Volume 15.25 | Jun 30

Issue 15.25 June 30, 2014
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Autologous Hematopoietic Stem Cell Transplantation vs Intravenous Pulse Cyclophosphamide in Diffuse Cutaneous Systemic Sclerosis
High-dose immunosuppressive therapy and autologous hematopoietic stem cell transplantation (HSCT) have shown efficacy in systemic sclerosis in Phase I and small Phase II trials. Investigators compared efficacy and safety of HSCT vs 12 successive monthly intravenous pulses of cyclophosphamide. [JAMA] Abstract | Press Release
Your Free Copy of the Nature Neuroscience Poster "Neural Stem Cells" Awaits
PUBLICATIONS (Ranked by impact factor of the journal)
A Novel Self-Lipid Antigen Targets Human T Cells against CD1c+ Leukemias
Researchers report that CD1c self-reactive T cells recognize a novel class of self-lipids, identified as methyl-lysophosphatidic acids (mLPAs), which are accumulated in leukemia cells. mLPA-specific T cells efficiently kill CD1c+ acute leukemia cells, poorly recognize nontransformed CD1c-expressing cells, and protect immunodeficient mice against CD1c+ human leukemia cells. [J Exp Med] Abstract | Press Release

Activity of Broad-Spectrum T Cells as Treatment for AdV, EBV, CMV, BKV, and HHV6 Infections after HSCT
Scientists demonstrate the feasibility and clinical utility of rapidly generated single-culture virus-specific T cells that recognize 12 immunogenic antigens from five viruses (Epstein-Barr virus, adenovirus, cytomegalovirus, BK virus, and human herpesvirus 6) that frequently cause disease in immunocompromised patients. [Sci Transl Med] Abstract | Press Release | Video

Degradable Lipid Nanoparticles with Predictable In Vivo siRNA Delivery Activity
The authors synthesized 1,400 degradable lipidoids and evaluated their transfection ability and structure-function activity. They showed that lipidoid nanoparticles mediate potent gene knockdown in hepatocytes and immune cell populations on IV administration to mice. [Nat Commun] Abstract

Allele-Specific Genome Editing and Correction of Disease-Associated Phenotypes in Rats Using the CRISPR-Cas Platform
Investigators employed CRISPR/Cas for accurate and efficient genome editing in rats. The synthetic chimeric guide RNAs discriminate a single-nucleotide polymorphism difference in rat embryonic fibroblasts, allowing allele-specific genome editing of the dominant phenotype in (F344 × DA)F1 hybrid embryos. [Nat Commun] Full Article

HucMSC-Exosome Mediated -Wnt4 Signaling Is Required for Cutaneous Wound Healing
The authors examined the benefit of human umbilical cord mesenchymal stem cell derived exosome (hucMSC-Ex) in cutaneous wound healing using a rat skin burn model. They found that hucMSC-Ex-treated wounds exhibited significantly accelerated re-epithelialization, with increased expression of CK19, PCNA, collagen I (compared to collagen III) in vivo. [Stem Cells] Abstract | Full Article

Poly(γ-Glutamic Acid) Based Combination of Water-Insoluble Paclitaxel and TLR7 Agonist for Chemo-Immunotherapy
Immuno-stimulating agent toll-like receptor-7 (TLR7) agonist-imiquimod and low dose chemotherapeutic agent-paclitaxel were synergized to demonstrate tumor therapy along with anti-tumor memory effect. [Biomaterials] Abstract

Chronic Restraint Stress Decreases the Repair Potential from Mesenchymal Stem Cells on Liver Injury by Inhibiting TGF-β1 Generation
Researchers investigated the effect of chronic restraint stress on therapeutic potential of mesenchymal stem cells on carbon tetrachloride-induced liver injury in mice. [Cell Death Dis] Full Article

Radiation-Induced Autophagy Potentiates Immunotherapy of Cancer via Up-Regulation of Mannose 6-Phosphate Receptor on Tumor Cells in Mice
Researchers report that radiation therapy potentiates the effect of immune therapy via induction of autophagy and resultant trafficking of mannose-6-phopsphate receptor (MPR) to the cell surface. Irradiation of different tumor cells caused substantial up-regulation of MPR on the cell surface in vitro and in vivo. [Cancer Immunol Immunother] Abstract

Manufacturing of Highly Functional and Specific T Cells for Adoptive Immunotherapy against Virus from Granulocyte Colony-Stimulating Factor-Mobilized Donors
Researchers successfully manufactured highly specific, functional and cytotoxic cytomegalovirus-specific T cells from granulocyte colony-stimulating factor-mobilized donor peripheral blood mononuclear cells. [Cytotherapy] Abstract

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Complexity in the Therapeutic Delivery of RNAi Medicines: An Analytical Challenge
The authors reflect on the complexity in the therapeutic delivery of RNA interference-based drugs emerging from the recent clinical experiences and report the actual technological and analytical advances introduced to solve it. [Expert Opin Drug Deliv] Abstract

Visit our reviews page to see a complete list of reviews in the cell, gene and immunotherapy research field.

Learn more: StemSpan™ CD34+ Cell Expansion Supplement
Researchers Demonstrate Promise of Dicerna Investigational Therapy in Preclinical Model of Primary Hyperoxaluria Type 1 (PH1)
Dicerna Pharmaceuticals, Inc., a leader in the development of RNAi-based therapeutics, announced the presentation of preclinical data demonstrating the promise of DCR-PH1, the company’s therapeutic candidate for the treatment of PH1, a rare inherited liver disorder that often results in progressive and severe kidney damage. [Press release from Dicerna Pharmaceuticals, Inc. discussing research presented at the 11th International Primary Hyperoxaluria Workshop, Chicago] Press Release

Actinium’s Actimab-A and Alpha-Particle Immunotherapy Platform Further Validated
Actinium Pharmaceuticals, Inc. provided highlights from an educational session featuring Actimab-A, a radiolabeled antibody currently in a Phase I/II clinical trial. [Press release from Actinium Pharmaceuticals, Inc. discussing research presented at the 2014 Society for Nuclear Medicine and Molecular Imaging (SNMMI) Annual Meeting, St. Louis] Press Release

Cell Culture World Congress Asia 2014
Australian Scientists Take to the Streets to Protest Job Cuts
Abandoning their usual reserve, nearly 1000 scientists across the country downed instruments and grabbed placards to protest pending job losses at the nation’s leading research organization, the Commonwealth Scientific and Industrial Research Organization. [ScienceInsider] Editorial

U.S. Aging Researchers Prepare for Loss of Hungry Mouse Colony
The National Institute on Aging, part of the U.S. National Institutes of Health, revealed earlier this month that it will be phasing out its colony of calorie-restricted rodents. Although most researchers who study aging won’t be affected by the decision, some scientists will have to pay substantially more for experimental mice, and some may be priced out of the field. [ScienceInsider] Editorial
Bone Therapeutics Starts Patient Treatment in First Ever Clinical Trial with Allogeneic Bone Cell Therapy Product
Bone Therapeutics announced that the first patient has been treated with its novel allogeneic osteoblastic (bone-forming) cell therapy product ALLOB® in its Phase I/IIa study for the treatment of delayed union fractures. [Bone Therapeutics] Press Release

Merck’s Investigational Anti-PD-1 Antibody, Pembrolizumab, under Regulatory Review in Europe for the Treatment of Advanced Melanoma
Merck announced the European Medicines Agency has accepted for review a marketing authorization application for pembrolizumab (MK-3475), the company’s investigational anti-PD-1 antibody, for the treatment of advanced melanoma. [Merck] Press Release

Phase III First-Line Melanoma Study of Nivolumab, an Investigational PD-1 Checkpoint Inhibitor, Demonstrates Superior Overall Survival Compared to Dacarbazine; Study Stopped Early
Bristol-Myers Squibb Company announced that a randomized blinded comparative Phase III study evaluating nivolumab versus dacarbazine in patients with previously untreated BRAF wild-type advanced melanoma was stopped early because an analysis conducted by the independent data monitoring committee showed evidence of superior overall survival in patients receiving nivolumab compared to the control arm. [Bristol-Myers Squibb Company] Press Release

Calimmune Approved to Treat Second Group in HIV Stem Cell Gene
Calimmune, Inc. announced that encouraging results from a first group of participants indicates the company is ready to begin treating a second cohort in a clinical trial involving the use of Cal-1, an innovative gene-based stem cell therapy to help protect individuals infected with HIV from progressing to AIDS. [Calimmune, Inc.]
Press Release

Pluristem Selects Rotator Cuff Repair as New Orthopedic Indication
Pluristem Therapeutics Inc. announced it has selected rotator cuff repair as the second indication in its orthopedic clinical program. The company’s placental expanded cells offer potential as the first off-the-shelf cell therapy product for muscle and tendon regeneration that can be used to enhance repair after rotator cuff surgery. [Pluristem Therapeutics Inc.] Press Release

Yale Stem Cell Researchers Receive $5.6 Million in State Grants
Yale researchers received a total of $5.6 million for stem cell research out of $10 million given to 18 state-based researchers. The largest sum, $1.2 million, is a group grant given to Yale University, led by principal investigator Joseph Craft, for research into the therapeutic role of induced pluripotent stem cells in human lupus, an autoimmune disease. [The New Haven Register] Article

Multiple Myeloma Research Foundation (MMRF) Launches Linda Rodgers Emory Fund for Accelerating Immune Therapy in Multiple Myeloma
MMRF announced the generous donation of $1 million by Linda Rodgers Emory, a longtime supporter of the MMRF and recognized philanthropist. The Linda Rodgers Emory Fund for Accelerating Immune Therapy in Multiple Myeloma will urgently advance a pipeline of immunotherapeutic agents to benefit myeloma patients and enable critical research into the basic biology and mechanisms of immune response in myeloma. [Multiple Myeloma Research Foundation] Press Release

Cellectis’ UCART19 Receives Advanced-Therapy Medicinal Product Classification from EMA
Cellectis announced it received a scientific recommendation from the European Medicines Agency (EMA), in consultation with the European Commission, for UCART19, its lead product candidate in adoptive immunotherapy against CD19 expressing leukemias and lymphomas. [Cellectis] Press Release

Frederic Bushman, Ph.D. Receives Pioneer Award for Advancing Therapeutic Gene Delivery Methods
Frederic D. Bushman’s early pioneering work in understanding how HIV reproduces by inserting its genetic material into the DNA of a host cell led to key advances in the ability to move pieces of DNA and whole genes between cells. In recognition of his scientific achievements and leadership in the field, Dr. Bushman is the recipient of a Pioneer Award from Human Gene Therapy. [Mary Ann Liebert, Inc. (EurekAlert!)] Press Release | Editorial
National Institute of General Medical Sciences Notice of Closed Meetings (FR Doc. No: 2014-14933)

National Center for Advancing Translational Sciences (NCATS): Cooperative Research and Development Agreement (CRADA) and Licensing Opportunity for Small Molecule Inhibitors of the Human USP1/UAF1 Complex(1) for the Treatment of Cancer (FR Doc. No: 2014-14719)
Summary Basis for Regulatory Action – Eloctate
Food and Drug Administration (United States)

Global Unique Device Identification Database; Guidance for Industry and Food and Drug Administration Staff; Availability (FR Doc. No: 2014-15020)

Blood Products Advisory Committee; Notice of Meeting (FR Doc. No: 2014-14922)
NEW Advances in Stem Cell Research 2014
September 16-17, 2014

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Cell Therapy Lab Assistant Supervisor (Fred Hutchinson Cancer Research Center)

Postdoctoral Fellow – AAV Vectors for Gene Therapy of Neurological and Other Disorders (University of Massachusetts Medical School)

Postdoctoral Scientist – Immune Focused In Vitro Analytics (Celentyx Ltd.)

Assistant or Associate Professor – Stem Cell Transplantation Program (Dana-Farber Cancer Institute)

PhD Studentship – Endogenous Retroviruses as Immunotherapy Targets (University of Plymouth)

Postdoctoral Fellow – Prostate Cancer Research (Henry Ford Hospital)

Postdoctoral Position – Neurotoxicity and Epigenetics (Thomas Jefferson University)

Research/Review Scientist – Cell Therapy and Regenerative Medicine (Center for Biologics Evaluation and Research)

Postdoctoral Position – Cell Death and Inflammation (Inflammation Research Center – VIB-UGent)

Postdoctoral Position – Role of Calreticulin in TGF-Beta Signaling in the Kidney Proximal Tubule under Diabetic Conditions (University of Alabama)

Postdoctoral Fellow – Transgene Immunity in AAV Vector-Based Antiviral and/or Gene Therapeutics (University of Massachusetts)

Postdoctoral Fellow – Neurophysiology of Addiction (Medical University of South Carolina)

Principal Investigator – Molecular Mechanisms of Inflammation (Inflammation Research Center – VIB-UGent)

Scientist – Pluripotent Stem Cell Media Development, High Throughput Screening (STEMCELL Technologies Inc.)

Scientist – Cell Culture Support Products (STEMCELL Technologies Inc.)

Scientist – Liver Cell Biology (STEMCELL Technologies Inc.)

Scientist – Immunology/Cell Separation (STEMCELL Technologies Inc.)

Research Associate – Cell Separation (STEMCELL Technologies Inc.)

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