Cell Therapy News Volume 14.30 | Aug 5 2013

Directed Differentiation of Human Embryonic Stem Cells into Thymic Epithelial Progenitor-Like Cells Reconstitutes the Thymic Microenvironment In Vivo
The authors report a stepwise protocol to direct the differentiation of human embryonic stem cells (hESCs) into thymic epithelial progenitor-like cells (TEPLCs) by mimicking thymus organogenesis with sequential regulation of Activin, retinoic acid, BMP, and WNT signals. The hESC-derived TEPLCs expressed the key thymic marker gene FOXN1 and could further develop in vivo into thymic epithelium expressing the functional thymic markers MHC II and AIRE upon transplantation. [Cell Stem Cell] Abstract | Graphical Abstract

Efficient Generation of Human iPSCs by a Synthetic Self-Replicative RNA
Researchers report a simple, highly reproducible RNA-based induced pluripotent stem cell (iPSC) generation approach that utilizes a single, synthetic self-replicating VEE-RF RNA replicon that expresses four reprogramming factors at consistent high levels prior to regulated RNA degradation. A single VEE-RF RNA transfection into newborn or adult human fibroblasts resulted in efficient generation of iPSCs with all the hallmarks of stem cells, including cell surface markers, global gene expression profiles, and in vivo pluripotency, to differentiate into all three germ layers. [Cell Stem Cell]
Abstract | Press Release | Graphical Abstract

Epoxyeicosanoids Promote Organ and Tissue Regeneration
Scientists showed that endothelial-derived epoxyeicosatrienoic acids (EETs) play a critical role in accelerating tissue growth in vivo, including liver regeneration, kidney compensatory growth, lung compensatory growth, wound healing, corneal neovascularization, and retinal vascularization. Administration of synthetic EETs recapitulated these results, whereas lowering EET levels, either genetically or pharmacologically, delayed tissue regeneration, demonstrating that pharmacological modulation of EETs can affect normal organ and tissue growth. [Proc Natl Acad Sci USA] Abstract | Full Article | Press Release

Reducing CTGF/CCN2 Slows mdx Muscle Dystrophy and Improves Cell-Therapy
Mdx mice with hemizygous connective tissue growth factor (CTGF)-deletion (mdx-Ctgf +/-), and mdx mice treated with a neutralizing anti-CTGF monoclonal antibody (FG-3019), performed better in an exercise endurance test, had better muscle strength in isolated muscles, and reduced skeletal muscle impairment, apoptotic damage and fibrosis. Moreover, both mdx-Ctgf+/-, and FG-3019 treated mdx mice had improved grafting upon intramuscular injection of dystrophin positive satellite cells. [Hum Mol Genet] Abstract

Comparison of Fetal Mesencephalic Grafts, AAV-Delivered GDNF, and Both Combined in an MPTP-Induced Non-Human Primate Parkinson’s Model
Researchers combined viral vector delivery of human glial-derived neurotrophic factor (GDNF) with the grafting of dopamine precursor cells from fetal ventral mesencephalon to determine whether these strategies would improve the anti-Parkinson’s effects in 1-methyl-4-phenyl-1,2,3,6-tetrahydropyridine (MPTP)-treated monkeys, an animal model for Parkinson’s disease. [Mol Ther] Abstract

Design of Composite Scaffolds and Three-Dimensional Shape Analysis for Tissue-Engineered Ear
Investigators previously demonstrated that a titanium wire framework within a composite collagen ear-shaped scaffold helped to maintain the gross dimensions of the engineered ear after implantation, resisting the deformation forces encountered during neocartilage maturation and wound healing. The ear geometry was redesigned to achieve a more accurate aesthetic result when implanted subcutaneously in a nude rat model. [J R Soc Interface] Full Article | Press Release

Generation of Tooth-Like Structures from Integration-Free Human Urine Induced Pluripotent Stem Cells
Investigators described the generation of tooth-like structures from integration-free human urine induced pluripotent stem cells (ifhU-iPSCs). Results demonstrated that ifhU-iPSCs can be used to regenerate patient specific dental tissues or even tooth for further drug screening or regenerative therapies. [Cell Regen]
Abstract | Full Article | Press Release

Cell Therapy for Cystic Fibrosis
This review provides an overview of the rapidly progressing field of cell therapy for cystic fibrosis (CF), focusing on the various cell types utilized and current strategies that show promise to improve life expectancy and quality of life for CF patients. [J Tissue Eng Regen Med] Abstract

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

Medistem Presents Its ERC-124 Stem Cell Product
Researchers presented the Company’s cryopreservation process in the manufacturing and shipment of its ERC-124 product, an allogeneic universal donor stem cell derived from the endometrium. [Press release from Marketwire, Incorporated discussing research presented at the CRYO 2013 Conference, Bethesda]
Press Release

Brazil’s Science Investment Reaches Record High
Brazil’s investment in science has reached an all-time high, with the science ministry’s budget increasing from 8.6 billion reals (about US$3.8 billion) in 2012 to 12.7 billion reals (around US$5.6 billion) this year, according to science minister Marco Antonio Raupp. [Science Minister, Brazil] Press Release

Strict Limits on Animal Research Stun Italian Scientists
In a major victory for animal rights activists, the Italian Parliament approved a bill that puts drastic limitations on animal testing. Scientists have warned that the provisions, voted by the Senate earlier, will severely hurt biomedical research in their country – but they haven’t given up hope yet that the measures will be canceled. [Italian Parliament, Italy] Press Release

Australia’s Top Scientist Calls for a National Strategic Science Policy
Australia’s top science adviser warned the nation that it was time to feel a “sense of urgency” about its slipping science and technology expertise or risk falling behind the rest of the world scientifically and economically. Ian Chubb, the Australian government’s chief scientist, made the remarks while unveiling a position paper calling for a strategic plan to advance science, technology, engineering, and mathematics in the nation. [Chief Scientist, Australia] Press Release

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Expert Consortium Awarded £2 Million to Advance Trachea Replacement Technology
A tracheal replacement project, led by Videregen Ltd and involving a team of UK experts, has been awarded almost £2m by the UK Technology Strategy Board under its Regenerative Medicine and Cell Therapy competition. The consortium will use the funds to progress a promising clinical prototype through a rigorous development process and early clinical trials for the treatment of severe structural airway disease. [Technology Strategy Board] Press Release

uniQure to Build 55,000 Square Foot State-of-the-Art Gene Therapy Production Plant in US to Leverage AAV-Manufacturing Strength
uniQure B.V. announced it has commenced build-out of a state-of-the-art 55,000 square foot US manufacturing plant in Lexington, Massachusetts for the production of commercial grade Adeno Associated Virus (AAV)-based gene therapy products to complement its current EMA-approved site in Amsterdam, the Netherlands. The Company expects the facility to be operational by early 2015. [uniQure B.V.] Press Release

StemCells, Inc. Launches Alzheimer’s Disease Program Supported by California Institute for Regenerative Medicine
StemCells, Inc. announced that it has formally launched its Alzheimer’s disease program, which is being supported by the California Institute for Regenerative Medicine. The goal of the project is to file, within four years, an Investigational New Drug application with the U.S. Food and Drug Administration to evaluate the Company’s proprietary HuCNS-SC® product candidate (purified human neural stem cells) as a potential therapeutic in the treatment of Alzheimer’s disease. [StemCells, Inc.] Press Release

BrainStorm Submits Favorable Interim Safety Report for Second Group of Patients in Its Phase IIa Dose-Escalating Trial of NurOwn™ in ALS
BrainStorm Cell Therapeutics announced that it has submitted a favorable safety report to the hospital Helsinki Committee for the second group of patients in its ongoing Phase IIa ALS clinical trial at the Hadassah Medical Center in Jerusalem, Israel. In this Phase IIa dose-escalating trial, currently underway at Hadassah under the direction of Principal Investigator Professor Dimitrios Karussis, 12 ALS patients are receiving combined intramuscular and intrathecal administration of NurOwn™ cells, in three cohorts, with increasing doses. [BrainStorm Cell Therapeutics] Press Release

uniQure Announces Start of Parkinson’s Disease Gene Therapy Phase I Trial by UCSF/NIH to Test Its GDNF Gene in Patients
uniQure B.V. announced the start of a human trial in Parkinson’s disease with glial cell line-derived neurotrophic factor (GDNF). The gene therapy developed by researchers at the University of California, San Francisco (UCSF), uses the GDNF gene, which uniQure licensed from Amgen. [uniQure B.V.] Press Release

Factor Granted First Patent for RNA Reprogramming Technology
The United States Patent and Trademark Office granted Factor Bioscience the first patent covering methods for reprogramming cells using RNA, a technology with wide-ranging applications in biological research and personalized medicine. The patented methods can transform patient skin cells into stem cells more rapidly, efficiently, and safely than previous techniques. [Factor Bioscience Inc.] Press Release

Cancer Immunotherapy Platform Targets Broad Range of Metastatic Cancers
California Stem Cell, Inc. has announced the completion of an internal study demonstrating that the company’s proprietary immunotherapy manufacturing process can be applied to multiple cancer types. Originally developed for late stage metastatic melanoma, the platform tissue culture technology has also been shown to work with tumor tissues from hepatocellular carcinoma, ovarian cancer, glioblastoma multiforme, and other solid tumor cancers. [California Stem Cell, Inc.] Press Release

Avita Medical Enrolls First Patients in ReCell® Trial for Chronic Leg Ulcers
Regenerative medicine Company Avita Medical Ltd. announced the initial enrollment of three patients in the RESTORE trial, a multi-center, randomized European clinical trial evaluating the use of ReCell® Spray-on Skin® in the management of patients with venous leg ulcers. The company believes the management and treatment of lower limb ulcers represents a large and growing market opportunity for ReCell. [Avita Medical Ltd.]
Press Release

HEMACORD®, the First FDA-Licensed Stem Cell Product, Nominated for Prix Galien USA “Best Biotechnology Product” Award
The New York Blood Center’s Milstein Cord Blood Program is proud to acknowledge the nomination of its first-in-class hematopoietic stem cell product, HEMACORD® (HPC, Cord Blood) for the 2013 “Best Biotechnology Product” Award by Prix Galien USA. [PR Newswire Association LLC] Press Release  

National Institute of Biomedical Imaging and Bioengineering; Notice of Closed Meeting (FR Doc No: 2013-18586)  

Update on Reshaping the Organization of the European Medicines Agency

Therapeutic Goods Administration (Australia)

TGA Structure  

NEW IBC’s 3rd Annual Cell Therapy Bioprocessing
October 21-22, 2013
Bethesda, United States

NEW Cell Based Assays
November 18-19, 2013
London, United Kingdom

NEW IBC’s Biopharmaceutical Development & Production (BDP) Week: Methods & Molecules
March 24-27, 2014
San Diego, United States

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

NEW Postdoctoral Fellow – Human Stem Cell Biology and Hematopoiesis (Johns Hopkins University School of Medicine)

NEW Tenure-Track Position – Cell Biologist (McGill University)

NEW Postdoctoral Positions – AIDS Vaccine and Human Gene Therapy (GeneCure Biotechnologies)

NEW Postdoctoral Appointee – Cell Cycle Regulation in Tumor Stem Cells (Rutgers University, Rutgers Cancer Institute of New Jersey)

Research Associate – Cardioprotection and Myocardium Repair by Gene or Cell Therapy (University of Louisville)

Postdoctoral Position – Stem Cell Therapy and Epigenetics of Retina (University of Pittsburgh, Ophthalmology)

Postdoctoral Fellow – Neural Tissue Engineering (School of Chemical & Biomedical Engineering, Nanyang Technological University)

Postdoctoral Fellowship – Immunology (Johns Hopkins University School of Medicine)

Research Scientist/Senior Research Officer – Monoclonal Antibodies (Bioprocessing Technology Institute/A*STAR)

Director of Cell Processing Facility (S L Collins Associates, Inc.)

Postdoctoral Researcher – Hematopoietic Stem Cells (Albert Einstein College of Medicine)

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