Cell Therapy News Volume 19.25 | Jul 23 2018

TOP STORY

Allogeneic Mesenchymal Stem Cell Therapy for the Treatment of HBV Related Acute-on-Chronic Liver Failure

The authors demonstrated significant survival benefit of allogeneic bone marrow mesenchymal stem cells for hepatitis B virus (HBV)-related acute-on-chronic liver failure in comparison with standard medical treatment. [Hepatology] Abstract

(Ranked by impact factor of the journal)

Remission of Spontaneous Canine Tumors after Systemic Cellular Viroimmunotherapy

Scientists present the treatment of 27 canine cancer patients with canine mesenchymal stem cells infected with ICOCAV17, a canine oncolytic adenovirus. No significant adverse effects were found. The response rate was 74%, with 14.8% showing complete responses, including total remissions of lung metastasis. [Cancer Res] Abstract

Longitudinal In Vivo Monitoring of CNS Demonstrates the Efficacy of Gene Therapy in a Sheep Model of CLN5 Batten Disease

Single administrations of brain-directed lentiviral or recombinant single stranded adeno-associated virus 9 vectors expressing ovine CLN5 into six pre-clinically affected sheep with a naturally-occurring CLN5 neuronal ceroid lipofuscinose resulted in long-term disease attenuation. [Mol Ther] Abstract

Allogeneic HSCT Transfers Wild Type Cystinosin to Non-Hematological Epithelial Cells in Cystinosis: First Human Report

The authors treated a 16-year-old male suffering from infantile cystinosis and side effects of cysteamine therapy with hematopoietic stem cell transplantation (HSCT). They demonstrated successful transfer of the wild type cystinosin protein and CTNS mRNA to non-hematological epithelial cells in the recipient, as well as a decrease in the tissue cystine-crystal burden. [Am J Transplant] Abstract

Autologous Transplantation of Adipose-Derived Stem Cells Improves Functional Recovery of Skeletal Muscle without Direct Participation in New Myofiber Formation

Intramuscular transplantation of adipose-derived stem cells (ADSCs) into crushed tibialis anterior muscle leads to an improved muscle regeneration with ADSCs residing in the damaged area. [Stem Cell Res Ther] Abstract

Allogeneic Cell Therapy Using Umbilical Cord MSCs on Collagen Scaffolds for Patients with Recurrent Uterine Adhesion: A Phase I Clinical Trial

Twenty-six patients who suffered from infertility caused by recurrent intrauterine adhesion were enrolled in this prospective, non-controlled, Phase I clinical trial with a 30-month follow-up. During the procedure, 1×10⁷ umbilical cord-derived mesenchymal stromal cells, loaded onto a collagen scaffold, were transplanted into the uterine cavity following an adhesion separation procedure. [Stem Cell Res Ther] Full Article

Urocortin 3 Gene Transfer Increases Function of the Failing Murine Heart

Scientists investigated whether UCn3 gene transfer, which provides sustained elevation of plasma UCn3 levels, increases function of the failing heart. [Hum Gene Ther] Abstract

Bone Marrow Derived Mesenchymal Stem Cell-Mediated Dual-Gene Therapy for Glioblastoma

Investigators developed an efficient bone marrow mesenchymal stem cells (BMSCs) based therapeutic strategy that simultaneously allows killing of tumor cells, inhibiting of tumor angiogenesis and assessment and eradication of implanted BMSCs after treatment of glioblastoma. [Hum Gene Ther] Abstract

VEGF-B Electrotransfer Mediated Gene Therapy Induces Cardiomyogenesis in a Rat Model of Cardiac Ischemia

Researchers hypothesized that VEGF-B, critical during fetal heart development, could promote cardiomyogenesis in adult ischemic hearts. Gene electrotransfer, a physical method of in vivo gene delivery, was adapted to the rat model of myocardial infarction. [Bioelectrochemistry] Abstract

Intra-Cavity Stem Cell Therapy Inhibits Tumor Progression in a Novel Murine Model of Medulloblastoma Surgical Resection

Scientists developed an image-guided mouse model of medulloblastoma (MB) surgical resection and investigated intra-cavity neural stem cell therapy for post-operative MB. [PLoS One] Full Article

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Cell Reprogramming Approaches in Gene- and Cell-Based Therapies for Parkinson’s Disease

The authors discuss how current gene and cell therapy strategies hold great promise for the treatment of Parkinson’s disease (PD) and how the use of stem cells and recent developments in cellular reprogramming could contribute to open a new avenue in PD therapy. [J Control Release] Abstract | Graphical Abstract

The Expansion of Targetable Biomarkers for CAR T Cell Therapy

Researchers discuss biomarkers currently under investigation and point out several promising biomarkers in the preclinical stage of development that may be useful as targets. [J Exp Clin Cancer Res] Full Article

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

Vyriad Announces Collaboration with Merck KGaA, Darmstadt, Germany, and Pfizer to Evaluate Oncolytic Virus, Voyager-V1, in Combination with Anti-PD-L1 Antibody, Avelumab, in Phase I Clinical Study for Metastatic Colorectal Cancer

Vyriad Inc. announced a collaboration agreement with Merck KGaA and Pfizer to expand its ongoing Phase I clinical trial program in solid tumors to include a combination study of its lead asset, Voyager-V1, with avelumab, a human anti-PD-L1 antibody. [Vyriad Inc.] Press Release

Pfizer Initiates Pivotal Phase III Program for Investigational Hemophilia B Gene Therapy

Pfizer Inc. and Spark Therapeutics announced that Pfizer initiated a Phase III open-label, multi-center, lead-in study to evaluate the efficacy and safety of current factor IX prophylaxis replacement therapy in the usual care setting. [Pfizer Inc.] Press Release

Hemostemix Announces First U.S. Patients Treated in Phase II Clinical Trial

Hemostemix Inc. announced that it has treated three additional patients including its first two patients in the United States under its continuing Phase II Clinical Trial for critical limb ischemia. [Hemostemix Inc.] Press Release

Sangamo Announces Treatment of First Patient in Phase I/II Clinical Trial Evaluating SB-318 Investigational In Vivo Genome Editing Therapy for MPS I

Sangamo Therapeutics, Inc. announced treatment of the first patient in the Phase I/II clinical trial evaluating SB-318, an investigational in vivo genome editing therapy for patients with mucopolysaccharidosis type I. [Sangamo Therapeutics, Inc.] Press Release

Poseida Awarded $3.99 Million CIRM Grant to Support Preclinical Development of P-PSMA-101, a T Stem Cell Memory CAR-T Therapy for Prostate Cancer

Poseida Therapeutics Inc. announced that the California Institute for Regenerative Medicine (CIRM) awarded a $3.99 million CLIN 1 grant to support the late stage preclinical development of Poseida’s P-PSMA-101 product candidate. [Poseida Therapeutics Inc.] Press Release

PTC Therapeutics to Acquire Agilis Biotherapeutics

PTC Therapeutics, Inc. announced that it has entered into an agreement to acquire Agilis Biotherapeutics, Inc., a biotechnology company advancing an innovative gene therapy platform for rare monogenic diseases that affect the central nervous system. [PTC Therapeutics, Inc.] Press Release

NanoGenics Acquired to Advance Gene Therapy

Ryboquin announced the acquisition of Nanogenic Solutions to form a new product and platform company, Nanogenics Limited. [Ryboquin (Pharmafield)] Press Release

Sangamo Therapeutics to Acquire TxCell

Sangamo Therapeutics, Inc. and TxCell S.A. announced that they have entered into a definitive agreement on July 20, 2018 pursuant to which Sangamo will, following the completion of the contemplated acquisition of a majority stake of TxCell, file a simplified cash tender offer for the purchase of all then outstanding ordinary shares of TxCell, at a price of €2.58 per share in cash, or approximately €72 million, on a debt-free and cash-free basis. [Sangamo Therapeutics, Inc.] Press Release

Trump and Brexit Could Lure More Scientists to Ireland, Says Research Chief

Uncertainty in the UK and US scientific communities because of combined effects of Brexit and Donald Trump’s controversial policies could provide Ireland with a competitive advantage, according to the director general of Science Foundation Ireland. [The Irish Times] Editorial

University of Edinburgh Demands Retraction of Researcher’s Papers

The University of Edinburgh has asked for the retraction of five papers by cell biologist and former faculty member Irina Stancheva following an internal investigation into scientific misconduct allegations, according to Times Higher Education. [The Scientist] Editorial

Philippines Sweetens Deal for Scientists Who Return Home

A renewed government effort to draw Filipino scientists back to the Philippines by paying them to set up their own research labs or teach has met with a mixed reception. [Nature News] Editorial

NEW LabRoots: 2^nd Annual Cell Biology Virtual Event
September 26-27, 2018
Online, None

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

NEW Postdoctoral Scientist – Novel T Cell Based Cancer Therapies (Netherlands Cancer Institute – Antoni van Leeuwenhoek)

Research Associate – Neuroimmunology (Thomas Jefferson University)

Research Assistant or Associate – Cell Therapy (California Institute for Biomedical Research)

Postdoctoral Fellow – DNA Damage Repair and Pediatric Sarcoma Therapy (University of Texas Health Science Center at San Antonio)

Postdoctoral Position – Corneal Diseases (University of California, Los Angeles)

PhD Student – Cellular Immunotherapy (Glycostem Therapeutics)

Research Associate/PhD Position – iPS Cell-Derived Neurons to Model Parkinson’s Disease (TU Dresden)

Postdoctoral Fellow – Cell and Gene Therapies for Cardiac Arrhythmias and Heart Failure (Duke University)

Scientist/Senior Scientist – Lentivirus Production (Obsidian Therapeutics)

Postdoctoral Fellow – Immunology, Immunotherapy, Gene Therapy (City of Hope)

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