Cell Therapy News Volume 17.20 | May 30 2016

TOP STORY

A Humanized Bone Marrow Ossicle Xenotransplantation Model Enables Improved Engraftment of Healthy and Leukemic Human Hematopoietic Cells

Researchers showed that a xenotransplantation model bearing subcutaneous humanized ossicles with an accessible bone marrow (BM) microenvironment, formed by in situ differentiation of human BM-derived mesenchymal stromal cells, enables the robust engraftment of healthy human hematopoietic stem and progenitor cells, as well as primary acute myeloid leukemia samples, at levels much greater than those in unmanipulated mice. [Nat Med] Abstract

(Ranked by impact factor of the journal)

Osteoblast-Targeting-Peptide Modified Nanoparticle for siRNA/microRNA Delivery

Investigators developed osteoblast-targeting peptide (SDSSD)-modified polyurethane (PU) nanomicelles encapsulating siRNA/microRNA that delivers drugs to osteoblasts; the data showed that SDSSD-PU could selectively target not only bone-formation surfaces but also osteoblasts without overt toxicity or eliciting an immune response in vivo. [ACS Nano] Abstract

Phase I Open Label Liver-Directed Gene Therapy Clinical Trial for Acute Intermittent Porphyria

Researchers aimed to investigate the safety of a recombinant adeno-associated vector expressing porphobilinogen deaminase administered for the first time in humans for the treatment of acute intermittent porphyria. [J Hepatol] Full Article

Non-invasive, Neuron-Specific Gene Therapy by Focused Ultrasound-Induced Blood-Brain Barrier Opening in Parkinson’s Disease Mouse Model

Investigators previously demonstrated that a separated gene-carrying liposome and microbubbles (MBs) administration plus focused ultrasound exposure can deliver genes into the brain, with the successful expression of the reporter gene and glial cell line-derived neurotrophic factor (GDNF) gene. In this study, they further modified the delivery system by conjugating gene-carrying liposomes with MBs to improve the GDNF gene-delivery efficiency, and to verify the possibility of using this system to perform treatment in the 1-Methyl-4-phenyl-1,2,3,6-tetrahydropyridine-induced animal disease model. [J Control Release] Abstract

Development of Novel Small Hairpin RNAs That do not Require Processing by Dicer or AGO2

The authors developed a 30-nucleotide single-strand RNA, termed “guide hairpin RNA (ghRNA, ghR)”, that has a physiological function similar to that of miRNA and siRNA. The ghR caused no innate cytokine response either in vitro or in vivo. [Mol Ther] Abstract

Vascular Regeneration in Ischemic Hindlimb by Adeno-Associated Virus Expressing Conditionally Silenced Vascular Endothelial Growth Factor

In a BALB/c mouse model of CLI, scientists showed that gene therapy with AAV-conditionally silenced-human vascular endothelial growth factor, but not unregulated AAV or plasmid, vectors conferred limb salvage, protection from necrosis, and vascular regeneration when delivered via intramuscular or intra-arterial routes. [J Am Heart Assoc] Full Article

Design of a Phase I Clinical Trial to Evaluate M032, a Genetically Engineered HSV-1 Expressing IL-12, in Patients with Recurrent/Progressive Glioblastoma Multiforme, Anaplastic Astrocytoma, or Gliosarcoma

This clinical protocol outlines the dose-escalating Phase I study for evaluation of M032 in patients with recurrent or progressive malignant glioma. [Hum Gene Ther Clin Dev] Abstract

Neural Stem Cells Derived from Human Parthenogenetic Stem Cells Engraft and Promote Recovery in a Nonhuman Primate Model of Parkinson’s Disease

The authors report the results of a 12-month transplantation study of parthenogenetic stem cellderived neural stem cells in 1-methyl-4-phenyl-1,2,3,6-tetrahydropyridine-lesioned African green monkeys with moderate to severe clinical Parkinsonian symptoms. [Cell Transplant] Full Article

In Vivo Performance of an Acellular Disc-Like Angle Ply Structure (DAPS) for Total Disc Replacement in a Small Animal Model

Researchers evaluated a strategy in which, after in vivo implantation, endogenous cells could colonize the acellular DAPS and form an extracellular matrix organized by the DAPS topographical template. To do so, acellular DAPS were implanted into the caudal spines of rats and evaluated over 12 weeks by mechanical testing, histology and microcomputed tomography. [J Orthop Res] Abstract

Mesenchymal Stem Cell Therapy to Promote Limb Transplant Functional Recovery

Orthotopic syngeneic hindlimb transplants were performed in Lewis rats, followed by topical and intravenous injections of syngeneic mesenchymal stem cells or vehicle. Transplanted limb sensory and motor functions were tested by cutaneous pain reaction and walking track analysis, respectively. [Microsurgery] Abstract

Optimizing T Cell Receptor Gene Therapy for Hematologic Malignancies

The reviewers propose that antigen expression in non-malignant cells may contribute to the efficacy of T cell therapy by maintaining effector function and promoting memory. While chimeric antigen receptor recognition is limited to cell surface structures, T cell receptors can recognize intracellular proteins. [Blood] Abstract

Clinical Development of Gene Therapy: Results and Lessons from Recent Successes

Multiple clinical studies reported successful treatments of pediatric patients. Design of gene therapy vectors and their clinical development are advancing rapidly. This article reviews some of the major successes in clinical gene therapy of recent years. [Mol Ther Methods Clin Dev] Full Article

CAR Models: Next-Generation CAR Modifications for Enhanced T-Cell Function

The authors discuss the recent major advances in chimeric antigen receptor (CAR) design and how these modifications will impact its clinical application. [Mol Ther Oncolytics] Full Article

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

Spark Therapeutics and Pfizer Announce Data from Initial Subjects in Hemophilia B Trial Demonstrating Consistent Therapeutic Levels of Factor IX Expression

Spark Therapeutics and Pfizer Inc. announced that new data will be presented. These data will show encouraging initial observations for the first subjects dosed in the Phase I/II clinical trial of SPK-9001, the lead investigational compound in the SPK-FIX program, which is being studied for the treatment of Hemophilia B. [Press release from Spark Therapeutics discussing research presented at the European Hematology Association’s (EHA) 21^st Congress, Copenhagen] Press Release

Purdue Research May Expand Engineered T-Cell Cancer Treatment

Low and Purdue graduate student Yong Gu Lee collaborated with Endocyte Inc. scientist Haiyan Chu to design genetically engineered CAR T cells that must be activated and targeted by a small molecule adaptor before they can kill cancer cells. The technology has been tested in animal models but no human trials have been performed. [Press release from Purdue University (EurekAlert!) discussing research presented at the 2016 American Association for Cancer Research (AACR) Annual Meeting, New Orleans] Press Release

Biogen Announces Collaboration with University of Pennsylvania on Multiple Gene Therapy Programs

Biogen announced a broad collaboration and alliance with the University of Pennsylvania to advance gene therapy and gene editing technologies. The expansive research and translational development collaboration has multiple objectives, but will primarily focus on the development of therapeutic approaches that target the eye, skeletal muscle and the central nervous system. [Biogen] Press Release

State Stem Cell Agency Awards Stanford Researchers

Albert Wong receives $2.9 million to develop vaccine for glioblastoma; four others awarded $240,000 each to study bladder, heart and eye conditions. [Stanford School of Medicine] Press Release

MJFF Supports Stem Cell Projects to Explore Therapies and Provide Research Tools

The Michael J. Fox Foundation (MJFF) announced funding for two projects leveraging the promise of engineered stem cells to speed new therapies and deeper understanding of Parkinson’s disease. [The Michael J. Fox Foundation] Press Release

Novel Immunotherapy Trial for Lymphoma Offers Hope to Patients at Sylvester

Researchers are testing a novel cellular immunotherapy approach to treating patients with diffuse large B-cell lymphoma who have failed standard therapy. This investigational anti-CD19 chimeric antigen receptor T cell therapy, known as KTE-C19, is being studied in a Phase II clinical trial for patients with aggressive non-Hodgkin’s lymphoma. [University of Miami Miller School of Medicine] Press Release

Asterias Biotherapeutics Announces Positive New Long-Term Follow-Up Results for AST-OPC1

Asterias Biotherapeutics, Inc. announced new positive long-term follow-up results from its Phase I clinical trial assessing the safety of AST-OPC1 (oligodendrocyte progenitor cells) in patients with spinal cord injury. [Asterias Biotherapeutics, Inc.] Press Release

FDA Grants Roche’s Cancer Immunotherapy Tecentriq (Atezolizumab) Accelerated Approval for People with a Specific Type of Advanced Bladder Cancereneration CAR Modifications for Enhanced T-Cell Function

Roche announced that the U.S. Food and Drug Administration (FDA) granted accelerated approval to Tecentriq^® for the treatment of people with locally advanced or metastatic urothelial carcinoma. [F. Hoffmann-La Roche Ltd.] Press Release

Nano Dimension and Accellta Joined Forces to Successfully BioPrint Stem Cell-Derived Tissues

Nano Dimension Ltd. announced it has successfully lab-tested a proof of concept 3D Bioprinter for stem cells. The trial was conducted in collaboration with Accellta Ltd. [Nano Dimension Ltd. (PR Newswire Association LLC.)] Press Release

Regen BioPharma, Inc. Announces ucVax: Universal Donor Cancer Cellular Immunotherapy

Regen BioPharma, Inc. and announced initiation of a preclinical development program aimed at creating the first cord blood based cancer immunotherapeutic product leveraging its NR2F6 immunological checkpoint. [Regen BioPharma, Inc. (PR Newswire Association LLC.)] Press Release

VM BioPharma Announces FDA Fast Track Designation Granted for Investigational Gene Therapy VM202 for Patients with Amyotrophic Lateral Sclerosis (ALS)

VM BioPharma announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for the company’s lead investigational drug, VM202, a Phase II novel gene therapy for the potential treatment of Amyotrophic Lateral Sclerosis. [VM BioPharma] Press Release

Cryoport to Provide Cold Chain Logistics Support for International Stem Cell Corporation’s Phase I Clinical Trial for the Treatment of Parkinson’s Disease

Cryoport, Inc. announced that it will provide global logistics support to International Stem Cell Corporation’s (ISCO) Phase I clinical trial in Australia for the treatment of moderate to severe Parkinson’s disease. ISCO commenced patient enrollment for the study earlier this month. [Cryoport, Inc.] Press Release

WPI Team Awarded Patent for Reprograming Skin Cells

Cell therapies for a range of serious conditions, including heart attacks, diabetes, and traumatic injuries, will be accelerated by research at Worcester Polytechnic Institute (WPI) that yielded a newly patented method of converting human skin cells into engines of wound healing and tissue regeneration. [Worcester Polytechnic Institute] Press Release

Caladrius Biosciences Licenses Cell Therapy Technology for Ovarian Cancer and Subleases Irvine Facility to AiVita Biomedical

Caladrius Biosciences, Inc. announces that it has licensed to AiVita Biomedical, Inc. the exclusive global rights to its tumor cell/dendritic cell technology for the treatment of ovarian cancer. [Caladrius Biosciences] Press Release

Why the Historic Deal to Expand US Chemical Regulation Matters

A rare bipartisan compromise endorsed by industry and the White House will give the US government new authority to ensure that chemicals are safe. [Nature News] Editorial | Guidelines

In Dramatic Statement, European Leaders Call for ‘Immediate’ Open Access to All Scientific Papers by 2020

In what European science chief Carlos Moedas calls a “life-changing” move, European Union member states agreed on an ambitious new open access target. All scientific papers should be freely available by 2020, the Competitiveness Council—a gathering of ministers of science, innovation, trade, and industry—concluded after a two-day meeting in Brussels. [Science Insider] Editorial | Press Release

FDA

Agency Information Collection Activities; Proposed Collection; Comment Request; E6(R2) Good Clinical Practice; International Council for Harmonisation (FR Doc. No:2016-12651) Notice

Collaboration in Regulatory Systems Strengthening and Standardization Activities To Increase Access to Safe and Effective Biological Products (FR Doc. No:2016-12685) Notice

Considerations for Use of Histopathology and Its Associated Methodologies To Support Biomarker Qualification; Guidance for Industry; Availability (FR Doc. No:2016-11438) Notice

NEW 2^nd Annual Cell & Gene Therapy Congress 2016
November 3-4, 2016
London, United Kingdom

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

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

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

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

Scientist – Bioengineering (STEMCELL Technologies Inc.)

Professor – Hematology and Cell Therapy (Karolinska University Hospital)

Postdoctoral Fellow – 3D Microtissues from Pluripotent Stem Cells (Gladstone Institutes)

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

PhD Studentship – Various Projects (Vienna Biocenter)

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

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

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

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