Cell Therapy News Volume 18.08 | Mar 13 2017

TOP STORY

Infusion of Mesenchymal Stromal Cells after Deceased Liver Transplantation: A Phase I-II, Open-Label, Clinical Study

Mesenchymal stromal cell (MSC) infusion could be a mean to establish tolerance in solid organ recipients. The aim of this prospective, controlled, Phase I study was to evaluate the feasibility, safety and tolerability of a single infusion of MSCs in liver transplant recipients. [J Hepatol] Full Article

(Ranked by impact factor of the journal)

Electroresponsive and Cell-Affinitive Polydopamine/Polypyrrole Composite Microcapsules with a Dual-Function of On-Demand Drug Delivery and Cell Stimulation for Electrical Therapy

Results indicated that polydopamine (PDA) and the microporous structure not only enhance the biocompatibility of PDA-polypyrrole microcapsules (PDA-PPy-MCs) but also strengthen the effect of electrical stimulation. In vivo implantation showed that PDA-PPy-MCs have good biocompatibility. [NPG Asia Materials] Full Article

The Balance between CD8⁺ T Cell-Mediated Clearance of AAV-Encoded Antigen in the Liver and Tolerance Is Dependent on the Vector Dose

Researchers found that hepatic transfer of limited doses of an AAV-ovalbumin vector rapidly induced antigen-specific CD8⁺ T cells that only became functionally competent after >2 months. At this time, CD8⁺ T cells had downregulated negative checkpoint markers, e.g., the programmed death 1 receptor, and upregulated expression of relevant cytokines. [Mol Ther] Full Article | Graphical Abstract

An Innovative Approach for Enhancing Bone Defect Healing Using PLGA Scaffolds Seeded with Extracorporeal-Shock-Wave-Treated Bone Marrow Mesenchymal Stem Cells (BMSCs)

Histological and micro-CT results showed that poly-lactic-co-glycolic acid (PLGA) scaffolds seeded with ESW-treated BMSCs produced more bone-like tissue with commitment to the osteogenic lineage when subcutaneously implanted in vivo, as compared to control group. Significantly greater bone formation with a faster mineral apposition rate inside the defect site was observed in the ESW group compared to control group. [Sci Rep] Full Article

Discovery of BVDU as a Promising Drug for Autoimmune Diseases Therapy by Dendritic-Cell-Based Functional Screening

Investigators assessed the in vivo therapeutic potentials of BVDU on mouse models including EAE and STZ-induced T1D, and found that BVDU treated EAE mice exhibited significantly lower EAE clinical scores, decreased leukocyte infiltration in central nervous system lesions, and reduced demyelination. [Sci Rep] Full Article

Short-Course Rapamycin Treatment Enables Engraftment of Immunogenic Gene-Engineered Bone Marrow under Low-Dose Irradiation to Permit Long-Term Immunological Tolerance

Bone marrow (BM) or hematopoietic stem and progenitor cell-expressing OVA ubiquitously or targeted to MHC II+ cells was transferred using low-dose total body irradiation. Recipients were administered rapamycin, cyclosporine or vehicle for three weeks commencing at BM transfer. [Stem Cell Res Ther] Full Article

Pulp Regeneration by Transplantation of Dental Pulp Stem Cells in Pulpitis: A Pilot Clinical Study

Five patients with irreversible pulpitis were enrolled and monitored for up to 24 weeks following mobilized dental pulp stem cells (MDPSCs) transplantation. The MDPSCs were isolated from discarded teeth and expanded based on good manufacturing practice. The quality of the MDPSCs at passages 9 or 10 was ascertained by karyotype analyses. The MDPSCs were transplanted with granulocyte colony-stimulating factor in atelocollagen into pulpectomized teeth. [Stem Cell Res Ther] Full Article

Effective and Persistent Antitumor Activity of HER2-Directed CAR-T Cells against Gastric Cancer Cells In Vitro and Xenotransplanted Tumors In Vivo

Investigators showed that the expanded chimeric antigen receptor (CAR)-T cells, expressing an increased central memory phenotype, were activated by the specific recognition of HER2 antigens in an MHC-independent manner, and effectively killed patient-derived HER2-positive gastric cancer cells. In HER2-positive xenograft tumors, CAR-T cells exhibited considerably enhanced tumor inhibition ability, long-term survival, and homing to targets, compared with those of non-transduced T cells. [Protein Cell] Full Article

Mesenchymal Stromal Cell Infusion to Treat Steroid-Refractory Acute GvHD III/IV after Hematopoietic Stem Cell Transplantation

The authors report the results of 46 patients treated with mesenchymal stromal cell (MSC) infusion as salvage therapy for steroid-refractory acute GvHD (aGvHD) III/IV. Patients received a median cumulative dose of MSCs of 6.81 × 10⁶/kg in a median of three infusions. Median time between the onset of aGvHD and the first MSC infusion was 25.5 days. [Bone Marrow Transplant] Abstract

Systemic Injection of AAV9-GDNF Provides Modest Functional Improvements in the SOD1^G93A ALS Rat but Has Adverse Side Effects

Systemic AAV9-glial cell line-derived neurotrophic factor (GDNF) was delivered via tail vein injections to young rats to determine whether this could be a safe and functional strategy to treat the SOD1^G93A rat model of ALS and, therefore, translated to a therapy for ALS patients. [Gene Ther] Full Article

Transplant Genetics and Genomics

Genetic analysis in transplantation, when linked to demographic and clinical outcomes, has the potential to drive personalized medicine by enabling individualized risk stratification and immunosuppression through the identification of variants associated with immune-mediated complications, post-transplant disease or alterations in drug-metabolizing genes. [Nat Rev Genet] Abstract

Platelet-Rich Plasma for Regeneration of Neural Feedback Pathways around Dental Implants: A Concise Review and Outlook on Future Possibilities

The authors present known growth factors contributing to the biological efficacy of platelet-rich plasma (PRP) and illustrates basic and (pre-)clinical evidence regarding the use of PRP and its relevant products in peripheral nerve regeneration. In addition, the potential of local application of PRP for structural and functional recovery of injured peripheral nerves around dental implants is discussed. [Int J Oral Sci] Full Article

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

Northwell Health and United Therapeutics Announce Strategic Partnership to Advance Bioelectronic Medicine and Cell Therapy

Northwell Health’s Feinstein Institute for Medical Research and United Therapeutics Corporation announced a strategic partnership focused on the application of bioelectronic medicine and cell therapy to cardiology, hypertension and post-transplant tolerance induction. [United Therapeutics Corporation] Press Release

Bio-Techne Signs Exclusive License Agreement with Multiclonal Therapeutics for Adult “Ground-State” Stem Cell Technology

Bio-Techne has entered into an exclusive license agreement with Multiclonal Therapeutics to commercialize reagents and media systems in the research reagent field intended for cloning, propagating, and differentiating adult “ground-state” stem cells from healthy and diseased epithelial tissues. [Bio-Techne] Press Release

SenzaGen and AstraZeneca Initiate Technology Development Collaboration

SenzaGen has signed a collaboration agreement with the global biopharmaceutical company AstraZeneca. The collaboration focuses on testing and development of SenzaGen’s sensitization test GARDair, in order to distinguish between allergens and irritants in the respiratory tract. [SenzaGen] Press Release

TiGenix Announces Top-Line Phase I/II Results of AlloCSC-01 in Acute Myocardial Infarction

TiGenix NV announced top-line one-year results from the CAREMI clinical trial, an exploratory Phase I/II study of donor-derived expanded cardiac stem cells (AlloCSCs) in acute myocardial infarction (AMI). CAREMI is the first-in-human clinical trial with the primary objective being safety and evaluating the feasibility of an intracoronary infusion of 35 million of AlloCSCs in patients with AMI and left ventricular dysfunction treated within the first week post-AMI. [TiGenix NV] Press Release (Download)

ProtoKinetix Announces Start of Phase I & Phase II Clinical Trials for the Use of AAGP^™ PKX-001 Treated Islet Cells in the Treatment of Type 1 Diabetes

ProtoKinetix, Incorporated announced the start of a Phase I first-in-human clinical trial of AAGP^™ PKX-001 treated islet cells used in conjunction with the Edmonton Protocol for the treatment of Type 1 diabetes. The first patient has been treated under this protocol. [ProtoKinetix, Incorporated (Business Wire, Inc.)] Press Release

Nature Cell Started Commercial Clinical Trials Phase I and II ‘ASTROSTEM,’ Stem Cell Drug for Alzheimer’s Disease Treatment in U.S.

Nature Cell announced that it had started to recruit patients for its clinical trials of ‘ASTROSTEM,’ a stem cell drug for Alzheimer’s disease treatment, in the U.S. [Nature Cell (Business Wire, Inc.)] Press Release

BioTime Expands OpRegen^® Clinical Trial in Dry-AMD with Opening of First US Sites

BioTime, Inc. announced the expansion of its ongoing Phase I/IIa clinical trial for OpRegen^® in the advanced dry form age-related macular degeneration (Dry-AMD) by naming the first two sites that will treat patients in the U.S. [BioTime, Inc.] Press Release

Servier and Pfizer Announce FDA Clearance of IND Application for UCART19 in Adult Relapsed/Refractory Acute Lymphoblastic Leukemia

Servier, together with Pfizer Inc. and Cellectis announced that the U.S. Food and Drug Administration (FDA) has granted Servier with an Investigational New Drug (IND) clearance to proceed in the U.S. with the clinical development of UCART19, an allogeneic, gene-edited cellular therapy candidate to treat relapsed/refractory acute lymphoblastic leukemia. [Pfizer Inc.] Press Release

Canada’s New Genetic Privacy Law Is Causing Huge Headaches for Justin Trudeau

A vote in Canada’s Parliament to approve a genetic privacy bill is creating a self-inflicted political headache for Prime Minister Justin Trudeau’s Liberal government—and could result in a relatively rare and unusual court case. [ScienceInsider] Editorial

Physician with Drug-Industry Ties Is Trump’s FDA Pick

President Donald Trump has nominated Scott Gottlieb — a conservative pundit, physician and venture capitalist — to head the US Food and Drug Administration (FDA). [Nature News] Editorial

Patchy Progress on Fixing Global Gender Disparities in Science

Although women are publishing more studies, being cited more often, and securing more coveted first-author positions than they were in the mid-1990s, overall progress towards gender parity in science varies widely by country and field. This is according to a massive report released on 8^th March that is the first to examine such a broad swath of disciplines and regions of the world over time. [Nature News] Editorial

Open-Data Contest Unearths Scientific Gems — and Controversy

When a prestigious medical journal challenged scientists to analyze data from a pivotal blood-pressure study in search of new findings, hundreds of researchers around the world rushed to sign up. The contest, sponsored by the New England Journal of Medicine, offered scientists a rare opportunity to access detailed trial data that otherwise might have remained proprietary for another year — if not indefinitely. But the competition, whose winners were announced on 7^th March, also illustrates the tension between speeding access to data and protecting the interests of those who labored to collect them. [Nature News] Editorial

FDA

United States Food and Drug Administration and Health Canada Joint Regional Consultation on International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use; Public Meeting and Webcast; Request for Comments (FR Doc. No:2017-04839) Notice

Joint Meeting of the Drug Safety and Risk Management Advisory Committee and the Anesthetic and Analgesic Drug Products Advisory Committee; Amendment of Notice (FR Doc. No:2017-04529) Notice

NEW International Society for Cellular Therapy (ISCT) 2017
May 3-6, 2017
London, United Kingdom

NEW American Society of Gene & Cell Therapy (ASGCT)
May 10-13, 2017
Washington, D.C., United States

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

NEW Postdoctoral Fellow – Radioprotectant to Alleviate Physiological Damage Due to Ionizing Radiation Exposure (Canadian Nuclear Laboratories)

NEW Postdoctoral Fellow – Gene and Stem Cell Therapy Program (Centenary Institute)

Research Scientist – Immunotherapies for Cancer (GammaDelta Therapeutics Limited)

Postdoctoral Research Fellow – Preclinical Models of Cancer Immunotherapy (Providence Cancer Center)

Principle Investigator and Postdoctoral Positions – Various Projects (Guangzhou Women and Children’s Medical Center)

BIH Chair for Cell and Gene Therapy and Coordinator of the Advanced Therapies Program (Berlin Institute of Health)

Assistant Professor – Medicine by Design Junior Chair in Immunobioengineering (University of Toronto)

Associate/Full Professor – Loretta Rogers Chair in Immunobioengineering (University of Toronto)

Medical Director – Clinical Research Physician (Celgene Corporation)

Postdoctoral Fellow – Various Projects (University of Oklahoma)

Staff Research Associate III – Translational Research to Clinical Cell Therapies (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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