Cell Therapy News Volume 17.22 | Jun 13 2016

TOP STORY

Immunoablation and Autologous Hemopoietic Stem-Cell Transplantation for Aggressive Multiple Sclerosis: A Multicenter Single-Group Phase II Trial

Scientists addressed whether near-complete immunoablation followed by immune cell depleted autologous hemopoietic stem-cell transplantation would result in long-term control of multiple sclerosis. [Lancet] Abstract | Press Release

(Ranked by impact factor of the journal)

Non-Genotoxic Conditioning for Hematopoietic Stem Cell Transplantation Using a Hematopoietic-Cell-Specific Internalizing Immunotoxin

Autologous hematopoietic stem cell transplantation using genetically corrected cells would avoid the risk of graft-versus-host disease, but the genotoxicity of conditioning remains a substantial barrier to the development of this approach. The authors report an internalizing immunotoxin targeting the hematopoietic-cell-restricted CD45 receptor that effectively conditions immunocompetent mice. [Nat Biotechnol] Abstract | Press Release

RNAi Targeting Multiple Cell Adhesion Molecules Reduces Immune Cell Recruitment and Vascular Inflammation After Myocardial Infarction

To counteract the resulting arterial leukocyte recruitment, investigators developed nanoparticle-based RNA interference (RNAi) that effectively silences five key adhesion molecules. [Sci Transl Med] Abstract | Press Release

A Universal System to Select Gene-Modified Hepatocytes In Vivo

Researchers described a universally applicable system for in vivo selection and expansion of gene-modified hepatocytes in any genetic background. In this system, the therapeutic transgene is coexpressed with a short hairpin RNA that confers modified hepatocytes with resistance to drug-induced toxicity. [Sci Transl Med] Abstract | Press Release

Clinical Responses with T Lymphocytes Targeting Malignancy-Associated κ Light Chains

Investigators developed a strategy to target B cell malignancies more selectively by taking advantage of B cell light Ig chain restriction. They generated a chimeric antigen receptor (CAR) that is specific for the κ light chain (κ.CAR) and therefore recognizes κ-restricted cells and spares the normal B cells expressing the nontargeted λ light chain, thus potentially minimizing humoral immunity impairment. [J Clin Invest] Full Article | Press Release

Autologous Hematopoietic Cell Transplantation for HIV-Related Lymphoma: Results of the (BMT CTN) 0803/(AMC) 071 Trial

The BMT CTN 0803/AMC 071 trial is a multicenter Phase II study of autologous hematopoietic cell transplant for patients with HIV-related lymphoma (HRL). Eligible patients had chemotherapy-sensitive relapsed/persistent HRL, were > 15 years of age, and had treatable HIV infection. Patients were prepared using BEAM (carmustine, etoposide, cytarabine, melphalan) and received consistent management of peri-transplant antiretroviral treatment. [Blood] Abstract | Press Release

IGF-1-Containing Multi-Layered Collagen-Fibrin Hybrid Scaffolds for Bladder Tissue Engineering

Scientists developed a multi-layered scaffold consisting of a bioactive fibrin layer laminated between two collagen sheets all having undergone plastic compression. With this construct they performed bladder augmentation in a nude rat model after partial bladder excision and evaluated the morphological and functional behavior of the implant. [Acta Biomater] Abstract

Effect of Chemistry on Osteogenesis and Angiogenesis Towards Bone Tissue Engineering Using 3D Printed Scaffolds

Researchers report the presence of magnesium and silicon in direct three dimensional printed (3D) tricalcium phosphate scaffolds promotes in vivo osteogenesis and angiogenesis when tested in rat distal femoral defect model. [Ann Biomed Eng] Abstract

Bone Marrow Transplantation for CVID-Like Humoral Immune Deficiency Associated with Red Cell Aplasia

The authors describe a case of common variable immunodeficiency (CVID)-like humoral immune deficiency in a child who initially presented with red cell aplasia and ultimately developed progressive bone marrow failure. Although bone marrow transplantation (BMT) has been associated with high mortality in CVID, their patient was successfully treated with a matched sibling BMT and engrafted with greater than 98% donor chimerism and the development of normal antibody titers to diphtheria and tetanus toxoids. [Pediatr Blood Cancer] Abstract

Current Status of Ex Vivo Gene Therapy for Hematological Disorders: A Review of Clinical Trials in Japan Around the World

Gene therapies are classified into two major categories, namely, in vivo and ex vivo. Clinical trials of human gene therapy began with the ex vivo techniques. Based on the initial successes of gene-therapy clinical trials, these approaches have spread worldwide. The number of gene therapy trials approved worldwide increased gradually starting in 1989, reaching 116 protocols per year in 1999, and a total of 2210 protocols had been approved by 2015. [Int J Hematol] Abstract

Regulatory T Cell Therapy: An Option to Induce Operational Tolerance in Liver Transplantation

Several ongoing clinical trials are currently testing the effects of donor-specific or non-specific regulatory T cell infusion with the goal to induce this state of operational tolerance a few months after liver transplantation. The preliminary results of two of these trials have been recently published, and raise a number of comments and issues. [Clin Res Hepatol Gastroenterol] Abstract

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

Use of an Insulin-Only Bionic Pancreas Effectively Controls Blood Glucose Levels

Use of an insulin-only bionic pancreas effectively controls blood glucose levels, with no difference in episodes of hypoglycemia, compared to a bihormonal (insulin plus glucagon) bionic pancreas, when both insulin-only and bihormonal configurations used a target blood sugar of 130 mg/dl. [Press release from the American Diabetes Association discussing research presented at the American Diabetes Association’s 76th Scientific Sessions, New Orleans] Press Release

EXAMINE Trial Post-Hoc Analyses of Mortality Data Regarding Cardiovascular Safety of Alogliptin

Takeda Pharmaceutical Company Limited announced the publication of a post-hoc analysis from the global EXAMINE cardiovascular (CV) safety outcomes trial, which suggested that in patients with Type 2 diabetes and recent acute coronary syndrome, the risk of death, including CV death, was not higher with the dipeptidyl peptidase-4 inhibitor alogliptin compared to placebo during a median follow-up period of 18 months. [Press release from Takeda Pharmaceutical Company Limited discussing research presented at the American Diabetes Association’s 76th Scientific Sessions, New Orleans] Press Release

Seattle Genetics Highlights Vadastuximab Talirine (SGN-CD33A) Data in Acute Myeloid Leukemia (AML)

Seattle Genetics, Inc. highlighted data evaluating vadastuximab talirine in combination with hypomethylating agents in frontline patients with acute myeloid leukemia who had declined intensive therapy. [Press release from Seattle Genetics, Inc. discussing research presented at 2016 European Hematology Association (EHA) Annual Congress, Copenhagen] Press Release

Kite Pharma to Highlight Key Data from CAR T-Cell Therapy Pipeline in Oral Presentations

Kite Pharma, Inc. announced three oral presentations relating to its clinical programs were delivered at a conference. Updated data from Phase I of Kite’s ZUMA-1 study of KTE-C19 in patients with chemorefractory non-Hodgkin lymphoma (NHL) were presented in an oral presentation. [Press release from Kite Pharma, Inc. discussing research presented at 2016 European Hematology Association (EHA) Annual Congress, Copenhagen] Press Release

Boston Biomedical Featured Seven Clinical Posters on Cancer Stem Cell Pathway Inhibitors Napabucasin and Amcasertib in Multiple Cancer Types

Boston Biomedical featured clinical data on investigational compounds napabucasin and amcasertib in multiple tumor types. Data highlighted the potential of napabucasin – an orally administered investigational cancer stemness inhibitor designed to inhibit cancer stem cell pathways by targeting STAT3 – to promote anti-cancer activity when used in combination with other chemotherapeutics. [Press release from Boston Biomedical discussing research presented at the 2016 American Society of Clinical Oncology (ASCO) Annual Meeting, Chicago] Press Release

Latest Penn Studies of Personalized Cell Therapies Define Optimal Doses

More precise dosing methods and cellular engineering techniques show promise in the effort to improve treatment of aggressive cancers with personalized cellular therapies, according to new studies from researchers. Those findings are among results of six studies of investigational chimeric antigen receptor T cells for both adult and pediatric leukemias, adult lymphomas, and ovarian cancer. [Press release from Penn Medicine discussing research presented at the 2016 American Society of Clinical Oncology (ASCO) Annual Meeting, Chicago] Press Release

TxCell and the Lübeck Institute of Experimental Dermatology Launch Collaboration to Develop CAR-Tregs in Bullous Pemphigoid

TxCell SA announces the signature of a strategic R&D collaboration agreement with the Lübeck Institute of Experimental Dermatology, part of the University of Lübeck in Germany. This specific collaboration agreement covers the development of a CAR-Treg-based cellular immunotherapy for bullous pemphigoid, a rare, potentially fatal autoimmune disease characterized by tense inflammatory skin blisters and in some patients, erosions on mucous membranes [TxCell SA] Press Release

BloodCenter of Wisconsin and San Diego Blood Bank Announce Collaborative Offering Called Celluvative

BloodCenter of Wisconsin and the San Diego Blood Bank have created a strategic partnership to accelerate advancements in research and patient care. Together, BloodCenter of Wisconsin and San Diego Blood Bank will offer Celluvative™ – a portfolio of products and clinical trial services that will include esoteric laboratory testing, blood products for research and manufacturing applications, cell processing, and access to a broad database of diverse donors who have expressed interest in clinical trials. [Celluvative] Press Release

CRISPR Therapeutics and Anagenesis Biotechnologies Announce Strategic In-Licensing and Collaboration Agreement to Develop CRISPR/Cas9-Based Cell Therapies for Muscle Diseases

CRISPR Therapeutics and Anagenesis Biotechnologies announced a strategic in-licensing and collaboration agreement, which grants CRISPR Therapeutics exclusive worldwide license to Anagenesis’ proprietary Paraxial Mesoderm Multipotent Cells (P2MCs) technology for cell therapy for all human muscle diseases. The P2MC technology allows for the efficient, reproducible and chemically defined differentiation of pluripotent cells into skeletal muscle stem cells, also known as satellite cells. [CRISPR Therapeutics] Press Release

Magnetic Insight Signs Supply Agreement with Meito Sangyo Co., Ltd. to Enable Cell Therapy Research with Magnetic Particle Imaging

Magnetic Insight Inc. announced that they have entered into a strategic supply agreement with Meito Sangyo. In the agreement, Magnetic Insight will develop and market VivoTrax™, a superparamagnetic iron oxide nanoparticle coated with carboxydextran, as a tracer to support stem cell and immune cell tracking in research for both magnetic particle imaging and magnetic resonance imaging applications. [Magnetic Insight Inc.] Press Release

BioTime Announces $2.2 Million Grant for Further Development of Dry-AMD Program

BioTime, Inc. and its subsidiary Cell Cure Neurosciences Ltd. (“Cell Cure”) announced that Cell Cure has been awarded a new grant for 2016 of 8.4 million shekels (approximately $2.2 million) from the Israel Innovation Authority of the Ministry of Economy and Industry. The grant provides continuing funding for the development of OpRegen^®, a cell-based therapeutic product that consists of animal product-free retinal pigment epithelial cells with high purity and potency. [BioTime, Inc.] Press Release

ImmunoCellular Therapeutics Treats First Patient in ICT-107 Phase III Registrational Trial in Newly Diagnosed Glioblastoma

ImmunoCellular Therapeutics, Ltd. announced that the first patient has been treated in the Phase III registrational trial of ICT-107, the company’s lead cancer immunotherapy product candidate, in newly diagnosed glioblastoma. ICT-107 is a patient-specific, dendritic cell-based immunotherapy targeting multiple tumor-associated antigens on glioblastoma stem cells. [ImmunoCellular Therapeutics, Ltd.] Press Release

Amarantus-Sponsored Training for Phase II Severe Burn Trial Completed with U.S. Army Clinical Site in Late April

Amarantus BioScience Holdings, Inc. announced that the current Good Manufacturing Practices manufacturing process for its Engineered Skin Substitute program is now qualified at Lonza Walkersville, Inc., a premier contract manufacturer providing cell and tissue-based products for clinical development. [Amarantus BioScience Holdings, Inc.] Press Release

Brammer Bio Establishes Dedicated Late Phase Development and Commercial Manufacturing Facility for Advanced Cell and GeneTherapies in Lexington, Massachusetts

Brammer Bio announced plans to build-out a 50,000 sq. ft. facility dedicated to late phase development, including Phase III and commercial production of viral vector-based and cell-based advanced therapies. The facility will be qualified and operational by Q2 of 2017. [Brammer Bio] Press Release

BioTime’s Subsidiary Cell Cure Neurosciences Receives DSMB Approval to Start Second Patient Cohort in Clinical Trial for Dry-AMD

BioTime, Inc. and Cell Cure Neurosciences Ltd. (Cell Cure) announced that the Data Safety Monitoring Board (DSMB), an independent group of medical experts closely monitoring the clinical trial, has reviewed the initial safety data from the first cohort in the Phase I/IIa clinical trial of OpRegen^®. [BioTime, Inc.] Press Release

South Korean Scientists Fight Plan to Scrap Military Exemptions

A proposal from the South Korean defense ministry to scrap exemptions from military service for science and engineering students is being met with fierce resistance by researchers and students alike. [Nature News] Editorial

Roadmap for Advanced Cell Manufacturing Shows Path to Cell-Based Therapeutics

An industry-driven consortium has developed a national roadmap designed to chart the path to large-scale manufacturing of cell-based therapeutics for use in a broad range of illnesses including cancer, neuro-degenerative diseases, blood and vision disorders and organ regeneration and repair. [Georgia Tech] Editorial | Resource

FDA

Determination of Regulatory Review Period for Purposes of Patent Extension; POMALYST (FR Doc. No:2016-13796) Notice

Determination of Regulatory Review Period for Purposes of Patent Extension; TANZEUM (FR Doc. No:2016-13797) Notice

Agency Information Collection Activities; Proposed Collection; Comment Request; Biological Products: Reporting of Biological Product Deviations and Human Cells, Tissues, and Cellular and Tissue-Based Deviations in Manufacturing; Forms FDA 3486 and 3486A (FR Doc. No:2016-13366) Notice

NEW AACR’s Colorectal Cancer: From Initiation to Outcomes
September 17-20, 2016
Tampa, United States

NEW Cancer Stem Cell Conference 2016
September 20-23, 2016
Cleveland, United States

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

NEW 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)

NEW Scientist – Target Validation (Innate Pharma)

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

Scientist – Bioengineering (STEMCELL Technologies Inc.)

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

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

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

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