Volume 16.25 | Jun 29

Issue 16.25 June 29, 2015
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Experimental Treatment Sends Deadly Leukemia into Remission
An experimental new treatment approach for a rare, deadly leukemia can send the disease into remission even in patients for whom the standard therapy has failed, buying them more time to have the stem cell transplant that could save their lives, a small pilot study has found. [Press release from University of Virginia Health System discussing online prepublication in Science Translational Medicine] Press Release | Abstract
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PUBLICATIONS (Ranked by impact factor of the journal)
Plerixafor+G-CSF-Mobilized CD34+ Cells Represent an Optimal Graft Source for Thalassemia Gene Therapy
CD34+ cells from 31 thalassemic patients mobilized with Hydroxyurea+G-CSF, G-CSF, Plerixafor or Plerixafor+G-CSF were transduced with the TNS9.3.55 β-globin lentivector and compared for transducibility and globin expression in vitro, as well as engraftment potential in a xenogeneic model after partial myeloablation. [Blood] Abstract

Viral Infection of Tumors Overcomes Resistance to PD-1-Immunotherapy by Broadening Neoantigenome-Directed T-Cell Responses
Investigations on neoepitope-specific T-cell responses in tumor-bearing mice demonstrated that PD-1 immunotherapy was insufficient whereas viral oncolysis elicited cytotoxic T-cell responses to a conserved panel of neoepitopes. [Mol Ther] Abstract

Amniotic Fluid Stem Cells Provide Considerable Advantages in Epidermal Regeneration: B7H4 Creates a Moderate Inflammation Microenvironment to Promote Wound Repair
In vivo, in a mouse excisional wound model, GFP-positive human amniotic fluid stem (hAFS) cells participate in wound repair. Co-localization of GFP/K14 and GFP/K10 in the repaired epidermis demonstrated that hAFS cells can differentiate into keratinocytes. [Sci Rep] Full Article

Antitumor and Adjuvant Activity of λ-Carrageenan by Stimulating Immune Response in Cancer Immunotherapy
Researchers found that intratumoral injection of λ-carrageenan could inhibit tumor growth in B16-F10 and 4T1 bearing mice and enhance tumor immune response by increasing the number of tumor-infiltrating M1 macrophages, DCs and more activated CD4+CD8+ T lymphocytes in spleen. [Sci Rep] Full Article

Conditioning with Rabbit versus Horse ATG Dramatically Alters Clinical Outcomes in Identical Twins with Severe Aplastic Anemia Transplanted with the Same Allogeneic Donor
The authors report a case of two monozygotic twins with constitutional severe aplastic anemia that evolved to myelodysplastic syndrome who both underwent allogeneic peripheral blood stem cell transplantation from the same single HLA antigen mismatched sibling donor with the only difference in the transplant regimen being the type of antithymocyte globulin (ATG) used in the preparative regimen. [J Hematol Oncol] Abstract | Full Article

Adipose- and Bone Marrow-Derived Mesenchymal Stem Cells Prolong Graft Survival in Vascularized Composite Allotransplantation
Investigators compared the in vitro and in vivo immunomodulatory efficacy of adipose-derived mesenchymal stem cells (MSCs) and bone marrow-derived MSCs in a rat vascularized composite allotransplantation model. [Transplantation] Abstract

Cardiac Engraftment of Genetically-Selected Parthenogenetic Stem Cell-Derived Cardiomyocytes
Following engraftment into isogenic recipient hearts, selected cardiomyocytes formed a functional syncytium with the host myocardium as evidenced by the presence of entrained intracellular calcium transients. [PLoS One] Full Article

Intratumoral INF-γ Triggers an Antiviral State in GL261 Tumor Cells: A Major Hurdle to Overcome for Oncolytic Vaccinia Virus Therapy of Cancer
Scientists used a comparison of different tumor locations and different immunologic and genetic backgrounds to determine the replication efficacy and oncolytic potential of the vaccinia virus LIVP 1.1.1, an attenuated wild-type isolate of the Lister strain, in murine GL261 glioma models. [Mol Ther Oncolytics] Full Article

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IL-12 and IL-23 Cytokines: From Discovery to Targeted Therapies for Immune-Mediated Inflammatory Diseases
The authors discuss the therapeutic rationale for targeting cytokines, the unintended consequences for host defense and tumor surveillance and potential ways in which therapies can be applied to treat additional immune disorders. [Nat Med] Full Article

Mesenchymal Stromal Cells and Hematopoietic Stem Cell Transplantation
The authors critically discuss recent advances in mesenchymal stromal cell therapy by reporting on the most relevant studies in the field of hematopoietic stem cell transplantation. [Immunol Lett] Abstract

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

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Preclinical Data on Neuralstem’s Genetically Modified HK532-IGF-1 Stem Cells in Alzheimer’s Disease Presented at International Society for Stem Cell Research Annual Meeting
Neuralstem, Inc. announced that the poster “Human Neural Stem Cells Expressing IGF-1: A Novel Cellular Therapy for Alzheimer’s Disease” was presented. [Press release from Neuralstem, Inc. discussing research presented at the International Society for Stem Cell Research (ISSCR) Annual Meeting, Stockholm] Press Release

Baxalta Reports Continued Progress on Phase I/II Clinical Trial of BAX335, Investigational Gene Therapy Treatment for Hemophilia B
Baxalta Incorporated reported continued progress on the Phase I/II open-label clinical trial assessing the safety and optimal dosing level of BAX 335, an investigational factor IX gene therapy treatment for hemophilia B. [Press release from Baxter International Inc. discussing research presented at the 2015 International Society on Thrombosis and Haemostasis (ISTH) Congress, Toronto] Press Release

Stem Cells & Regenerative Medicine Congress 2015
What to Do with $1 Billion in Cancer Funding? Oregon University to Provide Answer
The Oregon Health & Science University in Portland is gearing up to see how far $1 billion will go toward better cancer detection. The university declared victory in a challenge launched in 2013 by Nike shoe mogul Phil Knight and his wife, Penny, who offered up a $500 million donation if the university could match that amount through fundraising. [ScienceInsider] Editorial

Researchers Frustrated by Failure to Roll Out ‘Game-Changing’ Ebola Test
A test kit that diagnoses Ebola rapidly using just a finger prick of blood could save lives in the ongoing epidemic in West Africa. But researchers are perplexed as to why the diagnostic kit has not been deployed in the field, despite both the World Health Organization and the US Food and Drug Administration approving it four months ago for emergency use. [Nature News] Editorial

Senate Panel Approves $2 Billion Raise for NIH in 2016
A Senate panel approved a bill that would bestow a generous $2 billion increase on the National Institutes of Health (NIH) in 2016, or what appears to be a 6% raise, to $32 billion. [ScienceInsider] Editorial

E.U. Science Chief Wants to Work ERC Magic on Innovation
The European Union’s research commissioner Carlos Moedas has proposed setting up a European Innovation Council to fund applied research and innovation. Inspired by the well-loved European Research Council (ERC), this idea is one of several measures announced to boost innovation across the union. [ScienceInsider] Editorial

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Services and Research Collaboration Agreement between CellResearch Corporation Pte Ltd and the University of Colorado School of Medicine
CellResearch Corporation Pte Ltd has entered into a Services and Research Collaboration Agreement with the University of Colorado School Of Medicine and ClinImmune Labs. The Service and Research Collaboration Agreement has the objective of achieving US FDA approval for therapeutic products of umbilical cord lining membrane progenitor/stem cells. [CellResearch Corporation Pte Ltd] Press Release

Lilly and Immunocore Announce Immunotherapy-Based Clinical Trial Collaboration in Melanoma
Eli Lilly and Company and Immunocore Limited announced that they have entered into an immunotherapy-based clinical trial collaboration to explore the utility of Immunocore’s lead T cell receptor-based investigational therapeutic, IMCgp100, in combination with Lilly’s galunisertib and merestinib for the treatment of melanoma. [Eli Lilly and Company] Press Release

Teva and Active Biotech Announce Completion of Patient Enrollment in Laquinimod Phase III CONCERTO Trial
Teva Pharmaceutical Industries Ltd. and Active Biotech announced that the patient enrollment for the pivotal Phase III CONCERTO trial has been finalized, as well as a planned sample size re-assessment analysis of the study. [Teva Pharmaceutical Industries Ltd.] Press Release

In-Man Trials of Manufactured Blood within Two Years
Lab produced red blood cells are set to be transfused into humans by 2017, NHS Blood and Transplant announced. The in-man clinical trials of manufactured blood form a key part of the blood and organ service’s 2020 Research and Development program. [NHS Blood and Transplant] Press Release

Hans Clevers Awarded McEwen Award for Innovation at ISSCR 2015
The International Society for Stem Cell Research (ISSCR) has presented EuroStemCell partner, Hans Clevers, with the McEwen Award for Innovation at the opening of its annual meeting. [EuroStemCell] Press Release

Celladon Corporation Provides Business Update
Celladon Corporation confirmed its plans to suspend further research or development of its MYDICAR (AAV1/SERCA2a) program and its other pre-clinical programs including the Stem Cell Factor gene therapy and SERCA2b small molecule programs. [Celladon Corporation] Press Release

CellResearch Corporation Receives New US Patent Approval for Exclusive Stem Cell Technology Amidst New Partnerships
CellResearch Corporation Pte Ltd has received approval for its latest patent from the US Patent and Trademark Office. [CellResearch Corporation Pte Ltd] Press Release

RepliCel Life Sciences Inc. Announces Closing of Brokered and Non-Brokered Private Placement
RepliCel Life Sciences Inc., a clinical stage regenerative medicine company focused on the development of autologous cell therapies, announced it has completed its financing announced on June 1, 2015 [RepliCel Life Sciences Inc.] Press Release

New CIRM-Funded Stem Cell Clinic at City of Hope Treats Its First Patient
A City of Hope brain tumor patient was the first to be treated by City of Hope’s Alpha Clinic for Cell Therapy and Innovation, part of the state’s Alpha Clinics network, created to speed the development and delivery of stem cell therapies to patients who desperately need them. [City of Hope (Business Wire)] Press Release

TxCell Announces a Temporary Partial Hold of Activities at Its Pilot Manufacturing Unit
TxCell SA, a biotechnology company developing personalized T cell immunotherapies using antigen specific regulatory T-cells for severe chronic inflammatory and autoimmune diseases, announced that it has temporarily put its pilot manufacturing facility activities at Besançon, France on partial hold. [TxCell SA] Press Release
National Cancer Institute; Notice of Closed Meetings (FR Doc. No: 2015-15310)

National Institute of Neurological Disorders and Stroke; Notice of Closed Meetings (FR Doc. No: 2015-15306)

Prospective Grant of an Exclusive License Agreement: Development of Bispecific and Multi-Specific Fusion Proteins for the Treatment of ROR1 Expressing Human Cancers (FR Doc. No: 2015-15655)
Food and Drug Administration (United States)

Allowable Excess Volume and Labeled Vial Fill Size in Injectable Drug and Biological Products; Guidance for Industry; Availability (FR Doc. No: 2015-15637)
NEW Cell Manufacturing and Gene Therapy Congress 2015
December 2-3, 2015
Brussels, Belgium

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Postdoctoral Position – Splicing in Hematopoiesis (Northwestern University)

Scientist – Reprogramming and Pluripotent Stem Cell Biology (STEMCELL Technologies Inc.)

Primary Cell Transduction Process Scientist (GlaxoSmithKline)

Postdoctoral Fellow – Antibody Engineering/Immuno-Oncology (California Institute for Biomedical Research)

Postdoctoral Fellow – Stem Cell Biology (INSERM)

MICA Stem Cell Scientist (Medicines and Healthcare Products Regulatory Agency)

Stem Cell Scientist (Medicines and Healthcare Products Regulatory Agency)

Cell Therapy GMP Manager (Paramount Recruitment)

Postdoctoral Fellow – Neural Stem Cells (Children’s Hospital of Philadelphia)

Lead Device and Delivery Scientist (Cell Therapy Catapult)

Postdoctoral Fellow – Immune Reconstitution in Hematopoietic Stem Cell Transplantation (Stanford University)

Postdoctoral Fellow – Immune Tolerance (Stanford University)

Postdoctoral Fellow Positions – iPSC Research and Banking (University of Magna Graecia)

Process Facilitator (Opexa Therapeutics)

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