Cell Therapy News Volume 17.25 | Jul 4 2016

TOP STORY

New Therapy Treats Autoimmune Disease without Harming Normal Immunity

In a study with potentially major implications for the future treatment of autoimmunity and related conditions, scientists from have found a way to remove the subset of antibody-making cells that cause an autoimmune disease, without harming the rest of the immune system. [Press release from the University of Pennsylvania discussing online prepublication in Science] Press Release | Abstract

(Ranked by impact factor of the journal)

Generation of a Nonhuman Primate Model of Severe Combined Immunodeficiency using Highly Efficient Genome Editing

The authors describe the generation of a marmoset model of severe combined immunodeficiency. Their study optimized zinc-finger nucleases and transcription activator-like effector nucleases to target interleukin-2 receptor subunit gamma in pronuclear stage marmoset embryos. [Cell Stem Cell] Abstract | Graphical Abstract

AAV9 Delivery of ShRNA to the Mouse Heart

The authors describe protocols for use of recombinant adeno-associated viruses (rAAVs) as a cargo to deliver miRNA backbone-based shRNA controlled by a cardiac-specific promoter into the mouse heart. [Curr Protoc Mol Biol] Abstract

Allogeneic Mesenchymal Stem Cell Therapy Promotes Osteoblastogenesis and Prevents Glucocorticoid-Induced Osteoporosis

Scientists investigated whether systemic infusion of allogeneic mesenchymal stem cells without genetic manipulation could prevent glucocorticoid-induced murine osteoporosis, whether anabolic and anticatabolic effects existed, and whether homing or immunomodulation underlay the putative therapeutic effects. [Stem Cells Transl Med] Abstract

Efficient Production of Multi-Modified Pigs for Xenotransplantation by ‘Combineering’, Gene Stacking and Gene Editing

Investigators used two methods to colocate xenoprotective transgenes at one locus, sequential targeted transgene placement – ‘gene stacking’, and cointegration of multiple engineered large vectors – ‘combineering’, to generate pigs carrying modifications considered necessary to inhibit short to mid-term xenograft rejection. [Sci Rep] Full Article

Identification of Adeno-Associated Viral Vectors (AAV) That Target Neonatal and Adult Mammalian Inner Ear Cell Subtypes

AAVs have become the vector of choice for gene delivery in animal models in vivo. To identify AAV vectors that target inner ear cell subtypes, researchers systemically screened 12 AAV vectors with different serotypes including AAV1, 2, 5, 6, 6.2, 7, 8, 9, rh.8, rh.10, rh.39 and rh.43 that carry a reporter gene GFP in neonatal and adult mice by microinjection in vivo. [Hum Gene Ther] Abstract

Autologous Stem Cell Transplant for Multiple Myeloma Patients 70 Years or Older

Autologous stem cell transplant (Auto-SCT) is increasingly used in older patients with multiple myeloma (MM), despite lack of Phase III trials in this age-defined population. For 207 consecutive MM patients who underwent Auto-SCT and were 70 years or older at transplant, data were analyzed and compared with a younger cohort. [Bone Marrow Transplant] Abstract

Inhibitory Effect of Endostatin Gene Therapy Combined with Phosphorus-32 Colloid on Tumor Growth in Wistar Rats

The effect of endostatin combined with a small dose of ³²P-colloidal on tumour growth in vivo was evaluated and the potential mechanism underlying the combined therapy was explored. The authors found that ³²P-colloid combined with endostatin exhibited higher inhibitory effect upon tumour growth compared with application of ³²P-colloid or endostatin alone, although three therapies all significantly inhibited tumour growth compared with saline control group. [Biosci Rep] Full Article

Synthesis of and In Vitro and In Vivo Evaluation of a Novel TGF-β1-SF-CS Three-Dimensional Scaffold for Bone Tissue Engineering

The role of transforming growth factor-β1 (TGF-β1) in normal human fracture healing has been previously demonstrated. Investigators examined the biocompatibility of TGF-β1-silk fibroin-chitosan (TGF-β1-SF-CS) three-dimensional scaffolds in order to construct an ideal scaffold for bone tissue engineering. [Int J Mol Med] Abstract

Chondroitinase Administration and pcDNA3.1-BDNF-BMSC Transplantation Promote Motor Functional Recovery Associated with NGF Expression in Spinal Cord-Transected Rat

Scientists evaluated whether a combination of chondroitinase administration and brain-derived neurotrophic factor (BDNF)-mesenchymal stem cell transplantation could provide an optimal effect for the treatment of spinal cord injury subjected to complete transection. [Spinal Cord] Abstract

Multilineage Transduction of Resident Lung Cells In Vivo by AAV2/8 for α1-Antitrypsin Gene Therapy

Researchers describe sustained pulmonary transgene expression for at least 52 weeks after a single intratracheal instillation of adeno-associated virus (AAV)2/8 and characterize the multiple cell types transduced within the lung utilizing this approach. They demonstrated that lung-directed AAV2/8 was able to achieve therapeutic α-1 antitrypsin (AAT) protein levels within the lung epithelial lining fluid and that AAT gene delivery ameliorated the severity of experimental emphysema in mice. [Mol Ther Methods Clin Dev] Full Article

Widespread AAV1- and AAV2-Mediated Transgene Expression in the Nonhuman Primate Brain: Implications for Huntington’s Disease

The use of RNA interference to inhibit huntingtin expression could represent a disease-modifying therapy. The potential of two recombinant adeno-associated viral vectors (AAV), AAV1 and AAV2, to transduce the cortico-striatal tissues that are predominantly affected in Huntington’s disease was explored. [Mol Ther Methods Clin Dev] Full Article

Stem Cell Therapies in Clinical Trials: Progress and Challenges

Clinical investigations using stem cell products in regenerative medicine are addressing a wide spectrum of conditions using a variety of stem cell types. To date, there have been few reports of safety issues arising from autologous or allogeneic transplants. [Cell Stem Cell] Full Article

Liver Regeneration — Mechanisms and Models to Clinical Application

The reviewers describe the mechanisms underpinning liver regeneration, the models used to study this process, and discusses areas in which failed or compromised liver regeneration is clinically relevant. [Nat Rev Gastroenterol Hepatol] Abstract

Acute Myeloid Leukemia: A Comprehensive Review and 2016 Update

Despite advances in supportive care, the backbone of therapy remains a combination of cytarabine- and anthracycline-based regimens with allogeneic stem cell transplantation for eligible candidates. Elderly patients are often unable to tolerate such regimens, and carry a particularly poor prognosis. The authors review the major recent advances in the treatment of acute myeloid leukemia. [Blood Cancer J] Full Article

Visit our reviews page to see a complete list of reviews in the cell therapy research field.

BioTime Presents Online Resource for Applying Artificial Intelligence to Stem Cell Biology at Mensa 2016 Annual Gathering

BioTime, Inc. announced that Co-CEO Dr. Michael D. West, will give a lecture at the Mensa Annual Gathering titled, “Hayflick Rewound: Implications of Reversing the Aging of Human Cells.” Dr. West discusses the impact of aging on society, as well as fundamental advances in understanding its causes and strategies to intervene using regenerative medicine. [Press release from BioTime, Inc. discussing research presented at the 2016 Mensa Annual Gathering, San Diego] Press Release

Bristol-Myers Squibb and PsiOxus Therapeutics Announce Immuno-Oncology Clinical Collaboration to Evaluate the Combination of Opdivo and Enadenotucirev

Bristol-Myers Squibb Company and PsiOxus Therapeutics, Ltd. announced an exclusive clinical collaboration agreement to evaluate the safety, tolerability, and preliminary efficacy of PsiOxus’ enadenotucirev, a systemically administered oncolytic adenovirus therapeutic, in combination with Bristol-Myers Squibb’s Immuno-Oncology agent Opdivo (nivolumab) to treat a range of tumor types in late-stage cancer patients. [Bristol-Myers Squibb Company] Press Release

Helen F. Graham Cancer Center & Research Institute and The Wistar Institute Partner to Expand Research in Gene Editing

To accelerate breakthrough cancer research in the human genome, the Gene Editing Institute at Christiana Care’s Helen F. Graham Cancer Center & Research Institute has entered into an agreement with The Wistar Institute. [Christiana Care Health System] Press Release

VM BioPharma Announces First Patient Dosed in Phase III Study of Gene Therapy Candidate, VM202, in Painful Diabetic Peripheral Neuropathy

VM BioPharma, the United States division of ViroMed Co., Ltd. in Seoul, Korea announced the first patient was dosed in the recently initiated Phase III clinical study evaluating VM202, a proprietary DNA based biopharmaceutical, in patients with painful diabetic peripheral neuropathy. [ViroMed Co., Ltd.] Press Release

Celyad Announces Results for the CHART-1 Phase III Clinical Trial Evaluating C-Cure^® Cell Therapy

Celyad announced headline results for CHART-1, its European Phase III clinical trial for its lead cardiovascular disease product candidate. [Celyad] Press Release

Biocon, Quark Announce Initiation of Pivotal Phase II/III Study of QPI-1007 in Rare Eye Disease in India

Biocon Ltd. announced the randomization of the first patient in India in the pivotal global Phase II/III study of QPI-1007, a novel small interfering RNA drug candidate for ocular neuroprotection. [Biocon Ltd.] Press Release

Ibrutinib (IMBRUVICA^®) Granted Breakthrough Therapy Designation by U.S. Food and Drug Administration (FDA) for the Development of a Treatment for Chronic Graft-Versus-Host Disease (cGVHD)

The FDA has granted a fourth Breakthrough Therapy Designation for ibrutinib: as monotherapy for the treatment of patients with cGVHD after failure of one or more lines of systemic therapy, Janssen Research & Development, LLC announced. The FDA also granted the therapy Orphan Drug Designation for cGVHD. [Janssen Pharmaceutical Companies] Press Release

Statement From FDA Commissioner Robert Califf, M.D. Announcing the Acting Director of the FDA Oncology Center of Excellence

The Food and Drug Administration (FDA) is honored to be an integral part of the Vice President’s National Cancer Moonshot Initiative, which calls on the agency to leverage the combined skills of regulatory scientists and reviewers with expertise in drugs, biologics and devices to create the Oncology Center of Excellence. [U.S. Food and Drug Administration] Press Release

Kite Pharma Opens State-of-the-Art T-Cell Therapy Manufacturing Facility

Kite Pharma, Inc. marked the official opening of its new commercial manufacturing facility in El Segundo, California. The facility has been designed to produce chimeric antigen receptor (CAR) and T-cell receptor product candidates for clinical trials, as well as for the potential launch and commercialization of Kite’s lead CAR T-cell product candidate, KTE-C19, which is in clinical study for the treatment of chemorefractory diffuse large B-cell lymphoma and other B-cell malignancies. [Kite Pharma, Inc.] Press Release

Mirna Therapeutics Appoints Perry Nisen, M.D., Ph.D., to Its Board of Directors

Mirna Therapeutics, Inc. announced the appointment of Perry Nisen, M.D., Ph.D., to the Company’s Board of Directors. Dr. Nisen currently serves as the Chief Executive Officer and Donald Bren Chief Executive Chair of the Sanford Burnham Prebys Medical Discovery Institute, an independent biomedical research institute. [Mirna Therapeutics, Inc.] Press Release

NIH Should Abandon Controversial Effort to Update Human Research Rules, Says National Academies Panel

Stop what you are doing—now!—and wait for more discussion and instruction. That’s the blunt message that a National Academies of Sciences, Engineering, and Medicine committee on reforming federal regulation of U.S. research sent to the National Institutes of Health. [Science Insider] Editorial | Resource

Selling Stem Cells in the USA: Assessing the Direct-to-Consumer Industry

Businesses marketing putative stem cell interventions have proliferated across the U.S. This commercial activity generates a host of serious ethical, scientific, legal, regulatory, and policy concerns. [Cancer Stem Cell] Editorial

FDA

Early Clinical Trials With Live Biotherapeutic Products: Chemistry, Manufacturing, and Control Information; Guidance for Industry; Availability (FR Doc. No:2016-15664) Notice

Erythropoietic Protoporphyria; Scientific Workshop (FR Doc. No:2016-15662) Notice

NEW 2016 National Cancer Research Institute (NCRI) Cancer Conference
November 6-9, 2016
Liverpool, United Kingdom

Visit our events page to see a complete list of events in the cell therapy community.

NEW Resource Technologist B – Cellular Manufacturing Activities for Cell and Gene Therapy (University of Pennsylvania)

NEW Postdoctoral Fellow – Pluripotent Stem Cell Technology (University of California Davis)

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

NEW Associate Member – Stem Cell and Gene Therapy (Fred Hutchinson Cancer Research Center)

Scientist – Bioengineering (STEMCELL Technologies Inc.)

Faculty Position – Cancer Immunology (University of New Mexico)

Postdoctoral Fellow – Pathogenic Mechanisms of Rare Bone Disorders (University of Connecticut Health)

Postdoctoral Fellow – Oxidative Lung Injury (Temple University)

Postdoctoral Fellow – Lentivirus-Mediated FOXP3 Gene Transfer (Stanford University School of Medicine)

Postdoctoral Fellowship – Deciphering the Link between Adhesion and Pluripotency (The Danish Stem Cell Center at Faculty of Health & Medical Sciences at the University of Copenhagen)

Scientist – Target Validation (Innate Pharma)

Postdoctoral Fellow – Data Analysis and Stem Cell Research (Nestle Institute of Health Sciences)

Postdoctoral Fellow – Stem Cell Research and Neurosensory Regeneration (Universitatsklinikum Tubingen)

Postdoctoral Fellow – Stem Cells to Study Disease (Hamad bin Khalifa University)

Postdoctoral Fellow – Protein and Antibody Engineering (Hamad bin Khalifa University)

Postdoctoral Fellow – Cancer Immunology and Immunotherapy (Hamad bin Khalifa University)

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

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

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