Cell Therapy News Volume 14.21 | Jun 3 2013

Significant Improvement in Survival after Allogeneic Hematopoietic Cell Transplantation during a Period of Significantly Increased Use, Older Recipient Age, and Use of Unrelated Donors
Over the past four decades, allogeneic hematopoietic cell transplantation has evolved as a curative modality for patients with hematologic diseases. Researchers described changes in use, technique, and survival in a population-based cohort. [J Clin Oncol] Abstract | Press Release

Positive Selection of mC46-Expressing CD4⁺ T Cells and Maintenance of Virus Specific Immunity in a Primate AIDS Model
In 2009, an HIV⁺-patient was effectively cured of HIV following allogeneic transplantation of hematopoietic stem cells from a CCR5^-/- donor. The utility of this approach, however, is severely limited due to the difficulties in finding matched donors. Researchers studied the potential of HIV/SHIV-resistant stem cells in the autologous setting in a nonhuman primate AIDS model and incorporated a fusion inhibitor (mC46) as the means for developing infection-resistant cells. [Blood] Abstract

Innate Pro-B-Cell Progenitors Protect against Type 1 Diabetes by Regulating Autoimmune Effector T Cells
Investigators provided evidence for innate pro-B cells that emerged within the bone marrow both in vitro and in vivo upon Toll-like receptor-9 activation and whose adoptive transfer protected nonobese diabetic mice against type 1 diabetes. [Proc Natl Acad Sci USA] Abstract

Intranasal Antibody Gene Transfer in Mice and Ferrets Elicits Broad Protection against Pandemic Influenza
Adeno-associated virus (AAV) vectors based on serotype 9 were engineered to express a modified version of the previously isolated broadly neutralizing monoclonal antibody to influenza A, FI6. Researchers demonstrated that intranasal delivery of AAV9.FI6 into mice afforded complete protection and log reductions in viral load to 100 LD₅₀ (median lethal dose) of three clinical isolates of H5N1 and two clinical isolates of H1N1, all of which have been associated with historic human pandemics (including H1N1 1918). [Sci Transl Med]
Abstract | Press Release

Synergistic Effects of GDNF and VEGF on Lifespan and Disease Progression in a Familial ALS Rat Model
Human mesenchymal stem cells (hMSCs) engineered to secrete glial cell line-derived neurotrophic factor (GDNF) (hMSC-GDNF), vascular endothelial growth factor (hMSC-VEGF), insulin-like growth factor-I (hMSC-IGF-I), or brain-derived neurotrophic factor (hMSC-BDNF), were prepared and transplanted bilaterally into three muscle groups. hMSC-GDNF and hMSC-VEGF prolonged survival and slowed the loss of motor function, but hMSC-IGF-I and hMSC-BDNF did not have any effect. [Mol Ther] Abstract | Press Release

Mediation of Protection and Recovery from Experimental Autoimmune Encephalomyelitis by Macrophages Expressing the Human Voltage-Gated Sodium Channel NaV1.5
Investigators developed a novel transgenic mouse model, in which the human macrophage NaV1.5 splice variant is expressed in vivo in mouse macrophages. These mice were protected from experimental autoimmune encephalomyelitis, the mouse model of multiple sclerosis. NaV1.5-positive macrophages that were adoptively transferred into wild-type recipients with established experimental autoimmune encephalomyelitis homed to lesions and promoted recovery. [J Neuropathol Exp Neurol] Abstract | Press Release

Amelioration of Motor/Sensory Dysfunction and Spasticity in a Rat Model of Acute Lumbar Spinal Cord Injury by Human Neural Stem Cell Transplantation
Researchers characterized the effect of intraspinal grafting of clinical grade human fetal spinal cord-derived neural stem cells on the recovery of neurological function in a rat model of acute lumbar compression injury. [Stem Cell Res Ther] Abstract | Press Release

Randomized, Controlled Phase II Study of Post-Surgery Radiotherapy Combined with Recombinant Adenoviral Human p53 Gene Therapy in Treatment of Oral Cancer
Scientists evaluated clinical benefits of recombinant adenoviral human p53 gene therapy combined with radiotherapy in prevention of oral cancer recurrence after a radical resection. [Cancer Gene Ther] Abstract

Combined Delivery of Bone Marrow-Derived Mononuclear Cells in Chronic Ischemic Heart Disease: Rationale and Study Design
Researchers aimed to demonstrate that the combination of intramyocardial and intracoronary injection of bone marrow-derived mononuclear cells is safe and improves left ventricular function in patients with chronic ischemic heart disease. [Clin Cardiol] Full Article

Transient Proteolytic Modification of Mesenchymal Stromal Cells Increases Lung Clearance Rate and Targeting to Injured Tissue
One of the main obstacles especially in intravenous delivery of cells is a massive cell retention in the lungs, which impairs homing to the target tissue and may decrease the therapeutic outcome. Investigators showed that an alternative cell detachment of mesenchymal stromal/stem cells with pronase instead of trypsin significantly accelerated the lung clearance of the cells and, importantly, increased their targeting to an area of injury. [Stem Cells Transl Med] Abstract

The Role of Gene Therapy in Regenerative Surgery: Updated Insights
In the past two decades, regenerative surgeons have focused increasing attention on the potential of gene therapy for treatment of local disorders and injuries. Gene transfer techniques may provide an effective local and short-term induction of growth factors without the limits of other topical therapies. In 2002, Tepper and Mehrara accurately reviewed the topic: given the substantial advancement of research on this issue, an updated review is provided. [Plast Reconstr Surg] Abstract | Press Release

Mesenchymal Stem Cells as Vectors for Lung Cancer Therapy
Despite recent advances in treatment, lung cancer accounts for one third of all cancer-related deaths, underlining the need of development of new therapies. Mesenchymal stem cells (MSCs) possess the ability to specifically home into tumors and their metastases. This review outlines various agents which can be delivered by MSCs with substantial emphasis on TRAIL (tumor necrosis factor-related apoptosis-inducing ligand). [Respiration]
Full Article

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

Phase I/II Data of VB-111 Demonstrate Anti-Tumor Effects with Increase in Overall Survival with This Anti-Angiogenic Gene Therapy, in Patients with Recurrent Glioblastoma Multiforme
Data from the trial evaluating the effect of VB-111 on 28 patients with recurrent Glioblastoma Multiforme demonstrate that VB-111 was safe and well tolerated with repeat doses of up to 1×10¹³ viral particles. Tumor responses and significant attenuation of tumor growth rate were seen. VB-111 is a novel anti-angiogenic gene-therapy tool that targets endothelial cells in the tumor vasculature. [Press release from VBL Therapeutics discussing research presented at the Annual Meeting of the American Society of Clinical Oncology, Chicago] Press Release

Nektar Presents Positive Preclinical Data for NKTR-214, a Novel Cancer Immunotherapy
Nektar Therapeutics announced positive preclinical data for NKTR-214, an investigational cancer immunotherapy which targets the IL-2 receptor complex and is being developed as a potential treatment for multiple cancers. [Press release from Nektar Therapeutics discussing research presented at the Annual Meeting of the American Society of Clinical Oncology, Chicago] Press Release

Dendreon Announces Presentation of PROVENGE® (sipuleucel-T) Data
Dendreon Corporation announced that four abstracts featuring PROVENGE® (sipuleucel-T) data from ongoing Phase II sequencing studies and the PROCEED registry, and two abstracts highlighting preliminary Phase II data of DN24-02, an investigational active cellular immunotherapy in patients with surgically resected HER2+ urothelial cancer, will be presented. [Press release from Dendreon Corporation discussing research to be presented at the Annual Meeting of the American Society of Clinical Oncology, Chicago] Press Release

Top European Life Science Institutes Join Forces
Ten top European life sciences institutes have launched a new alliance to help themselves become more competitive and have a strong, unified voice in European science policy. [EU-Life, European Union]
Press Release

Japan Aims High for Growth
Prime Minister Shinzo Abe is riding high since his Liberal Democratic Party swept back to power. He has stimulated the economy, ending 15 years of deflation, jumpstarted the stock market and weakened the yen to spur exports. His reform platform includes a new growth strategy, and central to that strategy is innovation in science and technology. [Prime Minister, Japan] Press Release

Russian Academy of Sciences Elects Reformer as President
The Russian Academy of Sciences (RAS) has elected itself a new president. Yuri Osipov, who has headed RAS since the collapse of the Soviet Union, has stepped down and RAS members chose Vladimir Fortov, head of the RAS Joint Institute for High Temperatures in Moscow, to replace him. [Russian Academy of Sciences, Russia] Press Release

France’s Message to Science: Help Us Fix the Economy
The National Assembly approved a new law that aims to simplify the national landscape for research and higher education and make it more efficient, better able to address societal and economic challenges, and more competitive at the European level. The bill, which comes hand in hand with a new strategic plan for France’s research priorities, also gives the government a greater role in coordinating research. The bill and the road map have been sharply criticized by various groups of researchers and university professors. [National Assembly, France] Press Release

Research Funders Propose Steps to Promote Open Access
A group of research funding organizations from around the world today put its weight behind open access to the scientific literature but stopped short of making concrete policy recommendations for its members. [Global Research Council] 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) announced the execution of a Services Agreement under which PCT will provide cGMP (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

Johannes Gutenberg University Mainz Obtains New Collaborative Research Center on “Nanodimensional Polymer Therapeutics for Tumor Therapy”
The German Research Foundation has established a new Collaborative Research Center on “Nanodimensional polymer therapeutics for tumor therapy” (CRC 1066) at Johannes Gutenberg University Mainz and the Max Planck Institute for Polymer Research. Starting in October 2013, the Collaborative Research Center will receive grants totaling approximately EUR 11 million over four years to develop a nanoparticle-based cancer therapy to combat melanoma as an immunogenic tumor model. The Mainz scientists will focus on a form of cancer immunotherapy that is specifically suitable for permanently eliminating minimal residual disease, such as hidden metastases. [Johannes Gutenberg-Universität Mainz] Press Release

Grant Funds Research into Cardiac Stem Cells as Treatment for Heart Disease
Newport Beach-based nonprofit Coalition Duchenne has awarded a $150,000 grant to a Cedars-Sinai Heart Institute team investigating whether an experimental cardiac stem cell treatment could be used to treat Duchenne muscular dystrophy patients who have developed heart disease. [Cedars-Sinai] Press Release

Bioheart Announces First Patients Successfully Enrolled and Treated in Angel Trial
Bioheart, Inc. announced that they have successfully enrolled and treated the first patients in the ANGEL Trial. This phase I ANGEL study will provide necessary safety and preliminary efficacy of adipose derived stem cells in patients with congestive heart failure. [Bioheart, Inc.] Press Release

StemCells, Inc.’s Spinal Cord Injury Trial Approved by Health Canada
StemCells, Inc. announced that Health Canada has authorized the Company to expand its Phase I/II clinical trial for chronic spinal cord injury into Canada. The Phase I/II trial, currently underway in Zurich, Switzerland, is designed to evaluate the safety and preliminary efficacy of the Company’s proprietary HuCNS-SC® product candidate (purified human neural stem cells) as a treatment for chronic spinal cord injury. [StemCells, Inc.]
Press Release

FDA-Approved Clinical Trial to Test Effectiveness of Fat-Derived Regenerative Cells on Heart Failure Patients
Doctors at the Texas Heart Institute at St. Luke’s Episcopal Hospital announced that they have performed an investigational procedure on six patients in a new FDA-approved clinical trial to evaluate therapy that uses regenerative cells derived from a patients’ own adipose tissue to treat a severe form of heart failure. [Texas Heart Institute] Press Release

Arrowhead Files to Begin Phase I Trial of RNAi Therapeutic ARC-520 against Chronic Hepatitis B Infection
Arrowhead Research Corporation announced that it recently filed an application for approval to begin a phase I clinical trial of its RNAi-based therapeutic candidate, ARC-520, against chronic hepatitis B virus infection. [Arrowhead Research Corporation] Press Release

Licenses Granted for the Manufacture of Human Cell Therapy Products
Roslin Cells Ltd, the Scottish National Blood Transfusion Service and the University of Edinburgh announced that the GMP Cellular Therapy Manufacturing Facility within the Scottish Centre for Regenerative Medicine has recently been licensed by the Medicines and Healthcare products Regulatory Agency for the manufacture of human cell therapy products. [Scottish National Blood Transfusion Service] Press Release

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Draft Guidance for Industry: Changes to an Approved Application: Biological Products: Human Blood and Blood Components Intended for Transfusion or for Further Manufacture

May 30, 2013 Approval Letter – ALLOCORD

European Medicines Agency (United Kingdom)

Revised Version of Application Form for Initial Evaluation of Human Medicines Available

Food and Drug Administration (United States)

Agency Information Collection Activities; Proposed Collection; Comment Request; Requirements for Submission of Labeling for Human Prescription Drugs and Biologics in Electronic Format (FR Doc No: 2013-12825)

NEW The Jackson Laboratory: Seeds of Hope – New and Emerging Mouse Models of Parkinson’s Disease
June 6, 1:00PM (ET in US)
Webinar

NEW Hydra IX Summer School on Stem Cell Biology
September 7-14, 2013
Hydra, Greece

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