Cell Therapy News Volume 13.48 | Dec 10 2012

Generation of Integration-Free Neural Progenitor Cells from Cells in Human Urine
Scientists combined an episomal system to deliver reprogramming factors with a chemically defined culture medium to reprogram epithelial-like cells from human urine into neural progenitor cells. Although functional in vivo analysis is still needed, they reported that the cells survive and differentiate upon transplant into newborn rat brain. [Nat Methods] Abstract | Press Release

Sustained Effector Function of IL-12/15/18-Preactivated NK Cells against Established Tumors
Recently, mouse IL-12/15/18-preactivated natural killer (NK) cells were shown to persist with sustained effector function in vivo. This study investigated the antitumor activity of such NK cells. A single injection of syngeneic IL-12/15/18-preactivated NK cells, but neither naive nor IL-15- or IL-2-pretreated NK cells, combined with irradiation substantially reduced growth of established mouse tumors. [J Exp Med] Abstract | Press Release

Autologous Mesenchymal Stem Cell-Derived Dopaminergic Neurons Function in Parkinsonian Macaques
Investigators showed that autologous engraftment of A9 dopaminergic neuron-like cells induced from mesenchymal stem cells leads to long-term survival of the cells and restoration of motor function in hemiparkinsonian macaques. [J Clin Invest] Full Article | Press Release

In Vivo Generation of Transplantable Human Hematopoietic Cells from Induced Pluripotent Stem Cells
Researchers report a novel in vivo system in which human induced pluripotent stem cells differentiate within teratomas to derive functional myeloid and lymphoid cells. Similarly, hematopoietic stem/progenitor cells can be isolated from teratoma parenchyma and reconstitute a human immune system when transplanted into immunodeficient mice. [Blood] Abstract

Double Unit Grafts Successfully Extend the Application of Umbilical Cord Blood Transplantation in Adults with Acute Leukemia
The transplantation of two partially HLA-matched umbilical cord blood (UCB) units has been adopted as a simple approach for increasing the total nucleated cells. The authors sought to determine whether the relative safety and efficacy of this approach was comparable to a single UCB transplant. [Blood] Abstract

Injectable Bone Tissue Engineering Using Expanded Mesenchymal Stem Cells
Researchers investigated whether injectable tissue-engineered bone composed of mesenchymal stem cells and platelet rich plasma was able to regenerate functional bone in alveolar deficiencies. [Stem Cells] Abstract

Pro-Angiogenic and Pro-Survival Functions of Glucose in Human Mesenchymal Stem Cells upon Transplantation
Investigators challenged the current paradigm of human mesenchymal stem cell (hMSC) survival, which assigned a pivotal role to oxygen, by testing the hypothesis that exogenous glucose may be key to hMSC survival. Ectopically implanted tissues constructs supplemented with glucose exhibited four- to five-fold higher viability and were more vascularized compared to those without glucose at day 14. [Stem Cells] Abstract | Full Article

Alpharetroviral Vector-Mediated Gene Therapy for X-CGD: Functional Correction and Lack of Aberrant Splicing
Researchers chose X-linked chronic granulomatous disease (X-CGD) as a model to evaluate the potential of self-inactivating alpharetroviral vectors for gene therapy of monogenic diseases. They combined the alpharetroviral vector backbone with the elongation factor-1α short promoter, both considered to possess a low genotoxic profile, to drive transgene (gp91^phox) expression. [Mol Ther] Abstract

Short-Term, Long-Term and Paracrine Effect of Human Umbilical Cord-Derived Stem Cells in Lung Injury Prevention and Repair in Experimental Bronchopulmonary Dysplasia
Human umbilical cord-derived perivascular cells and mesenchymal stem cells exert short- and long-term therapeutic benefit without adverse lung effects in an experimental bronchopulmonary dysplasia model and offer new therapeutic options for lung diseases characterized by alveolar damage. [Thorax] Abstract | Press Release

Effect of Cardiac Stem Cells on Left Ventricular Remodeling in a Canine Model of Chronic Myocardial Infarction
Scientists used a chronic large animal model of myocardial infarction with substantial reductions in left ventricular ejection fraction and adverse remodeling to examine the effect of late autologous cardiac stem cell intramyocardial injection on long term cardiac structure and function. [Circ Heart Fail] Abstract

Perspectives of Stem Cell Therapy in Duchenne Muscular Dystrophy
The authors describe different types of myogenic stem cells and their possible use for the progression of cell therapy in DMD. [FEBS J] Abstract | Full Article

Allogeneic Stem Cell Transplantation for Ischemic Myocardial Dysfunction
Allogeneic stem cell populations have demonstrated therapeutic promise in ischemic heart disease, without a requirement for immunosuppressive drugs, and offer the potential to create large-scale, cryopreserved banks of cells for ‘off the shelf’ utility. [Curr Opin Organ Transplant] Abstract

Engineered Immune Cells Produce Complete Response in Child with an Aggressive Pediatric Leukemia
By reprogramming a seven-year-old girl’s own immune cells to attack an aggressive form of childhood leukemia, a pediatric oncologist has achieved a complete response in his patient, who faced grim prospects when she relapsed after conventional treatment. The innovative experimental therapy used bioengineered T cells, custom-designed to multiply rapidly in the patient, and then destroy leukemia cells. [Press release from The Children’s Hospital of Philadelphia discussing research presented at the 54^th American Society of Hematology Annual Meeting, Atlanta] Press Release

Alnylam Presents New Pre-Clinical Data on RNAi Therapeutics for the Treatment of Hemophilia and Other Bleeding Disorders
Alnylam scientists presented data showing that ALN-AT3, a subcutaneously administered RNAi therapeutic targeting antithrombin (AT), yields potent, dose-dependent, and durable knockdown of AT in non-human primates with an up to four-fold increase in thrombin generation. [Press release from Alnylam Pharmaceuticals, Inc. discussing research presented at the 54^th American Society of Hematology Annual Meeting, Atlanta]
Press Release

Sarepta Therapeutics Announces a Continued Benefit on Walking Test through 62 Weeks in Phase IIb Open-Label Extension Study of Eteplirsen in Duchenne Muscular Dystrophy
Sarepta Therapeutics announced updated data from Study 202, its open-label, Phase IIb extension study of eteplirsen for the treatment of Duchenne muscular dystrophy. Patients treated with eteplirsen for 62 weeks and evaluable on ambulatory measures maintained a statistically significant clinical benefit on the primary clinical outcome measure, the six-minute walk test, compared to patients who received placebo for 24 weeks followed by 38 weeks of eteplirsen treatment. [Press release from Sarepta Therapeutics discussing research to be presented at the European Neuromuscular Centre Workshop] Press Release

U.K. Government Injects Nearly $1 Billion into Research
The United Kingdom’s finance minister announced a new lump of money, some £600 million, for scientific research. The new funding will be spent on big data and energy efficient computing, synthetic biology, energy storage, and advance materials. [U.K. Finance Minister, United Kingdom] Press Release

NSF Graduate Fellows Get Chance to Work Overseas
As many as 400 U.S. graduate students will be able to work abroad in 2014 under a new program at the National Science Foundation (NSF). NSF officials unveiled the Graduate Research Opportunities Worldwide program as part of a celebration of the 60th anniversary of its Graduate Research Fellowship Program. [National Science Foundation, United States] Press Release

National Panel Says California Stem Cell Agency Has Room for Improvement
A review by the Institute of Medicine (IOM) praises the California Institute for Regenerative Medicine as a “bold social innovation” that provided a creative new source of funding that has turned the state into an international hub of stem cell research. But the IOM panel authoring the report also concluded that the funding agency’s organization and governance is not optimal. [Institute of Medicine, United States] Press Release

Pakistan’s New Science Policy Raises Hopes
As they hail Pakistan’s first comprehensive national science, technology and innovation policy, the country’s science leaders are hopeful of effective implementation and funding. The ‘National Science, Technology and Innovation Policy-2012’, launched with support from the Pakistan Council for Science and Technology and the ministry of science and technology, is expected to help Pakistan emerge as a scientifically sensitive nation. [Pakistan Council for Science and Technology, Pakistan] Press Release

Roche and the Innovative Medicines Initiative Join Forces to Promote Use of Nobel Prize-Winning Stem Cell Technology to Enhance Drug Development
Roche and the Innovative Medicines Initiative announced the launch of StemBANCC, a new academic-industry partnership that unites ten pharmaceutical companies and 23 academic institutions. Initiated and coordinated by Roche and managed by Oxford University, StemBANCC aims to use human induced pluripotent stem cells as research tools for drug discovery with the goal of using this ground-breaking new technology to develop human disease models and enhance drug development. [F. Hoffmann-La Roche Ltd] Press Release

WiCell Teams with Agilent Technologies to Offer High-Resolution CGH+SNP Microarray Service
Agilent Technologies Inc. and WiCell announced that WiCell is now offering comparative genomic hybridization plus single nucleotide polymorphism microarray analysis using the Agilent SurePrint G3 Human Genome CGH+SNP Microarray. [WiCell Research Institute, Inc.] Press Release

UT MD Anderson, GlaxoSmithKline to Collaborate on New Approach to Cancer Immune Therapy; Success Could Earn Cancer Center $335 Million Plus Royalties
The University of Texas (UT) MD Anderson Cancer Center and GlaxoSmithKline have signed a research collaboration and license agreement to develop new therapeutic antibodies that promote an immune system attack against cancer. [The University of Texas MD Anderson Cancer Center] Press Release

GE Healthcare Life Sciences Opens New £3 Million Laboratories for Cell Science in Cardiff, Wales
GE Healthcare Life Sciences announced the opening of its new £3 million cell science laboratories in Cardiff, Wales. The state-of-the-art investment at the company’s Maynard Centre creates a world-class facility for research and technology innovation in cell science, to help advance the rapidly emerging fields of cell therapy and cell bioprocessing, and to support the development of new, more effective and safer medicines. [GE Healthcare Life Sciences] Press Release

Moderna Announces $40 Million in Financing to Advance Development of New Biotherapeutic Modality: Messenger RNA Therapeutics^TM
Moderna Therapeutics announced that it has closed more than $40 million in financing to date, led by Flagship Ventures and private investors, which will be used to advance multiple programs toward clinical stage development. [Moderna Therapeutics] Press Release

Gene Therapy as a New Option for Bone Defects
Gene therapy involving modified stem cells obtained from fatty tissue and bone marrow could represent a new option for the treatment of severe orthopedic injuries to the extremities. The new method involves altering the body’s own stem cells, obtained from fat or bone marrow, with BMP-2 genes which are known to promote bone healing. [Medical University of Vienna] Press Release

Stem Cell Success in Treating Glaucoma
Risk factors for glaucoma are raised pressure in the eye and a weakness in the optic nerve. Researchers have undertaken an experimental treatment that potentially means one can use the patient’s own stem cells. By transplanting a small number of olfactory ensheathing cells into the region of the optic nerve, researchers have been able to halve the loss of optic nerve fibers caused by raised eye pressure and reduce the damage to the optic nerve tissue. [The UK Stem Cell Foundation] Press Release

Cytomedix Announces Landmark Bright Cell Therapy Study in Peripheral Arterial Disease
Cytomedix, Inc. announced the signing of an agreement with NIH to collaborate on a Phase II clinical study in patients with intermittent claudication (IC). The Phase II PACE (Patients with Intermittent Claudication Injected with ALDH Bright Cells) study is an 80 patient, double-blind, placebo-controlled clinical trial intended to demonstrate the safety and efficacy of ALD-301 (Bright Cells) in patients diagnosed with IC. [Cytomedix, Inc.] Press Release

uniQure Licenses RNA Interference Technology to Advance Huntington’s Disease Program
uniQure B.V. announced a non-exclusive cross-licensing agreement with Benitec Biopharma Ltd. giving uniQure access to Benitec’s proprietary DNA-directed RNA interference technology in Huntington’s disease. [uniQure B.V.] Press Release

Nuvilex Subsidiary, Austrianova Singapore, Publishes Advantages of Its Encapsulated Cell Therapy for the Cell-Based Therapeutic Treatment of Diabetes
Nuvilex, Inc. announced its subsidiary Austrianova Singapore has published a new manuscript that describes the use of the Company’s proprietary cell encapsulation technology for studies designed to create a cell based therapeutic treatment for diabetes. [Nuvilex, Inc.] Press Release

NIH Promises to Improve Biomedical Research Training
Reacting to a steep rise in the number of young biomedical scientists seeking scarce academic jobs, the National Institutes of Health (NIH) plans to launch programs to prepare scientists for nonacademic careers, move students through their PhDs faster, and bolster the pay of postdocs. Press Release

Notice to Extend PA-10-009 Bioengineering Research Grants (BRG) (R01) (NOT-EB-13-001)

National Cancer Institute; Notice of Closed Meeting (FR Doc No: 2012-29559)

National Institute of Biomedical Imaging and Bioengineering; Notice of Meeting (FR Doc No: 2012-29300)

Food and Drug Administration (United States)

Cellular, Tissue and Gene Therapies Advisory Committee; Notice of Meeting (FR Doc No: 2012-29706)

Request for Notification from Industry Organizations Interested in Participating in the Selection Process for Nonvoting Industry Representatives and Request for Nominations for Nonvoting Industry Representatives on the Device Good Manufacturing Practice Advisory Committee (FR Doc No: 2012-29165)


Therapeutic Goods Administration (Australia)

Therapeutic Goods Information Specifications

NEW The Jackson Laboratory: Disaster Prevention – Smart Ways to Protect Your Research
December 11, 1:00PM (ET in US)
Webinar

NEW The Jackson Laboratory: Improving Mouse Colony Management Efficiency
December 13, 1:00PM (ET in US)
Webinar

NEW StemCONN 2013: Realizing the Promise
April 3, 2013
New Haven, United States

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

Product Quality Scientist (STEMCELL Technologies, Inc.)

Scientist – Endothelial Cell Research (STEMCELL Technologies, Inc.)

Research Technologist – Cell Separation (STEMCELL Technologies, Inc.)

Scientist or Engineer – hPSC Bioengineer (STEMCELL Technologies, Inc.)

Quality Control Analyst (STEMCELL Technologies, Inc.)

Postdoctoral Positions in Cell Biology, Biophysics and Neurobiology (University of Missouri – Kansas City)

Postdoctoral Position – Tumor Stem Cells (Cancer Institute of New Jersey)

Quality Assurance Manager (The University of Texas Medical School at Houston)

Researcher (ESR8) – Regulatory T Cell Therapy (Università Vita-Salute San Raffaele)

Postdoctoral Position – iPSC Research (University of Washington)

Postdoctoral Position (Karolinska Institute, Department of Medicine, Huddinge)

Senior QC Analyst/Assay Development Associate (StemCells, Inc.)

Postdoctoral Position (Sandra Rotman Centre for Global Health)

Postdoctoral Fellow – Molecular Biology (Cleveland Clinic Foundation)

Recruit Top Talent: Reach more than 55,000 potential candidates by posting your organization’s career opportunities on the Connexon Creative Job Board at no cost.