Volume 16.34 | Sep 14

Issue 16.34 September 14, 2015
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Researchers Report Sustained Remission after Treatment with Investigational Personalized Cellular Therapy in Patient with Multiple Myeloma
A multiple myeloma patient whose cancer had stopped responding after nine different treatment regimens experienced a complete remission after receiving an investigational personalized cellular therapy known as CTL019. The investigational treatment was combined with chemotherapy and an autologous stem cell transplant – a new strategy designed to target and kill the cells that give rise to myeloma cells. [Press Release from the University of Pennsylvania discussing online publication in the New England Journal of Medicine]
Press Release | Abstract
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PUBLICATIONS (Ranked by impact factor of the journal)
Chimeric Antigen Receptor T Cells Persist and Induce Sustained Remissions in Relapsed Refractory Chronic Lymphocytic Leukemia
Researchers report the mature results from their initial chimeric antigen receptor (CAR) T cell trial. CAR T cell persistence correlated with clinical responses, and these cells were functional up to four years after treatment. [Sci Transl Med] Abstract | Press Release

Efficacy of Systemic Adoptive Transfer Immunotherapy Targeting NY-ESO-1 for Glioblastoma
Investigators utilized human glioblastoma cell cultures to induce expression of New York–esophageal squamous cell carcinoma (NY-ESO-1) following in vitro treatment with the demethylating agent decitabine. They examined the in vivo application of this immune therapy using an intracranially implanted xenograft model for in situ T cell trafficking, survival, and tissue studies. [Neuro Oncol] Abstract | Press Release

Bioengineering Vascularized Tissue Constructs Using an Injectable Cell-Laden Enzymatically Crosslinked Collagen Hydrogel Derived from Dermal Extracellular Matrix
Investigators tested whether collagen-phenolic hydroxyl hydrogels could support the formation of vascularized engineered tissue graft by human blood-derived endothelial colony-forming cells and bone marrow-derived mesenchymal stem cells in vivo. [Acta Biomater] Abstract

Adult Lung Spheroid Cells Contain Progenitor Cells and Mediate Regeneration in Rodents with Bleomycin-Induced Pulmonary Fibrosis
Investigators report a rapid and robust method to generate therapeutic resident lung progenitors from adult lung tissues. In a syngeneic rat model of pulmonary fibrosis, lung spheroid cells outperformed adipose-derived mesenchymal stem cells in reducing fibrotic thickening and infiltration. [Stem Cells Transl Med]
Abstract | Press Release

Human Satellite Cell Transplantation and Regeneration from Diverse Skeletal Muscles
Xenotransplantation into mice of 50–70 fiber-associated, or 1,000–5,000 FACS-enriched CD56+/CD29+ human satellite cells led to stable engraftment and formation of human-derived myofibers. Human cells with characteristic PAX7, CD56, and CD29 expression patterns populated the satellite cell niche beneath the basal lamina on the periphery of regenerated fibers. [Stem Cell Rep] Full Article

GDNF-Transfected Macrophages Produce Potent Neuroprotective Effects in Parkinson’s Disease Mouse Model
Scientists evaluated genetically-modified macrophages for active targeted brain delivery of glial cell-line derived neurotropic factor (GDNF). A systemic administration of GDNF-expressing macrophages significantly ameliorated neurodegeneration and neuroinflammation in Parkinson’s disease mice. [PLoS One] Full Article | Press Release

Preclinical Safety of Human Embryonic Stem Cell-Derived Oligodendrocyte Progenitors Supporting Clinical Trials in Spinal Cord Injury
Investigators characterized the preclinical safety profile of a human embryonic stem cell-derived oligodendrocyte progenitor cell therapy product (AST-OPC1) in support of its use as a treatment for spinal cord injury. The phenotype and functional capacity of AST-OPC1 was characterized in vitro and in vivo. [Regen Med]
Full Article | Press Release

An HPV-E6/E7 Immunotherapy Plus PD-1 Checkpoint Inhibition Results in Tumor Regression and Reduction in PD-L1 Expression
Researchers investigated if immunotherapy against human papilloma virus (HPV) using a viral gene delivery platform to immunize against HPV 16 genes E6 and E7 combined with programmed death-ligand 1 blockade could increase therapeutic effect as compared to the vaccine alone. [Cancer Gene Ther] Abstract

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Lentivirus Technologies for Modulation of the Immune System
Twenty years of vector development have successfully brought lentiviral vectors to the clinic, with follow up studies of clinical trials treating primary immunodeficiencies now being reported. Growing interest in gene modified adoptive T cell transfer as an alternative strategy has driven further technology innovation, including characterization of novel viral envelopes. [Curr Opin Pharmacol] Abstract

Cancer Gene Therapy with T Cell Receptors and Chimeric Antigen Receptors
The authors present strategies to optimize chimeric antigen receptors and T cell receptor function, and discuss the importance of target antigen selection to enhance tumor specificity, while reducing on-target and off-target toxicity. [Curr Opin Pharmacol] Abstract

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

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Genentech to Present Data from 15 Medicines across Its Portfolio of Cancer Immunotherapy and Targeted Medicines
Genentech announced that data from 138 abstracts will be presented. Results will be presented from several clinical studies that are supporting ongoing regulatory discussions for three investigational medicines across specific types of lung, bladder and skin cancer. [Press release from Genentech, Inc. discussing research to be presented at the European Cancer Congress (ECC), Vienna] Press Release

Cellular Biomedicine Group to Release CAR-T for Non-Small Cell Lung Cancer Phase I Clinical Trial Data
Cellular Biomedicine Group Inc. (CBMG) announced a scheduled poster presentation. The presentation will detail PLAGH/CBMG’s Phase I clinical trial data using chimeric antigen receptor-modified T-Cells (CAR-T) targeting HER-1 for the treatment of patients with HER-1 expressing advanced relapsed/refractory non-small cell lung cancer. [Press release from Cellular Biomedicine Group Inc. (Globe Newswire, Inc.) discussing research to be presented at the European Cancer Congress (ECC), Vienna] Press Release

Transgene Announces Final Overall Survival Data from Phase IIb TIME Trial with TG4010 Immunotherapy in Non-Small Cell Lung Cancer
Transgene SA announced the presentation of final data from the Phase IIb part of the TIME trial with TG4010 MUC1 targeted immunotherapy in non-small cell lung cancer. [Press release from Transgene SA discussing research presented at the 16th World Conference on Lung Cancer (WCLC), Denver] Press Release

OSE PHARMA Unveils Clinical and Immunological Outcomes in Patients with Brain Metastases Treated with Tedopi® in a Phase II Trial
OSE Pharma SA unveiled encouraging results of survival and T-specific immune response in the patients with brain metastases treated with the company’s T-specific immunotherapy. [Press release from OSE Pharma SA discussing research presented at the 16th World Conference on Lung Cancer (WCLC), Denver] Press Release

New Cohorts from CheckMate -012 Assess Optimal Dosing of Opdivo+Yervoy in the First-Line Treatment of Patients with Advanced Non-Small Cell Lung Cancer
Bristol-Myers Squibb Company announced updated results from the Opdivo (nivolumab)+Yervoy (ipilimumab) arms in CheckMate -012, a multi-arm Phase Ib trial evaluating Opdivo in patients with chemotherapy-naïve advanced non-small cell lung cancer. [Press release from Bristol-Myers Squibb Company discussing research presented at the 16th World Conference on Lung Cancer (WCLC), Denver] Press Release

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Medicine’s Wild West — Unlicensed Stem-Cell Clinics in the United States
Although stem-cell therapy may hold great potential, the field is less advanced than the public has been led to believe. Stem-cell clinics in the United States and abroad have capitalized on this confusion by selling treatments that are not approved by the Food and Drug Administration, supported by clinical studies, or covered by insurers. [New Engl J Med] Editorial

Research on Gene Editing in Embryos Is Justified
Genetic editing of human embryos “has tremendous value” to help solve important scientific questions, and should proceed despite potential worries about use of the technique in the clinic, an influential bioethics group said today in a statement. [ScienceInsider] Editorial

New Education Think Tank Wants Research to Drive U.S. Policy
The United States conducts more education research than any other country. But that output hasn’t translated into a world-class education system. Now, a prominent academic has formed a California-based think tank with the goal of improving that sorry record by putting research results to better use. [ScienceInsider] Editorial

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Cancer Research UK and MedImmune Launch Ground-Breaking Biotherapeutics Research Centre in Cambridge
A new laboratory that will focus on the discovery and development of novel biologic cancer treatments and diagnostics has been opened this week in Cambridge. The state-of-the-art CRUK-MEDI Alliance Laboratory is an innovative collaboration between Cancer Research UK, its commercial arm Cancer Research Technology, and MedImmune, the global biologics research and development arm of AstraZeneca. [Cancer Research UK] Press Release

Kite Pharma Expands Collaboration with Netherlands Cancer Institute
Kite Pharma, Inc. announced that it has expanded its collaboration with the Netherlands Cancer Institute (NKI). Kite and the NKI have entered into an agreement under which Kite will receive from the NKI the exclusive option to license multiple T cell receptor gene sequences for the development and commercialization of cancer immunotherapy candidates targeting solid tumors. [Kite Pharma, Inc.] Press Release

Immunicum Provides Ad5PTDf35 Adenovirus Technology to Rutgers Cancer Institute
Immunicum AB has signed a research agreement with Rutgers Cancer Institute of New Jersey to supply the institution with the company’s adenovirus (Ad5PTDf35) technology. [Immunicum AB] Press Release

Stem Cell Specialists BioEden to Enter Strategic Alliance with Precious Cells Group
BioEden is teaming up with Precious Cells Group, a London based Medtech Company whose facilities specialize in enabling the harvesting and banking of stem cells from cord blood and peripheral blood derived stem cells amongst others platform technologies. [BioEden (PRWeb)] Press Release

International Stem Cell Corporation Announces Next Phase of Research Collaboration with Rohto Pharmaceutical Co., Ltd. of Japan
International Stem Cell Corporation (ISCO) announced that it had entered into the second phase of the existing research agreement with Rohto Pharmaceutical Co., Ltd. After successfully completing preliminary studies of ISCO’s human parthenogenetic neural stem cells, Rohto acknowledged that ISCO’s proprietary cells demonstrate consistent high quality and are suitable for further use in Rohto’s research. [International Stem Cell Corporation] Press Release

Childhood Cancer Research at UCSF to Transcend Tissue Types with Innovative Grant
Researchers at UC San Francisco (UCSF) are leading a five-year, $10 million research project dedicated to pediatric cancer, funded by the first grant of its kind to focus on a molecular pathway that underlies many cancers rather than on a cancer in a particular organ or tissue in the body. [UC San Francisco] Press Release

Baylor College of Medicine Receives Grant to Study Experimental Immunotherapy in HPV-Related Throat Cancer
Researchers at Baylor College of Medicine have received a $1.1 million grant from the U.S. Food and Drug Administration to advance clinical studies of an experimental immunotherapy for human papillomavirus (HPV)-related throat cancer. [Baylor College of Medicine] Press Release

Ogawa-Yamanaka Prize Founded at the Gladstone Institutes to Support Translational Stem Cell Research
The Gladstone Institutes presented the inaugural Ogawa-Yamanaka Stem Cell Prize to Masayo Takahashi, MD, PhD, a faculty member and project leader in the Laboratory for Retinal Regeneration at the RIKEN Centre for Developmental Biology in Japan. Dr. Takahashi was awarded the prize for her trailblazing work leading the first clinical trial to use induced pluripotent stem cells in humans. [Gladstone Institutes] Press Release

ASTRO Awards $33,500 in Individual Grants to 41 Researchers Presenting at 2015 Annual Meeting
The American Society for Radiation Oncology (ASTRO) has selected 41 recipients to receive a total of $33,500 for the 2015 Annual Meeting Abstract Awards. [American Society for Radiation Oncology] Press Release
Center for Scientific Review; Notice of Closed Meetings (FR Doc. No: 2015-22504)
Use of Donor Screening Tests to Test Donors of Human Cells, Tissues and Cellular and Tissue-Based Products for Infection with Treponema pallidum (Syphilis)
Food and Drug Administration (United States)

Agency Information Collection Activities; Proposed Collection; Comment Request; Guidance for Industry on Safety Labeling Changes-Implementation of Section 505 of the Federal Food, Drug, and Cosmetic Act (FR Doc. No: 2015-21645)
NEW BioChina International 2015 Summit
October 27-28, 2015
Shanghai, China

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Postdoctoral Researcher – Epigenetic Modifiers (Van Andel Research Institute)

NEW Senior Cell Biologist (Adaptimmune Inc.)

NEW Postdoctoral Fellow – Immunotherapy/T Cell Biology (California Institute for Biomedical Research)

Postdoctoral Position – Spinal Cord Injury Research (Indiana University-Purdue University)

Postdoctoral Fellow – HMGA Chromatin Remodeling Proteins in Cancer and Stem Cell Biology (Johns Hopkins University School of Medicine)

Senior Research Technician (Qu Biologics Inc.)

Postdoctoral Fellow Position – Genome Editing, Synthetic Biology, and Cell Evolution and Engineering (University of California – Irvine)

Research Associate – Cell Biology (Editas Medicine)

Senior Research Associate (Editas Medicine)

Cell Biology Lab Manager (Harvard Apparatus Regenerative Technology)

Science Writer – Cardiovascular Regeneration (Houston Methodist Research Institute)

Aseptic Manufacturing Associate (Lonza Biologics, Inc.)

Postdoctoral Fellow – Stem Cell Biology (INSERM)

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