Cell Therapy News Volume 14.22 | Jun 10 2013

RD3 Gene Delivery Restores Guanylate Cyclase Localization and Rescues Photoreceptors in the Rd3 Mouse Model of Leber Congenital Amaurosis 12
To investigate the role of RD3 in photoreceptors and explore gene therapy as a potential treatment for Leber Congenital Amaurosis 12, scientists delivered adeno-associated viral vector with a Y733F capsid mutation and containing the mouse Rd3 cDNA under the control of the human rhodopsin kinase promoter to photoreceptors of 14 day old Rb(11.13)4Bnr/J and In (5)30Rk/J strains of rd3 mice by subretinal injections. [Hum Mol Genet] Abstract

Recovery From Experimental Parkinsonism by Semaphorin-Guided Axonal Growth of Grafted Dopamine Neurons
Scientists asked whether intranigral transplantation of dopamine (DA) neurons, combined with either degradation of chondroitin sulphate proteoglycans or with grafts of Semaphorin 3-expressing cells, towards the striatum, is effective in establishing a new nigrostriatal dopaminergic pathway in rats with unilateral depletion of DA neurons. They found depolarization-induced DA release in dorsal striatum, DA axonal projections from substantia nigra to striatum, and concomitant behavioral improvement in Semaphorin 3-treated animals. [Mol Ther] Abstract

Myeloid/Microglial Driven Autologous Hematopoietic Stem Cell Gene Therapy Corrects a Neuronopathiclysosomal Disease
The authors previously demonstrated improved neuropathology in mucopolysaccharidosistype IIIA (MPSIIIA) mice using lentiviral vectors (LV) overexpressing N-sulfoglucosaminesulfohydrolase in wild type hematopoietic stem cell transplants (LV-HSCT), achieved via donor monocyte/microglial engraftment in the brain. However, neurological disease was not corrected using LV in autologous MPSIIIA HSCT. In this study, to improve brain expression via monocyte/microglial specificity, LV expressing eGFP under ubiquitous PGK, or myeloid-specific promoters were compared in transplanted HSCs. [Mol Ther] Abstract

Pericytes Derived from Adipose-Derived Stem Cells Protect against Retinal Vasculopathy
Adipose-derived stem cells (ASCs) differentiate into pericytes, suggesting they may be useful as a protective and regenerative cellular therapy for retinal vascular disease. Researchers found that ASCs express pericyte-specific markers in vitro. When injected intravitreally into the murine eye subjected to oxygen-induced retinopathy, ASCs were capable of migrating to and integrating with the retinal vasculature. [PLoS One]
Full Article | Press Release

Adult Bone Marrow Neural Crest Stem Cells and Mesenchymal Stem Cells Are Not Able to Replace Lost Neurons in Acute MPTP-Lesioned Mice
Whereas the in vivo integration and differentiation of bone marrow stromal cells in neurons into the central nervous system is currently matter of debate, researchers report that once injected into the striatum of 1-methyl-4-phenyl-1,2,3,6-tetrahydropyridine(MPTP)-treated mice, pure populations of either bone marrow neural crest stem cells or mesenchymal stem cells survived only transiently into the lesioned brain. [PLoS One] Full Article

Systemic Treatment with Adipose-Derived Mesenchymal Stem Cells Ameliorates Clinical and Pathological Features in the Amyotrophic Lateral Sclerosis Murine Model
Researchers assessed the efficacy of the systemic administration of adipose-derived mesenchymal stem cells (ASC), a previously untested stem cell population, in superoxide-dismutase 1 (SOD1)-mutant transgenic mice, the animal model of familial amyotrophic lateral sclerosis. The administration of ASC to SOD1-mutant mice at the clinical onset significantly delayed motor deterioration for four-six weeks, as shown by clinical and neurophysiological tests. [Neuroscience] Abstract

Cyclosporine-Assisted Adipose-Derived Mesenchymal Stem Cell Therapy to Mitigate Acute Kidney Ischemia-Reperfusion Injury
Researchers tested the hypothesis that cyclosporine-supported syngeneic adipose-derived mesenchymal stem cell (ADMSC) therapy offered superior attenuation of acute ischemia-reperfusion (IR) kidney injury than either therapy alone. They concluded that combination therapy using cyclosporine-plus-ADMSCs offers improved protection against acute IR kidney injury. [Stem Cell Res Ther] Abstract | Full Article

Inflammatory Hypertrophic Cauda Equina following Intrathecal Neural Stem Cell Injection
The authors describe a woman who presented with progressive bilateral leg pain, numbness, and gait difficulties. A prior stroke, macular degeneration, osteoarthritis, and depression, led her to receive intrathecal neural stem cell therapy overseas one year prior to onset of symptoms. Imaging showed marked enlargement of lumbosacral roots of the cauda equina, which was not seen prior to stem cell treatment. Electrodiagnostic studies confirmed chronic multiple lumbosacral radiculopathies. [Muscle Nerve] Abstract

Growing Self-Organizing Mini-Guts from a Single Intestinal Stem Cell: Mechanism and Applications
Single Lgr5 stem cells derived from the intestine can be cultured to build epithelial structures that retain hallmarks of the in vivo epithelium. The authors review the mechanisms that support this notable example of self-organization and discuss applications of this technology for stem cell research, disease modeling (e.g., for colorectal cancer and cystic fibrosis), and regenerative medicine. [Science] Abstract

Cardiac Stem Cell Therapy and the Promise of Heart Regeneration
The authors consider how cardiac stem cell biology has led into clinical trials, and suggest that achieving true cardiac regeneration in patients may ultimately require resolution of critical controversies in experimental cardiac regeneration. [Cell Stem Cell] Abstract | Full Article

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

Generex Announces ASCO Presentations of AE37 Vaccine Data; Potential Leading Immunotherapy Option for Breast Cancer Patients
Generex Biotechnology Corporation announced two presentations showing the strengths of AE37 as a viable and promising treatment option for patients who have had breast cancer. The two presentations focused on the safety and long-term immunity that can be achieved with AE37 treatment. [Press release from Generex Biotechnology Corporation discussing research presented at the 2013 Annual Meeting of the American Society of Clinical Oncology (ASCO), Chicago] Press Release

Actinium Pharmaceuticals’ Acute Myeloid Leukemia Clinical Data Presented at 8^th International Symposium on Targeted Alpha Therapy Conference
Actinium Pharmaceuticals, Inc. announced a presentation devoted to its Acute Myeloid Leukemia clinical programs. Dr. Joseph Jurcic, delivered the oral presentation of the abstract entitled “Alpha-Particle Immunotherapy for Acute Myeloid Leukemia with Bismuth-213 and Actinium-225.” [Press release from Actinium Pharmaceuticals, Inc. discussing research presented at the 8^th International Symposium on Targeted Alpha Therapy Conference, Oak Ridge] Press Release

The Right to Care and the Expectations of Society-Controversial Stem Cell Therapy in Italy
A debate has started about a controversial stem cell therapy in Italy that exposes the difficult interaction between scientists, health care, doctors’ duties and patients’ rights. The case involves an experimental therapy based on adult mesenchymal stem cells that can differentiate into bone and connective tissue, and that were administered to a pediatric patient suffering from metachromatic leukodystrophy. [EMBO Reports] Press Release

US Scientists Chafe at Restrictions on New Stem-Cell Lines
The US National Institutes of Health, which distributes the majority of federal funding for stem-cell research, prohibits research on cells taken from embryos created solely for research – a category that includes the six stem-cell lines developed by Shoukhrat Mitalipov, a reproductive-biology specialist at the Oregon Health and Science University in Beaverton, and his colleagues. [National Institutes of Health] Press Release

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Surgeons at Duke University Hospital Implant Bioengineered Vein
In a first-of-its-kind operation in the United States, a team of doctors at Duke University Hospital helped create a bioengineered blood vessel and implanted it into the arm of a patient with end-stage kidney disease. The procedure, the first U.S. clinical trial to test the safety and effectiveness of the bioengineered blood vessel, is a milestone in the field of tissue engineering. [Duke University Health System] Press Release

Advancing Neuroscience: MRI Interventions, IMRIS and Tocagen Combine Technologies to Precisely Deliver Potential Cancer-Fighting Drug to Brain Tumors
MRI Interventions, Inc. and IMRIS Inc. announced that a surgical team at a leading U.S. neuroscience institution has delivered the investigational gene therapy drug Toca 511 directly into a glioblastoma brain tumor using real-time intraoperative MRI guidance within an IMRIS VISIUS^® Surgical Theatre. MRI Interventions’ ClearPoint^® Neuro Intervention System served as the navigation platform and the ClearPoint SmartFlow^® large-bore cannula as the vehicle for delivery of the therapeutic agent. [MRI Interventions, Inc.] Press Release

NeoStem’s Subsidiary, PCT, Enters Into a Manufacturing Agreement with ImmunoCellular Therapeutics, Ltd. for Dendritic Cell Vaccines Targeting Brain and Other Cancers
NeoStem, Inc., its subsidiary, Progenitor Cell Therapy LLC (PCT), and ImmunoCellular Therapeutics, Ltd. (IMUC), a clinical-stage biotechnology company that is developing immune-based therapies for the treatment of brain and other cancers, announced the execution of a Services Agreement under which PCT will provide current good manufacturing practices manufacturing services to support research and development of IMUC’s ICT-121 cell therapy product candidate, a dendritic cell vaccine targeting CD133 cells. [NeoStem Inc.]
Press Release

Cell Therapy Companies Make Strong Progress from October 2012 to March 2013 Amid Mixed Stock Market Sentiment
During Q4 2012 and Q1 2013, the cell therapy industry made strong progress in translation and commercialization. Continued development of the companies included in a dedicated stock market index suggests emergence of this industry as a distinct healthcare sector. [Cell Stem Cell] Summary

Sosei Announces Establishment of a Regenerative Medicine Fund
Sosei Group announced at the meeting of the Board of Directors that it resolved to invest in the regenerative medicine fund run by the newly-established subsidiary Sosei Corporate Venture Capital (Sosei CVC). Sosei Group will pursue a new venture through Sosei CVC. [Sosei Co. Ltd.] Press Release

Arcturus Therapeutics Raises $1.3 Million in Series Seed Funding
Arcturus Therapeutics, Inc., an industry leader in RNAi technologies for the treatment of disease, announced it has raised $1.3 million in a seed funding round led by multiple high net-worth private investors from the United States and Canada. [Arcturus Therapeutics, Inc.] Press Release

Allied’s CardioCel® $1.9M Grant from Commercialization Australia
Allied Healthcare Group announced that Commercialization Australia will soon announce that Allied’s CardioCel^® is the recipient of a $1.9M grant for the commercialization and launch of the groups lead regenerative tissue product CardioCel^®. CardioCel^® is the group’s lead regenerative tissue product for the repair and reconstruction of cardiovascular defects including repairing heart valves. [Allied Healthcare Group Ltd.] Press Release

SanBio Announces FDA Clearance for the Initiation of a Phase I/IIa Clinical Trial Testing Their Cell Therapy Product, SB623, in Patients with Traumatic Brain Injury
SanBio Inc. announced that the U.S. Food and Drug Administration (FDA) has approved their Investigational New Drug application for the use of SB623, a novel allogeneic stem cell therapy product, in patients suffering from traumatic brain injuries. This approval allows the company to proceed with a Phase I/IIa clinical trial testing the safety and feasibility of the therapy. The clinical trial is expected to be carried out at several major hospitals in the United States. [PR Newswire Association LLC] Press Release

Key Mesenchymal Precurser Cell Patent Granted in Japan
Mesoblast Limited announced that it has been granted a key patent by the Japanese Patent Office. Japanese patent number 5265190 provides Mesoblast with exclusive commercial rights in Japan through to September 2025 to all compositions-of-matter and uses of its Mesenchymal Precursor Cell (MPC) technology platform, irrespective of the MPC tissue source, including bone marrow, adipose, placenta, umbilical cord and dental pulp. [Mesoblast, Inc.] Press Release

Pluristem to Receive Clinical-Hold Notice from the U.S. FDA
Pluristem Therapeutics Inc. announced that it has received notification from the U.S. Food and Drug Administration (FDA) that its United States phase II Intermittent Claudication study (IND 15038) has been placed on clinical hold due to a serious allergic reaction in a case which required hospitalization. [Pluristem Therapeutics Inc.] Press Release

Fact Sheet: Impact of Sequestration on the National Institutes of Health
On March 1, 2013, as required by statute, President Obama signed an order initiating sequestration. The sequestration requires the National Institutes of Health (NIH) to cut 5 percent or $1.55 billion of its fiscal year 2013 budget. NIH must apply the cut evenly across all programs, projects, and activities, which are primarily NIH institutes and centers. This means every area of medical research will be affected. Press Release

National Cancer Institute; Notice of Closed Meetings (FR Doc No: 2013-13371)

Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2013-13618)

European Medicines Agency (United Kingdom)

Revised Guideline on Biosimilars Containing Biotechnology-Derived Proteins Published for Public Consultation

Food and Drug Administration (United States)

Draft Guidance for Industry on Human Immunodeficiency Virus-1 Infection: Developing Antiretroviral Drugs for Treatment; Availability (FR Doc No: 2013-13288)

Agency Information Collection Activities; Proposed Collection; Comment Request; Biological Products: Reporting of Biological Product Deviations and Human Cells, Tissues, and Cellular and Tissue-Based Product Deviations in Manufacturing; Forms FDA 3486 and 3486A (FR Doc No: 2013-13279)

Therapeutic Goods Administration

Revised Code of GMP for Human Blood and Tissues

NEW The Jackson Laboratory: Light Up Your Research – Use and Selection of Optogenetic Mouse Models
June 13, 1:00PM (ET in US)
Webinar

NEW ICCSCE 2013: International Conference on Cell and Stem Cell Engineering
September 26-27, 2013
Rome, Italy

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

NEW Postdoctoral Fellow/Research Scientist – Tissue Engineering (Institute of Bioengineering and Nanotechnology)

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

Postdoctoral Position – Developmental Neurobiology (North Carolina College of Veterinary Medicine)

Senior Postdoctoral Research Position – Molecular Cardiovascular and Renal Research (University of Oklahoma Health Sciences Center)

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

Senior Director of Regulatory Affairs (Celgene Cellular Therapeutics, Celgene Corporation)

Postdoctoral Position – Bone and Cartilage Tissue Engineering (University of California, Los Angeles)

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

Postdoctoral Research Fellow – Immunology (Beth Israel Deaconess Medical Center, Harvard Medical School)


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