Cell Therapy News Volume 17.21 | Jun 6 2016

TOP STORY

In Vivo Tracking of Human Hematopoiesis Reveals Patterns of Clonal Dynamics during Early and Steady-State Reconstitution Phases

Using integration site analysis, investigators longitudinally tracked >89,000 clones from 15 distinct bone marrow and peripheral blood lineages purified up to four years after transplant in four Wiskott-Aldrich syndrome patients treated with hematopoietic stem/progenitor cells gene therapy. [Cell Stem Cell] Full Article

(Ranked by impact factor of the journal)

In Vivo Hepatic Reprogramming of Myofibroblasts with AAV Vectors as a Therapeutic Strategy for Liver Fibrosis

As a strategy to generate new hepatocytes and limit collagen deposition in the chronically injured liver, scientists developed in vivo reprogramming of myofibroblasts into hepatocytes using adeno-associated virus (AAV) vectors expressing hepatic transcription factors. [Cell Stem Cell] Abstract | Graphical Abstract | Press Release

Photothermal Combined Gene Therapy Achieved by Polyethyleneimine-Grafted Oxidized Mesoporous Carbon Nanospheres

Combining controllable photothermal therapy and efficacious gene therapy in a single platform holds great promise in cancer therapy due to the enhanced combined therapeutic effects. Herein, polyethyleneimine-grafted oxidized mesoporous carbon nanospheres were developed for combined photothermal combined gene therapy in vitro and in vivo. [Biomaterials] Abstract

Enhanced Immunoregulation of Mesenchymal Stem Cells by IL-10-Producing Type 1 Regulatory T Cells in Collagen-Induced Arthritis

Through a comparative analysis of regulatory T (Treg) and IL-10-producing type 1 regulatory T (Tr1) cells, researchers hypothesized that Tr1 cells enhance the immunoregulatory functions of mesenchymal stem cells, and that a combinatorial approach to cell therapy may exert synergistic immunomodulatory effects in an experimental animal model of rheumatoid arthritis. [Sci Rep] Full Article

Combinatorial Therapy with Three-Dimensionally Cultured Adipose-Derived Stromal Cells and Self-Assembling Peptides to Enhance Angiogenesis and Preserve Cardiac Function in Infarcted Hearts

Combinatorial therapy with three-dimensional cell masses (3DCMs) and self-assembling peptides was tested in a rat myocardial infarction model. Spheroid-type 3DCMs, which are vascular differentiation-induced cells, were prepared by culturing human adipose-derived stromal cells on a fibroblast growth factor-immobilized surface. [J Tissue Eng Regen Med] Abstract

Quantitative Proteome Profiling of Dystrophic Dog Skeletal Muscle Reveals a Stabilized Muscular Architecture and Protection against Oxidative Stress after Systemic Delivery of MuStem Cells

MuStem cell-based therapy represents a promising approach for clinical applications to cure Duchenne Muscular Dystrophy (DMD). To expand our previous studies collected in the clinically relevant DMD animal model, researchers decided to investigate the skeletal muscle proteome four months after systemic delivery of allogenic MuStem cells. [Proteomics] Abstract

Mesenchymal Stromal/Stem Cells Do Not Ameliorate Experimental Autoimmune Encephalomyelitis and Are Not Detectable in the Central Nervous System of Transplanted Mice

Scientists made use of the established experimental autoimmune encephalomyelitis mouse model to investigate migration and therapeutic efficacy of murine bone-marrow derived mesenchymal stromal/stem cells. [Stem Cells Dev] Abstract

Improved Anti-Leukemia Activities of Adoptively Transferred T Cells Expressing Bispecific T-Cell Engager in Mice

The authors compared CD19 bispecific T-cell engager-transferred T cells that had been transfected by RNA electroporation with CD19 chimeric antigen receptors RNA-transferred T cells both in vitro and in an aggressive Nalm6 leukemia mouse model. [Blood Cancer J] Full Article

Concurrent Progress of Reprogramming and Gene Correction to Overcome Therapeutic Limitation of Mutant ALK2-iPSC

Investigators developed a simplified one-step procedure by simultaneously introducing reprogramming and gene-editing components into human fibroblasts derived from patient with fibrodysplasia ossificans progressiva syndrome, and genetically treated it. [Exp Mol Med] Full Article

Extracellular Matrix (ECM) Multilayer Membrane as a Sustained Releasing Growth Factor Delivery System for RhTGF-β3 in Articular Cartilage Repair

Scientists have developed a novel drug delivery system that continuously releases recombinant human transforming growth factor beta-3 (rhTGF-β3) using a multilayered ECM membrane. They hypothesize that the sustained release of rhTGF-β3 could activate stem cells and result in enhanced repair of cartilage defects. [PLoS One] Full Article

In Vitro Models for Assessing Therapeutic Angiogenesis

Therapeutic angiogenesis aims to stimulate new blood vessel formation via the local administration of proangiogenic agents or cell therapy products. In this review, the authors provide a summary of the current understanding of in vitro models for assessing the angiogenic potential of a product. [Drug Discov Today] Abstract

Haematopoietic Stem Cell Transplantation for Sickle Cell Disease – Current Practice and New Approaches

To date, allogeneic haematopoietic stem cell transplant remains the only available curative therapy for sickle cell disease. With declining incidences of rejection and transplant-related mortality, disease-free survival after human leucocyte antigen-identical sibling transplant exceeds 90 percent. [Br J Haematol] Abstract

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

City of Hope Presents New Cancer Research at American Society of Clinical Oncology Meeting

City of Hope doctors and researchers will present their latest research on immunotherapy, precision medicine and other topics. [Press release from City of Hope discussing research presented at the 52^nd American Society of Clinical Oncology (ASCO) Annual Meeting, Chicago.] Press Release

Novartis Data Show More Than 50 Percent of Eligible Ph⁺ CML Patients Maintain Treatment-Free Remission (TFR) After Stopping Tasigna^®

Novartis announced the first results from the Tasigna (nilotinib) TFR clinical trial program. These studies evaluated the potential to maintain molecular response after stopping therapy in adult patients with Philadelphia chromosome-positive (Ph⁺) chronic myeloid leukemia in the chronic phase who achieved a sustained deep level of molecular response with Tasigna – a concept called TFR3. [Press release from Novartis Pharmaceuticals Corporation discussing research presented at the 52^nd American Society of Clinical Oncology (ASCO) Annual Meeting, Chicago.] Press Release

Kite/NCI Anti-CD19 CAR T-Cell Therapy Demonstrates Durable Complete Remissions in Advanced Non-Hodgkin Lymphoma

Kite Pharma, Inc. announced results from a study of low-dose chemotherapy conditioning followed by anti-CD19 chimeric antigen receptor (CAR) T-cell therapy. The results showed that CAR T-cell therapy was effective in inducing a high response rate in patients with advanced non-Hodgkin lymphoma. [Press release from Kite Pharma, Inc. discussing research presented at the 52^nd American Society of Clinical Oncology (ASCO) Annual Meeting, Chicago.] Press Release

Juno Therapeutics’ Investigational CAR T Cell Product Candidate JCAR015 Shows High Response Rates in Adults with B-cell ALL

Juno Therapeutics, Inc. announced that encouraging clinical data from JCAR015, a chimeric antigen receptor (CAR) T cell product candidate, support its strategic approach towards the commercialization of its first CAR T therapy. [Press release from Juno Therapeutics, Inc. discussing research presented at the 52^nd American Society of Clinical Oncology (ASCO) Annual Meeting, Chicago.] Press Release

Bellicum Pharmaceuticals Enters into Research Collaboration with Leiden University Medical Center to Discover Natural High-Affinity TCRs

Bellicum Pharmaceuticals, Inc. announced it has entered into a research agreement with Leiden University Medical Center, to discover and validate natural high-affinity TCR (T cell receptor) product candidates targeting several cancers. [Bellicum Pharmaceuticals, Inc.] Press Release

Kite Pharma and Cell Design Labs Announce Exclusive Research Collaboration Using Molecular ‘On/Off Switches’ for CAR T-Cell Immunotherapies

Kite Pharma, Inc. and Cell Design Labs, Inc. announced a research collaboration and license agreement to develop next generation, precision-controlled chimeric antigen receptor (CAR) product candidates that incorporate Cell Design Labs’ molecular on/off switch technology. [Kite Pharma, Inc.] Press Release

CEVEC and Paragon Bioservices Enter into Comprehensive Collaboration to Roll Out CAP^® Technologies in North America

CEVEC Pharmaceuticals GmbH (CEVEC) and Paragon Bioservices, Inc. announced that the companies signed a collaboration and license agreement to jointly establish CEVEC’s proprietary CAP^® technologies as a new industry standard for the scalable production of viral gene therapy vectors in North America. [CEVEC Pharmaceuticals GmbH] Press Release

DCGI Gives Limited Approval to Market First Allogeneic Cell Therapy Product in India Stempeutics Puts India on The “World Map of Regenerative Medicine”

Stempeutics Research announced that the Drugs Controller General India (DCGI) has granted limited approval for manufacturing & marketing of stem cell based biological product Stempeucel^® for the treatment of Buerger’s disease. [Stempeutics Research] Press Release

Kiadis Pharma Announces Its Decision to File for Marketing Authorization with the European Medicines Agency (EMA) for ATIR101™ in Blood Cancers

Kiadis Pharma N.V. announces a regulatory strategy update that, based on positive Phase II data, it has taken the decision to submit a Marketing Authorization Application to the European Medicines Agency for its lead product ATIR101™ for use in blood cancers to reduce relapse rates, Transplant Related Mortality and Graft-versus-Host-Disease in the context of a hematopoietic stem cell transplantation using a haploidentical donor. [Kiadis Pharma] Press Release

StemCells, Inc. Announces Termination of Phase II Pathway Study Following Review of Data

StemCells, Inc. announced its decision to terminate the Company’s Phase II Pathway Study in spinal cord injury following an in-depth review of data from the study and after obtaining the concurrence of the study’s Interim Analysis Data Monitoring Committee. While the results showed overall improvement in patients treated with the company’s proprietary cells, the magnitude of the effect and the perceived trend of the effect over time did not justify continuing the study. [StemCells, Inc.] Press Release

Kite Pharma Granted Access to Priority Medicines (PRIME) Regulatory Support for KTE-C19 in the Treatment of Chemorefractory Diffuse Large B-Cell Lymphoma (DLBCL)

Kite Pharma, Inc. announced that the European Medicines Agency Committee for Medicinal Products for Human Use and Committee for Advanced Therapies has granted access to its newly established Priority Medicines (PRIME) regulatory initiative for KTE-C19 in the treatment of patients with refractory diffuse large B-cell lymphoma (DLBCL). KTE-C19 is an investigational therapy in which a patient’s T-cells are genetically modified to express a chimeric antigen receptor designed to target the antigen CD19, a protein expressed on the cell surface of B-cell lymphomas and leukemias. [Kite Pharma, Inc.] Press Release

Takeda Receives Positive CHMP Opinion for ADCETRIS^® (Brentuximab Vedotin) as Consolidation Treatment in Post-Transplant Hodgkin Lymphoma

Takeda Pharmaceutical Company Limited announced that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for the extension of the current conditional approval of ADCETRIS^® and recommended its approval for the treatment of adult patients with CD30+ Hodgkin lymphoma at increased risk of relapse or progression following autologous stem cell transplantation. [Takeda Pharmaceutical Company Limited] Press Release

Americord Announces Increasing Stem Cell Yields, Including Record Collection

Americord, the fastest growing cord blood bank in the country and a leader in the advancement of umbilical cord blood, cord tissue, and placenta tissue banking, announced that it has reached several milestones, including record cord blood collection volumes and record yields of total nucleated cell counts. [Americord Registry] Press Release

European Research Universities Form a New Lobby Organization—but Did They Need It?

Nine European universities that invest heavily in research have set up a new group to lobby the European Union’s institutions. The Guild of European Research Intensive Universities includes the University of Oslo, the University of Groningen in the Netherlands, and the University of Bologna in Italy. It’s seeking new members before its official launch in November. [Science News] Editorial

FDA

E18 Genomic Sampling and Management of Genomic Data; International Council for Harmonisation; Draft Guidance for Industry; Availability (FR Doc. No:2016-13168) Notice

Charging for Investigational Drugs Under an Investigational New Drug Application-Questions and Answers; Guidance for Industry; Availability (FR Doc. No:2016-13166) Notice

NEW 2016 Tissue Engineering and Regenerative Medicine International Society – Asia Pacific Meeting (TERMIS-AP 2016)
September 3-6, 2016
Taipei, Taiwan

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

NEW Clinical Specialist – Chimeric Antigen Receptor Cell Products (Celgene Corporation)

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

Scientist – Bioengineering (STEMCELL Technologies Inc.)

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

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

PhD Studentship – Various Projects (Vienna Biocenter)

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

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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