Volume 14.11 | Mar 25

Issue 14.11 March 25, 2013
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T-Cell Therapy Eradicates an Aggressive Leukemia in Two Children
Two children with an aggressive form of childhood leukemia had a complete remission of their disease, showing no evidence of cancer cells in their bodies, after treatment with a novel cell therapy that reprogrammed their immune cells to rapidly multiply and destroy leukemia cells. [Press release from Penn Medicine discussing online prepublication in The New England Journal of Medicine] Press Release | Abstract

Gain Hands-On Training. New Course: Differentiation of hPSCs to Neural Progenitor Cells

PUBLICATIONS (Ranked by impact factor of the journal)

Coagulation Factor X Shields Adenovirus Type 5 from Attack by Natural Antibodies and Complement
Researchers demonstrated that the ability of coagulation factor X (FX) to enhance liver transduction by adenovirus type 5 (Ad5) vectors is due to an unexpected ability of FX to protect Ad5 from attack by the classical complement pathway. [Nat Med] Abstract

Amniotic Fluid Stem Cells Improve Survival and Enhance Repair of Damaged Intestine in Necrotizing Enterocolitis via a COX-2 Dependent Mechanism
Investigators tested the hypothesis that amniotic fluid stem cells exerted a beneficial effect in a neonatal rat model of necrotizing enterocolitis. [Gut] Abstract | Press Release

An Epigenetic Component of Hematopoietic Stem Cell Aging Amenable to Reprogramming into a Young State
The authors applied induced pluripotent stem (iPS) cell reprogramming of aged hematopoietic progenitors and allowed the resulting aged-derived iPS cells to reform hematopoiesis via blastocyst complementation. Next, they functionally characterized iPS-derived hematopoietic stem cells (HSCs) in primary chimeras and following the transplantation of ‘re-differentiated’ HSCs into new hosts; the gold standard to assess HSC function. [Blood] Abstract | Press Release

In Vitro and In Vivo Delivery of Genes and Proteins Using the Bacteriophage T4 DNA Packaging Machine
The bacteriophage T4 DNA packaging machine consists of a molecular motor assembled at the portal vertex of an icosahedral head. The ATP-powered motor packages the 56-µm-long, 170-kb viral genome into 120 nm × 86 nm head to near crystalline density. Researchers engineered this machine to deliver genes and proteins into mammalian cells. [Proc Natl Acad Sci USA] Abstract

Generation of Induced Neurons via Direct Conversion In Vivo
Scientists showed that transplanted human fibroblasts and human astrocytes, which are engineered to express inducible forms of neural reprogramming genes, convert into neurons when reprogramming genes are activated after transplantation. [Proc Natl Acad Sci USA] Abstract

Intracellular Aggregation of Multimodal Silica Nanoparticles for Ultrasound-Guided Stem Cell Implantation
Researchers described a multimodal, silica-based nanoparticle that can be used for cell sorting (fluorescence), real-time guided cell implantation ultrasound, and high-resolution, long-term monitoring by magnetic resonance imaging. [Sci Transl Med] Abstract | Press Release

CD19-Targeted T Cells Rapidly Induce Molecular Remissions in Adults with Chemotherapy-Refractory Acute Lymphoblastic Leukemia
Scientists treated five relapsed B cell acute lymphoblastic leukemia subjects with autologous T cells expressing a CD19-specific CD28/CD3ζ second-generation dual-signaling chimeric antigen receptor termed 19-28z. [Sci Transl Med] Abstract | Press Release

Nanoparticle-Mediated Gene Silencing Confers Radioprotection to Salivary Glands In Vivo
Investigators introduced a preclinical mouse model for small-interfering RNA-based gene silencing to provide protection of salivary glands from radiation-induced apoptosis. [Mol Ther] Abstract

Adenoviral Gene Transfer Corrects the Ion Transport Defect in the Sinus Epithelia of a Porcine CF Model
Scientists investigated the ion transport defect in the cystic fibrosis (CF) sinus and found that CF porcine sinus epithelia lack cyclic AMP-stimulated anion transport. The authors asked whether they could restore CF transmembrane conductance regulator gene current in the porcine CF sinus epithelia by gene transfer. [Mol Ther] Abstract

Multifunctional T-Cell Analyses to Study Response and Progression in Adoptive Cell Transfer Immunotherapy
Adoptive cell transfer (ACT) of genetically engineered T cells expressing cancer-specific T-cell receptors (TCR) is a promising cancer treatment. Researchers investigated the in vivo functional activity and dynamics of the transferred cells by analyzing samples from three representative patients with melanoma enrolled in a clinical trial of ACT with TCR transgenic T cells targeted against the melanosomal antigen MART-1. [Cancer Discov]
Abstract | Press Release

Webinar: Groundbreaking Applications for Feeder-Free hESC & hiPSC Culture. Watch Now.


The Rise of Cell Therapy Trials for Stroke: Review of Published and Registered Studies
Different clinical trials for stroke, the majority of them small, non-randomized and uncontrolled, have been reported and their results indicate that cell therapy seems safe and feasible in these conditions. In the last two years, the number of published and registered trials has dramatically increased. The authors review the main findings available in the field, with emphasis on clinical results. [Stem Cell Dev] Abstract | Full Article

Interaction between Natural Killer Cells and Regulatory T Cells: Perspectives for Immunotherapy
This review discusses the recent information on the interaction between regulatory T (Treg) cells and natural killer cells under healthy and pathologic conditions, to describe the specific conditions in which this interaction takes place, the effect of Treg cells on hematopoietic stem cell differentiation and the consequences of this interaction on the optimization of immunotherapeutic protocols. [Cell Mol Immunol] Abstract

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

StemSpan ACF: The WORLD'S FIRST Plasma Component-Free Hematopoietic Expansion Medium. Come learn more.


OPTiM Trial Results Are First Positive Phase III Oncological Virus Cancer Immunotherapy Study in Melanoma
Results from the Oncovex Pivotal Trial in Melanoma (OPTiM) have been announced, finding that a genetically modified version of herpes simplex virus type 1, designated talimogene laherparepvec, shrank tumors of the deadly skin cancer melanoma in patients who were in the late stages of the disease. [Society for Immunotherapy of Cancer] Press Release

A Revolutionary Treatment for Burns & Bone Fractures
A team of multi-disciplinary researchers from Koç University developed artificial skin and bone tissue for the first time in Turkey. This study will further help in treatment of burns and bone fractures. The laboratory phase of the study has been completed and results are proved to be promising. Next steps are tests on animals and clinical trials. [PR Newswire Association LLC] Press Release

11th Annual International Cord Blood Symposium | San Francisco


Congress Completes Work on 2013 Spending Bill
U.S. research agencies finally know what they have to spend for the rest of the 2013 fiscal year after Congress completed work on a bill to fund the government through 30 September. [House of Representatives, United States] Press Release

Canadian Budget Targets Industrial Applications
Canadian scientists are feeling a decided chill in the air after federal Finance Minister Jim Flaherty unveiled the government’s fiscal blueprint for 2013 to 2014. The new budget promises stiffer competition for a smaller pool of research grants. What little new money is made available will again be funneled into targeted “industry-academic” partnerships. [Finance Minister, Canada] Press Release

New French Science Law Falls Short of Expectations
Months of consultation with the French scientific community culminated when the science minister presented a draft bill for a new higher education and research law that France’s Parliament will soon consider. [French Parliament, France] Press Release

U.K. Agency Cautiously Endorses Mitochondria Replacement
There is broad public support in the United Kingdom for allowing a new type of IVF treatment that could prevent mitochondrial diseases, the country’s Human Fertilization and Embryology Authority (HFEA) announced. The techniques would introduce new DNA into an embryo, and so it has raised thorny ethical questions. At the same time, the authority advised the government that several safeguards should be included in any proposals for new regulations that would permit clinics to perform the technique. [Human Fertilization and Embryology Authority, United Kingdom] Press Release

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Stem Cell Agency Banks on $32 Million New Approach to Advance Research
When you need money you go to the bank. But when researchers need high quality stem cells where do they go to get those? Soon they’ll be able to go to a stem cell bank set up by California’s stem cell agency, the California Institute for Regenerative Medicine (CIRM). The governing Board of the agency voted to approve nine applications to create the cells that will go in that bank and to run it. [California Institute for Regenerative Medicine] Press Release

bluebird bio Announces Global Strategic Collaboration with Celgene to Advance Gene Therapy in Oncology
bluebird bio announced the formation of a broad, global strategic collaboration with Celgene Corporation to discover, develop and commercialize novel disease-altering gene therapies in oncology. The collaboration will focus on applying gene therapy technology to genetically modify a patient’s own T-cells, known as chimeric antigen receptor T-cells, to target and destroy cancer cells. [bluebird bio] Press Release

Sanofi and Transgene Announce Innovative Long-Term Collaboration for the Production of Immunotherapy Treatments
Sanofi and Transgene SA announced a collaboration agreement for the creation of a new state-of-the-art industrial platform dedicated to the production of immunotherapy products including Transgene’s therapeutic products. [Sanofi SA] Press Release

Benitec Selects the Duke Clinical Research Unit as a Site for Hepatitis C Phase I/II Clinical Trial
RNAi-based therapeutics company Benitec Biopharma Limited announced the selection of the Duke Clinical Research Unit, the early phase unit of the Duke Clinical Research Institute Durham, North Carolina, USA as a site for its upcoming phase I/II first-in-man trial for TT-034 in Hepatitis C. TT-034 is being developed as a potential “one-shot-cure” for Hepatitis C. [Benitec Biopharma Limited] Press Release

Bone Therapeutics Awarded Manufacturing Authorization and European Approval to Produce Allogeneic Bone Cell Therapy Products
BONE THERAPEUTICS announced that it has been awarded both ‘Tissue Establishment and ‘GMP’ Accreditation for the manufacturing of its ALLOB® allogeneic bone cell therapy product, being initially developed for the treatment of impaired fractures. [Bone Therapeutics SA] Press Release

European Patent Office to Grant Patent for Macrocure’s CureXcell®
Macrocure announced that the European Patent Office intends to grant a European patent covering Marcocure’s flagship product, CureXcell®. The patent will be officially granted under an application titled “Activate Leukocyte Composition” and will provide patent coverage for CureXcell® in Europe through March 2030. [MacroCure]
Press Release

St. Jude Children’s Research Hospital Announces Issuance of U.S. Patent for Genetically Modified Human Immune Cells for Cancer Therapy
The United States Patent and Trademark Office awarded St. Jude Children’s Research Hospital U.S. patent number 8,399,645 for St. Jude’s invention of compositions for genetically modifying human immune cells so they can destroy some of the most common forms of cancer in children and adults. [St. Jude Children’s Research Hospital] Press Release

NW Bio Receives U.S. Patent on Broad Processes for Producing More Potent Dendritic Cells
Northwest Biotherapeutics (NW Bio) announced that it has been issued a key U.S. patent (#8389278) covering novel processes for manufacturing human dendritic cells. The cells produced through these processes are much more potent in their capacity to activate the immune system than those produced by the standard methods used throughout academia and industry today to produce dendritic cells. [Northwest Biotherapeutics, Inc.]
Press Release

Regen BioPharma Receives IND # from FDA for HemaXellerate
Regen BioPharma, Inc. announced issuance of IND number 15376 for its proposed Phase I/II clinical trial evaluating use of its stem cell product, HemaXellerate, for treatment of aplastic anemia. [Marketwire]
Press Release

Dicerna Announces Patent Issuance for Dicer Substrate siRNA Directed at the KRAS Gene, a Key Cancer Regulator
Dicerna Pharmaceuticals, Inc. announced that the United States Patent and Trademark Office has issued patent claims specific to double-stranded RNAs directed against the important oncology gene target KRAS. [Dicerna Pharmaceuticals, Inc.] Press Release


Notice of NIAMS’ Participation in PAR-13-137 “Bioengineering Research Grants (BRG)(R01)”(NOT-AR-13-016)

National Institute of Biomedical Imaging and Bioengineering; Meeting (FR Doc No: 2013-06686)

Office of the Director, National Institutes of Health; Notice of Closed Meeting (FR Doc No: 2013-06249)

National Heart, Lung, and Blood Institute; Notice of Closed Meeting (FR Doc No: 2013-06241)


Food and Drug Administration (United States)

Risk Communications Advisory Committee; Notice of Meeting (FR Doc No: 2013-06415)

Public Hearing Before a Public Advisory Committee; Technical Amendments (FR Doc No: 2013-06354)


NEW The Jackson Laboratory: Lupus – Explore Our Innovative Suite of Mouse Research Strains, Services and Expertise
April 4, 1:00PM (ET in US)

NEW Clinical Trials: Moving Stem Cell Based Therapies to the Clinic
April 15, 10:00AM (PT in US)

NEW Advanced Summer School – Interrogations at the Biointerface: Inflammation/Repair Interface
June 25-28, 2013
Porto, Portugal

NEW Gordon Research Seminar – Biomaterials & Tissue Engineering
July 27-28, 2013
Holderness, United States

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


Faculty Position – Stem Cell, Vascular and Developmental Biology in Ophthalmology (Zhongshan Ophthalmic Center, Sun Yat-Sen University)

Postdoctoral Position – Adult Neurogenesis and Neural Survival Signaling (University of Kentucky)

Cord Blood Laboratory Manager (Puget Sound Blood Center)

Postdoctoral Fellow – Regenerative Medicine (The University of Texas Health Science Center at Houston)

Senior Scientist – Stem Cell Therapy: Metabolic Diseases (Paramount Recruitment)

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

Postdoctoral Research Fellow – Regenerative Medicine (Loma Linda University)

Postdoctoral Research Associate (Washington State University)

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