination of Cobimetinib plus Zelboraf® (Vemurafenib) Provided Significant Benefit to People with Advanced Melanoma over Zelboraf Alone
Roche announced positive data from the coBRIM Phase III study. The results show that people with previously untreated BRAF V600 mutation-positive, advanced melanoma who received the MEK inhibitor cobimetinib plus Zelboraf lived significantly longer without their disease worsening or death compared to Zelboraf alone. [Press release from Roche Ltd. discussing research presented at the European Society of Medical Oncology (ESMO) 2014 Congress, Madrid] Press Release

Data Show PS-Targeting Antibodies Enhance the Anti-Tumor Activity of Immune Checkpoint Inhibitors by Decreasing Levels of Myeloid Derived Suppressor Cells (MDSC) in the Tumor Microenvironment
Peregrine Pharmaceuticals, Inc. announced the presentation of preclinical data related to the company’s immuno-oncology development program and its lead drug candidate bavituximab, a phosphatidylserine (PS)-targeting antibody. [Press release from Peregrine Pharmaceuticals, Inc. discussing research to be presented at the 22nd Annual International Cancer Immunotherapy Symposium, New York] Press Release

AABB Annual Meeting Cellular Therapy Workshop
Fountain of Youth: A Congressman’s Plan to Make NIH Grantees Younger
A member of Congress has waded into the thorny issue of the graying of U.S. biomedical researchers with a radical solution: He wants to order the National Institutes of Health (NIH) to bring down the average age at which new investigators receive their first grant by four years within a decade. [ScienceInsider] Editorial

E.U. Medicines Agency Hands Victory to Advocates of Trial Data Sharing
After an 18-month saga, the European Medicines Agency has approved the details of a new system allowing researchers to scrutinize unpublished data from clinical trials. [ScienceInsider] Editorial

End of the Road for Rogue Stem-Cell Therapy in Italy
The Italian health minister Beatrice Lorenzin declared that her government will not support a clinical trial of a controversial stem cell therapy that it promised last year. [Nature News] Editorial

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Bristol-Myers Squibb and MD Anderson Cancer Center Announce Novel Research Collaboration in Immuno-Oncology Focused on Leukemia and Hematologic Malignancies
Bristol-Myers Squibb Company and The University of Texas MD Anderson Cancer Center announced a novel clinical research collaboration to evaluate multiple immunotherapies as potential treatment options for acute and chronic leukemia as well as other hematologic malignancies. [Bristol-Myers Squibb Company] Press Release

LCT Collaborates to Develop Novel Neurological Treatments
Living Cell Technologies Limited (LCT) has formed a collaboration with the Center for Brain Research. The research collaboration will identify additional neurodegenerative disease targets for clinical studies of LCT’s lead product NTCELL®. [Living Cell Technologies Limited] Press Release

NIBSC Signs Agreement with UCL
A Memorandum of Understanding has been signed between the National Institute for Biological Standards and Control (NIBSC) and University College London (UCL). The memorandum recognizes the tremendous promise of gene therapy, stem cell therapy and tissue engineering in preventing and treating illness, restoring health and saving lives. [National Institute for Biological Standards and Control] Press Release

FDA Awards Grants to Stimulate Drug, Device Development for Rare Diseases
The U.S. Food and Drug Administration (FDA) announced it has awarded 15 grants totaling more than $19 million to boost the development of medical device, drug, and biological products for patients with rare diseases, with at least a quarter of the funding going to studies focused solely on pediatrics. [U.S. Food and Drug Administration] Press Release

ExxonMobil Donates $18 Million to World-Renowned Houston Health Institutions
ExxonMobil announced $18 million in grants to Houston health care facilities. From the engineering of artificial hearts to advancements in regenerative medicine, which uses patients’ own adult stem cells to create whole hearts, the Texas Heart Institute is offering new hope to thousands of patients worldwide who are not candidates for a heart transplant. [ExxonMobil Corporation] Press Release

International Stem Cell Corporation Provides Update on the Status of Its Parkinson’s Disease Program
International Stem Cell Corporation provided an update on the status of Parkinson’s disease preclinical program. To date no adverse events or pathological reactions have been observed in any of the completed preclinical pharmacology/toxicology studies. [International Stem Cell Corporation] Press Release

Tokyo Electron Launches Its Open Innovation Stem Cell Technology Centre (STC) in the UK to Develop Smart Cell Processing Technologies
Tokyo Electron Limited opened the company’s open innovation center at Stevenage Bioscience Catalyst in the United Kingdom. The goal is to jointly, with ecosystem partners from academia and industry, develop standardized smart cell processing technologies for cell culture and inspection through open innovation. [Tokyo Electron Limited] Press Release

Horwitz Prize Awarded for Work on Therapy that Uses the Immune System to Destroy Cancer Cells
Columbia University will award the 2014 Louisa Gross Horwitz Prize to James P. Allison, PhD, of the University of Texas MD Anderson Cancer Center, for his work on understanding the process of T-cell activation and for developing therapies that harness the immune system to fight cancer. [Columbia University] Press Release

Midwestern Regional Medical Center Recognized for Exemplary Stem Cell Treatment and Cell Therapy Program
Cancer Treatment Centers of America at Midwestern Regional Medical Center has been recognized by the Foundation for the Accreditation of Cellular Therapy (FACT) at the University of Nebraska Medical Center for demonstrating compliance with the FACT-JACIE International Standards for Cellular Therapy Product Collection, Processing and Administration. [Cancer Treatment Centers of America] Press Release
National Institute of General Medical Sciences; Notice of Closed Meetings (FR Doc. No: 2014-23732)

National Cancer Institute; Notice of Closed Meeting (FR Doc. No: 2014-23737)
FDA Study Identifies Occurrence and Risk Factors for TRALI, A Post-Transfusion Respiratory Complication, among the U.S. Elderly during 2007 through 2011
European Medicines Agency (European Union)

Publication of Clinical Reports
NEW Global Controls in Stem Cells
November 5-7, 2014
Singapore, Singapore

NEW Biologics Congress 2015
February 2-3, 2015
Berlin, Germany

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Postdoctoral Position – Leukemic Stem Cells (BC Cancer Agency)

NEW 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 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)

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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