Cell Therapy News Volume 19.04 | Feb 5 2018

TOP STORY

Tisagenlecleucel in Children and Young Adults with B-Cell Lymphoblastic Leukemia

The authors conducted a Phase II, single-cohort, 25-center, global study of tisagenlecleucel in pediatric and young adult patients with CD19⁺ relapsed or refractory B-cell acute lymphoblastic leukemia. [N Engl J Med] Abstract | Press Release

(Ranked by impact factor of the journal)

Long-Term Follow-Up of CD19 CAR Therapy in Acute Lymphoblastic Leukemia

Researchers conducted a Phase I trial involving adults with relapsed B-cell acute lymphoblastic leukemia who received an infusion of autologous T cells expressing the 19-28z CAR at the Memorial Sloan Kettering Cancer Center. Safety and long-term outcomes were assessed, as were their associations with demographic, clinical, and disease characteristics. [N Engl J Med] Abstract | Press Release

Adenoviral Vector with Shield and Adapter Increases Tumor Specificity and Escapes Liver and Immune Control

Scientists engineered a high-affinity protein coat, shielding the most commonly used vector in clinical gene therapy, human adenovirus type 5. When the shielded particles were equipped with adaptor proteins, the virions delivered their payload genes into human cancer cells expressing HER2 or EGFR. The combination of shield and adapter also increased viral gene delivery to xenografted tumors in vivo, and reduced liver off-targeting and immune neutralization. [Nat Commun] Full Article | Press Release

Immunological Synapse Predicts Effectiveness of Chimeric Antigen Receptor Cells

Investigators predict the effectiveness of CAR-modified cells by evaluating the quality of the CAR-mediated immunological synapse (IS), by quantitation of F-actin, clustering of tumor antigen, polarization of lytic granules, and distribution of key signaling molecules within the IS. [Mol Ther] Abstract | Full Article | Graphical Abstract

In Vivo Expansion and Antitumor Activity of Coinfused CD28- and 4-1BB-Engineered CAR-T Cells in Patients with B-Cell Leukemia

The authors interrogated and compared the impact of different costimulatory signal domains on CAR-T cells in vivo. Patients with B-cell leukemia were infused with a mixture of two types of CD19-specific CAR-T cells, individually bearing CD28 and 4-1BB costimulatory signaling domains. [Mol Ther] Abstract

Functional Outcome of Human Adipose Stem Cell Injections in Rat Anal Sphincter Acute Injury Model

Investigators evaluated the effect of human adipose stem cell (hASC) injections in a rat model for anal sphincter injury, which is the main cause of anal incontinence in humans. They tested if the efficacy of hASCs could be improved by combining them with polyacrylamide hydrogel carrier, Bulkamid^®. [Stem Cells Transl Med] Full Article

Gene Therapy for Human Glioblastoma Using Neurotropic JC Virus-Like Particles as a Gene Delivery Vector

A possible gene therapy strategy for glioblastoma multiforme using JC polyomavirus (JCPyV) virus-like particles (VLPs) as a gene delivery vector was investigated. The authors found that JCPyV VLPs were able to deliver the green fluorescent protein reporter gene into tumor cells for expression. [Sci Rep] Full Article

Severe Toxicity in Nonhuman Primates and Piglets following High-Dose Intravenous Administration of an AAV Vector Expressing Human SMN

Scientists treated three juvenile nonhuman primates and three piglets with an intravenous injection of an AAVhu68 vector carrying a human survival of motor neuron (SMN) transgene at a dose similar to that employed in the spinal muscular atrophy clinical trial. [Hum Gene Ther] Abstract | Editorial

Clinical Trial of MGMT(P140K) Gene Therapy in the Treatment of Pediatric Patients with Brain Tumors

In this Phase I trial, seven patients received gene-modified hematopoietic stem cells following myelo-suppressive conditioning, but with only transient low level engraftment of O6-methyl-guanine-methyl-transferase (MGMT(P140K)) gene-modified cells detectable in four patients. [Hum Gene Ther] Abstract

Functions of NKG2D in CD8⁺ T Cells: An Opportunity for Immunotherapy

The authors focus on the similarities and differences between natural killer group 2 member D (NKG2D) and CD28; less well-described characteristics of NKG2D, such as the potential role of NKG2D in CD8⁺ T-cell memory formation, cancer immunity and autoimmunity; and the opportunities for targeting NKG2D in immunotherapy. [Cell Mol Immunol] Abstract

Preclinical Models in Chimeric Antigen Receptor-Engineered T Cell Therapy

Careful model selection based on the primary goals of the study is a crucial step in evaluating CAR-T treatment. Advancements are being made in preclinical models, with the ultimate objective of providing safer, more effective CAR-T therapy to patients. [Hum Gene Ther] Abstract

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

Valerion to Present Initial Clinical Data with VAL-1221 in Pompe Disease

Valerion Therapeutics announced that a poster highlighting initial results from the first cohort of its ongoing Phase I/II clinical study with VAL-1221 in patients with late-onset Pompe disease will be presented. [Press release from Valerion Therapeutics discussing research to be presented at 14^th Annual WORLD^Symposium™ 2018, San Diego] Press Release

Anika Therapeutics Announces Strategic Collaboration with the Institute of Integrative Biology at the University of Liverpool to Develop an Innovative Therapy for Osteoarthritis

Anika Therapeutics, Inc. announced an agreement with the Institute of Integrative Biology at the University of Liverpool to collaborate on research to develop an injectable mesenchymal stem cell therapy for the treatment of osteoarthritis. [Anika Therapeutics, Inc.] Press Release

Children’s Hospital Los Angeles Joins National Consortium to Study Use of Stem Cells in Treating a Rare Cardiac Defect

Children’s Hospital Los Angeles announced participation in the first-ever clinical trial using stem cells from umbilical cord blood to delay or even prevent heart failure in children born with a rare congenital heart defect that leaves them with half a heart. [Children’s Hospital Los Angeles] Press Release

Alnylam Announces FDA Acceptance of New Drug Application (NDA) and Priority Review Status for Patisiran, an Investigational RNAi Therapeutic for the Treatment of Hereditary ATTR (hATTR) Amyloidosis

Alnylam Pharmaceuticals, Inc. announced that the FDA has accepted for filing its NDA for patisiran, an investigational RNAi therapeutic targeting transthyretin (TTR) for the treatment of hATTR amyloidosis. [Alnylam Pharmaceuticals, Inc.] Press Release

Bellicum Pharmaceuticals Announces Clinical Hold on BPX-501 Clinical Trials in the United States

Bellicum Pharmaceuticals, Inc. announced that the company has received notice from the FDA that U.S. studies of BPX-501 have been placed on a clinical hold following three cases of encephalopathy deemed as possibly related to BPX-501. [Bellicum Pharmaceuticals, Inc.] Press Release

CardiAMP Cell Therapy Receives FDA Approval for Pivotal Trial in Chronic Myocardial Ischemia

BioCardia^®, Inc. announced that the FDA has approved an Investigational Device Exemption for the CardiAMP Chronic Myocardial Ischemia Trial to treat patients with refractory angina. [BioCardia^®, Inc.] Press Release

CAR T-Cell Immunotherapy Named Advance of the Year in Annual ASCO Report

A new and unique new way to treat cancer – CAR T-cell therapy – is poised to transform the outlook for children and adults with certain otherwise incurable cancers. The American Society of Clinical Oncology (ASCO) named this type of adoptive cell immunotherapy the Advance of the Year in its annual report. [American Society of Clinical Oncology] Press Release

Tessa Therapeutics Announces Opening of Recruitment Sites Across the United States for Its Phase III Nose Cancer Immunotherapy Trial

Tessa Therapeutics announced that the Stanford Cancer Institute recently joined the company’s trial network, which now consists of seven hospitals across the United States. The company’s Phase III trial targets advanced nose cancer, or nasopharyngeal cancer, with T cell immunotherapy. [Tessa Therapeutics Pte Ltd.] Press Release

CytRx Corporation Highlights Second NantCell Inc. Clinical Trial Evaluating Immuno-Oncology Agents and Cell-Based Therapies in Combination with Aldoxorubicin in Patients with Advanced Squamous Cell Carcinoma

CytRx Corporation highlighted that aldoxorubicin licensee NantCell, Inc. has dosed the first patient in the Phase Ib portion of a Phase Ib/II clinical trial for patients with advanced squamous cell carcinoma of either the head and neck or non-small cell lung cancer. [CytRx Corporation (PR Newswire Association LLC.)] Press Release

Capricor Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for Duchenne Muscular Dystrophy Therapy

Capricor Therapeutics announced that the FDA has granted CAP-1002, its lead investigational cell therapy for the treatment of Duchenne muscular dystrophy, the designation. [Capricor Therapeutics] Press Release

Fibrocell Announces Submission of Investigational New Drug Application for FCX-013 for the Treatment of Moderate to Severe Localized Scleroderma

Fibrocell Science, Inc. announced the submission of an Investigational New Drug Application with the FDA for FCX-013, the company’s gene therapy candidate for the treatment of moderate to severe localized scleroderma. [Fibrocell Science, Inc.] Press Release

Pubmed Commons Closes Its Doors to Comments

The US National Institutes of Health announced that it will discontinue PubMed Commons, a tool that allows scientists to comment on articles indexed in the agency’s popular PubMed database of biomedical literature. The agency said low levels of engagement and the growth of alternate venues for public discussion of published papers contributed to its decision to close PubMed Commons after five years. [Nature News] Editorial

Nature Journals Tighten Rules on Non-Financial Conflicts

What makes a conflict of interest in science? Definitions differ, but broadly agree on one thing: an influence that can cloud a researcher’s objectivity. For some people, that influence can be money. But there are other influences that can interfere, such as institutional loyalty, personal beliefs and ambition. [Nature News] Editorial

Bulgaria in the Cold as European Union Freezes Its Funding

European Union (EU) science ministers are due to meet in their bloc’s poorest member state — Bulgaria — to discuss future EU research policy. For the host nation, it was supposed to be a chance to showcase its ambitious plans to boost economic growth by attracting international research institutes to the country. [Nature News] Editorial

Kid Co-Authors in South Korea Spur Government Probe

The South Korean government is expanding an investigation into researchers who named their children as co-authors on papers. The extended probe comes after a government report identified 82 academic papers on which authors had named their children or relatives — many of them in middle or high school — as co-authors on the publications. [Nature News] Editorial

NEW International Conference on Lymphocyte Engineering
September 13-15, 2018
Madrid, Spain

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

NEW Postdoctoral Researcher – Immunotherapy (Karolinska Institutet)

Lecturer or Senior Lecturer – Gene Therapy (Imperial College London)

Postdoctoral Position – Pancreas Development and Stem Cells (Helmholtz Zentrum München)

Postdoctoral Research Scientist – Autophagy and Cell Death in Cancer (Beatson Institute for Cancer Research)

Postdoctoral Fellowships – Cancer Research (University of Miami)

Postdoctoral Scientist – Systems Oncology (Cancer Research UK Manchester Institute)

Tenure-Track Group Leader Position – Pancreatic Cancer Biology (University of Copenhagen)

Postdoctoral Research Fellows – Bioengineering and Nanotechnology (University of Queensland)

Research Associate – Immunology and Gene Therapy (Fred Hutchinson Cancer Research Center)

Postdoctoral Associate – Gene Therapy (University of Massachusetts)

Professor and Faculty Positions – Various Projects (Academy of Medical Sciences, Zhengzhou University)

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