Cell Therapy News Volume 14.32 | Aug 19 2013

Complement Anaphylatoxin C3a Is a Potent Inducer of Embryonic Chick Retina Regeneration
Scientists showed that complement fragment C3a is sufficient to induce complete regeneration of the embryonic chick retina from stem/progenitor cells present in the eye, independent of fibroblast growth factor receptor signaling. Instead, C3a induces retina regeneration via STAT3 activation, which in turn activates the injury- and inflammation-responsive factors, IL-6, IL-8 and TNF-α. [Nat Commun] Full Article

Transplants of Adult Mesenchymal and Neural Stem Cells Provide Neuroprotection and Behavioral Sparing in a Transgenic Rat Model of Huntington’s Disease
Investigators compared the efficacy of transplants of mesenchymal stem cells (MSCs), adult neural stem cells (aNSCs), or co-transplants of MSCs and aNSCs for reducing deficits in a transgenic rat model of Huntington’s disease. [Stem Cells] Abstract

The miR-223/Nuclear Factor I-A Axis Regulates Glial Precursor Proliferation and Tumorigenesis in the CNS
Previously, the authors identified nuclear factor I-A (NFIA) as a key regulator of developmental gliogenesis, while miR-223 has been shown to repress NFIA expression in other systems. Using this relationship as a starting point, they found that miR-223 can suppress glial precursor proliferation via repression of NFIA during chick spinal cord development [J Neurosci] Abstract

Polydopamine-Assisted Osteoinductive Peptide Immobilization of Polymer Scaffolds for Enhanced Bone Regeneration by Human Adipose-Derived Stem Cells
Investigators report a biologically inspired strategy to prepare functional polymer scaffolds that efficiently regulate the osteogenic differentiation of human adipose-derived stem cells. [Biomacromolecules] Abstract

Adipose Tissue-Derived Stromal Stem Cell Therapy in Murine ConA-Derived Hepatitis Is Dependent on Myeloid-Lineage and CD4⁺ T-Cell Suppression
Researchers investigated the immunomodulatory effect of adipose tissue-derived stromal stem cells (ADSCs) on hepatitis using an acute ConA C57BL/6 murine hepatitis model. Intravenous administration of ADSCs simultaneously or 3 hours post injection prevented and treated ConA-induced hepatitis. [Eur J Immunol] Abstract

Human Umbilical Cord Mesenchymal Stem Cell Therapy for Patients with Active Rheumatoid Arthritis: Safety and Efficacy
Scientists assessed the safety and efficacy of human umbilical cord mesenchymal stem cells (UC-MSCs) in the treatment of rheumatoid arthritis (RA). In this ongoing cohort, 172 patients with active RA who had inadequate responses to traditional medication were enrolled. Patients were divided into two groups for different treatment: Disease modifying anti-rheumatic drugs (DMARDs) plus medium without UC-MSCs, or DMARDs plus UC-MSCs group (4×10⁷ cells per time) via intravenous injection. [Stem Cells Dev] Abstract

Adipose Tissue-Derived Stem Cells (ADSCs) Transplantation Promotes Regeneration of Expanded Skin Using a Tissue Expansion Model
Roles of ADSCs transplantation in expanded-skin regeneration have remained unknown. To clarify the roles, researchers used a tissue expansion model. The study comprised three groups of 13 rats in each group: the ADSCs group, the fibroblast group, and the control group. [Wound Repair Regen] Abstract

Enhancing In Vivo Survival of Adipose-Derived Stromal Cells through Bcl-2 Overexpression Using a Minicircle Vector
The Bcl-2 protein is a prosurvival member of a family of proteins that regulate the mitochondrial pathway of apoptosis. Using several strategies to overexpress the Bcl-2 protein, investigators demonstrated a decrease in the mediators of apoptosis in vitro and in vivo. This was shown through the use of two different clinical tissue repair models. [Stem Cell Transl Med] Abstract

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Grafix^® Demonstrates Overwhelming Efficacy in Landmark Stem Cell Study for the Treatment of Diabetic Foot Ulcers
Osiris Therapeutics, Inc. reported that its multi-center, randomized, controlled clinical trial comparing the safety and effectiveness of Grafix^® to standard of care in patients with chronic diabetic foot ulcers had met the pre-specified stopping rules for overwhelming efficacy as determined by the data monitoring committee during a planned interim analysis. [Osiris Therapeutics, Inc.] Press Release

$250 Million Regenerative Medicine Plan Part of Rudd Labor Health Policy
Scientists would be able to “grow” kidneys for patients under a plan by Prime Minister Kevin Rudd to develop regenerative medicine in Australia. Mr Rudd will outline his plan for the nation to play a leading role in the next frontier of healthcare in Brisbane, where he will announce a $250 million investment fund to take medical research breakthroughs from the lab to the patient. [Government of Australia] Press Release

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Stem Cell Research Lab to Open at Tehnopol
A stem cell research lab was opened at Tehnopol, a science park in Tallinn. In cooperation with the Cell Therapy Cluster, the Enterprise Estonia and EU-funded lab will be used to develop high-quality cell treatment products. [Estonian Public Broadcasting] Press Release

Cystinosis Research Foundation Awards $1.29 Million in Grants to Find a Cure for Deadly Genetic Disease
Six grants totaling $1,295,377 were issued to cystinosis researchers investigating better treatments and a cure for the disease by the Cystinosis Research Foundation in its first round of proposals in 2013. Included is funding for additional research on stem cell and gene therapy, which holds the greatest promise to cure the rare, deadly metabolic and genetic disease. [Marketwire, Inc.] Press Release

FDA Approves MSC-NP Therapy as Investigational New Drug in MS Clinical Trial: A Research Milestone
The stem cell research division of the Tisch MS Research Center of New York announced Food and Drug Administration (FDA) approval of autologous, mesenchymal stem cell-derived neural progenitor cells (MSC-NPs) as an Investigational New Drug for an open label, Phase I clinical trial in the treatment of multiple sclerosis. [Tisch MS Research Center of New York] Press Release

Alnylam Receives Orphan Drug Designation from U.S. Food & Drug Administration for ALN-AT3, an RNAi Therapeutic for the Treatment of Hemophilia
Alnylam Pharmaceuticals, Inc. announced that the U.S. Food & Drug Administration has granted an Orphan Drug Designation to ALN-AT3 as a therapeutic for the treatment of hemophilia B. Alnylam is developing ALN-AT3, a subcutaneously administered RNAi therapeutic targeting antithrombin, for the treatment of hemophilia – including hemophilia A, hemophilia B, and hemophilia A or B with “inhibitors” – and other Rare Bleeding Disorders. [Alnylam Pharmaceuticals, Inc.] Press Release

AlloSource Launches AlloMend: Allograft Offers New Possibilities in Soft Tissue Repair
AlloSource announced the launch of a new proprietary allograft, AlloMend Acellular Dermal Matrix. The new allograft can be used in a broad range of soft tissue surgical procedures, including ventral hernia repair and post-mastectomy reconstruction. [AlloSource] Press Release

MRI Interventions’ ClearPoint System Enables Precise Delivery of Promising Investigational Gene Therapy in Parkinson’s Disease Phase I Clinical Trial
MRI Interventions, Inc. announced treatment of the first patient in a Phase I clinical trial utilizing uniQure B.V.’s glial cell line-derived neurotrophic factor for treatment of Parkinson’s disease. [MRI Interventions, Inc.]
Press Release

Celldex Announces Expansion of Phase II ReACT Study in Recurrent Glioblastoma
Celldex Therapeutics, Inc. recently completed enrollment in an initial cohort of Avastin^® refractory patients in the company’s ongoing ReACT study of rindopepimut in EGFRvIII-positive glioblastoma. [Celldex Therapeutics, Inc.] Press Release

Medistem Announces Patent Application on Stem Cell Secreted Nanoparticles (Exosomes)
Medistem, Inc. announced the publication of a patent application entitled “Therapeutic Immune Modulation by Stem Cell Secreted Exosomes.” The patent application covers the use of stem cell derived nanoparticles, termed “exosomes”, for the treatment of autoimmune conditions, including Type 1 Diabetes, multiple sclerosis, rheumatoid arthritis, and lupus. [Medistem, Inc.] Press Release | Patent

Verastem Issued U.S. Patent Covering VS-4718 Composition of Matter
Verastem, Inc. announced that the U.S. Patent and Trademark Office issued U.S. Patent No. 8,501,763 claiming composition of matter for VS-4718, the Company’s novel, small molecule inhibitor of cancer stem cells through blockade of focal adhesion kinase. [Verastem, Inc.] Press Release

Arcturus Therapeutics Acquires Cutting-Edge UNA Patent Estate from Marina Biotech
Arcturus Therapeutics, Inc. announced it has acquired the patented portfolio of Unlocked Nucleobase Analog intellectual property from Marina Biotech. The use of UNAs in RNA interference to silence aberrant gene expression is an emerging and promising approach in the treatment of disease. [Arcturus Therapeutics, Inc.] Press Release

Prospective Grant of Exclusive License: Development of Brachyury Tumor Associated Antigens as Cancer Vaccines for Colorectal Cancer (FR Doc No: 2013-20057)

National Center for Advancing Translational Sciences; Notice of Meetings (FR Doc No: 2013-19899)

Clarification of the Scope for PAR-13-094 “Differentiation and Integration of Stem Cells (Embryonic and Induced-Pluripotent) into Developing or Damaged Tissues (R01)

NEW Clinical Translation of Stem Cells
April 21-22, 2014
Palm Desert, United States

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Research Technologist – Human Pluripotent Stem Cell Products (STEMCELL Technologies Inc.)

Postdoctoral Position – Neurosteroids and Neural Stem Cells in Developing Brain (Children’s National Medical Center)

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

Tenure-Track Position – Cell Biologist (McGill University)

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

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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