Volume 15.47 | Dec 1

Newsletter Issue
Issue 15.47 December 1, 2014
Cell Therapy News - Your Industry in an Instant
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Publications | Reviews | Science | Policy | Business | NIH | CBER | Regulatory | Events | Jobs
Protein Predicts Response to New Immunotherapy Drug
The presence of an immune-suppressing protein in non-cancerous immune cells may predict how patients with different types of cancer respond to treatment, a multi-center Phase I study using an investigational immune therapy drug has found. [Press release from Yale University discussing online prepublication in Nature]
Press Release | Abstract
Free Wallchart: Small Molecules, Big Impact in PSC Research
PUBLICATIONS (Ranked by impact factor of the journal)
Human COL7A1-Corrected Induced Pluripotent Stem Cells for the Treatment of Recessive Dystrophic Epidermolysis Bullosa
Researchers generated patient-derived COL7A1-corrected epithelial keratinocyte sheets for autologous grafting. Induced pluripotent stem cell-derived keratinocytes were produced with minimal heterogeneity, and these cells secreted wild-type type VII collagen, resulting in stratified epidermis in vitro in organotypic cultures and in vivo in mice. [Sci Transl Med] Abstract | Press Release

Silk-Based Resorbable Electronic Devices for Remotely Controlled Therapy and In Vivo Infection Abatement
Researchers present a fully degradable, remotely controlled, implantable therapeutic device operating in vivo to counter a Staphylococcus aureus infection that disappears once its function is complete. [Proc Natl Acad Sci USA] Abstract

Bystander Immunotherapy as a Strategy to Control Allergen-Driven Airway Inflammation
Allergen-specific immunotherapy is unsuitable for plurisensitized patients, and highly immunogenic allergens cannot be used. To overcome these hurdles, investigators sought to induce regulatory CD4+ T cells specific to an exogenous antigen that could be later activated as needed in vivo to control allergic responses. [Mucosal Immunol] Abstract

Derivation and High Engraftment of Patient-Specific Cardiomyocyte-Sheet Using Induced Pluripotent Stem Cells Generated from Adult Cardiac Fibroblast
Researchers show that human cardiac induced pluripotent stem cells (hciPSCs) can be generated from cardiac fibroblasts and subsequently differentiated into exceptionally pure sheets of cardiomyocytes (CMs). When using the fibrin gel enhanced delivery of hciPSC-CM sheets at the site of injury in infarcted mouse hearts, the engraftment rate was 31.91% at day 28 post transplantation. [Circ Heart Fail] Abstract

Serial In Vivo Imaging Using a Fluorescence Probe Allows Identification of Tumor Early Response to Cetuximab Immunotherapy
Scientists investigated whether near-infrared fluorescence imaging of tumor vascular endothelial growth factor expression could be a biomarker for tumor early response to cetuximab therapy in preclinical wild-type and mutant tumor models of the KRAS gene. [Mol Pharm] Abstract | Graphical Abstract

AAV-Mediated and Pharmacological Induction of Hsp70 Expression Stimulates Survival of Retinal Ganglion Cells following Axonal Injury
Scientists show that a single intravitreal injection of 17-AAG (17-N-allylamino-17-demethoxygeldanamycin) results in an increased survival of optic nerve crush-injured retinal ganglion cells by approximately 49% compared with the vehicle-treated animals. [Gene Ther] Abstract

Employing a Gain-of-Function Factor IX Variant R338L to Advance the Efficacy and Safety of Hemophilia B Human Gene Therapy: Preclinical Evaluation Supporting an Ongoing AAV Clinical Trial
Investigators used a scalable manufacturing process to produce GMP grade AAV8 expressing the factor IX (FIX)R338L gain-of-function variant with minimal empty capsid and performed comprehensive dose/response, bio-distribution and safety evaluations in clinically-relevant hemophilia models. [Hum Gene Ther] Abstract

RNA Interference Targeting Hypoxia-Inducible Factor 1α via a Novel Multifunctional Surfactant Attenuates Glioma Growth in an Intracranial Mouse Model
Using an orthotopic mouse model with U87-LucNeo cells, the authors used RNA interference to knock down hypoxia-inducible factor 1α in vivo. Stereotactic injection was used to deliver small interfering RNA (siRNA) locally through a guide-screw system, and delivery/uptake was verified by imaging of fluorescently labeled siRNA. [J Neurosurg] Abstract

Precise Correction of the Dystrophin Gene in Duchenne Muscular Dystrophy Patient Induced Pluripotent Stem Cells by TALEN and CRISPR-Cas9
To restore dystrophin protein, scientists performed three correction methods (exon skipping, frameshifting, and exon knockin) in Duchenne muscular dystrophy-patient-derived induced pluripotent stem cells, and found that exon knockin was the most effective approach. [Stem Cell Rep] Full Article | Graphical Abstract | Press Release

NEW MesenPure™ Highly Enriched Mouse MSC Cultures Without the Wait FREE Sample Request
The New Frontier of Genome Engineering with CRISPR-Cas9
The authors review the history of CRISPR (clustered regularly interspaced palindromic repeat) biology from its initial discovery through the elucidation of the CRISPR-Cas9 enzyme mechanism, which has set the stage for remarkable developments using this technology to modify, regulate, or mark genomic loci in a wide variety of cells and organisms from all three domains of life. [Science] Abstract | Press Release

Aerosol Gene Delivery Using Viral Vectors and Cationic Carriers for In Vivo Lung Cancer Therapy
Aerosol delivery is a noninvasive method for gene delivery to the lungs, although efficient and biocompatible vectors need to be developed for lung cancer therapy. This review summarizes recent advances in airway gene delivery for lung cancer treatment in animal models using viral vectors or cationic polymers. [Expert Opin Drug Deliv] Abstract

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

A New System for High-Throughput Cell Isolation Directly from Whole Blood
In Landmark Study of Cell Therapy for Heart Attack, More Cells Make a Difference
Patients who receive more cells get significant benefits. Doctors treated heart attack patients with their own bone marrow cells, selected for their healing potential and then reinjected into the heart, in an effort to improve the heart’s recovery. [Press release from Emory University discussing research presented at the American Heart Association Scientific Sessions 2014, Chicago] Press Release

High-Dose Interleukin-2 Effective in mRCC Pre-Treated with VEGF-Targeted Therapies
High-dose interleukin-2 can be effective in selected metastatic renal cell cancer (mRCC) patients pre-treated with vascular endothelial growth factor (VEGF)-targeted agents. [Press release from The European Society for Medical Oncology (ESMO) discussing research presented at the ESMO Symposium on Immuno-Oncology, Geneva]
Press Release

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Germany Poised to Say Yes to €1.1 Million a Patient Gene Therapy Drug
The western world’s first gene therapy drug is set to go on sale in Germany, with a price tag that could amount to an £870,000 cost to treat a single patient. Glybera, a treatment for the rare genetic condition lipoprotein lipase deficiency, which clogs the blood with fat, has been developed by Dutch biotech firm UniQure and Italian marketing partner Chiesi. [Guardian News and Media Limited] Editorial

E.U. Commission Wants to Divert Horizon 2020 Money into New Investment Fund
The new boss of the European Commission has sparked criticism with plans to raid the European Union’s science budget to fodder a new investment fund aimed at boosting Europe’s sluggish economy and creating new jobs. [ScienceInsdier] Editorial
OncoSec Medical and Plexxikon Collaborate to Explore Combination Immunotherapy in Preclinical Tumor Studies
OncoSec Medical Inc. announced a preclinical collaboration with Plexxikon Inc. to test the combination of Plexxikon’s selective CSF-1R inhibitor with OncoSec’s Immunopulse pIL-12. [OncoSec Medical Inc.]
Press Release

UC San Francisco and OncoSec Medical Collaborate to Evaluate Investigational Combination of ImmunoPulse and Anti-PD-1 Treatment
OncoSec Medical Inc. has entered a clinical collaboration with the University of California, San Francisco to evaluate the safety, tolerability and efficacy of the combination of KEYTRUDA® (pembrolizumab), Merck’s anti-PD-1 therapy, and OncoSec’s ImmunoPulse (intratumoral IL-12) in metastatic melanoma. [OncoSec Medical Inc.]
Press Release

Amgen and AstraZeneca Announce Positive Results from Third and Final Pivotal Phase III Study of Brodalumab in Patients with Moderate-to-Severe Plaque Psoriasis
Amgen and AstraZeneca announced that AMAGINE-2TM, a pivotal, multi-arm Phase III trial evaluating two doses of brodalumab in more than 1,800 patients with moderate-to-severe plaque psoriasis, met its primary endpoints when compared with both Stelara® (ustekinumab) and placebo at week 12. [Amgen Inc.] Press Release

Regulatory Update on Transaction with Novartis
The US Federal Trade Commission has voted to approve GlaxoSmithKline plc. (GSK)’s proposed acquisition of Novartis’s vaccines business (excluding influenza vaccines) and the proposed creation of a consumer healthcare joint venture between GSK and Novartis. [GlaxoSmithKline plc.] Press Release

Unum Therapeutics Announces Start of First Phase I Clinical Trial of Cellular Immunotherapy Targeting CD20+ Chronic Lymphocytic Leukemia and Non-Hodgkin Lymphoma
Unum Therapeutics announced that recruitment has begun in the first clinical trial of the ATTCK20 therapy. The Phase I study will examine the feasibility, safety and potential efficacy of infusing the ATTCK20 combination therapy in patients with B-cell malignancies and persistent disease following standard therapy. [Unum Therapeutics] Press Release

Pluristem Granted Patent in Singapore for its 3D Cell Expansion Technology
Pluristem Therapeutics Inc. announced it has been granted a patent in Singapore titled “Methods for Cell Expansion and Uses of Cells and Conditioned Media Produced Thereby for Therapy.” [Pluristem Therapeutics Inc.] Press Release

New IMBRUVICA® (Ibrutinib) Application Accepted by European Medicines Agency for Waldenstrom’s Macroglobulinemia
Pharmacyclics, Inc. announced the acceptance of a Type II variation application for IMBRUVICA® (ibrutinib) by the European Medicines Agency. This submission represents a potential label expansion for IMBRUVICA in the European Union (EU) for the treatment of adult patients with Waldenstrom’s macroglobulinemia, a rare type of B-cell lymphoma for which treatment options are limited in the EU. [Pharmacyclics, Inc.] Press Release

CPRIT Awards UTSW Faculty $22.5 Million for Recruitment and Research in Liver Cancer, Leukemia, and Immunotherapy
UT Southwestern Medical Center faculty was awarded seven grants totaling more than $22 million from the Cancer Prevention and Research Institute of Texas (CPRIT) for investigations into leukemia, liver cancer, and immunotherapy, as well as to recruit new faculty. [UT Southwestern Medical Center] Press Release

City of Hope Researchers Unlock the Potential of the Immune System to Fight Cancer
City of Hope is using chimeric antigen receptor, or CAR-T cell therapy, in clinical trials for leukemia, lymphoma and other hematologic malignancies. [City of Hope] Press Release
National Institute of Neurological Disorders and Stroke; Notice of Closed Meeting (FR Doc. No: 2014-27666)
Approval History, Letters, Reviews, and Related Documents – TRUMENBA
Food and Drug Administration (United States)

The Drug Supply Chain Security Act Standards for the Interoperable Exchange of Information for Tracing of Certain Human, Finished, Prescription Drugs: How To Exchange Product Tracing Information; Availability (FR Doc. No: 2014-28085)

New Clinical Trials Demographic Data; Availability for Comment (FR Doc. No: 2014-27732)
NEW Gordon Research Conference: Stem Cells & Cancer
February 15-20, 2015
Ventura, United States

NEW 4th Annual Cell Line Engineering & Development Asia
May 19-22, 2015
Shanghai, China

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Postdoctoral Researcher – Cell Based Therapy for ALS (University of Warmia and Mazury)

Mesenchymal Stem Cell (MSC) Laboratory Technician (California Cryobank)

Clinical Laboratory Scientist – Umbilical Cord Blood Processing (California Cryobank)

Postdoctoral Researcher – Chromatin and Epigenetic Regulation (Van Andel Research Institute)

Animal Surgery – Regenerative Medicine for Cardiac and Skeletal Muscle Disease (Duke University)

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

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)

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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