Volume 16.03 | Jan 26

Issue 16.03 January 26, 2015
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NIH Researchers Tackle Thorny Side of Gene Therapy
National Institutes of Health (NIH) researchers have uncovered a key factor in understanding the elevated cancer risk associated with gene therapy. They conducted research on mice with a rare disease similar to one in humans, hoping their findings may eventually help improve gene therapy for humans. [Press release from the National Institutes of Health discussing online publication in Journal of Clinical Investigation]
Press Release | Full Article | Video
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PUBLICATIONS (Ranked by impact factor of the journal)
Vitiligo-Like Depigmentation in Patients with Stage III-IV Melanoma Receiving Immunotherapy and Its Association with Survival: A Systematic Review and Meta-Analysis
Scientists conducted a systematic review of patients with stage III to IV melanoma treated with immunotherapy to determine the cumulative incidence of vitiligo-like depigmentation and the prognostic value of vitiligo development on survival. [J Clin Oncol] Abstract

Enhancing Anti-Tumor Efficacy of Chimeric Antigen Receptor T Cells through Constitutive CD40L Expression
Researchers enhanced the anti-tumor efficacy of chimeric antigen receptor T cells through the constitutive expression of CD40 ligand (CD40L). T cells genetically modified to constitutively express CD40L demonstrated increased proliferation and secretion of pro-inflammatory TH1 cytokines. [Mol Ther] Abstract

Designer Self-Assemble Peptides Maximize the Therapeutic Benefits of Neural Stem Cell Transplantation for Alzheimer’s Disease via Enhancing Neuron Differentiation and Paracrine Action
Investigators undertook in vitro and in vivo works to examine whether a designer self-assemble peptide, which contains one functional domain Tyr-Ile-Gly-Ser-Arg derived from laminin, promotes the survival and neuronal differentiation of neural stem cells and behavioral improvement. [Mol Neurobiol] Full Article

Co-Immunization with DNA and Protein Mixture: A Safe and Efficacious Immunotherapeutic Strategy for Alzheimer’s Disease in PDAPP Mice
Scientists describe the immunological characterization and protective efficacy of co-immunization with a 6Aβ15-T DNA and protein mixture without adjuvant as an Alzheimer’s disease immunotherapeutic strategy. [Sci Rep]
Full Article

Clinical Outcomes of a Novel Therapeutic Vaccine with Tax Peptide-Pulsed Dendritic Cells for Adult T Cell Leukemia/Lymphoma in a Pilot Study
Researchers developed a novel therapeutic vaccine designed to augment an human T cell leukemia virus type-I Tax-specific cytotoxic T lymphocyte response that has been implicated in anti-adult T cell leukemia/lymphoma effects, and conducted a pilot study to investigate its safety and efficacy. [Br J Haematol] Abstract

Allogeneic Hematopoietic Stem Cell Transplantation for Adult Patients with Mixed Phenotype Acute Leukemia: Results of a Matched-Pair Analysis
Investigators retrospectively assessed the efficacy of allogeneic stem cell transplantation for mixed phenotype acute leukemia (MPAL). Eighteen patients with MPAL were identified from the transplant outcome database of KSGCT. [Eur J Haematol] Abstract

Oncolytic HSV Virotherapy in Murine Sarcomas Differentially Triggers an Antitumor T-Cell Response in the Absence of Virus Permissivity
Scientists tested three syngeneic murine sarcoma models that displayed varied degrees of permissiveness to oncolytic herpes simplex virus (HSV) replication and cytotoxicity in vitro, with the most permissive being comparable to some human sarcoma tumor lines. The in vivo antitumor effect ranged from no or modest response to complete tumor regression and protection from tumor rechallenge. [Mol Ther Oncolytics]
Full Article | Press Release

Combined Immune Checkpoint Protein Blockade and Low Dose Whole Body Irradiation as Immunotherapy for Myeloma
Researchers hypothesized that blocking a combination of checkpoint receptors in the context of low-dose, lymphodepleting whole body radiation would boost anti-tumor immunity. Myeloma-bearing mice were treated with a low dose of whole body irradiation and combinations of blocking antibodies to PD-L1, LAG-3, TIM-3, CD48 (the ligand for 2B4) and CTLA4. [J Immunother Cancer] Full Article

Free Cell Stem Cell Poster: Directed Differentiation of ESCs/iPSCs
Preclinical and Clinical Evidence for Stem Cell Therapies as Treatment for Diabetic Wounds
Diabetic foot ulcers occur in 15% of all patients with diabetes and precede 84% of all diabetes-related lower leg amputations. Experimental preclinical evidence for the successful use of adult stem cell therapies for diabetic wounds gives new hope for the development of effective treatments for use in the clinic. [Drug Discov Today] Abstract

Gene and Cell Therapy for Pancreatic Cancer
The authors focus on Phase I–III clinical trials of gene and cell therapy for pancreatic cancer and on future implications of recent translational research. [Expert Opin Biol Ther] Abstract

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

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Aduro Biotech Reports Long-Term Survival and Immune Biomarker Data from Phase II Clinical Trial of Its Immuno-Oncology Regimen in Patients With Pancreatic Cancer
Aduro Biotech, Inc. announced that seven pancreatic cancer patients treated with its combination immuno-oncology regimen in a 93-patient Phase IIa clinical trial continue in survival follow-up, with two of those patients remaining on the company’s combination therapy for more than two years with no evidence of cumulative toxicity. [Press release from Aduro Biotech, Inc. discussing research presented at American Society of Clinical Oncology (ASCO) Gastrointestinal Cancers Symposium, San Francisco] Press Release

Kite Pharma Announces Presentations Highlighting Cancer Immunotherapy T Cell Manufacturing Process
Kite Pharma, Inc. announced presentations on the company’s rapid, six-day manufacturing process for the production of Kite’s lead product candidate, KTE-C19. The manufacturing technology enables a patient’s T cells to be genetically modified using a gammaretroviral vector to express a chimeric antigen receptor designed to target the antigen CD19, a protein expressed on the cell surface of B cell lymphomas and leukemias. [Press release from Kite Pharma, Inc. discussing research presented at Scale-Up and Manufacturing of Cell-Based Therapies IV, San Diego] Press Release

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US Precision-Medicine Proposal Sparks Questions
During his State of the Union address to Congress, President Barack Obama announced a program called the Precision Medicine Initiative. The White House is remaining tight-lipped about the details of the program, declining to answer questions from Nature — as is the US National Institutes of Health, which is expected to be a key partner in the effort. [Nature News] Editorial

University of Oxford Named United Kingdom’s Global Health Front-Runner
Only a handful of U.K. universities are deeply involved in the fight to improve global health, according to a new ranking table released at the United Kingdom’s Houses of Parliament. The idea behind the list — which follows a similar ranking for U.S. and Canadian universities and another one for pharmaceutical companies — is to encourage spending on global health research and to increase the pressure on stragglers to step up their efforts. [ScienceInsider] Editorial
IntelliCell BioSciences Announces Collaboration to Develop Closed Processing System for Cell Therapy Manufacturing
IntelliCell BioSciences, Inc. announced an agreement for the development of a new closed processing system for its cellular therapy manufacturing with Hielscher Ultrasonics. Under the agreement, Hielscher Ultrasonics will provide system design and engineering development for ultrasonic cavitation manufacturing, and IntelliCell Biosciences will use this system in its commercial application of its stromal vascular fraction cellular product. [IntelliCell BioSciences, Inc. (PR Newswire Association LLC)] Press Release

Syncona LLP and UCLB Announce the Formation of Autolus Limited, a Cancer Immunotherapy Company
Syncona LLP and University College London Business PLC (UCLB) announce the creation of Autolus Limited, a biopharmaceutical company focused on the development and commercialization of next-generation engineered T-cell therapies for hematological and solid tumors. [University College London Business PLC] Press Release

King’s College London Signs License Agreement with UCB in Immunology and Type 1 Diabetes
King’s College London announced that it has entered an exclusive license agreement with UCB that grants the company the rights to develop a peptide-based immunotherapy program for type 1 diabetes. [King’s College London] Press Release

Kite Pharma Announces Expanded Agreement with Tel Aviv Sourasky Medical Center and Pioneering Researcher Professor Zelig Eshhar to Develop Novel Chimeric Antigen Receptor (CAR) Approaches for Cancer Immunotherapy
Kite Pharma, Inc. announced that the company has expanded its agreement with Tel Aviv Sourasky Medical Center to research and develop novel approaches to CAR T cell therapy, the technology underlying Kite’s most advanced programs in cancer immunotherapy. [Kite Pharma, Inc.] Press Release

Health Canada Clears Immunovaccine to Conduct Phase I Clinical Trial of Respiratory Syncytial Virus (RSV) Vaccine
Immunovaccine Inc. announced that it has received clearance from Health Canada to conduct a Phase I clinical study of its RSV vaccine in healthy adults. The RSV vaccine is formulated in Immunovaccine’s proprietary DepoVax™ adjuvanting platform and is initially being developed to protect the elderly population from infection. [Immunovaccine Inc.] Press Release

Tekmira Initiates Phase I Clinical Trial of TKM-HBV
Tekmira Pharmaceuticals Corporation announced that it has dosed the first subject in a Phase I clinical trial of TKM-HBV, a therapeutic agent designed to reduce hepatitis B surface antigen in patients chronically infected with hepatitis B virus. [Tekmira Pharmaceuticals Corporation] Press Release

Advaxis Announces FDA Acceptance of Its Investigational New Drug Application to Commence First-in-Human Clinical Trials of ADXS-HER2
Advaxis, Inc. announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug application to conduct a Phase I clinical study of ADXS-HER2 for the treatment of patients with metastatic HER2 expressing solid tumors. [Advaxis, Inc.] Press Release

Athersys and Cell Therapy Catapult Announce Grant to Support Clinical Development of Stem Cell Therapy for Severe Acute Respiratory Condition
Athersys Limited and the Cell Therapy Catapult have announced that Athersys Limited has been awarded a grant from Innovate UK which will support a Phase IIa clinical study evaluating the administration of MultiStem® cell therapy to acute respiratory distress syndrome patients. [Athersys Limited] Press Release

New Animal Models Faithfully Reproduce the Tumor of Each Patient
A team from the Catalan Institute of Oncology and Bellvitge Biomedical Research Institute has developed a new animal models that reproduce faithfully the evolution and malignancy of different human tumors. [Bellvitge Biomedical Research Institute] Press Release

New Book Explores Current State of Regenerative Medicine
From offering new cancer-fighting options to re-growing damaged organs and nerves, regenerative techniques have a use in nearly every area of medicine. These diverse applications are explored in the new book Stem Cells, Tissue Engineering and Regenerative Medicine, edited by David Warburton, director of Developmental Biology and Regenerative Medicine at The Saban Research Institute of Children’s Hospital Los Angeles. [University of Southern California] Press Release
National Institute of Diabetes and Digestive and Kidney Diseases; Notice of Closed Meetings (FR Doc. No: 2015-00654)
Guidance Agenda: Guidance Documents CBER is Planning to Publish During Calendar Year 2015
Food and Drug Administration (United States)

Agency Information Collection Activities; Proposed Collection; Comment Request; Requirements on Content and Format of Labeling for Human Prescription Drug and Biological Products (FR Doc. No: 2015-00761)
NEW 1st Central European Conference on Regenerative Medicine (CECRM)
March 14-15, 2015
Bydgoszcz, Poland

NEW World Conference on Regenerative Medicine
October 21-23, 2015
Leipzig, Germany

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Postdoctoral Fellow – Vascular Bioengineering (University of Pittsburgh)

NEW Assistant/Associate Professor – Gastroenterology (North Carolina State University College of Veterinary Medicine)

Director (California Institute for Regenerative Medicine)

Postdoctoral Position – Immunology Animal Models (The Technical University of Denmark)

Postdoctoral Position – Inflammatory Bowel Disease (The Technical University of Denmark)

Postdoctoral Fellow – Wnt Signaling in Development and Disease (Van Andel Research Institute)

Cell Therapy Manufacturing Scientist (SLS Services Ltd.)

Postdoctoral Fellowship – CNS Inflammation (Rutgers University)

Postdoctoral Fellow – Radiation Therapy and Immunotherapy (Georgia Regents University Cancer Center)

PhD Student – Experimental Leukemia Research (University Children’s Hospital Basel)

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

Scientist – PSC Biology and Bioengineering (STEMCELL Technologies Inc.)

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.)

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