Cell Therapy News Volume 17.33 | Sep 12 2016

TOP STORY

Lineage-Specific BCL11A Knockdown Circumvents Toxicities and Reverses Sickle Phenotype

Researchers found that using optimized shRNAs embedded within a miRNA (shRNA^miR) architecture to achieve ubiquitous knockdown of BCL11A profoundly impaired long-term engraftment of both human and mouse hematopoietic stem cells despite a reduction in nonspecific cellular toxicities. [J Clin Invest] Full Article | Press Release

(Ranked by impact factor of the journal)

GUCY2C-Directed CAR-T Cells Oppose Colorectal Cancer Metastases without Autoimmunity

T cells expressing chimeric antigen receptors (CARs) directed by a GUCY2C-specific antibody fragment recognized GUCY2C, quantified by expression of activation markers and cytokines. Further, GUYC2C CAR-T cells lysed GUCY2C-expressing, but not GUCY2C-deficient, mouse colorectal cancer cells. [Oncoimmunology] Abstract

Contributions of Mouse and Human Hematopoietic Cells to Remodeling of the Adult Auditory Nerve after Neuron Loss

Scientists investigated the contribution of mouse and human hematopoietic stem cells to cellular diversity in the auditory nerve following the destruction of neuron cell bodies, also known as spiral ganglion neurons (SGNs). Exposure of the adult mouse cochlea to ouabain selectively killed type I SGNs and disrupted the blood-labyrinth barrier. [Mol Ther] Full Article

Lentiviral Vector Gene Therapy Protects XCGD Mice from Acute Staphylococcus aureus Pneumonia and Inflammatory Response

Gene therapy, with hematopoietic stem cells transduced with regulated lentiviral vectors, restored the functional activity of NADPH oxidase complex and saved mice from death caused by S. aureus, significantly reducing the bacterial load and lung damage, similarly to wildtype mice even at low vector copy number. [Mol Ther] Abstract

Adipose-Derived Mesenchymal Stem Cells Promote the Survival of Fat Grafts via Crosstalk Between the Nrf2 and TLR4 Pathways

Enrichment of fat grafts with adipose-derived stem cells inhibited inflammatory cytokine production, enhanced growth factor levels, increased fat graft survival, downregulated NADPH oxidase 1 and 4 expression, increased vascularization and reduced ROS production in a manner dependent on toll-like receptor-4 and nuclear factor erythroid 2-related factor 2 (Nrf2) expression. [Cell Death Dis] Full Article

One-Year Outcomes of Pachymetry and Epithelium Thicknesses after Accelerated (45 mW/cm2) Transepithelial Corneal Collagen Cross-Linking for Keratoconus Patients

The thickness of corneal pachymetry and the epithelium after accelerated (45 mW/cm²) transepithelial corneal collagen cross-linking for keratoconus were assessed in this prospective case series study. Twenty-eight patients were treated for keratoconus. [Sci Rep] Full Article

Chondrogenic Commitment of Human Umbilical Cord Blood-Derived Mesenchymal Stem Cells in Collagen Matrices for Cartilage Engineering

The authors aimed to characterize and determine the chondrogenic differentiation potential of human umbilical cord blood-mesenchymal stem cells for cartilage tissue engineering using an approach combining 3D culture in type I/III collagen sponges and chondrogenic factors. [Sci Rep] Full Article

3D Printed PCL-Based Scaffolds Provide an Advantageous Environment for Osteogenic Differentiation of Human Adipose-Derived Stem Cells

Investigators proposed to use scaffolds based on polycaprolactone, which is widely considered a suitable mesenchymal stem cells (MSCs) delivery system, as a 3D culture environment promoting osteogenic differentiation of adipose-derived MSCs. [J Tissue Eng Regen Med] Abstract

First Report of Pediatric Hematopoietic Stem Cell Transplantation Activities in the Eastern Mediterranean Region From 1984 to 2011: On Behalf of the Pediatric Cancer Working Committee of the Eastern Mediterranean Blood and Marrow Transplantation Group

To describe the hematopoietic stem cell transplantation activities for children in the Eastern Mediterranean (EM) region, data on transplants performed for children less than 18 years of age between 1984 and 2011 in eight EM countries were collected. A total of 5187 transplants were performed, of which 4513 were allogeneic and 674 were autologous. [Bone Marrow Transplant] Abstract

Multi-Center Analysis of the Effect of T-Cell Acute Lymphoblastic Leukemia Subtype and Minimal Residual Disease on Allogeneic Stem Cell Transplantation Outcomes

Researchers provide a detailed analysis of allogeneic stem cell transplantation outcomes in a large T-cell acute lymphoblastic leukemia cohort with a specific emphasis on the effects of pre-transplant minimal residual disease and disease subtype, including the aggressive early-thymic precursor subtype. [Bone Marrow Transplant] Abstract

Implantable Amyloid Hydrogels for Promoting Stem Cell Differentiation to Neurons

Scientists report a new class of amyloid-inspired peptide hydrogels that was designed and based on α-synuclein protein for which hydrogel formation is triggered by various stimuli, such as heating/cooling or changes in pH. The amyloid hydrogels facilitated the attachment and neuronal differentiation of mesenchymal stem cells (MSCs) and assist in the delivery and engraftment of MSCs in the substantia nigra and caudate putamen of a Parkinsonian mouse model. [NPG Asia Materials] Full Article

Genome Editing in Cardiovascular Diseases

The reviewers discuss the applications of genome-editing technology throughout cardiovascular disease research and the prospect of in vivo genome-editing therapies in the future. They also describe some of the existing limitations of genome-editing tools that will need to be addressed if cardiovascular genome editing is to achieve its full scientific and therapeutic potential. [Nat Rev Cardiol] Abstract

Striving to Cure Adult T-Cell Leukemia/Lymphoma: A Role for Allogeneic Stem Cell Transplant?

The authors discuss the rationale for and issues surrounding allogeneic stem cell transplant in adult T-cell leukemia/lymphoma in the context of conventional and emerging therapies. [Bone Marrow Transplant] Abstract

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

Asterias Biotherapeutics Announces Interim Efficacy Data from SCiStar Clinical Trial of AST-OPC1 in Complete Cervical Spinal Cord Injury Patients to be Presented at the 55th ISCoS Annual Scientific Meeting

Asterias Biotherapeutics, Inc. announced that Edward Wirth, MD, PhD, Chief Medical Officer, will present interim efficacy data from the 10 million cell cohort in the company’s ongoing AST-OPC1 SCiSTAR Phase I/IIa clinical study in complete cervical spinal cord injury patients. [Press release from Asterias Biotherapeutics, Inc.discussing research to be presented at the 55^th International Spinal Cord Society (ISCoS) Annual Scientific Meeting, Vienna] Press Release

Cynata and FUJIFILM Sign Non-Binding Development and Commercialization Term Sheet

Cynata Therapeutics Limited announced the execution of a term sheet with FUJIFILM Corporation of Japan for the development and commercialization of certain Cynata technology, including Cynata’s lead induced pluripotent stem cell derived therapeutic mesenchymal stem cell product, CYP-001. [Cynata Therapeutics Limited] Press Release

Lonza to Offer Novel Anc-AAV Gene Therapy Technology through Exclusive Licensing Agreement with Massachusetts Eye and Ear

Lonza Houston, Inc. and Massachusetts Eye and Ear have entered into a strategic agreement that provides customers the ability to in-license Anc80 and other Anc-AAVs for the clinical development and commercialization of novel gene therapies. [Massachusetts Eye and Ear] Press Release

UTHealth Receives $ 6.8 Million From DOD to Study Stem Cell Therapy for TBI

A research team led by Charles S. Cox, Jr., M.D., at The University of Texas Health Science Center at Houston (UTHealth) has been awarded $6.8 million from the U.S. Department of Defense to assess the safety and efficacy of using autologous stem cell therapy in adults with emergent traumatic brain injury (TBI). [University of Texas Health Science Center at Houston] Press Release

MiMedx Announces Nationwide Launch of AmnioFill™

MiMedx Group, Inc. announced its plans for the nationwide launch of AmnioFill^™, the first product in the MiMedx placental collagen matrix product family to be commercially launched. AmnioFill is a collagenous matrix derived from the placenta and comprised of placental extracellular matrix tissue. AmnioFill is a tissue allograft containing ECM proteins, growth factors, cytokines and other specialty proteins present in placental tissue. [MiMedx Group, Inc.] Press Release

Asterias Biotherapeutics Receives Safety Clearance to Begin Administering the Highest Dose of AST-OPC1 in the SCiStar Phase I/IIa Clinical Trial in Cervical Spinal Cord Injury Patients

Asterias Biotherapeutics, Inc. announced that its Data Monitoring Committee has reviewed the safety data from the initial cohort of three patients dosed with 2 million cells, and a subsequent five patients in the second cohort dosed with 10 million cells, and has cleared the company to now begin dosing a third cohort of 5-8 complete cervical injury patients with the highest dose of 20 million cells. [Asterias Biotherapeutics, Inc. (PR Newswire Association LLC.)] Press Release

Gradalis^®, Inc. Announces Dosing of First Patient in Pilot Study Combining Vigil® Engineered Autologous Tumor Cell Immunotherapy and Durvalumab in Advanced Breast Cancer

Gradalis^® Inc. announced that the first patient has been dosed in a pilot study combining Vigil^® Engineered Autologous Tumor Cells with durvalumab in advanced breast cancer. [Gradalis^® Inc.] Press Release

StemGenex Announces Steven A. Brody, M.D., Ph.D. Chief Scientific Officer

StemGenex recently announced it has named Steven A. Brody, M.D., Ph.D. Chief Scientific Officer. Dr. Brody is an internationally recognized physician-scientist specializing in regenerative and restorative medicine, bioidentical hormone therapy, and genomic methods to enhance health. [StemGenex (PR Newswire Association LLC.)] Press Release

Experimental Stem Cell Therapy Helps Paralyzed Man Regain Use of Arms and Hands

The Keck Medical Center of the University of Southern California (USC) announced that a team of doctors became the first in California to inject its patient with an experimental treatment made from stem cells, AST-OPC1, as part of a multi-center clinical trial. Charles Liu, MD, PhD, director of the USC Neurorestoration Center, led the surgical team, working in collaboration with the Rancho Los Amigos National Rehabilitation Center and Keck Medicine of USC, that injected an experimental dose of 10 million AST-OPC1 cells directly into Boesen’s cervical spinal cord in early April. [University of Southern California] Press Release

Reactor Shutdown Threatens World’s Medical-Isotope Supply

Next month, Canada’s Chalk River nuclear research reactor will halt production of a medical isotope that is widely used in diagnostic scans. Any unplanned outages at the world’s remaining production sites could lead to severe shortages of the tracer technetium-99m until new facilities come online in 2017 and 2018, the US National Academies of Science, Engineering and Medicine warn in a report released. [Nature Breaking News] Editorial

Use Antimicrobials Wisely

The United Nations must reframe action on antimicrobial resistance as the defence of a common resource, argue Peter S. Jørgensen, Didier Wernli and colleagues. [Nature Comment] Editorial

Moscow Monument Proposed to Immortalize Peer Review

Sociologist hopes to raise cash to carve concrete sculpture. [Nature News] Editorial

FDA

E17 General Principles for Planning and Design of Multi-Regional Clinical Trials; International Council for Harmonisation; Draft Guidance for Industry; Availability (FR Doc. No:2016-21689) Notice

ICH S3A Guidance: Note for Guidance on Toxicokinetics: The Assessment of Systemic Exposure in Toxicity Studies-Questions and Answers; International Council for Harmonisation; Draft Guidance for Industry; Availability (FR Doc. No:2016-21552) Notice

Agency Information Collection Activities; Proposed Collection; Comment Request; Medical Device: Current Good Manufacturing Practice Quality System Regulations (FR Doc. No:2016-21553) Notice

NEW Cell and Gene Therapy Bioprocessing and Commercialization
October 5-7, 2016
Boston, United States

NEW Cell Culture and Downstream Processing World Congress
February 21-22, 2017
Munich, Germany

NEW World Advanced Therapies and Regenerative Medicine Congress
May 17-19, 2017
London, United Kingdom

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

NEW Postdoctoral Fellow – Novel Systems for Treatment of Type 1 Diabetes (Toronto General Research Institute & McEwen Centre for Regenerative Medicine)

NEW Group Leader – Stem Cells and Ageing (University of Queensland)

NEW Assistant Professor – Developmental Biology (Florida State University)

NEW Visiting Research Specialist – Physiology and Biophysics (University of Illinois at Chicago)

Research Associate – Cellular Therapy (California Institute for Biomedical Research)

Research Scientist – Downstream Bioprocess for Vaccine, Gene Therapy and Biosimilars (Univercells)

PhD Studentship – Fibrosis (National University of Ireland, Galway)

Postdoctoral Fellow – Tumor Immunology and Head and Neck Cancer (University of Lausanne)

Postdoctoral Fellow – Tumor Immunology and T Cell Differentiation (University of Lausanne)

Research Fellow – Hematology (Mayo Clinic)

Postdoctoral Fellow – Pluripotent Stem Cell Technology (University of California Davis)

Associate Member – Stem Cell and Gene Therapy (Fred Hutchinson Cancer Research Center)

Postdoctoral Fellow – Pathogenic Mechanisms of Rare Bone Disorders (University of Connecticut Health)

Postdoctoral Fellow – Oxidative Lung Injury (Temple University)

Postdoctoral Fellow – Lentivirus-Mediated FOXP3 Gene Transfer (Stanford University School of Medicine)

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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