Cell Therapy News Volume 17.18 | May 9 2016

Phase I Studies of Central-Memory-Derived CD19 CAR T Cell Therapy Following Autologous HSCT in Patients with B-Cell NHL
In two Phase I studies designed to improve long-term remission rates, investigators administered adoptive T-cell immunotherapy post-hematopoietic stem cell transplantation (HSCT), utilizing ex vivo-expanded autologous central-memory-enriched T cells transduced with lentivirus expressing CD19-specific chimeric antigen receptors (CARs). [Blood] Abstract

Extensive Graft-Derived Dopaminergic Innervation Is Maintained 24 Years after Transplantation in the Degenerating Parkinsonian Brain
Researchers describe the histopathological analysis of a unique patient with Parkinson’s disease who underwent unilateral cell transplantation in the putamen with human embryonic mesencephalic tissue at 24 years before death. [Proc Natl Acad Sci USA] Full Article | Press Release

Pervasive Supply of Therapeutic Lysosomal Enzymes in the CNS of Normal and Krabbe-Affected Non-Human Primates by Intracerebral Lentiviral Gene Therapy
Previous studies established lentiviral gene therapy as a rapid and effective intervention to provide pervasive supply of therapeutic lysosomal enzymes in CNS tissues of metachromatic leukodystrophy and globoid cell leukodystrophy mice. Here, researchers investigated whether this strategy is similarly effective in juvenile non-human primates. [EMBO Mol Med] Full Article

Chimeric Antigen Receptor T Cells Secreting Anti-PD-L1 Antibodies More Effectively Regress Renal Cell Carcinoma in a Humanized Mouse Model
Researchers used a single bicistronic lentiviral vector to develop a new combination immunotherapy that consists of human anti-carbonic anhydrase IX-targeted chimeric antigen receptor T cells engineered to secrete human anti-programmed death ligand 1 (PD-L1) antibodies at the tumor site. [Oncotarget] Full Article

Safe and Efficient Preclinical Gene Therapy for Pyruvate Kinase Deficiency
Researchers provided a preclinical gene therapy for pyruvate kinase deficiency (PKD) based on a lentiviral vector harboring the hPGK eukaryotic promoter that drives the expression of the PKLR cDNA. This therapeutic vector was used to transduce mouse PKD hematopoietic stem cells that were subsequently transplanted into myeloablated PKD mice. [Mol Ther] Abstract

Augmenter of Liver Regeneration Gene Therapy Using a Novel Minicircle DNA Vector Alleviates Liver Fibrosis in Rats
The authors evaluated augmenter of liver regeneration/ growth factor ERV1-like (ALR/GFER) gene therapeutic effect mediated by a novel minicircle vector (MC-hALR). The results in liver fibrotic rats that received MC-hALR through hydrodynamics-based transfection for eight weeks indicated that the minicircle DNA vector produced a more effective gene therapy effect than traditional plasmids. [Hum Gene Ther] Abstract

Haploidentical HCT Using an αβ T-Cell-Depleted Graft with Targeted αβ+ Cells by Add-Back After a Reduced Intensity Preparative Regimen Containing Low-Dose TBI
Between 2012 and 2015, 42 pediatric patients underwent haploidentical hematopoietic cell transplantation using an αβ+ T-cell-depleted graft with targeted αβ cells at 1–5 × 105/kg by add-back; 31 had hematologic malignancy, 8 had non-malignant disease and 3 had solid tumors. [Bone Marrow Transplant] Abstract

Autologous CD34⁺ Cell Therapy for Refractory Angina: 2 Year Outcomes from the ACT34-CMI Study
In ACT34-CMI, patients treated with autologous CD34⁺ stem cells had improvements in angina and exercise time at six and twelve months compared to placebo; however, the longer-term effects of this treatment are unknown. [Cell Transplant] Full Article

Periodontal Wound Healing by Transplantation of Jaw Bone Marrow-Derived Mesenchymal Stem Cells in Chitosan/Anorganic Bovine Bone Carrier into One-Wall Infrabony Defects in Beagles
Scientists evaluated the performance of chitosan/anorganic bovine bone scaffold seeded with human jaw bone marrow-derived mesenchymal stem cells in supporting the healing/repair of one-wall critical size periodontal defects. [J Periodontol] Abstract

Inhibitory Effect of Genetically Engineered Mesenchymal Stem Cells with Apoptin on Hepatoma Cells In Vitro and In Vivo
Researchers constructed recombinant adenovirus which expresses Apoptin and then infects human bone marrow mesenchymal stem cells (MSCs), and explored the migration of MSCs to the hepatoma cells and inhibitory effect of genetically engineered MSCs with Apoptin on hepatoma cells in vitro and in vivo. [Mol Cell Biochem] Abstract

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

Intraventricular Transplantation of Autologous Bone Marrow Mesenchymal Stem Cell in Hemorrhagic Stroke
Winner of the American Association of Neurological Surgeons (AANS) International Travel Scholarship, Asra Al Fauzi, MD, IFAANS, presented his research, “Intraventricular Transplantation of Autologous Bone Marrow Mesenchymal Stem Cell in Hemorrhagic Stroke”. [Press release from the American Association of Neurological Surgeons (AANS) (NewsWise) discussing research presented at the 2016 AANS Annual Scientific Meeting, Chicago] Press Release

First Skin-to-Eye Stem Cell Transplant in Humans Successful
Researchers have safely transplanted stem cells derived from a patient’s skin to the back of the eye in an effort to restore vision. [Press release from the Association for Research in Vision and Ophthalmology (ARVO) (NewsWise) discussing research presented at the 2016 ARVO Annual Meeting, Seattle] Press Release

Oncolytics Biotech^® Inc. Announces Presentation of REOLYSIN® Preclinical Research
Oncolytics Biotech^® Inc. announced a poster presentation by researchers, covering preclinical work in squamous cell carcinoma of the head and neck. [Press release from Oncolytics Biotech^® Inc. discussing research presented at the 19^th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), Washington, DC.] Press Release

Asterias Biotherapeutics Announces Oral Presentation
Asterias Biotherapeutics, Inc. announced that clinical data from its AST-VAC1 (antigen-presenting autologous dendritic cells) immunotherapy clinical program will be presented during an oral session. [Press release from Asterias Biotherapeutics discussing research presented at the 19^th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), Washington, DC.] Press Release

Gene Therapy Halts Rare Brain Disease in Young Boys
In a new study, all but one of 17 boys with adrenoleukodystrophy remained relatively healthy for up to two years after having an engineered virus deliver into their cells a gene to replenish a missing protein needed by the brain. [Press release from ScienceInsider discussing research presented at the 19^th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), Washington, DC.] Press Release

Follistatin Gene Therapy Increases Function in Inclusion Body Myositis Patients
Milo Biotechnology described promising safety and initial efficacy data in sporadic inclusion body myositis (sIBM) patients treated with follistatin gene therapy. In total, nine sIBM patients received follistatin gene therapy treatments and have been followed up to four years. [Press release from Milo Biotechnology discussing research presented at the 19^th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), Washington, DC] Press Release

Processed Fat Cells Show Potential as Treatment for Refractory Ischemia Patients
Patients treated with processed autologous adipose-derived regenerative cells injected into the heart muscle demonstrated symptomatic improvement and a trend towards lower rates of heart failure hospitalizations and angina, despite no improvement in left ventricle ejection fraction or ventricular volumes. [Press release from the Society for Cardioovascular Angiography and Interventions (SCAI) discussing research presented at the 2016 SCAI Scientific Sessions, Orlando] Press Release

BEAT BioTherapeutics Announces Scientific Presentation
BEAT BioTherapeutics Corporation announced that co-founder and Scientific Advisory Board member Dr. Michael Regnier presented “AAV6-Mediated Cardiac-Specific Overexpression of Ribonucleotide Reductase Enhances Myocardial Contractility via Myosin Activation.” [Press release from BEAT BioTherapeutics (Business Wire) discussing research to be presented at the 19^th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), Washington, DC] Press Release

Stem Cell Therapy Shows Potential for Difficult-to-Treat RA Patient Population
A study using a stem cell therapy to treat challenging refractory angina (RA) patients demonstrated promising results, including improved exercise time, reduced angina and reduced mortality. The RENEW results were presented as a late-breaking clinical trial. [Press release from the Society for Cardiovascular Angiography and Interventions (SCAI) discussing research to be presented at the 2016 SCAI Scientific Sessions, Orlando] Press Release

Embryology Policy: Revisit the 14-Day Rule
Studies of human development in vitro are on a collision course with an international policy that limits embryo research to the first two weeks of development, warn Insoo Hyun, Amy Wilkerson and Josephine Johnston. [Nature News & Comment] Editorial

In Survey, Most Give Thumbs-Up to Pirated Papers
Last week, a feature article in Science analyzed 28 million download requests over 6 months from Sci-Hub, a popular repository of pirated scientific literature. The data showed widespread use of Sci-Hub around the world, in countries rich and poor, and in all fields of science. [ScienceInsider] Editorial

France’s Research Minister Lays out His Priorities
Thierry Mandon on cutting red tape, French researchers’ self-effacement and shaking up social science. [Nature News: Q&A] Editorial

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NIH Funds Pitt Study of Culture-Free Fat Cell-Based Vascular Grafts in Cardiovascular Disease Patients
The National Institutes of Health (NIH) have awarded David Vorp with a two-year, $417,838 R21 grant for research into the use of cells from a patient’s own adipose as vascular grafts in arterial bypass surgery. [University of Pittsburgh] Press Release

ImmunoCellular Therapeutics Receives Regulatory Approval in Canada, the UK and the Netherlands to Initiate ICT-107 Phase III Trial in Newly Diagnosed Glioblastoma
ImmunoCellular Therapeutics, Ltd. announced that the company has received approval from regulatory authorities in Canada, the United Kingdom and the Netherlands to initiate the ICT-107 Phase IIII registration trial in patients with newly diagnosed glioblastoma. [ImmunoCellular Therapeutics, Ltd.] Press Release

TG Therapeutics, Inc. Launches First Clinical Trial in Multiple Sclerosis (MS) for TG-1101 (Ublituximab)
TG Therapeutics, Inc. announced the opening of its Phase II clinical study of ublituximab, the company’s glycoengineered anti-CD20 monoclonal antibody, in patients with relapsing remitting MS, a chronic demyelinating disease of the central nervous system. [TG Therapeutics, Inc.] Press Release

Agenus Commences Phase I Clinical Trial of Its CTLA-4 Checkpoint Antibody to Treat Solid Tumors
Agenus Inc. announced that the first patient has been dosed in the company’s Phase I clinical trial of AGEN1884, an anti-CTLA-4 checkpoint antibody. The open-label, multicenter trial in patients with advanced or refractory cancer is designed to evaluate the safety of AGEN1884 and determine the estimated maximum tolerated dose. [Agenus Inc.] Press Release

Celsion Corporation Announces Positive Data from the First Cohort of Patients in the OVATION Study
Celsion Corporation announced data from the first cohort of patients in its Phase Ib dose escalating clinical trial (the OVATION Study) combining GEN-1, the company’s DNA-based immunotherapy, with the standard of care for the treatment of newly-diagnosed patients with advanced ovarian cancer who will undergo neoadjuvant chemotherapy followed by interval debulking surgery. [Celsion Corporation] Press Release

University of Wisconsin and Harvard Scientists Receive Awards from ASNTR
At the 23rd Annual Conference of the American Society of Neural Therapy and Repair (ASNTR), held April 28-30, 2016 in Clearwater Beach, Florida, ASNTR awarded The 2016 Bernard Sanberg Memorial Award for Brain Repair to Marina E. Emborg, MD, PhD, an associate professor of medical physics at the University of Wisconsin-Madison. ASNTR also presented The Molly and Bernard Sanberg Memorial Award to Eng H. Lo, PhD, professor of neurology and radiology at Harvard University and Massachusetts General Hospital. [University of Wisconsin (EurekAlert!)] Press Release

Axiogenesis Secures Major Funding for its Expansion Course
Axiogenesis has acquired a major investment from Sino-German High-Tech Fund to further expand product development, strengthen its disease modeling capabilities and increase market presence. Axiogenesis’ current product portfolio includes several validated induced pluripotent stem cell derived cardiac and neural cell types. [Axiogenesis AG (PR Newswire Association LLC)] Press Release

National Heart, Lung, and Blood Institute Notice of Closed Meeting (FR Doc. No: 2016-10456)

Chronic Hepatitis C Virus Infection: Developing Direct-Acting Antiviral Drugs for Treatment; Draft Guidance for Industry; Availability (FR Doc. No: FDA-2013-D-1170)

Medical Devices; Exemption From Premarket Notification: Method, Metallic Reduction, Glucose (Urinary, Non-Quantitative) Test System in a Reagent Tablet Format (FR Doc. No: FDA-2016-P-0159)

Natural History Studies for Rare Disease Product Development: Orphan Products Research Project Grant (R01) (FR Doc. No: FDA-2016-N-0012)

NEW Postdoctoral Fellow – Gene Therapy for Metabolic Homeostasis (Legacy REsearch Institute)

Scientist – Bioengineering (STEMCELL Technologies Inc.)

Scientist – Human Pluripotent Stem Cell Neural Differentiation (STEMCELL Technologies Inc.)

PhD Studentship – Various Projects (Vienna Biocenter)

Postdoctoral Fellow – Stem Cell Models of Parkinson’s (University of Oxford)

Group Leader – Development and Stem Cells (Monash University)

Postdoctoral Fellow – Immunology (The Pirbright Institute)

Director – Stem Cells (PharmaScouts)

Assistant Professor – Cell Factory Design (Chalmers University of Technology)

Postdoctoral Fellow – Non-Viral Gene Delivery (Nanyang Technological University)

Project Lead – Engineering (Cytonome)

Faculty Position – Tumor Immunology (John Wayne Cancer Institute at Providence Saint John’s Health Center)

Postdoctoral Fellow – Non-Coding RNAs in Malignant Progression (The University of Texas MD Anderson Cancer Center)

Postdoctoral Associate – Genetic Medicine (Cornell University)

Postdoctoral Fellow – Retinal Disease Genomics and Gene Therapy (Ocular Genomics Institute, Mass Eye and Ear and Harvard Medical School)

Associate Scientist – Hematopoietic Stem Cell Biology (Editas Medicine)

Research Associate – Hematopoietic Stem Cell Biology (Editas Medicine)

Postdoctoral Fellow – Hematology/Oncology (Boston Children’s Hospital)

Postdoctoral Position – Hematology/Oncology (Penn State College of Medicine)

Staff Scientist – Cell Therapy Process Development (Fred Hutchinson Cancer Research Center)

Postdoctoral Research Fellow – Cell Cycle Control and Tumorigenesis (Fred Hutchinson Cancer Research Center)

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