Cell Therapy News Volume 17.19 | May 16 2016

TOP STORY

Experimental Therapy Halts Treatment-Resistant Brain Tumors

Researchers report an experimental therapy that in laboratory tests on human cells and mouse models stops glioblastoma and high-grade gliomas. Testing a multi-step therapeutic strategy, they found a way to use a gene therapy to shut down a gene long-implicated in the formation of high-grade gliomas called Olig2. [Press release from the Cincinnati Children’s Hospital Medical Center discussing online publication in Cancer Cell] Press Release | Abstract | Graphical Abstract

(Ranked by impact factor of the journal)

Persistence of Long-Lived Plasma Cells and Humoral Immunity in Individuals Responding to CD19-Directed CAR T Cell Therapy

To explore plasma cell (PC) longevity and humoral immunity in humans, researchers investigated the fate of PCs and their antibodies in adult and pediatric patients who received chimeric antigen receptor-based adoptive T-cell immunotherapy targeting CD19 to treat B-cell lineage malignancies (CTL019). [Blood] Abstract

Preclinical Justification of pbi-shRNA EWS/FLI1 Lipoplex (LPX) Treatment for Ewing’s Sarcoma

Type 1 Ewing’s sarcoma xenograft modeling confirmed dose related safety and tumor response to pbi-shRNA EWS/FLI1 LPX. Toxicology studies in mini-pigs with doses comparable to the demonstrated in vivo efficacy dose resulted in transient fever, occasional limited hypertension at low and high dose assessment and transient liver enzyme elevation at high dose. [Mol Ther] Full Article

Mesenchymal Stromal Cells Generated from Pooled Mononuclear Cells of Multiple Bone Marrow Donors as a Rescue Therapy for Children with Severe Steroid-Refractory Graft versus Host Disease: A Multicenter Survey

To circumvent donor-to-donor heterogeneity, which may lead to inconsistent results after treatment of acute graft-versus-host disease with mesenchymal stromal cells generated from single donors, researchers developed a novel approach by generating these cells from pooled bone marrow mononuclear cells of eight healthy third-party donors. [Haematologica] Abstract

Finding a New Therapeutic Approach for No-Option Parkinsonisms: Mesenchymal Stromal Cells for Progressive Supranuclear Palsy

Researchers used mesenchymal stromal cells as a novel candidate therapeutic tool in a pilot Phase I study for patients affected by progressive supranuclear palsy, a rare, severe and no-option form of Parkinsonism. [J Transl Med] Full Article

Transplantation of Human Adipose-Derived Mesenchymal Stem Cells on a Bladder Acellular Matrix for Bladder Regeneration in a Canine Model

A bladder acellular matrix (BAM) or BAM loaded with human adipose-derived mesenchymal stem cells was used to repair partial cystectomy of the bladder in a canine model. [Biomed Mater] Abstract

Development of an AAV9 Coding for a 3XFLAG-TALE_frat#8^–-VP64 Able to Increase In Vivo the Human Frataxin in YG8R Mice

Researchers produced an adeno-associated virus 9 (AAV9) coding for a TALE_frat#8^– containing 13 repeat variable diresidues able to bind to the proximal promoter of human frataxin gene. [Gene Ther] Full Article

Inhibitory Effect of Endostatin Gene Therapy Combined with Phosphorus-32 Colloid on Tumor Growth in Wistar Rats

Investigators found that ³²P-colloid combined with endostatin exhibited higher inhibitory effect upon tumor growth compared with application of ³²P-colloid or endostatin alone, although these therapies all significantly inhibited tumor growth compared with saline control group. [Biosci Rep] Abstract

Intratracheal Transplantation of Mesenchymal Stem Cells Simultaneously Attenuates Both Lung and Brain Injuries in Hyperoxic Newborn Rats

Scientists investigated whether attenuation of hyperoxic lung injury with intratracheal transplantation of human umbilical cord blood-derived mesenchymal stem cells could simultaneously mitigate brain damage in neonatal rats. [Pediatr Res] Abstract

Therapeutic Anti-CD3 Monoclonal Antibodies: From Bench to Bedside

The authors review preclinical and clinical studies in which intravenous or mucosal administration of anti-CD3 monoclonal antibodies has been employed and provide an outlook on future developments to enhance the efficacy of this promising therapeutic approach. [Immunotherapy] Full Article

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

Dicerna Reports Potent Preclinical Activity with GalNAc-DsiRNA-EX Conjugates

Dicerna Pharmaceuticals, Inc. presented updated preclinical data demonstrating the potency of its proprietary GalNAc-DsiRNA-EX Conjugate technology in enabling direct delivery of RNA interference-based therapy to the liver via subcutaneous injection, with potential utility against multiple therapeutic targets. [Press release from Dicerna Pharmaceuticals, Inc discussing research presented at the 18^th Annual TIDES: Oligonucleotide and Peptide Research, Technology and Product Development Conference, Long Beach] Press Release

Tocagen to Present Clinical and Preclinical Data

Tocagen Inc., a clinical-stage, cancer-selective gene therapy company, announced that results from the clinical investigation of Toca 511 & Toca FC for the treatment of recurrent high grade glioma will be presented. [Press release from Dicerna Pharmaceuticals, Inc. discussing research to be presented at the 2016 American Society of Clinical Oncology (ASCO) Annual Meeting, Chicago] 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

SillaJen Announces Commercial Manufacturing Agreement with ABL Europe For Lead Oncolytic Virus, Pexa-Vec

ASillaJen, Inc. and ABL Europe have entered into a commercial manufacturing agreement for SillaJen’s lead product candidate, Pexa-Vec. SillaJen is currently conducting a multinational, randomized, Phase III, open-label study of Pexa-Vec in patients with advanced liver cancer. [SillaJen, Inc. (PR Newswire Association LLC)] Press Release

Bayer and BioNTech Join Forces to Develop Innovative New mRNA Vaccines and Therapeutics for Animal Health Applications

Bayer and BioNTech AG have entered into an agreement to develop novel, first-in-class mRNA vaccines and therapeutics specifically for Animal Health applications. [BioNTech AG] Press Release

HemaCare Signs Distribution Agreement with China-Based Partner

HemaCare Corporation has expanded its global capabilities through a strategic distribution agreement in China with Shanghai-based MT-Biological Science and Technology Co. Ltd. [HemaCare Corporation (Business Wire)] Press Release

Cell Therapy Ltd. Grants Japan License to Daiichi Sankyo for Its Heart Regeneration Medicine, Heartcel™

Cell Therapy Ltd. announced the granting of the Japan license for its innovative cardiac regeneration medicine, Heartcel™ (immuno-modulatory progenitor cells) to Daiichi Sankyo. [Cell Therapy Ltd.] Press Release

Stem Cell Gene Therapy for Fatal Childhood Disease Ready for Human Trial

Scientists who have developed a stem cell gene therapy to reverse a fatal childhood illness have agreed to work with a new therapeutics company to test it in a human trial. [The University of Manchester] Press Release

Aduro Biotech Announces Phase IIb ECLIPSE Trial Misses Primary Endpoint in Heavily Pretreated Metastatic Pancreatic Cancer

Aduro Biotech, Inc. announced that the Phase IIb ECLIPSE trial did not meet the primary endpoint of an improvement in overall survival for patients with pancreatic cancer who had failed at least two prior therapies in the metastatic setting. [Aduro Biotech, Inc.] Press Release

Leading Chinese CAR-T Cell Therapy Provider SiDanSai Changes Name to ICT in Preparation for Expansion into Overseas Market

As part of its strategy for market expansion, SiDanSai has officially changed its English name to Innovative Cellular Therapeutics. [Innovative Cellular Therapeutics Co., Ltd. (PR Newswire Association LLC)] Press Release

From our sponsor: Free wallchart from Nature Neuroscience – Neural Stem Cells. Request your copy.

Global Standards for Stem-Cell Research

In response to scientific progress and evolving ethical concerns, the International Society for Stem Cell Research (ISSCR) issued updated and extended guidelines for work involving the manipulation of stem cells and the translation of that work into medical therapies. [Nature News] Editorial | Guidelines

Contamination Created Controversial ‘Acid-Induced’ Stem Cells

Stem cells that were claimed to be created simply by exposing ordinary cells to stress were probably derived from embryonic stem cells, according to the latest investigation into an ongoing scientific scandal. How that contamination occurred, however, remains an open question. [Nature News] Editorial | Summary Report

CBER

Clinical Investigator Status (Biologics) Notice

FDA

Standard Preparations, Limits of Potency, and Dating Period Limitations for Biological Products (FR Doc. No:2016-10385) Notice

NIH

Prospective Grant of Exclusive License: Development of Autologous Tumor Infiltrating Lymphocyte Adoptive Cells for the Treatment of Lung, Breast, Bladder, and HPV-Positive Cancers (FR Doc. No:2015-12539) Notice

NEW ICCSCE 2016: 18^th International Conference on Cell and Stem Cell Engineering
September 15-16, 2016
Rome, Italy

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

NEW Professor – Hematology and Cell Therapy (Karolinska University Hospital)

Scientist – Bioengineering (STEMCELL Technologies Inc.)

Scientist – Human Pluripotent Stem Cell Neural Differentiation (STEMCELL Technologies Inc.)

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)

Group Leader – Development and Stem Cells (Monash University)

Postdoctoral Fellow – Immunology (The Pirbright Institute)

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

Postdoctoral Fellow – Non-Viral Gene Delivery (Nanyang Technological University)

Faculty Position – Tumor Immunology (John Wayne Cancer Institute at Providence Saint John’s Health Center)

Postdoctoral Fellow – Non-Coding RNAs in Malignant Progression (The University of Texas MD Anderson Cancer Center)

Postdoctoral Associate – Genetic Medicine (Cornell University)

Postdoctoral Fellow – Retinal Disease Genomics and Gene Therapy (Ocular Genomics Institute, Mass Eye and Ear and Harvard Medical School)

Associate Scientist – Hematopoietic Stem Cell Biology (Editas Medicine)

Research Associate – Hematopoietic Stem Cell Biology (Editas Medicine)

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

Staff Scientist – Cell Therapy Process Development (Fred Hutchinson Cancer Research Center)

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

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