Cell Therapy News Volume 14.15 | Apr 22 2013

Medial Ganglionic Eminence-Like Cells Derived from Human Embryonic Stem Cells Correct Learning and Memory Deficits
The authors presented a method for differentiating human embryonic stem cells to a nearly uniform population of NKX2.1⁺ medial ganglionic eminence (MGE)-like progenitor cells. Mice transplanted with MGE-like but not spinal progenitors showed improvements in learning and memory deficits. [Nat Biotechnol]
Abstract | Press Release

Generation of Oligodendroglial Cells by Direct Lineage Conversion
Researchers report the generation of induced oligodendrocyte precursor cells (iOPCs) by direct lineage conversion. iOPCs gave rise to mature oligodendrocytes that could ensheath multiple host axons when co-cultured with primary dorsal root ganglion cells and formed myelin after transplantation into shiverer mice. [Nat Biotechnol] Abstract | Press Release

Geometric Control of Vascular Networks to Enhance Engineered Tissue Integration and Function
Scientists used a microtissue molding approach to demonstrate that constructs containing highly aligned “cords” of endothelial cells triggered the formation of new capillaries along the length of the patterned cords. These vessels became perfused with host blood as early as 3 days post implantation and became progressively more mature through 28 days. [Proc Natl Acad Sci USA] Abstract

The Therapeutic Activities of Engrafted Neural Stem/Precursor Cells Are Not Dormant in the Chronically Injured Spinal Cord
Researchers performed temporal analysis of injured spinal cords and demonstrated their multiphasic cellular and molecular responses. To determine how these environmental differences affect engrafted cells, neural stem/precursor cells transplanted into acutely, subacutely, and chronically injured spinal cords were selectively isolated by flow-cytometry, and their whole transcriptomes were compared by RNA-sequencing. [Stem Cells] Abstract

Multipotent Stromal Cells Alleviate Inflammation, Neuropathology, and Symptoms Associated with Globoid Cell Leukodystrophy in the Twitcher Mouse
Investigators aimed to improve bone marrow-derived multipotent stromal cell (MSC) therapy for globoid cell leukodystrophy by increasing the cell dosage and comparing cell type, treatment timing, and administration route. The mice receiving peripheral MSC therapy had improved motor function, twitching symptoms, and weight compared to both the untreated and intracerebroventricular-treated mice. [Stem Cells] Abstract

Mouse Otocyst Transuterine Gene Transfer Restores Hearing in Mice with Connexin 30 Deletion-Associated Hearing Loss
Because mutations or deletions in the connexin (Cx) genes are common causes of profound congenital hearing loss in both humans and mice, the authors investigated whether gene supplementation therapy using the wild-type Cx gene could cure hearing loss. [Mol Ther] Abstract

CD34⁺ Expansion with Delta-1 and HOXB4 Promotes Rapid Engraftment and Transfusion Independence in a Macaca nemestrina Cord Blood Transplant Model
Researchers explored in a macaque cord blood transplant model expansion and engraftment kinetics of cells expanded with the combination of HOXB4 and Delta-1. [Mol Ther] Abstract

Sca-1+ Cardiac Progenitor Cell Therapy with Cells Overexpressing Integrin-Linked Kinase Improves Cardiac Function after Myocardial Infarction
Researchers investigated the effect of integrin-linked kinase on the transplantation efficiency of stem cell antigen-1-positive (Sca-1+) cardiac progenitor cells in a mouse myocardial infarction model. [Transplantation] Abstract

Articular Cartilage Repair with Magnetic Mesenchymal Stem Cells
Scientists hypothesized that magnetic-assisted delivery of magnetically labeled mesenchymal stem cells (m-MSCs) would be rapid, allowing for chondrogenic differentiation and functional joint repair without replacement. The m-MSCs had no adverse effect on chondrogenic differentiation, and m-MSCs delivered by magnetic field application repaired cartilage defects. [Am J Sport Med] Abstract

Percutaneous Gene Therapy Heals Cranial Defects
As the bone morphogenic protein and transforming growth factor beta pathways have been implicated in bone healing, researchers hypothesized that percutaneous Smad7 silencing would enhance signaling through both pathways and improve bone formation. [Gene Ther] Abstract

Immune Responses to AAV Vectors: Overcoming Barriers to Successful Gene Therapy
A comprehensive understanding of the determinants of immunogenicity of adeno-associated viral (AAV) vectors, and of the potential associated toxicities, is still lacking. Careful immunosurveillance conducted as part of ongoing clinical studies will provide the basis for understanding the intricacies of the immune response in AAV-mediated gene transfer, facilitating safe and effective therapies for genetic disease. [Blood] Abstract

Smelling the Roses and Seeing the Light: Gene Therapy for Ciliopathies
Growing understanding of ciliopathy genetics, coupled with recent advances in gene delivery and endogenous gene and transcript repair demonstrated thus far in tissues of the eye, nose, and airway, offers hope for curative measures in the near future. Researchers highlight these advances, as well as the challenges that remain with the development of personalized medicine for treating a very complex spectrum of disease, penetrant in a variety of organ systems. [Trends Biotechnol] Abstract

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

TiGenix Reports Positive Phase IIa Study Results in Refractory Rheumatoid Arthritis with Allogeneic Stem Cell Product Cx611
TiGenix announced positive six-month safety data of its Phase IIa study of Cx611 in rheumatoid arthritis, as well as a first indication of therapeutic activity on standard outcome measures and biologic markers of inflammation for at least three months after dosing. [TiGenix] Press Release

ReNeuron Presents New Pre-Clinical Data Demonstrating Therapeutic Effects of Nanoparticles Released from Its Stem Cells
ReNeuron announced important new pre-clinical data on the potential mechanisms by which its lead CTX stem cell line exerts its therapeutic effects. The Company’s researchers have identified that CTX cells release nanoparticles known as exosomes containing key proteins and micro-RNA that appear to influence neighboring cells to stimulate certain therapeutic mechanisms associated with tissue repair. [Press release from ReNeuron Group plc discussing research presented at the International Society for Extracellular Vesicles 2013 Conference, Boston] Press Release

Ceregene Reports Data from Parkinson’s Disease Phase IIb Study
Ceregene, Inc. announced the top-line data from its double-blind, randomized, controlled Phase IIb clinical study of CERE-120 (AAV-neurturin), a gene therapy product designed to deliver the neurotrophic factor neurturin, for Parkinson’s disease. [Ceregene, Inc.] Press Release

A Back Seat for Basic Science
With unemployment at a worrisome 7.6% and Congress insistent on deficit reduction, Obama’s budget is cautious. The US$3.8-trillion proposal would undo the across-the-board cuts known as sequestration, but includes just $143 billion for research and development, a mere 1% rise from 2012. The budget reflects not only caution, but also the Obama administration’s emerging attitude towards research: that science needs to be good for something, namely jobs and cures. [US President, United States] Press Release

NSF Peer Review Under Scrutiny by House Science Panel
The National Science Foundation (NSF) may soon be getting some unsolicited advice from Congress on how to improve its vaunted peer-review system. The new chairman of the House of Representatives Committee on Science, Space, and Technology floated the idea of having every NSF grant application include a statement of how the research, if funded, “would directly benefit the American people.” [House of Representatives Committee on Science, Space, and Technology, United States] Press Release

Romania to Replace National Research Council after Mass Resignation
The Romanian Ministry of Education, Research, Youth and Sport has asked universities to nominate replacements for the 19 members of the National Research Council, Romania’s main research funding agency. Council members resigned en masse to protest retroactive cuts in research grants. [Romanian Ministry of Education, Research, Youth and Sport, Romania] Press Release

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CT Catapult & Loughborough University to Collaborate on Innovative Manufacturing
The Cell Therapy Catapult and Loughborough University have agreed to work together on innovative manufacturing. The two organizations will collaborate to develop robust processes and new manufacturing and delivery techniques, removing the barriers associated with turning cell-based therapies into products, and providing training and skills development. [Cell Therapy Catapult] Press Release

Bone Therapeutics and Erasme University Hospital Start Phase IIa Trial in Osteoporosis with Its Cell Therapy Product PREOB^®
BONE THERAPEUTICS announced that its partner, the Rheumatology Department of Erasme University Hospital (Brussels, Belgium), has initiated a Phase IIa trial with its lead bone cell therapy product PREOB^® for the treatment of severe osteoporosis patients who do not respond to pharmacological treatment. [Bone Therapeutics SA] Press Release

StemCells, Inc. Adds Byers Eye Institute at Stanford as Second Site for Phase I/II Clinical Trial in Dry Age-Related Macular Degeneration
StemCells, Inc. announced the addition of the Byers Eye Institute at Stanford as a second site for the Company’s Phase I/II clinical trial of its proprietary HuCNS-SC^® product candidate (purified human neural stem cells) in dry age-related macular degeneration. [StemCells, Inc.] Press Release

JPT Peptide Technologies and Cancer Immunotherapy Trials Network Collaborate on Monitoring Cellular Immune Responses
JPT Peptide Technologies GmbH, a wholly-owned subsidiary of BioNTech AG, and the Cancer Immunotherapy Trials Network (CITN) agreed on the use of JPT’s PepMix™ Peptide Pool Platform to support CITN’s clinical cancer trials and cancer research related efforts. [JPT Peptide Technologies GmbH] Press Release

FDA Approves Phase II of Stem Cell Trial for ALS Led by U-M’s Dr. Eva Feldman
For nearly two years, University of Michigan (U-M) neurologist Eva Feldman, M.D., Ph.D. has led the nation’s first clinical trial of stem cell injections in patients with the deadly degenerative disease known as amyotrophic lateral sclerosis (ALS). Now, a new approval from the U.S. Food and Drug Administration paves the way for U-M to become the second site in the trial, pending approval of the U-M Institutional Review Board. To date, the first phase of the trial has taken place at Emory University, with Feldman serving as principal investigator. [University of Michigan Health System] Press Release

NW Bio’s Phase III Trial with DCVax®-L for Brain Cancer “Adopted” as a National Priority Trial in UK
Northwest Biotherapeutics (NW Bio) announced that the Company’s Phase III clinical trial for Glioblastoma multiforme brain cancer has been “adopted” as a national priority trial in the UK, under the “adoption” program managed by the National Institute for Health Research (NIHR), which is part of the UK’s National Health System. This “adoption” of the Company’s Phase III trial constitutes a significant validation, and also carries with it resources and operational support for the trial sites. [Northwest Biotherapeutics, Inc.] Press Release

Cardio-Thoracic Surgery Patients Receive Regenerative Medicine Treatment to Reduce Scarring, Speed Wound Healing
SironRX Therapeutics Inc. reported that it has completed enrollment of patients in Cohort 1 of its placebo-controlled, randomized, dose escalation double-blind clinical study evaluating the safety and efficacy of JVS-100 to reduce scar formation and improve wound healing in patients receiving surgical sternotomy incisions. Cohort 2 is actively being enrolled. [PR Newswire Association LLC] Press Release

Gene Therapy Developed for ALS Treatment: New Biotech Company Neuralgene Enters Evaluation Phase
Startup biotechnology company Neuralgene announced that it will begin animal studies in May to evaluate the efficacy of PRCN-829, its new gene therapy agent for the treatment of amyotrophic lateral sclerosis (ALS). PRCN-829 is the first gene therapy for sporadic ALS. [PR Newswire Association LLC] Press Release

HCV Clinical Trial Application Submitted to Recombinant DNA Advisory Committee (RAC)
RNAi-based therapeutics company Benitec Biopharma Limited announced that the company’s wholly owned US subsidiary, Tacere Therapeutics Inc., has submitted an application to the US National Institutes of Health’s RAC. The RAC submission document comprises the full clinical trial protocol for its first-in-man ddRNAi-based therapeutic for hepatitis C virus (HCV) infection, as well as the safety and toxicology data and responses to questions concerning the objective and rationale for the proposed trial. [Benitec Biopharma Limited]
Press Release

Veritas Bio’s RNA Delivery Patent Allowed by USPTO
Veritas Bio, LLC announced that the company has received a Notice of Allowance from the United States Patent and Trademark Office (USPTO) for U.S. Patent Application No. 12/514,237, “In Vivo Delivery Of Double Stranded RNA To a Target Cell”. [Veritas Bio, LLC] Press Release

House Committees Probe NIH Spending on Public Relations
In an unusual joint investigation, Republican members of two House of Representatives committees are asking the National Institutes of Health (NIH) for details on how much the $30 billion agency spends on communications and public relations. Some of this money could be cut to help the agency make up for funds lost this year to across-the-board spending cuts, the letter suggests. Press Release

NIH Reminds Grantee Institutions of the Requirement to Use the Research Performance Progress Report (RPPR) for All SNAP and Fellowship Progress Reports for Awards with Start Dates On or after July 1, 2013 (NOT-OD-13-061)

National Institute of Diabetes and Digestive and Kidney Diseases; Notice of Closed Meeting (FR Doc No: 2013-09320)

European Medicines Agency (European Union)

Stem Cell Therapy Treatments

Food and Drug Administration (United States)

Agency Information Collection Activities; Submission for Office of Management and Budget Review; Comment Request; Clinical Laboratory Improvement Amendments Waiver Applications (FR Doc No: 2013-09180)

Agency Information Collection Activities; Announcement of Office of Management and Budget Approval; Electronic Submission of Medical Device Registration and Listing (FR Doc No: 2013-09182)

NEW 2013 ASCO/AACR Workshop: Methods in Clinical Cancer Research
July 27-August 2, 2013
Vail, United States

NEW Joint Meeting of APHIA & HKSHI
July 31-August 2, 2013
Hong Kong

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)

Postdoctoral Position – Synthetic Biology, RNAi & Gene Therapy (University of Heidelberg Medical School)

Postdoctoral Fellowships – Muscular Dystrophy, Cardiomyopathy, Respiratory Failure, Gene Therapy (University of Pennsylvania)

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