Volume 15.43 | Nov 3

Issue 15.43 November 3, 2014
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Publications | Reviews | Science | Policy | Business | NIH | Events | Jobs
New Way of Genome Editing Could Cure Hemophilia in Mice
A technique developed by researchers could provide a safer, longer-lasting method of replacing faulty, disease-causing genes with working copies. [Press release from Stanford Medicine discussing online prepublication in Nature] Press Release | Abstract
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PUBLICATIONS (Ranked by impact factor of the journal)
Adiposity Induces Lethal Cytokine Storm after Systemic Administration of Stimulatory Immunotherapy Regimens in Aged Mice
Researchers determined the effects of adiposity on systemic immunotherapy (IT) tolerance in aged compared with young obese mice. Both young ob/ob– and diet-induced obese-mice generated proinflammatory cytokine levels and organ pathologies are comparable to those in aged ad libitum mice after IT, culminating in lethality. [J Exp Med] Abstract | Press Release

Insulin-Like Growth Factor-1 Stimulates Regulatory T Cells and Suppresses Autoimmune Disease
Scientists show that recombinant human insulin-like growth factor-1 stimulates proliferation of both human and mouse regulatory T cells in vitro and when delivered systemically via continuous minipump, it halts autoimmune disease progression in mouse models of type 1 diabetes and multiple sclerosis in vivo. [EMBO Mol Med]
Full Article | Press Release

Structure and Cancer Immunotherapy of the B7 Family Member B7x
Researchers developed an in vivo system to screen therapeutic monoclonal antibodies against B7x and found that the clone 1H3 significantly inhibited growth of B7x-expressing tumors in vivo via multiple mechanisms. Furthermore, the surviving mice given 1H3 treatment were resistant to tumor rechallenge. [Cell Rep]
Full Article | Graphical Abstract

Recurrence of Melanoma following T Cell Treatment: Continued Antigen Expression in a Tumor that Evades T Cell Recruitment
Researchers treated mouse melanomas with TCR-engineered T cells directed against a human peptide-MHC antigen in immune-competent mice. T cells resulted in significant tumor regression, which was followed by relapse in about 80-90% of mice. [Mol Ther] Abstract

Improving Single Injection CSF Delivery of AAV9-Mediated Gene Therapy for SMA – A Dose Response Study in Mice and Nonhuman Primates
Scientists evaluated the dosing and efficacy of self-complementary adeno-associated virus 9 (AAV9) carrying the human survival of motor neuron cDNA delivered directly to the cerebral spinal fluid (CSF) via single injection. They found widespread transgene expression throughout the spinal cord in mice and nonhuman primates when using a ten times lower dose compared to the intravenous injection application. [Mol Ther] Abstract

Tumor-Specific Suicide Gene Therapy for Hepatocellular Carcinoma by Transcriptionally Targeted Retroviral Replicating Vector
To design a tumor-specific retroviral replicating vector (RRV), scientists replaced the U3 region of the RRV ACE-GFP with a regulatory sequence consisting of the hepatitis B virus enhancer II (EII) and human α-fetoprotein (AFP) core promoter to produce ACE-GFP-EIIAFP, a hepatocellular carcinoma (HCC)-targeting RRV. In vivo, ACE-CD-EIIAFP-mediated suicide gene therapy efficiently suppressed HCC tumor growth and no detectable RRV signal was observed in extratumoral tissues. [Gene Ther] Abstract

Distribution of Mesenchymal Stem Cells and Effects on Neuronal Survival and Axon Regeneration after Optic Nerve Crush and Cell Therapy
Researchers investigated the therapeutic potential of mesenchymal stem cells (MSC) injected into the vitreous body in a model of optic nerve injury. Adult Lister Hooded rats underwent unilateral optic nerve crush followed by injection of MSC or the vehicle into the vitreous body. [PLoS One] Full Article

In Vivo Tissue-Engineered Allogenic Trachea Transplantation in Rabbits: A Preliminary Report
Scientists investigated in vivo tissue-engineered trachea regeneration from a decellularized cadaveric trachea matrix with seeded adult adipose tissue-derived mesenchymal stem cells and investigated the integration of the matrix into the recipient tracheal side. [Stem Cell Rev] Abstract

EBAG9 Modulates Host Immune Defense against Tumor Formation and Metastasis by Regulating Cytotoxic Activity of T Lymphocytes
MB-49 mouse bladder cancer cells were subcutaneously implanted into estrogen receptor-binding fragment-associated antigen 9-knockout (Ebag9KO) and control mice. Scientists found that tumor formation and metastasis to the lung by MB-49 cells were substantially reduced in Ebag9KO mice compared with control mice. [Oncogenesis] Full Article

Free Wallchart: Small Molecules, Big Impact in PSC Research
Epstein-Barr Virus and Multiple Sclerosis: Potential Opportunities for Immunotherapy
The rapidly accumulating evidence for a pathogenic role of Epstein-Barr virus (EBV) in multiple sclerosis (MS) provides ground for optimism that it might be possible to prevent and cure MS by effectively controlling EBV infection through vaccination, antiviral drugs or treatment with EBV-specific cytotoxic CD8+ T cells. [Clin Transl Immunol] Full Article

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

View Data: UM729, a Novel Small Molecule for Ex Vivo Expansion of Human hSCs
Phase II Objective Response Rate and Survival Data for Opdivo (Nivolumab) in Heavily Pre-Treated Advanced Squamous Cell Non-Small Cell Lung Cancer
Bristol-Myers Squibb Company announced results from CheckMate-063, a Phase II single-arm, open-label study of Opdivo, an investigational PD-1 immune checkpoint inhibitor, administered as a single agent in patients with advanced squamous cell non-small cell lung cancer who have progressed after at least two prior systemic treatments with 65% receiving three or more prior therapies. [Press release from Bristol-Myers Squibb Company discussing research presented at the 2014 Chicago Multidisciplinary Symposium on Thoracic Oncology, Chicago] Press Release

Celsion Presents Preclinical Data for its TheraSilence™ Platform
Celsion Corporation announced the presentation of data related to TheraSilence™, the company’s lung-specific RNA delivery technology. [Press release from Celsion Corporation discussing research presented at the miRNA World Conference Workshop on miRNA Delivery, Boston] Press Release

Lycera Announces Presentation of Data on Anti-Tumor Activity of Novel Synthetic RORγ Agonists
Lycera Corp. announced that data on the anti-tumor activity of novel synthetic RORγ agonist product candidates will be presented. [Press release from Lycera Corp. discussing research to be presented at the annual meeting of the Society for Immunotherapy of Cancer (SITC), National Harbor] Press Release

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European Union’s Pairing Plan for Science Proves Popular
A program designed to boost investments in the scientific infrastructure of Europe’s lagging regions by pairing them with elite institutes elsewhere on the continent has proved unexpectedly popular. The European Commission has received 169 scientific business plans for the scheme, dubbed Teaming, and may be able to advance only 16% of the proposals to the next round of the competition. [ScienceInsider] Editorial

China Ramps Up Efforts to Combat Ebola
In the unfolding Ebola crisis, much attention has focused on the relief efforts of Western countries and the nonprofit Doctors Without Borders. Out of the limelight, China is mounting one of its largest aid operations ever, driven in part by increasing political and business interests in Africa. [ScienceInsider] Editorial

Ministers Promise Basket of Gifts for German Science
Germany’s universities and large science organizations have been promised a continuing windfall to help them to cope with rising student enrolment and increasing competition for grant money. Political leaders have pledged €25.3 billion over the next six years to support special programs for research and higher education. [Nature News] Editorial
The Leukemia & Lymphoma Society Awards $15.6 Million to Help Advance Research from Lab to Clinic
The Leukemia & Lymphoma Society is awarding $15.6 million in academic grants to 26 scientists who have proposed innovative laboratory research that shows promise in leading to new treatments for blood cancer patients. [The Leukemia & Lymphoma Society] Press Release

UT Arlington Researcher Uses Microscaffolding Injections to Mend Cartilage, Prevent Osteoarthritis
A UT Arlington bioengineering professor has received a $1.04 million grant from the U.S. Army that aims to regenerate cartilage tissue and reduce osteoarthritis using a patient’s own stem cells, spurred through the injection of microscaffolding made of biodegradable polymers. [The University of Texas at Arlington] Press Release

OncoSec Medical Receives Phase I SBIR Funding for Development of Novel Electroporation Technologies
OncoSec Medical Inc., a company developing DNA-based intratumoral cancer immunotherapies, received a Phase I Small Business Innovation Research (SBIR) grant from the National Institute of Health. The grant will fund collaborative research between OncoSec and Old Dominion University to evaluate and develop novel electroporation technologies. [OncoSec Medical Inc. (Business Wire)] Press Release

Merck Receives FDA Breakthrough Therapy Designation for KEYTRUDA® (Pembrolizumab) in Advanced Non-Small Cell Lung Cancer
Merck announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to KEYTRUDA®, the company’s anti-PD-1 therapy, for the treatment of patients with epidermal growth factor receptor mutation-negative, and anaplastic lymphoma kinase rearrangement-negative non-small cell lung cancer whose disease has progressed on or following platinum-based chemotherapy. [Merck & Co., Inc.]
Press Release

City of Hope Announces New Hematologic Malignancies and Stem Cell Transplantation Institute
Cancers of the blood and immune system are considered to be among the most difficult-to-treat cancers. City of Hope is launching an institute specifically focused on researching and treating lymphoma, leukemia and myeloma, as well as other serious blood and bone marrow diseases. [City of Hope] Press Release

FDA Grants Kamada Orphan Drug Designation for the Treatment of Graft versus Host Disease
Kamada Ltd. announced that the U.S. Food and Drug Administration’s (FDA) Office of Orphan Products Development has granted orphan drug designation for Glassia®, the company’s proprietary human alpha-1 antitrypsin, to treat graft-versus-host-disease. [Kamada Ltd.] Press Release

Compugen Discloses Successful Validation Results for Novel B7/CD28-Like Cancer Immunotherapy Target Candidate
Compugen Ltd. disclosed successful experimental data for CGEN-15027, a Compugen-discovered immune checkpoint target candidate. The experimental results include its expression in the cancer microenvironment, both on cancer cells derived from lung, breast, and liver cancer patients, and on tumor infiltrating immune cells. [Compugen Ltd.] Press Release

Positive Phase II Preliminary Results of Validive® for the Prevention of Severe Oral Mucositis in Head and Neck Cancer Patients
Onxeo S.A. announced positive preliminary top-line results from its Phase II clinical trial of Validive® for prevention of severe oral mucositis. [Onxeo S.A.] Press Release

ViaCyte’s VC-01™ Investigational Stem Cell-Derived Islet Replacement Therapy Successfully Implanted into First Patient
ViaCyte, Inc. announced that the first patient in its Phase I/II study was successfully implanted with VC-01™, its embryonic stem cell-derived islet replacement product candidate being developed as a treatment for type 1 diabetes. [ViaCyte, Inc.] Press Release

HEMACORD®, the First FDA-Licensed Stem Cell Product, Wins Prix Galien USA “Best Biotechnology Product” Award
The New York Blood Center’s Milstein National Cord Blood Program announced that HEMACORD®, the first FDA-licensed hematopoietic stem cell product, has been awarded the prestigious “Best Biotechnology Product” Award by Prix Galien USA. [New York Blood Center] Press Release

Kiadis Pharma’s Lead Product ATIR™ Granted Orphan Drug Designation by EMA for the Treatment of Acute Myeloid Leukemia
Kiadis Pharma, a clinical stage biopharmaceutical company developing T-cell immunotherapy treatments for blood cancers, announced that its lead product ATIR™ has been granted orphan drug designation by the European Medicines Agency (EMA) for the treatment of acute myeloid leukemia. [Kiadis Pharma] Press Release

Stanford Launches New Online Course: Principles and Practices of Gene Therapy
A new Stanford online course, Principles and Practices of Gene Therapy, offers students a fundamental overview of gene therapy and an in-depth look at important trends, research and advances in the field. Students will gain a clear understanding of how gene therapy works, how it has developed and advanced, and how much potential it has. [Stanford Center for Professional Development (PR Newswire Association LLC)] Press Release
Center for Scientific Review; Notice of Closed Meetings (FR Doc. No: 2014-25504)

Center for Scientific Review; Notice of Closed Meetings (FR Doc. No: 2014-26020)

Government-Owned Inventions; Availability for Licensing (FR Doc. No: 2014-25874)
NEW 7th Annual World Congress of Regenerative Medicine & Stem Cells 2014
November 13-16, 2014
Haikou, China

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Postdoctoral Fellow – Developmental, Stem Cell, and/or Cancer Biology (North Carolina State University)

Postdoctoral Fellow – Collaborative Training Program in Musculoskeletal Health Research (Western University)

Postdoctoral Position – Muscle Cell Therapy (I-Stem)

Graduate Student – Pediatric Cancers (University of Calgary)

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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