Volume 14.24 | Jun 24

Issue 14.24 June 24, 2013
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No Danger of Cancer through Gene Therapy Virus
The first modified adeno-associated virus was recently approved for clinical gene therapy in the Western world. Scientists have shown that no cancer risk emanates from the virus used for gene delivery. [Press release from the German Cancer Research Center discussing online prepublication in Nature Medicine]
Press Release | Abstract

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PUBLICATIONS (Ranked by impact factor of the journal)

iPSC-Derived ß Cells Model Diabetes Due to Glucokinase Deficiency
To determine whether stem cell-derived ß cells recapitulate molecular-physiological phenotypes of a diabetic subject, researchers generated induced pluripotent stem cells (iPSCs) from subjects with maturity-onset diabetes of the young type 2, which is characterized by heterozygous loss of function of the gene encoding glucokinase. These stem cells differentiated into ß cells with efficiency comparable to that of controls and expressed markers of mature ß cells, including urocortin-3 and zinc transporter 8, upon transplantation into mice. [J Clin Invest]
Full Article | Press Release

Safety and Clinical Efficacy of Rapidly-Generated Trivirus-Directed T Cells as Treatment for Adenovirus, EBV and CMV Infections after Allogeneic Hematopoietic Stem Cell Transplant
Investigators showed that T cells targeting a range of viral antigens derived from Epstein-Barr virus (EBV), cytomegalovirus (CMV), and adenovirus (Adv) can be reproducibly generated in a single culture over a 2-3 week period, using methods that exclude all viral components and employ a much-simplified culture technology. When administered to recipients of haploidentical, matched unrelated, mismatched unrelated or matched related transplants with active CMV, Adv, EBV, EBV+Adv or CMV+Adv infections, the cells produced complete virological responses in 80%, including all patients with dual infections. [Mol Ther] Abstract

Maturation and Function of Human Embryonic Stem Cell-Derived Pancreatic Progenitors in Macroencapsulation Devices following Transplant into Mice
Researchers previously demonstrated that human embryonic stem cell (hESC)-derived pancreatic progenitor cells matured under the kidney capsule in a mouse model of diabetes into glucose-responsive insulin-secreting cells capable of reversing diabetes. However, the formation of cells resembling bone and cartilage was a major limitation of that study. Therefore, they developed an improved differentiation protocol that aimed to prevent the formation of off-target mesoderm tissue following transplantation. [Diabetologia] Abstract

Efficacy of Epicardially-Delivered Adipose Stroma Cell Sheets in Dilated Cardiomyopathy
The authors assessed the effects of epicardially-delivered adipose tissue-derived stroma cell sheets in a mouse model of dilated cardiomyopathy based on cardiac-specific and tamoxifen-inducible invalidation of serum response factor. [Cardiovasc Res] Abstract

Bone Marrow Transplantation in Dysferlin Deficient Mice Results in a Mild Functional Improvement
Scientists evaluated the effect of bone marrow transplantation using the A/J Dysfprmd mouse model of dysferlinopathy. [Stem Cells Dev] Abstract

Beneficial Effects of Parenteral GLP-1 Delivery by Cell Therapy in Insulin-Deficient Streptozotocin Diabetic Mice
Researchers assessed the effects of chronic glucagon-like peptide-1 (GLP-1) delivery by cell therapy, using the GLP-1-secreting GLUTag cell line, in normoglycemic and streptozotocin-induced diabetic mice. GLUTag cell aggregates were transplanted into the subscapular region of mice. Over 30 days, cellular transplantation gave rise to encapsulated and well-vascularized growths, which contained immunoreactive GLP-1. [Gene Ther] Abstract

Mitigation of Lethal Radiation Syndrome in Mice by Intramuscular Injection of 3D Cultured Adherent Human Placental Stromal Cells
In the current study C3H/HeN mice were total body irradiated by 7.7 Gy. Twenty four hours and five days after irradiation 2×106 cells from different preparations of human derived 3D expanded adherent placental stromal cells were injected intramuscularly. [PLoS One] Full Article | Press Release

Endothelial Cells Overexpressing IL-8 Receptor Reduce Cardiac Remodeling and Dysfunction following Myocardial Infarction
Investigators tested the hypothesis that targeted delivery of endothelial cells overexpressing neutrophil membrane interleukin-8 (IL8) receptors IL8RA and IL8RB reduces acute myocardial infarction-induced left ventricular remodeling and dysfunction and increases neo-vascularization in the area at risk surrounding the infarcted tissue. [Am J Physiol Heart Circ Physiol] Abstract

Combining CAR T Cells and the Bcl-2 Family Apoptosis Inhibitor ABT-737 for Treating B-Cell Malignancy
Gene engineered chimeric antigen receptor (CAR) T cells show promise in B-cell malignancy, and as they induce apoptosis via the extrinsic pathway, researchers hypothesized that small-molecule inhibitors of the Bcl-2 family may potentiate the efficacy of CAR T cells by engaging both apoptosis pathways. [Cancer Gene Ther] Abstract

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Adult Mesenchymal Stromal Cell Therapy for Inflammatory Diseases: How Well Are We Joining the Dots?
In this review the authors summarize some of the most significant new developments for each of the four areas that contribute to the process of translating mesenchymal stromal (stem) cell (MSC) research to the clinical arena. In the context of this recent progress, they discuss key challenges and specific knowledge gaps which, if not addressed in a coordinated fashion, may hinder the creation of robust “translational pipelines” for consolidating the status of MSC-based therapies. [Stem Cells] Abstract | Full Article

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

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Alnylam Scientists Present Pre-Clinical Data with ALN-CC5, an RNAi Therapeutic Targeting Complement Component C5 for the Treatment of Complement-Mediated Diseases
Alnylam scientists presented pre-clinical results showing potent, dose-dependent, and durable RNAi-mediated knockdown of serum C5 and inhibition of complement-mediated hemolytic activity of approximately 90% with a subcutaneously administered RNAi therapeutic. [Press release from Alnylam Pharmaceuticals, Inc. discussing research presented at the 6th International Conference on Complement Therapeutics, Kos] Press Release

Affimed Reports Phase I Clinical Data for Its Immunotherapy AFM13 in Relapsing/Refractory Hodgkin Lymphoma Patients
Affimed Therapeutics AG announced encouraging results from a phase I clinical trial of AFM13 as monotherapy for the treatment of patients with advanced relapsing/refractory Hodgkin lymphoma. AFM13 appears to be well tolerated with evidence of meaningful anti-tumor activity, including partial responses and stable diseases. AFM13 is a bispecific TandAb antibody recruiting host NK cells via its CD16A-binding domains to engage and kill CD30-positive malignant cells. [Press release from Affimed Therapeutics AG discussing research presented at the 12th International Conference on Malignant Lymphoma, Lugano] Press Release

Cell Culture World Congress USA 2013


Japan Minister Defends New Growth Strategy Emphasizing Targeted Research
Japanese scientists are proving a bit skeptical about the administration’s new strategy for economic growth, which includes an emphasis on applied biomedical research. Even before it was officially released, several life science-related academic societies raised questions about plans for a Japanese version of the U.S. National Institutes of Health that would “strongly support the commercialization of innovative medical technologies.” [Minister for Economic Revitalization, Japan] Press Release

Japan to Relax Ban on Chimeric Embryo Experiments
A scientist pioneering research toward generating human organs in pigs has cleared one hurdle in gaining approval to continue his research in Japan. But he is still concerned that finalizing new research guidelines will take too long, so he is considering conducting the key experiments in the United States. [Japanese Government, Japan] Press Release

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VGTI Florida® Scientists to Receive $3.6 Million in NIH Research Grants – “Translating Research Into Health®.”
Scientists at the Vaccine & Gene Therapy Institute of Florida (VGTI Florida) have been awarded three new federal research grants that will further support the Institute’s mission of “Translating Research Into Health®.” Dr. Elias Haddad, Ph.D. Associate Member and part of the HIV Vaccine Group at VGTI Florida has been awarded a 5-year, $3.17 million dollar RO1 grant from the National Institutes of Health (NIH) for work on “Boosting anti-HIV immunity through manipulation of Tfh (follicular helper T cells) Function.” [Vaccine & Gene Therapy Institute of Florida] Press Release

Grant Initiates New Stem Cell Research for Patients Suffering with Corneal Blindness
With a new vision for stem cell corneal transplantation, and a $1.25 million grant from the National Eye Institute, Cedars-Sinai co-investigators Alexander V. Ljubimov, PhD, FARVO, and Clive Svendsen, PhD, hope to treat previously untreatable patients suffering with corneal blindness. [Cedars-Sinai] Press Release

Health Center Researchers Awarded State Stem Cell Funds
Nine scientists at the University of Connecticut (UConn) Health Center received grant awards totaling $4.5 million from the Connecticut Stem Cell Research Program, making UConn the largest recipient of the $9.8 million total grants awarded this year. [University of Connecticut] Press Release

UH Case Medical Center Launches Novel Clinical Trial Using Stem Cells to Prevent Amputation
University Hospitals (UH) Case Medical Center clinical researchers have launched an innovative clinical trial, unique in its design, which will evaluate the ability of a patient’s own stem cells to prevent leg amputations in end stage peripheral arterial disease. [EurekAlert!] Press Release

Atu027 Update: Progression into Phase IIa
Silence announced that the Data Safety Monitoring Board has unanimously recommended the continuation of the Phase Ib/IIa study of Atu027. The Phase Ib/IIa study uses Atu027 in combination with gemcitabine, a chemotherapy agent in patients with advanced pancreatic cancer qualifying for first-line-treatment. [Silence Therapeutics plc] Press Release

BioRestorative Therapies Expands Its ThermoStem® Program to 150 Stem Cell Lines
BioRestorative Therapies, Inc. announced that it has expanded its ThermoStem® Program by collecting 150 samples of human brown adipose tissue. [BioRestorative Therapies, Inc.] Press Release

Sernova Receives Patent Allowance for Therapeutic Cell Immune Protection Technology
Sernova Corp. announced that the Australian Patent Office has issued a Notice of Patent Allowance to Sernova for a patent entitled “Adult sertoli cells and uses thereof”. Importantly, sertoli cells provide an immune privileged environment to protect donor therapeutic cells from immune system attack. [Sernova Corp.] Press Release

OncoSec Medical Announces Completion of Enrollment of Phase II Metastatic Melanoma Trial
OncoSec Medical Inc. announced the completion of patient enrollment in a Phase II clinical trial of ImmunoPulse for metastatic melanoma patients. [OncoSec Medical Inc.] Press Release

SCRM Building Wins New Laboratory Building of the Year Award
The Scottish Centre for Regenerative Medicine (SCRM) building has been named as best new laboratory building in the S-Lab Awards, which recognize laboratories for sustainability, safety and success. [MRC Centre for Regenerative Medicine] Press Release

Ontario Institute for Cancer Research (OICR) Appoints Robert N. Klein to Board of Directors
Dr. Calvin Stiller, Chair of the Board of Directors of the OICR announced the appointment of Robert N. Klein to OICR’s Board of Directors. Mr. Klein initiated California’s Proposition 71, a $3 billion ballot initiative that established the California Institute for Regenerative Medicine. [Ontario Institute for Cancer Research]
Press Release


Notice to Extend PAR-10-234 “Bioengineering Research Partnerships (BRP) (R01)” (NOT-EB-13-002)

Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2013-14937)

National Institute of Biomedical Imaging and Bioengineering; Notice of Closed Meetings (FR Doc No: 2013-14819)

National Institute of Allergy and Infectious Diseases; Notice of Closed Meeting (FR Doc No: 2013-14818)

Government-Owned Inventions; Availability for Licensing (FR Doc No: 2013-14821)


Summary Basis for Regulatory Action, May 30, 2013 – ALLOCORD

Instructions for Completing the Electronic Human Cell and Tissue Establishment Registration Form


NEW Bioprocess Miniaturization: Development and Optimization
November 26, 2013
London, United Kingdom

NEW Recovery and Purification of Biosynthetic Products
November 27, 2013
London, United Kingdom

NEW New Procedures and Innovations for Analytical Development of Biopharmaceuticals
November 28, 2013
London, United Kingdom

NEW Applications for Synthetic Biology in Industrial Biotechnology
November 29, 2013
London, United Kingdom

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

NEW Cardiac Stem Cell Faculty Member – Cell Therapy for Myocardial Repair (University of Louisville, Institute of Molecular Cardiology)

NEW Postdoctoral Fellow/Research Scientist – Tissue Engineering (Institute of Bioengineering and Nanotechnology)

Postdoctoral Fellowship – Organic Synthesis and Medicinal Chemistry (Masaryk University)

Research Scientist/Senior Research Officer – Monoclonal Antibodies (Bioprocessing Technology Institute/A*STAR)

Postdoctoral Fellow/Research Scientist – Tissue Engineering (Institute of Bioengineering and Nanotechnology)

Director of Cell Processing Facility (S L Collins Associates, Inc.)

Postdoctoral Position – Developmental Neurobiology (North Carolina College of Veterinary Medicine)

Senior Postdoctoral Research Position – Molecular Cardiovascular and Renal Research (University of Oklahoma Health Sciences Center)

Postdoctoral Researcher – Hematopoietic Stem Cells (Albert Einstein College of Medicine)

Postdoctoral Position – Bone and Cartilage Tissue Engineering (University of California, Los Angeles)

Postdoctoral Fellow – Stem Cell and Cancer Biology (Johns Hopkins University School of Medicine)

Postdoctoral Research Fellow – Immunology (Beth Israel Deaconess Medical Center, Harvard Medical School)

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