Cell Therapy News Volume 18.03 | Jan 30 2017

TOP STORY

Interspecies Organogenesis Generates Autologous Functional Islets

By injecting mouse pluripotent stem cells (PSCs) into Pdx-1-deficient rat blastocysts, researchers generated rat-sized pancreata composed of mouse-PSC-derived cells. Islets subsequently prepared from these mouse–rat chimeric pancreata were transplanted into mice with streptozotocin-induced diabetes. The transplanted islets successfully normalized and maintained host blood glucose levels for over 370 days in the absence of immunosuppression. [Nature] Abstract | Press Release

(Ranked by impact factor of the journal)

Interspecies Chimerism with Mammalian Pluripotent Stem Cells

Whether human pluripotent stem cells (hPSCs) can contribute to chimera formation in non-rodent species remains unknown. The authors systematically evaluated the chimeric competency of several types of hPSCs using a more diversified clade of mammals, the ungulates. They found that naïve hPSCs robustly engraft in both pig and cattle pre-implantation blastocysts but showed limited contribution to post-implantation pig embryos. [Cell] Abstract | Press Release

Improving T Cell Expansion with a Soft Touch

Polydimethylsiloxane (PDMS) was prepared in a microbead format and modified with activating antibodies to CD3 and CD28. A total of three different formulations of PDMS provided an extended proliferative phase in both CD4⁺-only and mixed CD4⁺–CD8⁺ T cell preparations. CD8⁺ T cells retained cytotoxic function, as measured by a set of biomarkers and an in vivo assay of targeted cell killing. [Nano Lett] Abstract

Multicomponent Injectable Hydrogels for Antigen-Specific Tolerogenic Immune Modulation

The authors explored the potential of an injectable, porous hydrogel to induce a regulatory T cell response by delivering a peptide antigen to dendritic cells in a noninflammatory context. Two methods were described for delivering the BDC peptide from pore-forming alginate gels in the nonobese diabetic mouse model of type 1 diabetes: encapsulation in poly(lactide-co-glycolide) microparticles, or direct conjugation to the alginate polymer. [Adv Healthc Mater] Abstract

Administration of Multipotent Mesenchymal Stromal Cells Restores Liver Regeneration and Improves Liver Function in Obese Mice with Hepatic Steatosis after Partial Hepatectomy

Evidence indicates that multipotent mesenchymal stromal cells (MSCs) administration can improve hepatic function and increase the potential for liver regeneration in patients with liver damage. Since hepatic steatosis (HS) is the most common form of chronic hepatic illness, scientists evaluated the role of MSCs in liver regeneration in an animal model of severe HS with impaired liver regeneration. [Stem Cell Res Ther] Full Article

Systemic and Persistent Muscle Gene Expression in Rhesus Monkeys with a Liver De-Targeted Adeno-Associated Virus (AAV) Vector

Investigators compared the muscle tropism and transduction efficiency of a liver de-targeted AAV variant to AAV9 following systemic administration in newborn rhesus monkeys. In vivo bioluminescence imaging was performed to monitor transgene expression post-administration. Results indicated comparable and sustained levels of systemic firefly luciferase gene expression in skeletal muscle over a period of two years. [Hum Gene Ther] Abstract

Transplantation of Hepatocytes from Genetically Engineered Pigs into Baboons

The authors explored survival of hepatocytes from genetically engineered pigs after direct injection into the spleen and other selected sites in immunosuppressed baboons to monitor the immune response and the metabolic function and survival of the transplanted hepatocytes. [Xenotransplantation] Abstract

In Vivo Guided Vascular Regeneration with a Non-Porous Elastin-Like Polypeptide Hydrogel Tubular Scaffold

Scientists demonstrated a new approach for small-caliber vascular reconstruction using a non-porous elastin-like polypeptide hydrogel tubular scaffold, based on the concept of guided vascular regeneration. The scaffolds were composed of elastin-like polypeptide, (Val-Pro-Gly-Ile-Gly)n, for compliance matching and antithrombogenicity and an Arg-Gly-Asp motif for connective tissue regeneration. [J Biomed Mater Res A] Abstract

Cryopreservation of GABAergic Neuronal Precursors for Cell-Based Therapy

Researchers describe a new standardized cryopreservation protocol for GABAergic neural precursors derived from the medial glanglionic eminence, a promising source of GABAergic neuronal progenitors for cell therapy against interneuron-related pathologies. Their migration capacity, acquisition of mature neuronal morphology, and potency to differentiate into multiple interneuron subtypes were confirmed in vivo after transplantation. [PLoS One] Full Article

Effects of Neural Stem Cell and Olfactory Ensheathing Cell Co-Transplants on Tissue Remodeling after Transient Focal Cerebral Ischemia in the Adult Rat

Neural stem cells (NSCs) are promising reparative candidates for stroke induced lesions, however, their survival and integration with the host-tissue post-transplantation is poor. Investigators addressed this challenge by testing whether co-grafting of NSCs with olfactory ensheathing cells, a special type of glia with proven neuroprotective, immunomodulatory, and angiogenic effects, can promote graft survival and host tissue remodeling. [Neurochem Res] Abstract

Chimeric Antigen Receptor T-Cell Therapy for Tumor Immunotherapy

The authors briefly summarize basic chimeric antigen receptor (CAR) structure, parts of reported tumor specific or associated antigens (TSAs/TAAs), results of the clinical trials of CAR T-cell therapies as well as two life-threatening side effects. Experiments in vivo or in vitro, ongoing clinical trials and the outlook for CAR T-cell therapies are also included. [Biosci Rep] Full Article

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Bill & Melinda Gates Foundation Boosts Vital Work of the University of Washington’s Institute for Health Metrics and Evaluation

The Bill & Melinda Gates Foundation and the University of Washington’s Institute for Health Metrics and Evaluation (IHME) announced the foundation’s commitment to invest $279 million in IHME to expand its work over the next decade. [University of Washington’s Institute for Health Metrics and Evaluation] Press Release

National Institutes of Health Honors Research Leaders from University Hospitals and Case Western Reserve University with Prestigious Outstanding Investigator Awards

Two internationally recognized medical research leaders from University Hospitals Cleveland Medical Center and Case Western Reserve University School of Medicine have received Outstanding Investigator Awards from the National Institutes of Health (NIH). The 7-year, $6.7 million dollar awards, which are the highest honor and largest individual support grants conferred by the NIH, will advance promising molecular studies of colon cancer and age-related cardiovascular risks, respectively. [University Hospitals Cleveland Medical Center] Press Release

PPMD Awards $250,000 Grant to UT Southwestern Medical Center to Explore CRISPR/Cas9 Technology

PPMD announced a $250,000 grant to be awarded to Dr. Eric Olson and the Department of Molecular Biology at UT Southwestern Medical Center. This grant, part of PPMD’s Gene Transfer Initiative, will support Dr. Olson’s ongoing study of CRISPR/Cas9 technology as a potential treatment for Duchenne. [Parent Project Muscular Dystrophy (PPMD)] Press Release

SpinalCyte, LLC Enrolls First Patient in Landmark Phase I Human Clinical Trial

SpinalCyte, LLC. announced the enrollment of its first patient in a study for the development of CybroCell, the first dermal fibroblast cell product for treatment of degenerative disc disease. [SpinalCyte, LLC.] Press Release

Vybion Huntington’s Disease Drug Patent Receives Track I Status

Vybion, Inc. announced that its patent application covering methods of use and composition for INT41 Gene Therapy received Track I status from the USPTO. The INT41 patent application covers several neurodegenerative diseases in addition to Huntington’s Disease including Spinal Cerebellar Ataxia, Spinal Muscular Atrophy, and Alzheimer’s. [Vybion, Inc. (PR Newswire Association LLC.)] Press Release

BrainStorm Announces Validation of NurOwn^® Cryopreservation Process

BrainStorm Cell Therapeutics Inc. announced that it has validated its cryopreservation process for NurOwn^® in preparation for the upcoming Phase III clinical study in Amyotrophic Lateral Sclerosis. The validation involved a comparison of NurOwn^® derived from fresh mesenchymal stem cells (MSC) to those derived from cryopreserved MSC. Company scientists were successful in showing that the MSC can be stored in the vapor phase of liquid nitrogen for prolonged periods of time while maintaining their characteristics. [BrainStorm Cell Therapeutics Inc.] Press Release

Exemplar Genetics Awarded Subcontract to Advance New Therapeutic Models for Sickle Cell Disease

Exemplar Genetics has been awarded a subcontract to create genetically engineered miniswine models of sickle cell disease as part of a national resource that could lead to new treatments for the disorder. The subcontract is with Leidos Biomedical Research, Inc. Work under the subcontract will support the National Institutes of Health National Center for Advancing Translational Sciences in creating genetically engineered miniswine models of sickle cell disease. [Exemplar Genetics] Press Release

Stephen Gottschalk, M.D., Has Been Named Chair of the Department of Bone Marrow Transplantation and Cellular Therapy at St. Jude Children’s Research Hospital

Stephen Gottschalk, M.D., has been named chair of the Department of Bone Marrow Transplantation and Cellular Therapy at St. Jude Children’s Research Hospital in Memphis, Tennessee. In his new position, Gottschalk will be responsible for management of the department’s clinical, research and educational activities. [St. Jude Children’s Research Hospital] Press Release

Biologics & Biosimilars Collective Intelligence Consortium (BBCIC) Announces Addition of Apobiologix to Consortium

BBCIC announced the addition of a new participating organization, Apobiologix. The organization supports the BBCIC’s mission to monitor biosimilars and their corresponding novel biologics for effectiveness and safety. The BBCIC is a science-based, non-profit, public service initiative that will monitor biosimilars and their corresponding novel biologics for effectiveness and safety. [Biologics & Biosimilars Collective Intelligence Consortium] Press Release

Intrexon to Acquire GenVec to Expand Industry-Leading Gene Delivery Platform

Intrexon Corporation announced that it has entered into a definitive agreement to acquire GenVec, Inc. a clinical-stage company and pioneer in the development of AdenoVerse^™ gene delivery technology. Intrexon intends to integrate and expand upon GenVec’s expertise in adenoviral vectors and cGMP drug product manufacturing to enhance its broad gene transfer capabilities that encompass multiple viral and non-viral platforms. [Intrexon Corporation] Press Release

Proposed U.S. Biotech Rules Raise Industry Hopes and Anxieties

Makers of genetically engineered products have long been on the lookout for changes in U.S. regulations. The system that divvies up the safety review of these products between the Food and Drug Administration, Environmental Protection Agency, and U.S. Department of Agriculture is in the middle of a years-long overhaul, in part to accommodate modern gene-editing technologies such as CRISPR. And a set of new proposals released last week offers hints about what those agencies still hope to change. [ScienceInsider] Editorial

Trump Agenda Threatens US Legacy of Science Diplomacy

A newly minted leader with no experience governing at home or establishing policy abroad now oversees the United States’ vast diplomatic enterprise. US President Donald Trump has a deep bench of scientific and technical expertise to tap across multiple government agencies — but it is not clear that he will use it. [Nature News] Editorial

FDA

Agency Information Collection Activities; Submission for Office of Management and Budget Review; Comment Request; Premarket Approval of Medical Devices (FR Doc. No:2017-01188) Notice

NEW Frontiers in Cancer Immunotherapy
February 27-28, 2017
New York City, United States

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February 27-March 1, 2017
Basel, Switzerland

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NEW Postdoctoral Researchers – Regenerative, Modular & Developmental Engineering Laboratory (NUI Galway)

NEW Medical Director – Clinical Research Physician (Celgene Corporation)

Stem Cell Scientist – Human Stem Cells as Advanced Therapy Medicinal Products (National University of Ireland Galway)

Postdoctoral Fellow – Various Projects (University of Oklahoma)

Scientist – Regulatory Support (Cook MyoSite, Inc.)

Scientist – Regulatory Affairs (Cook MyoSite, Inc.)

Senior Scientist – Engineered T Cells Used in Adoptive Cell Therapies (Immatics)

Senior Scientist – Cell Isolation, Culture, and Genetic Engineering of Human B Cells for Expression of Therapeutic Gene Products (Immusoft Corporation)

Tenure Track Faculty – Stem Cells and Regenerative Medicine (University of Notre Dame)

Staff Research Associate III – Translational Research to Clinical Cell Therapies (University of California Davis Health System)

Senior Research Scientist – Otolaryngology (Vanderbilt University Medical Center)

Laboratory Technician – Cellular Production Associate II-III (Fred Hutchinson Cancer Research Center)

Staff Research Associate III – Clinical Gene, Cellular, and Other Pharmaceutical Product Manufacturing (University of California Davis Health System)

GMP Manufacturing Director – Cell and Gene Therapy (Fred Hutchinson Cancer Research Center)

Assistant Professor – Molecular Therapeutics of Cancer (Dartmouth College)

Assistant or Associate Member – Stem CellGene Therapy (Fred Hutchinson Cancer Research Center)

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