Cell Therapy News Volume 18.22 | Jun 26 2017

TOP STORY

Tumor Microenvironment-Responsive Multistaged Nanoplatform for Systemic RNAi and Cancer Therapy

In vivo results demonstrated that the multistaged nanoparticle delivery of siRNA against bromodomain 4, a recently discovered target protein that regulates the development and progression of prostate cancer (PCa), can significantly inhibit PCa tumor growth. [Nano Lett] Abstract

(Ranked by impact factor of the journal)

Strontium- and Cobalt-Substituted Bioactive Glasses Seeded with Human Umbilical Cord Perivascular Cells to Promote Bone Regeneration via Enhanced Osteogenic and Angiogenic Activities

For in vivo study, human umbilical cord perivascular cell-seeded bioactive glasses were implanted into an animal’s body. Radiographic imaging, histology and immunohistology staining were performed on the harvested specimens at 4 and 12 weeks post-surgery. The in vivo evaluation of the samples showed that all the cell/glass constructs accelerated bone healing process in comparison with blank controls. [Acta Biomater] Abstract

Favorable Outcomes of Poor Prognosis Diffuse Large B-Cell Lymphoma Patients Treated with Dose-Dense Rituximab, High-Dose Methotrexate and Six Cycles of CHOP-14 Compared to First-Line Autologous Transplantation

Investigators report a 10-year single-centre study of 63 consecutively treated patients. Cohort 1 patients received four cycles R-CHOP-21 followed by first-line high-dose chemotherapy with autologous stem-cell support, resulting in two-year progression-free and overall survival of 60.6% and 67.9%. [Br J Haematol] Abstract

CRISPR/Cas9-Mediated Genome Editing via Postnatal Administration of AAV Vector Cures Hemophilia B Mice

Researchers describe treatment strategies for hemophilia B mice using the CRISPR/Cas9 system. Administration of adeno-associated virus (AAV) 8 vector harbouring Staphylococcus aureus Cas9 and single guide RNA to wild-type adult mice induced a double-strand break at the target site of F9 in hepatocytes, sufficiently developing hemophilia B. [Sci Rep] Full Article

Safe and Effective Administration of T-VEC in a Patient with Heart Transplantation and Recurrent Locally Advanced Melanoma

Investigators present the case of a patient with recurrent locally advanced cutaneous melanoma receiving salvage talimogene laherparepvec (T-VEC) therapy in the setting of orthotropic heart transplantation. [J Immunother Cancer] Full Article

Adoptive Cell Therapy Using PD-1⁺ Myeloma-Reactive T Cells Eliminates Established Myeloma in Mice

Adoptive transfer of ex vivo-expanded PD-1⁺ T cells together with a PD-L1 blocking antibody eliminated established myeloma in Rag-deficient mice. Both CD8 and CD4 T cell subsets were important for eradicating myeloma. [J Immunother Cancer] Full Article

Elevated Bone Marrow Eosinophil Count Is Associated with High Incidence of Severe Acute GvHD after Allogeneic Hematopoietic Stem Cell Transplantation

Scientists evaluated whether elevated eosinophil count in the bone marrow at one month after hematopoietic stem cell transplantation is associated with a high incidence of new and severe acute GvHD. [Bone Marrow Transplant] Abstract

Decorin Gene Upregulation Mediated by an Adeno-Associated Virus Vector Increases Intratumoral Uptake of Nab-Paclitaxel in Neuroblastoma via Inhibition of Stabilin-1

The anticancer effect of recombinant adeno-associated virus-DCN (rAAV-DCN) was assessed in vivo and in vitro. The effect of rAAV-DCN on the intratumoral uptake of paclitaxel was also studied in neuroblastoma-grafted nude mice. [Invest New Drugs] Abstract

The Effect of CD34⁺ Cell Telomere Length and hTERT Expression on the Outcome of Autologous CD34⁺ Cell Transplantation in Patients with Chronic Heart Failure

Researchers investigated the effect of CD34⁺ cell telomere length and hTERT expression on the clinical outcome of autologous CD34⁺ cell transplantation. [Mech Ageing Dev] Abstract

Carbon Nanotube Reinforced Collagen/Hydroxyapatite Scaffolds Improve Bone Tissue Formation In Vitro and In Vivo

The authors developed a new porous scaffold by adding the multi-walled carbon nanotube into collagen (Col)/hydroxyapatite (HA) composites. Data showed that 0.5%CNT/Col/HA group was approximately tenfolds stiffer than Col–HA, and it was superior in promoting bone marrow mesenchymal stem proliferation and spreading, mRNA and protein expressions of bone sialoprotein and osteocalcin than Col–HA group. [Ann Biomed Eng] Abstract

Precision Monitoring of Immunotherapies in Solid Organ and Hematopoietic Stem Cell Transplantation

The authors review recent and rapid advances in precision and genomic medicine approaches to monitoring of post-transplant immunotherapies. [Adv Drug Deliv Rev] Abstract

Sensory Response in Host and Engrafted Astrocytes of Adult Brain In Vivo

Glial transplantation would be a potentially interesting approach for the cell-based therapy for glia-related disorders. There are multiple cell sources for glial transplantation, including neural stem cells, glial progenitors, and pluripotent stem cells. [Glia] Abstract

Synthetic Nucleic Acid Analogues in Gene Therapy: An Update for Peptide-Oligonucleotide Conjugates

The authors sum up recent achievements in the delivery and in vivo administration of synthetic nucleic acid analogues focusing on POCs, and compare their efficiency. [Chembiochem] Abstract

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

TCR(2) Therapeutics Presents Positive Solid Tumor Data for its Novel TRuC™ Engineered T Cell Therapies at the World Preclinical Congress

TCR² Therapeutics Inc. announced data on two of its TRuC™ programs that showed superior persistence and anti-tumor activity of the company’s TRuC™-T cells compared to CAR-T cells in preclinical models. [Press release from TCR² Therapeutics Inc. discussing research presented at the World Preclinical Congress, Boston] Press Release

bluebird bio Announces Early Data from Phase III Northstar-2 (HGB-207) Study of LentiGlobin™ Drug Product

bluebird bio, Inc. announced early interim data from the ongoing Northstar-2 Phase III clinical study of LentiGlobin drug product in patients with transfusion-dependent β-thalassemia and non-β⁰/β⁰ genotypes. [Press release from bluebird bio discussing research presented at the European Hematology Association Annual Meeting, Madrid] Press Release

bluebird bio Presents New Data from HGB-205 Study of LentiGlobinTM Drug Product in Patients with Transfusion-Dependent β-Thalassemia (TDT) and Severe Sickle Cell Disease (SCD)

bluebird bio, Inc. announced new data from the ongoing HGB-205 clinical study evaluating its LentiGlobin gene therapy product candidate in patients with TDT and SCD. [Press release from bluebird bio discussing research presented at the European Hematology Association Annual Meeting, Madrid] Press Release

Xintela Participates in Multi-Million, Government Funded Initiative to Form a Cell and Gene Therapy Research Center

Xintela AB announced that the company is a partner in a project, financed by Vinnova and Vetenskapsrådet, to establish an international research center for effective production and development of Advanced Therapy Medicinal Products in cell and gene therapy. [Xintela AB] Press Release

Sarepta Therapeutics and Genethon Announce Gene Therapy Research Collaboration for Duchenne Muscular Disease (DMD) Therapies

Sarepta Therapeutics, Inc. and Genethon have signed a gene therapy research collaboration to jointly develop treatments for DMD. [Sarepta Therapeutics, Inc.] Press Release

FDA Approves Cryopreserved Formulation of ReNeuron’s Retinal Stem Cell Therapy Candidate

ReNeuron Group plc provided an update on its cell therapy development programs targeting degenerative diseases of the retina. They report that the FDA has approved the cryopreserved formulation of our human retinal progenitor cell therapeutic candidate and that they have now started treating patients with this formulation in their ongoing US Phase I/II study clinical trial in retinitis pigmentosa patients. [ReNeuron Group plc] Press Release

Sarepta Therapeutics Announces FDA Accelerated Approval of EXONDYS 51™ (Eteplirsen) Injection, an Exon Skipping Therapy to Treat Duchenne Muscular Dystrophy (DMD) Patients Amenable to Skipping Exon 51

Sarepta Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval for EXONDYS 51™ as a once weekly intravenous infusion of 30 milligrams per kilogram for the treatment of DMD in patients who have a confirmed mutation in the DMD gene that is amenable to exon 51 skipping. [Sarepta Therapeutics, Inc.] Press Release

Kadimastem Received Approval from the Hadassah Hospital IRB (Helsinki Committee) for Conducting a Clinical Trial in ALS Patients

Kadimastem announced that it had received approval from the IRB of the Hadassah Ein Kerem Hospital for the Phase I/IIa clinical trial in ALS patients using the cell therapy product developed by the company. [Kadimastem] Press Release

BrainStorm Awarded $2.1 Million Non-Dilutive Grant for 2017 by the Israel Innovation Authority

BrainStorm Cell Therapeutics Inc. announced that its wholly-owned subsidiary, Brainstorm Cell Therapeutics Ltd., has been awarded a new grant of approximately $2.1 million by the Israel Innovation Authority. The funds will support the development of NurOwn^®, BrainStorm’s innovative mesenchymal stem cell-based platform for the treatment of neurodegenerative diseases. [BrainStorm Cell Therapeutics Inc.] Press Release

Cell Medica Acquires WT1 Cancer Immunotherapy from Cell and Gene Therapy Catapult

Cell Medica announced the acquisition of Catapult Therapy TCR Limited and the initiation of a collaboration to establish cell therapy manufacturing for Cell Medica at CGT Catapult’s GMP manufacturing facility in Stevenage, UK. [Cell Medica] Press Release

Propagenix Inc. Lands Three Research Grants to Advance Cell Therapy Technology for Type I Diabetes and Lung Disease

Propagenix Inc. has been awarded three research grants totaling $950,000 in direct costs. Two of these grant awards are directed towards the use the company’s cell expansion technology to produce accessory epithelial cell cultures known to enhance the survival and function of pancreatic islets upon implantation in vivo. [Propagenix Inc. (PR Newswire Association LLC.)] Press Release

Scientists in Limbo as US Supreme Court Allows Modified Travel Ban

The US Supreme Court has reinstated a limited version of President Donald Trump’s temporary order banning travellers from six Muslim-majority countries from entering the United States. The court will hear a legal challenge to the ban in October. The court’s decision casts doubt on the fate of students and scientists from these countries who hope to study or work in the United States. [Nature News] Editorial

US Court Grants Elsevier Millions in Damages From Sci-Hub

One of the world’s largest science publishers, Elsevier, won a default legal judgement against websites that provide illicit access to tens of millions of research papers and books. A New York district court awarded Elsevier US$15 million in damages for copyright infringement by Sci-Hub, the Library of Genesis (LibGen) project and related sites. [Nature News] Editorial

University of Tokyo Scientist Hit by Anonymous Allegations Fights Back

Anonymous allegations of questionable data and images in 22 papers by six prominent groups at the prestigious University of Tokyo prompted the school to set up an investigating committee. Now, even before the panel completes its investigation, one of the accused researchers has mounted a staunch defense of his work. [ScienceInsider] Editorial

FDA

Agency Information Collection Activities: Proposed Collection; Comment Request; Guidance for Industry on Registration of Human Drug Compounding Outsourcing Facilities Under Section 503B of the Federal Food, Drug, and Cosmetic Act (FR Doc. No:2017-12838) Notice

Agency Information Collection Activities; Proposed Collection; Comment Request; Orphan Drugs; Common European Medicines Agency/Food and Drug Administration Application Form for Orphan Drug Medicinal Product Designation (FR Doc. No:2017-12620) Notice

NEW European Society for Medical Oncology (ESMO) 2017 Congress
September 8-12, 2017
Madrid, Spain

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

NEW PhD Scholarship – Virology/Gene Therapy (University of New South Wales)

Postdoctoral Scientist – Systems Oncology Group (CRUK Manchester Institute)

Quality Improvement Coordinator – Cellular Therapy Core (University of Texas Health Science Center at Houston)

Postdoctoral Position – Tumor Immunology and Cancer Immunotherapy (VIB)

Professor – Stem Cell Biology (University College London)

Specialist – Cell Procurement (Celgene Corporation)

Scientist – Analytical Development (KBI Biopharma)

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

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)

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

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

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