Cell Therapy News Volume 18.31 | Sep 18 2017

TOP STORY

Cortical Bone Stem Cell Therapy Preserves Cardiac Structure and Function after Myocardial Infarction

Investigators determined if post-myocardial infarction (MI) transendocardial injection of allogeneic cortical bone stem cells reduces pathological structural and functional remodeling and prevents the development of heart failure in a swine MI model. [Circ Res] Abstract

(Ranked by impact factor of the journal)

Checkpoint Blockade Immunotherapy Reshapes the High-Dimensional Phenotypic Heterogeneity of Murine Intratumoral Neoantigen-Specific CD8⁺ T Cells

Scientists showed that mass cytometry with multiplex combinatorial tetramer staining can identify and characterize neoantigen-specific CD8⁺ T cells in mice bearing T3 methylcholanthrene-induced sarcomas that are susceptible to checkpoint blockade immunotherapy. [Nat Commun] Full Article

Enhanced Cancer Immunotherapy by Chimeric Antigen Receptor-Modified T Cells Engineered to Secrete Checkpoint Inhibitors

Researchers engineered CAR-T cells secreting checkpoint inhibitors targeting PD-1 and evaluated their efficacy in a human lung carcinoma xenograft mouse model. They demonstrated that the secretion of anti-PD-1 enhanced the antitumor activity of CAR-T cells and prolonged overall survival. [Clin Cancer Res] Abstract

A Combination RNAi-Chemotherapy Layer-by-Layer Nanoparticle for Systemic Targeting of KRAS/P53 with Cisplatin to Treat Non-Small Cell Lung Cancer

In vivo tumor targeting and therapy was investigated in an orthotopic lung cancer model by microCT, fluorescence imaging, and immunohistochemistry. Systemic delivery of the nanoparticles indicated a preferential uptake in lung of the tumor-bearing mice. [Clin Cancer Res] Abstract | Full Article

Transplantation of Non-Cultured Stromal Vascular Fraction Cells of Adipose Tissue Ameliorates Osteonecrosis of the Jaw-Like Lesions in Mice

Transplantation of non-cultured stromal vascular fraction (SVF) cells has been demonstrated to be a useful method for regenerative medicine in place of stem cell therapy. Scientists investigated the effects of non-cultured SVF transplantation on tooth extraction socket healing in mice. [J Bone Miner Res] Abstract

Advanced Glycation End-Product (AGE)-Albumin from Activated Macrophage Is Critical in Human Mesenchymal Stem Cells Survival and Post-Ischemic Reperfusion Injury

For investigating the advanced glycation end-product-albumin (AGE-albumin) from activated macrophages is critical in both muscle cell and stem cell death, the authors evaluated the recovery of post-ischemic reperfusion injury-critical limb ischemia by injection of human bone marrow derived mesenchymal stem cells with or without soluble receptor for AGEs. [Sci Rep] Full Article

Physicochemical Stability and Transfection Efficiency of Cationic Amphiphilic Copolymer/pDNA Polyplexes for Spinal Cord Injury Repair

In situ gene therapy is a promising approach to address the limited availability of growth-promoting biomolecules at central nervous system injury sites. Scientists showed that poly (lactide-co-glycolide)-graft-polyethylenimine/pDNA polyplexes provide improved stability in the presence of competing polyanions and nuclease protection in serum relative to conventional branched polyethylenimine control. [Sci Rep] Full Article

An Engineered Biomarker System to Monitor and Modulate Immune Clearance of Cell Therapies

Researchers developed an engineered cell system that secretes a luciferase enzyme to sensitively detect cell transplants independent of their locale by a simple blood test. They studied a unique feature of cell transplant pharmacology—namely, immune clearance—using mesenchymal stromal cells as a proof-of-concept cell therapy. [Cytotherapy] Abstract

Enhancement of Mouse Hematopoietic Stem/Progenitor Cell Function via Transient Gene Delivery Using Integration-Deficient Lentiviral Vectors

The authors demonstrated the utility of short-term gene expression to improve functional potency of hematopoietic stem/progenitor cells (HSPCs) during transplantation by delivering HOXB4 and Angptl3 using integration-deficient lentiviruses (IdLVs) to enhance the engraftment of HSPCs. Constitutive overexpression of either of these genes is likely to be undesirable, but the transient nature of IdLVs reduced this risk and those associated with unsolicited gene expression in daughter cells. [Exp Hematol] Abstract

Chimeric Antigen Receptor (CAR)-Transduced Natural Killer Cells in Tumor Immunotherapy

T cells genetically modified with a CAR have demonstrated remarkable success in the treatment of hematological cancers. Compared to T cells, CAR-transduced NK cells exhibit several advantages, such as safety in clinical use, the mechanisms by which they recognize cancer cells, and their abundance in clinical samples. [Acta Pharmacol Sin] Abstract

Tools for Translation: Non-Viral Materials for Therapeutic mRNA Delivery

The authors provide an overview of the field of mRNA therapeutics and describe recent advances in the development of synthetic materials that encapsulate and deliver mRNA payloads. [Nat Rev Mater] Abstract

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

BioCardia’s CardiAMP Heart Failure Trial Design to Be Presented

BioCardia^®, Inc. announced that the trial design for its pivotal Phase III CardiAMP Heart Failure Trial was presented. [Press release from BioCardia^®, Inc. discussing research presented at the 2017 Texas Heart Institute International (THI) Symposium on Cardiovascular Regenerative Medicine, Houston] Press Release

Capricor Therapeutics to Present Six-Month Results from the Randomized HOPE Trial of CAP-1002 in Duchenne Muscular Dystrophy in a Late Breaking Session

Capricor Therapeutics, Inc. announced that it will present six-month follow-up results from the randomized Phase I/II HOPE-Duchenne clinical trial of CAP-1002 in boys and young men with Duchenne muscular dystrophy. [Press release from Capricor Therapeutics, Inc. discussing research to be presented at the 22^nd International Congress of the World Muscle Society (WMS), Saint Malo] Press Release

ZIOPHARM Oncology Announces Updated Findings from Phase I Study of Ad-RTS-hIL-12 + Veledimex in Recurrent Glioblastoma Presented

ZIOPHARM Oncology, Inc. announced that updated data from a subset of patients in its Phase I multicenter study of Ad-RTS-hIL-12 + veledimex in patients with recurrent or progressive glioblastoma was presented. [Press release from ZIOPHARM Oncology, Inc. discussing research presented at the 79^th Annual Meeting of the American Academy of Neurological Surgery (AANS), Santa Barbara] Press Release

TxCell: Presentation of New Transplantation CAR-Treg Data

TxCell SA announced that new preclinical data will be presented. An oral presentation entitled ‘Alloantigen-specific regulatory T-cells generated with a chimeric antigen receptor’ will be made. [Press release from TxCell SA discussing research to be presented at the 18^th Congress of the European Society for Organ Transplantation (ESOT), Barcelona] Press Release

UW–Madison to Partner in $20 Million Cell-Based Therapy Center

The National Science Foundation has awarded nearly $20 million to a consortium of universities, including the University of Wisconsin (UW)–Madison, to support a new engineering research center that will develop transformative tools and technologies for the consistent, scalable and low-cost production of high-quality living therapeutic cells. [University of Wisconsin–Madison] Press Release

Mustang Bio Announces Expansion of CAR T Pipeline into CD20-Directed Immunotherapies

Mustang Bio, Inc. announced the expansion of its pipeline of CAR T therapies into CD20-directed immunotherapies. Mustang has entered into an exclusive, worldwide licensing agreement with Fred Hutchinson Cancer Research Center for the use of a CAR T therapy related to autologous T cells engineered to express a CD20-specific chimeric antigen receptor. [Mustang Bio, Inc.] Press Release

BioAxone BioSciences Awarded NINDS SBIR Funding for the Development of Self-Delivering RNAi (sd-rxRNA^®) for the Treatment of Spinal Cord Injury

BioAxone BioSciences, Inc. announced that, as part of a collaborative effort with RXi Pharmaceuticals Corporation, it is the recipient of a grant award from the National Institute of Neurological Disorders and Stroke (NINDS), part of the agency’s SBIR Phase II funding program. This two-year grant provides funding for further development of BioAxone’s preclinical candidate BA-434, a novel sd-rxRNA compound that targets PTEN for the treatment of spinal cord injury. [BioAxone BioSciences, Inc.] Press Release

Autolus Announces First-Dose Cohort Completed in APRIL Study of AUTO2: A Phase I/II Study in Patients with Multiple Myeloma

Autolus Limited announced completion of the first-dose cohort of its Phase I/II study of its novel, dual-targeted CAR in patients with relapsed/refractory multiple myeloma. [Autolus Limited] Press Release

bluebird bio Announces First Patient Treated in Expansion Cohort of CRB-401, Phase I Study of Anti-BCMA CAR T Therapy bb2121

bluebird bio, Inc. announced that the expansion cohort of the CRB-401 Phase I study of bb2121, an anti-BCMA CAR T therapy, has been initiated. The objective of the CRB-401 study is to evaluate the safety and efficacy of bb2121 in patients with relapsed/refractory multiple myeloma and determine a recommended Phase II dose. [bluebird bio, Inc.] Press Release

Nektar Therapeutics Initiates PROPEL Clinical Study to Evaluate Combination of NKTR-214, a CD122-Biased Agonist, with TECENTRIQ^® (Atezolizumab) or KEYTRUDA^® (Pembrolizumab)

Nektar Therapeutics announced that it has begun dosing in the PROPEL clinical study which will evaluate the efficacy and safety of NKTR-214, the company’s lead immuno-oncology candidate in combination with approved checkpoint inhibitors, TECENTRIQ^® and KEYTRUDA^®. NKTR-214 is an investigational immuno-stimulatory therapy designed to expand specific cancer-fighting T cells and natural killer cells directly in the tumor microenvironment and increase expression of PD-1 on these immune cells. [Nektar Therapeutics] Press Release

Formula Pharmaceuticals Announces Initiation of the First Clinical Trial for Allogeneic CIK-CAR Cancer Immunotherapy

Formula Pharmaceuticals, Inc. announced the opening of the first clinical trial for allogeneic cytokine induced killer (CIK) cell-based CAR cancer immunotherapy. [Formula Pharmaceuticals, Inc. (Business Wire, Inc.)] Press Release

Can-Fite Files Patent Application to Treat Cytokine Release Syndrome, a Potentially Life-Threatening Complication of CAR-T Cell Therapy

Can-Fite BioPharma Ltd. announced it has filed a patent application to protect the use of its drugs and other ligands which target the A3 adenosine receptor to treat cytokine release syndrome. [Can-Fite BioPharma Ltd.] Press Release

Brammer Bio Completes Expansion of Cell and Gene Therapy Clinical Manufacturing Facilities in Florida

Brammer Bio announced completion of its clinical capacity expansion in Alachua, Florida. This doubles Brammer’s early phase manufacturing capacity to support clients’ Phase I/II cell and gene therapy clinical trials. [Brammer Bio (PR Newswire Association LLC.)] Press Release

U.S. House Approves 2018 Spending Bills, but Process Far from Finished

The U.S. House of Representatives took a major step toward setting federal science budgets for the 2018 fiscal year. But Congress is still far from the finish line, and final spending levels aren’t likely to be finalized until late this year at the earliest. [ScienceInsider] Editorial

After Uproar, U.K. Parliament’s Science Committee Now Has a Female Member

The House of Commons Science and Technology Committee tweeted that it was “pleased to announced our membership has been confirmed.” Parliamentary committees had been dissolved after the recent U.K. election, and now the panel was rolling out its new contingent of lawmakers. The only problem: All eight of the mostly smiling faces belonged to men. [ScienceInsider] Editorial

Congress Rejects Trump Proposals to Cut Health Research Funds

Congress has not only rejected the president’s National Institutes of Health proposal; lawmakers from both parties have joined forces to increase spending on biomedical research — and have bragged about it. [The New York Times] Editorial

FDA

Medical Devices Regulated by the Center for Biologics Evaluation and Research; Availability of Safety and Effectiveness Summaries for Premarket Approval Applications (FR Doc. No:2017-19607) Notice

NEW Stem Cell Society Singapore (SCSS) Symposium 2017
November 16-17, 2017
Singapore, Singapore

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

NEW Research Scientist – Immuno-Oncology (Kymab Ltd)

Research Technologist – Immunotherapy (STEMCELL Technologies Inc.)

Postdoctoral Position – NK Cell Therapy (Oslo University Hospital)

Technologist – Cell Therapy (Yale New Haven Health)

Researcher – Skeletal Tissue Engineering (KU Leuven)

Postdoctoral Fellow – Cell Biology, Tumor Immunology and Targeted Therapy (University of Minnesota)

Postdoctoral Associate – Enhancer RNA Therapy (Sylvester Comprehensive Cancer Center)

Postdoctoral Fellow – Cell and Gene Therapy Studies Targeting Heart Failure (Icahn School of Medicine at Mount Sinai)

Research Group Leaders (Centro de Investigación Príncipe Felipe)

Research Fellow – Functional Roles of Stem Cell Derived Extracellular Vesicles (Mayo Clinic)

Professor and Faculty Positions – Various Projects (Academy of Medical Sciences, Zhengzhou University)

Postdoctoral Fellow – Various Projects (University of Oklahoma)

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