Volume 14.07 | Feb 25

Issue 14.07 February 25, 2013
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How to Mend a Broken Heart: Advances in Parthenogenic Stem Cells
Investigators demonstrated that cells from a parthenogenote function as embryonic stem cells and maintain the capacity to develop into different types of tissue. Further, the authors used parthenogenic stem cells to make cardiomyocytes and engineered heart muscle that exhibited the structural and functional properties of normal myocardium. The engineered myocardium could then be used to engraft the mice that had contributed the eggs for parthenogenesis. [Press release from EurekAlert! discussing online prepublication in The Journal of Clinical Investigation] Press Release | Full Article

Free Interactive Tutorial: Explore Mammary Progenitor Cell Colonies

PUBLICATIONS (Ranked by impact factor of the journal)

Epigenomic Plasticity Enables Human Pancreatic α to β Cell Reprogramming
Using ChIP sequencing and RNA sequencing analysis, researchers determined the epigenetic and transcriptional landscape of human pancreatic α, β, and exocrine cells. Epigenomic findings suggested that α to β cell reprogramming could be promoted by manipulating the histone methylation signature of human pancreatic islets. The authors showed that treatment of cultured pancreatic islets with a histone methyltransferase inhibitor leads to colocalization of both glucagon and insulin and glucagon and insulin promoter factor 1 in human islets and colocalization of both glucagon and insulin in mouse islets. [J Clin Invest] Full Article | Press Release

Cotransplantation with Specific Populations of Spina Bifida Bone Marrow Stem/Progenitor Cells Enhances Urinary Bladder Regeneration
Investigators demonstrated the role of two specific populations of bone marrow stem/progenitor cells used in combination with a synthetic elastomeric scaffold that provides a unique and alternative means to current bladder regeneration approaches. [Proc Natl Acad Sci USA] Abstract | Press Release

An Open Label Dose Escalation Study to Evaluate the Safety of Administration of Non-Viral SDF-1 Plasmid to Treat Symptomatic Ischemic Heart Failure
JVS-100 is a DNA plasmid encoding human SDF-1. Researchers aimed to test in a Phase I open-label dose-escalation study with 12 months follow-up in subjects with ischemic cardiomyopathy to see if JVS-100 improves clinical parameters. [Circ Res] Abstract | Full Article | Press Release

Topical Administration of Allogeneic Mesenchymal Stem Cells Seeded in a Collagen Scaffold Augments Wound Healing and Increases Angiogenesis in the Diabetic Rabbit Ulcer
Researchers hypothesized that topically applied allogeneic mesenchymal stromal cells (MSCs) would improve wound healing by augmenting angiogenesis. Allogeneic non-diabetic bone-marrow derived MSCs were seeded in a collagen scaffold. The cells were applied to a full thickness cutaneous wound in the alloxan-induced diabetic rabbit ear ulcer model in a dose escalation fashion. [Diabetes] Abstract

Safety and Efficacy of an Injectable Extracellular Matrix Hydrogel for Treating Myocardial Infarction
Researchers have developed an injectable hydrogel derived from porcine myocardial extracellular matrix as a scaffold for cardiac repair after myocardial infarction. They established the safety and efficacy of this injectable biomaterial in large- and small-animal studies that simulate the clinical setting. [Sci Transl Med]
Abstract | Press Release

Treatment of Metastatic Renal Cell Carcinoma with CAIX CAR-Engineered T Cells: Clinical Evaluation and Management of On-Target Toxicity
Autologous T cells genetically modified to express a chimeric antibody receptor (CAR) against carboxy-anhydrase-IX (CAIX) were administered to 12 patients with CAIX-expressing metastatic renal cell carcinoma. This report shows that CAIX-targeting CAR T cells exerted antigen-specific effects in vivo and induced liver toxicity at the lowest dose of 0.2 × 109 T cells applied, illustrating the potency of receptor-modified T cells. [Mol Ther] Abstract

Whole Blood Interferon-γ Levels Predict the Therapeutic Effects of Adoptive T-Cell Therapy in Patients with Advanced Pancreatic Cancer
Researchers aimed to predict the therapeutic effects of adoptive T-cell therapy in patients with advanced pancreatic cancer. The findings of their study indicate that the assay of whole blood interferon-γ production offers promise for evaluating the clinical response of patients to cancer immunotherapy. [Int J Cancer] Abstract

Delivery of sTRAIL Variants by MSCs in Combination with Cytotoxic Drug Treatment Leads to p53-Independent Enhanced Antitumor Effects
Scientists engineered murine mesenchymal stem cells (MSCs) to express a secreted form of the TNF-related apoptosis-inducing ligand (TRAIL), which is a potent inducer of apoptosis in tumor cells, and tested these MSCs, termed MSC.sTRAIL, in combination with conventional chemotherapeutic drug treatment in colon cancer models. [Cell Death Dis] Full Article

High-Fidelity Tissue Engineering of Patient-Specific Auricles for Reconstruction of Pediatric Microtia and Other Auricular Deformities
Three-dimensional structures of normal pediatric ears were digitized and converted to virtual solids for mold design. Image-based synthetic reconstructions of these ears were fabricated from collagen type I hydrogels. Half were seeded with bovine auricular chondrocytes. Cellular and acellular constructs were implanted subcutaneously in the dorsa of nude rats and harvested after one and three months. [PLoS One]
Full Article | Press Release

Proliferation-Linked Apoptosis of Adoptively Transferred T Cells after IL-15 Administration in Macaques
Investigators showed in lymphoreplete macaca nemestrina, that proliferation of adoptively transferred central memory-derived CD8+ effector T cells is enhanced in vivo by administering IL-15. [PLoS One] Full Article

Gain Hands-On Training. Culture hPSCs and Differentiate to Definitive Endoderm or Neural Progenitor Cells


Myocardial Gene Transfer: Routes and Devices for Regulation of Transgene Expression by Modulation of Cellular Permeability
The success of gene therapy depends largely on the creation of a reliable and efficient delivery method. The authors describe a variety of physical and mechanical methods based on the transient disruption of the cell membrane which are applied in non-viral gene transfer. [Hum Gene Ther] Abstract

Generating β-Cells In Vitro: Progress Towards a Holy Grail
This review summarizes state-of-the-art efforts to generate scalable, functional β-cells to treat diabetes. [Curr Opin Endocrinol Diabetes Obes] Abstract

New TeSR™-E8™ is Here For Feeder-Free Culture of Human ES Cells and iPS Cells


Gamida Cell’s NiCord® Demonstrates Successful Engraftment in Patients with Hematological Malignancies in Phase I/II Study
Gamida Cell announced the successful results of the Phase I/II study of its second pipeline product NiCord, umbilical cord derived stem cells expanded using the company’s proprietary NAM technology. [Press release from Gamida Cell discussing research presented at the American Society for Blood and Marrow Transplantation 2013 BMT Tandem Meetings, Salt Lake City] Press Release

Designer Blood Clots: Artificial Platelets Could Treat Injured Soldiers on the Battlefield
Researchers exploring the complex stream of cellular signals produced by the body in response to a traumatic injury believe the initial response – formation of a blood clot – may control subsequent healing. Using that information, they’re developing new biomaterials, including artificial blood platelets laced with regulatory chemicals that could be included in an injector device the size of an iPhone. [Press release from the Georgia Institute of Technology discussing research presented at the 2013 American Association for the Advancement of Science (AAAS) Annual Meeting, Boston] Press Release


Expanding Public Access to the Results of Federally Funded Research
In a policy memorandum, Office of Science and Technology Policy Director John Holdren has directed Federal agencies with more than $100 million in R&D expenditures to develop plans to make the published results of federally funded research freely available to the public within one year of publication and requiring researchers to better account for and manage the digital data resulting from federally funded scientific research. [Office of Science and Technology Policy, The White House, United States] Press Release

New Breakthrough Prize Awards Millions to Life Scientists
Eleven scientists became multimillionaires when they were named the first winners of the new Breakthrough Prize in Life Sciences. Each researcher, whose specialties include genetics, stem cells, and cancer, will receive $3 million dollars, more than twice the maximum amount of a Nobel Prize. [ScienceInsider] Press Release


Florida Hospital Pepin Heart Institute and USF Health Team Up for the Groundbreaking Study of Novel Therapy for Heart Failure Patients
Cardiovascular disease specialists at Florida Hospital Pepin Heart Institute and Dr. Kiran C. Patel Research Institute affiliated with the University of South Florida (USF) announced they have enrolled their first two patients into a clinical trial testing a novel gene therapy for the treatment of heart failure after ischemic injury. [Florida Hospital Tampa Bay Division] Press Release

Stem Cell Therapeutics Announces Joint-Venture Agreement with Reneu Inc. and Nexgen Medical Systems, Inc.
Stem Cell Therapeutics Corp. (SCT) announced a joint-venture agreement with ReNeu Inc. (ReNeu) and NexGen Medical Systems, Inc. (NexGen). Subject to the definitive agreement, SCT and NexGen will license their respective technologies to ReNeu under world-wide, royalty-free licenses for the use of NexGen’s FDA-cleared EViTAR™ line of proprietary intracranial delivery systems and SCT’s NTx-265, -428 and -488 covering the proprietary use of drugs for the regeneration of endogenous neural stem cells. [Stem Cell Therapeutics Corp.] Press Release

BioMarin Licenses Factor VIII Gene Therapy Program for Hemophilia A from University College London and St. Jude Children’s Research Hospital
BioMarin Pharmaceutical Inc. announced that it has licensed a Factor VIII gene therapy program for hemophilia A from University College London and St. Jude Children’s Research Hospital. [BioMarin Pharmaceutical Inc.]
Press Release

Gladstone Scientist Named Inaugural Winner of Breakthrough Prize in Life Sciences
Gladstone Institutes’ Senior Investigator Shinya Yamanaka, MD, PhD, is one of 11 medical researchers to win a new, high-profile $3 million award from a group of high-tech sponsors, including the founders of Facebook and Google. [Gladstone Institutes] Press Release

HIV/AIDS Gene Therapy Work Continues with NIH Funds
Grant D. Trobridge of Washington State University has received a $1.6 million grant from the National Institutes of Health to continue investigating the use of gene therapy for HIV and AIDS, an area where he has had success. [Washington State University] Press Release

Osiris Therapeutics Receives Title of European Orphan Drug Designation for Prochymal®
Osiris Therapeutics, Inc. announced that the European Medicines Agency has designated Osiris as the Orphan Drug title holder for Prochymal® (remestemcel-L). The EU Commission adopted the decision to transfer the designation of Prochymal (ex vivo cultured adult human mesenchymal stem cells) as an orphan medicinal product under Regulation (EC) No 141/2000 of the European Parliament and of the Council. [Osiris Therapeutics, Inc.] Press Release

Osiris Therapeutics Successfully Defends Key Stem Cell Patent Challenge
Osiris Therapeutics, Inc. announced the successful conclusion of a patent challenge in Australia by an opponent whose identity under Australian law was not disclosed. The patent at issue covers the administration of mesenchymal stem cells, including Prochymal® (remestemcel-L), for the treatment of inflammatory conditions involving the gastrointestinal tract. [Osiris Therapeutics, Inc.] Press Release

Pluristem Receives FDA Orphan Drug Status Designation for Treatment of Aplastic Anemia
Pluristem Therapeutics Inc. announced that the U.S. Food and Drug Administration (FDA) has designated Pluristem’s placental expanded cells orphan drug status for the treatment of aplastic anemia. [Pluristem Therapeutics Inc.] Press Release

Bone Therapeutics Invests in Cell Therapy Manufacturing Facility
Bone Therapeutics announced that it has secured its future manufacturing requirements, allowing it to produce its unique bone cell therapy products on a commercial scale. Bone Therapeutics has made an investment in Skeletal Cell Therapy Support S.A, alongside the Walloon region and private investors, to fund the €10 million construction of a new manufacturing facility based in the Gosselies Scientific Park near Brussels, Belgium. [Bone Therapeutics SA] Press Release


NIH Director, Senator Mikulski Warn of Sequester’s Impact on Biomedical Research
The National Institutes of Health (NIH) and a key supporter in Congress warned about the damage to biomedical research if $85 billion in automatic cuts to all federal agencies goes into effect. NIH Director Francis Collins and Senator Barbara Mikulski (D-MD) said the so-called sequestration would slow scientific progress, delay clinical trials, and put a generation of young researchers at risk if NIH’s $31 billion budget for this year is trimmed by $1.5 billion. Press Release

Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2013-03802)

Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2013-03861)

National Heart, Lung, and Blood Institute; Notice of Closed Meetings (FR Doc No: 2013-04205)

Office of Biotechnology Activities; Recombinant DNA Research: Actions Under the NIH Guidelines for Research Involving Recombinant DNA Molecules (NIH Guidelines) (FR Doc No: 2013-03974)

Office of the Director, National Institutes of Health; Notice of Meeting (FR Doc No: 2013-03800)

Prospective Grant of Exclusive License: Development of MUC-1 Tumor Associated Antigens as Cancer Vaccines for Bladder Cancer, Breast Cancer, Colorectal Cancer, Gastric Cancer, Kidney Cancer, Liver Cancer, Lung Cancer, Ovarian Cancer, Prostate Cancer and Pancreatic Cancer (FR Doc No: 2013-03799)


2012 Biological License Application Approvals

2013 Biological Device Application Approvals


Food and Drug Administration (United States)

Guidance for Industry on Labeling for Human Prescription Drug and Biological Products–Implementing the Physician Labeling Rule Content and Format Requirements; Availability (FR Doc No: 2013-04195)

Human Subject Protection; Acceptance of Data from Clinical Studies for Medical Devices (FR Doc No: 2013-04201)

Medicines and Healthcare Products Regulatory Agency (United Kingdom)

European Commission Consultation Paper on the Regulation of Advanced Therapy Medicinal Products


NEW The Jackson Laboratory: We’ve Got Your Back – Smart Ways to Protect Your Research
February 26, 1:00PM (ET in US)

NEW The Jackson Laboratory: Humanized NSG Mice – Revolutionary Models of Human Infectious Disease
February 28, 1:00PM (ET in US)

NEW SMi’s Cell Culture 2013
July 3-4, 2013
London, United Kingdom

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 – Cardiovascular Regenerative Therapies (University of California, Los Angeles)

Postdoctoral Researcher (The University of Texas Health Science Center at San Antonio)

Postdoctoral Fellow – Transcriptional Regulation in CD8 T Lymphocytes (Université Libre de Bruxelles – Institute for Medical Immunology)

Postdoctoral Researcher Position in Bioinformatics (University of Oulu)

Physician-Scientist – Cancer Immunotherapy Program (The University of Chicago)

Postdoctoral Position – Center for Cell Engineering (Memorial Sloan-Kettering Cancer Center)

Senior Scientist (Spiber Technologies AB)

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

Postdoctoral Fellow – Pluripotent Stem Cells (Faculty of Medicine, University of Ottawa)

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