Volume 16.45 | Dec 7

Issue 16.45 December 7, 2015
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Phase I/II Trial of StemRegenin-1 Expanded Umbilical Cord Blood Hematopoietic Stem Cells Supports Testing as a Stand-Alone Graft
The authors evaluated the feasibility and safety of StemRegenin-1 (SR-1), an aryl hydrocarbon receptor antagonist that expands CD34+ cells, by placing one of the two units in expansion culture. SR-1 produced a 330-fold increase in CD34+ cells and led to engraftment in 17/17 patients at a median of 15 days for neutrophils and 49 days for platelets, significantly faster than in patients treated with unmanipulated umbilical cord blood. [Cell Stem Cell] Abstract | Graphical Abstract
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PUBLICATIONS (Ranked by impact factor of the journal)
Mouse Models of Human PIK3CA-Related Brain Overgrowth Have Acutely Treatable Epilepsy
Scientists generated mouse models expressing the most common activating catalytic subunit of phosphoinositide 3-kinase (Pik3ca) mutations in developing neural progenitors. They demonstrated that acute one hour-suppression of PI3K signaling despite the ongoing presence of dysplasia has dramatic anti-epileptic benefit. [eLife]
Abstract | Full Article | Press Release

Umbilical Cord Mesenchymal Stromal Cell with Autologous Bone Marrow Cell Transplantation in Established Type 1 Diabetes: A Pilot Randomized Controlled Open-Label Clinical Study to Assess Safety and Impact on Insulin Secretion
Researchers determined the safety and effects on insulin secretion of umbilical cord mesenchymal stromal cells plus autologous bone marrow mononuclear cell stem cell transplantation without immunotherapy in established type 1 diabetes. [Diabetes Care] Abstract | Full Article

Lack of Survival Advantage with Autologous Stem-Cell Transplantation in High-Risk Neuroblastoma Consolidated by Anti-GD2 Immunotherapy and Isotretinoin
High-risk neuroblastoma patients at a center received anti-GD2 antibody 3F8/GM-CSF + isotretinoin – but not myeloablative therapy with autologous stem-cell transplantation (ASCT). Post-ASCT patients referred from elsewhere also received 3F8/GM-CSF + isotretinoin. Scientists therefore accrued a study population of two groups treated during the same period and whose consolidative therapy, aside from ASCT, was identical. [Oncotarget] Full Article

Clonal Dynamics In Vivo of Virus Integration Sites of T Cells Expressing a Safety Switch
Researchers characterized the clonal diversity and dynamics of viral insertion sites in inducible caspase-9 gene-T cells pre- and post-chemical inducer of dimerization administration in four patients who developed graft versus host disease. [Mol Ther] Abstract

Human Embryonic Stem Cell-Derived Mesenchymal Cells Preserve Kidney Function and Extend Lifespan in NZB/W F1 Mouse Model of Lupus Nephritis
The authors showed that human embryonic stem cell-mesenchymal stromal cells (hESC-MSCs) prevent the progression of fatal lupus nephritis in NZB/W F1 mice. Treatment led to statistically significant reductions in proteinuria and serum creatinine and preserved renal architecture. Specifically, hESC-MSC treatment prevented disease-associated interstitial inflammation, protein cast deposition, and infiltration of CD3+ lymphocytes in the kidneys. [Sci Rep] Full Article | Press Release

Busulfan and Cyclosphamide Induce Liver Inflammation through NLRP3 Activation in Mice after Hematopoietic Stem Cell Transplantation
Investigators evaluated the role of NLRP3 inflammasome on busulfan combined with cyclosphamide-induced liver inflammation in mice after hematopoietic stem cell transplantation. [Sci Rep] Full Article

Immunoablation and Autologous Hematopoietic Stem Cell Transplantation in the Treatment of New-Onset Type 1 Diabetes Mellitus: Long-Term Observations
The activity of the autoimmune mechanism underlying type 1 diabetes mellitus can be suppressed when immunoablation and autologous hematopoietic stem cell transplantation are applied early in the course of the disease. Scientists report a single center experience with this treatment modality. [Bone Marrow Transplant] Abstract

Effects of Mesenchymal Stem Cell Treatment on the Expression of Matrix Metalloproteinases and Angiogenesis during Ischemic Stroke Recovery
The authors investigated the optimal time point of mesenchymal stem cell (MSC) transplantation. After MSC treatment, they also investigated the expression of matrix metalloproteinases, which play a role in vascular and tissue remodeling. [PLoS One] Full Article

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Interneuron Transplantation as a Treatment for Epilepsy
The authors review recent efforts to isolate and transplant cortical interneuron precursors derived from embryonic mouse and human cell sources. They also discuss some of the important challenges that must be addressed before stem-cell-based treatment for human epilepsy is realized. [Cold Spring Harb Perspect Med] Full Article

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SELLAS Life Sciences Reports Positive WT1 Cancer Vaccine Clinical Results in Mesothelioma and Acute Myeloid Leukemia (AML) Patients
SELLAS Life Sciences Group announced that positive data from the company’s clinical studies of its WT1 cancer vaccine in patients with malignant pleural mesothelioma and AML were reported. [Press release from SELLAS Life Sciences Group discussing research presented at the International WT1 Conference, Kyoto] Press Release

Bristol-Myers Squibb to Present New Data across Multiple Blood Cancers
Bristol-Myers Squibb will present data for elotuzumab in patients with relapsed or refractory (R/R) multiple myeloma; for Opdivo for investigational use in patients with R/R classical Hodgkin lymphoma; and for Sprycel in chronic myeloid leukemia. [Press release from Bristol-Myers Squibb Company discussing research to be presented at the 57th American Society of Hematology (ASH) Annual Meeting, Orlando] Press Release

City of Hope Researchers Present Study Results
Among the research presented by City of Hope scientists will be results of three Phase I clinical trials that lay the groundwork for novel leukemia or lymphoma treatments. [Press release from City of Hope discussing research to be presented at the 57th American Society of Hematology (ASH) Annual Meeting, Orlando] Press Release

Novartis Announces New CTL019 Study Data Demonstrating Overall Response in Adult Patients with Certain Types of Lymphoma
Findings from an ongoing Phase IIa study to evaluate the safety and efficacy of investigational chimeric antigen receptor T cell therapy CTL019 in certain types of relapsed or refractory non-Hodgkin lymphoma were presented. [Press release from Novartis AG discussing research presented at the 57th American Society of Hematology (ASH) Annual Meeting, Orlando] Press Release

Amgen Presents Data from Three Trials Evaluating BLINCYTO® (Blinatumomab) In Acute Lymphoblastic Leukemia
Amgen announced that new data from three Phase II trials support the efficacy and safety of BLINCYTO® in adults with acute lymphoblastic leukemia. [Press release from Amgen, Inc. discussing research presented at the 57th American Society of Hematology (ASH) Annual Meeting, Orlando] Press Release

Gene Therapy Restores Immunity in Children and Young Adults with Rare Immunodeficiency
Gene therapy can safely rebuild the immune systems of older children and young adults with X-linked severe combined immunodeficiency, a rare inherited disorder that primarily affects males, scientists have found. [Press release from National Institute of Allergy and Infectious Diseases discussing research presented at the 57th American Society of Hematology (ASH) Annual Meeting, Orlando] Press Release

Juno’s Investigational CAR T Cell Product Candidates JCAR014 and JCAR018 Demonstrate Encouraging Clinical Responses in Patients with B-Cell Cancers
Juno Therapeutics, Inc. announced, in partnership with its collaborators, that clinical data from a chimeric antigen receptor (CAR) T cell product candidate, JCAR014, demonstrated encouraging clinical responses in patients with relapsed or refractory non-Hodgkin lymphoma and chronic lymphocytic leukemia. [Press release from Juno Therapeutics, Inc. discussing research presented at the 57th American Society of Hematology (ASH) Annual Meeting, Orlando] Press Release

IMiD® Combination Therapies Form Backbone of Multiple Myeloma Research
Celgene Corporation announced that nearly 30 combination studies including IMiD® compounds, REVLIMID® and POMALYST®/IMNOVID®, across a range of patient segments within multiple myeloma are being presented. [Press release from Celgene Corporation discussing research presented at the 57th American Society of Hematology (ASH) Annual Meeting, Orlando] Press Release

bluebird bio Presents Pre-Clinical and Manufacturing Data from CAR T Oncology Programs
bluebird bio, Inc., a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic diseases and T cell-based immunotherapies for cancer, announced that pre-clinical data from its anti-B cell maturation antigen oncology program were presented. [Press release from bluebird bio, Inc. discussing research presented at the 57th American Society of Hematology (ASH) Annual Meeting, Orlando] Press Release

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Inside the Summit on Human Gene Editing: A Reporter’s Notebook
A congressman’s warning. A mother’s heart-wrenching appeal. The long shadow of eugenics. Philosophical, ethical, and moral debates. Uncertain science and patchy regulations. And a cast that included international delegations and the three scientists widely expected to share a Nobel Prize for a new DNA-changing technology, commonly called CRISPR. All was on display—and streaming live online—at the International Summit on Human Gene Editing. [ScienceInsider] Editorial

Italian Scientists Slam Selection of Stem-Cell Trial
Italian politicians have kindled the wrath of some biomedical scientists by hand-picking a stem-cell clinical trial for funding. The €3 million pot for the trial should be allocated through an open competition based on scientific merit rather than in an amendment to the country’s 2016 budget bill, say the researchers. [Nature News] Editorial

Pirate Research-Paper Sites Play Hide-and-Seek with Publishers
Operators of internet repositories that provide illicit free access to millions of research papers seem determined to keep up their services, despite being barred by an injunction. [Nature News] Editorial

The Truth about Fetal Tissue Research
An explosive climate has surrounded US research with fetal tissues since July, when an anti-abortion group called the Center for Medical Progress in Irvine, California, released covertly filmed videos in which senior physicians from the Planned Parenthood Federation of America bluntly and dispassionately discussed their harvesting of fetal organs from abortions for use in research. [Nature News] Editorial

Embryo Editing to Make Babies Would Be ‘Irresponsible,’ Says DNA Summit Statement
It would currently be “irresponsible” to use new DNA editing tools to alter the genomes of human embryos, eggs, or sperm in order to produce a baby, the organizing committee of an international summit on the issue concluded today in a statement. [ScienceInsider] Editorial
SQZ Biotech Announces Partnership to Fight Cancer with Novel Cell Engineering Technology
SQZ Biotech announced a partnership with Roche to develop a cell therapy platform that would empower a patient’s own immune cells to fight a broad range of cancers. [SQZ Biotech] Press Release

Adaptimmune and Universal Cells Announce Collaboration to Develop Allogeneic T-Cell Therapies
Adaptimmune Therapeutics plc and Universal Cells Inc. announced that they have entered into a collaboration and exclusive license agreement for the development of allogeneic T-cell therapies. [Adaptimmune Therapeutics plc] Press Release

TxCell Signs Strategic Agreement with MaSTherCell for European Manufacturing of Its Cell Therapy Products
TxCell SA announced it has signed a five-year agreement with MaSTherCell. TxCell already appointed MaSTherCell for the manufacturing of TxCell’s lead product Ovasave® for the ongoing CATS29 study. CATS29 is a Phase IIb trial of Ovasave conducted in refractory Crohn’s diseases patients. [TxCell SA] Press Release

Amgen and Merck Announce Cancer Immunotherapy Collaboration for Patients with Non-Hodgkin Lymphoma
Amgen and Merck announced a cancer immunotherapy collaboration to support a Phase Ib/III study investigating BLINCYTO® (blinatumomab) in combination with KEYTRUDA® (pembrolizumab) in patients with diffuse large B-cell lymphoma. [Amgen Inc.] Press Release

bluebird bio and ViroMed Enter into License Agreement for Novel Antibodies to Develop Chimeric Antigen Receptor T Cell Therapy
bluebird bio, Inc. and ViroMed Co., Ltd. announced that they have entered into an exclusive license agreement to research, develop and commercialize chimeric antigen receptor T cell therapies using ViroMed’s proprietary humanized antibody to an undisclosed cancer target for solid tumors. [bluebird bio Inc.] Press Release

CEL-SCI Reports Monthly Patient Enrolment in November for Its Phase III Head and Neck Cancer Trial
CEL-SCI Corporation announced that in the month of November it has enrolled 27 patients in its ongoing Phase III trial of its investigational immunotherapy Multikine in patients with advanced primary head and neck cancer. [CEL-SCI Corporation] Press Release

Cuban Cancer Vaccine Extended to Policlinics
The Cuban CIMAVAX EGF therapeutic vaccine produced in the Molecular Engineering Center has begun new clinical testing against lung cancer in policlinics throughout the country. [Molecular Engineering Center (Cuban News Agency)] Press Release

Partikula Reports Positive Results from a Multi-Checkpoint Protein Inhibition Study of Its First-in-Class Precision Tumor Microenvironment Modifiers (TMEM™)
Partikula LLC announced results from a study designed to assess the ability of its first-in-class compound, KULA101 to modify the tumor microenvironment and thereby increase immunological activation against cancer cells. [Partikula LLC (Business Wire)] Press Release

Texas Biomed Receives $5 Million Grant to Study Possible Functional Cure for Babies Born with HIV
The National Institutes of Health have awarded scientists at Texas Biomedical Research Institute and collaborators at the Food and Drug Administration, UCLA and the University of Pennsylvania a $5 million grant over the next four years to study a combination antiviral drugs and investigative AIDS vaccines aimed at treating infants and children affected by HIV. [Texas Biomedical Research Institute] Press Release
National Human Genome Research Institute; Notice of Closed Meeting (FR Doc. No: 2015-30517)
Food and Drug Administration (United States)

Recommendations for Assessment of Blood Donor Suitability, Donor Deferral and Blood Product Management in Response to Ebola Virus; Draft Guidance for Industry; Availability (FR Doc. No: 2015-30589)
NEW Cell & Gene Therapy World
January 25-27, 2016
Washington DC, United States

NEW Immunotherapy World
January 25-27, 2016
Washington DC, United States

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Professor – Regenerative Vision Research (University of Montreal)

NEW Assistant Professor – Regenerative Kidney Research (University of Montreal)

NEW PhD/MSc Position – Cancer Research and Wound Healing (Technion, Israel Institute of Technology)

NEW Postdoctoral Position – Cell Mechanobiology (Washington University in St. Louis)

NEW Open Scientist Positions – Gene and Cell Therapy (Autolus)

Postdoctoral Position – Neurosensory Stem Cell Biology and Regenerative Therapy of the Inner Ear (Universitätsklinikum Tübingen)

Research Technician – Preclinical and Translational Research (Qu Biologics Inc.)

Investigator – Cancer Biology (Wake Forest Baptist School of Medicine)

Postdoctoral Position – Scaffold Design & Drug/Gene Delivery for Neural Tissue Engineering (Nanyang Technological University)

Postdoctoral Position – Stem Cell Engineering Research (Nanyang Technological University)

Postdoctoral Position – hPSC-Derived Cardiomyocyte Grafts in Injured Hearts (University Health Network)

Group Leader – Translational Research (Cancer Research Center of Toulouse)

Group Leader Position – Cancer Research (Oslo University Hospital)

Research Technician – TCR Gene Therapy (Fred Hutchinson Cancer Research Center)

Assistant/Associate/Full Professor – Biomedical Engineering (Worcester Polytechnic Institute)

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