Volume 17.03 | Jan 25

Issue 17.03 January 25, 2016
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Long-Term Glycemic Control Using Polymer-Encapsulated Human Stem Cell–Derived Beta Cells in Immune-Competent Mice
Scientists report the first long-term glycemic correction of a diabetic, immunocompetent animal model using human glucose-responsive mature beta cells that are derived from human embryonic stem cells. [Nat Med] Abstract | Press Release
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PUBLICATIONS (Ranked by impact factor of the journal)
Gene Therapy Blockade of Dorsal Striatal p11 Improves Motor Function and Dyskinesia in Parkinsonian Mice
To focally inhibit p11 expression in the dorsal striatum, scientists injected an adeno-associated virus vector producing a short hairpin RNA. This intervention reduced the impairment in motor function on forced tasks, such as rotarod and treadmill tests, caused by substantia nigra lesioning in mice. [Proc Natl Acad Sci USA]
Abstract | Full Article

Larger Size of Donor Alloreactive NK Cell Repertoire Correlates with Better Response to NK Cell Immunotherapy in Elderly Acute Myeloid Leukemia Patients
Reearchers aimed at determining whether the composition of natural killer (NK) graft in terms of frequency of alloreactive NK cells influence the clinical response in a group of elderly acute myeloid leukemia patients undergoing NK immunotherapy. [Clin Cancer Res] Abstract

Expression of a Dominant T Cell Receptor Can Reduce Toxicity and Enhance Tumor Protection of Allogeneic T Cell Therapy
Adoptive transfer of allogeneic T cells expressing a dominant T-cell-receptors significantly reduced the graft versus host toxicity in recipient mice. Using two bone marrow transplant models, enhanced anti-tumor activity was observed in the presence of reduced graft-versus-host-disease. [Haematologica] Abstract | Full Article

Noninvasive, Targeted, and Non-Viral Ultrasound-Mediated GDNF-Plasmid Delivery for Treatment of Parkinson’s Disease
Researchers demonstrated that the focused ultrasound-triggered glial cell line-derived neurotrophic factor (GDNF)p-loaded cationic microbubbles platform can achieve non-viral targeted gene delivery via a noninvasive administration route, outperform intracerebral injection in terms of targeted GDNF delivery of high-titer GDNF genes, and has a neuroprotection effect in Parkinson’s disease (PD) animal models to successfully block PD syndrome progression and to restore behavioral function. [Sci Rep] Full Article

Combinatorial Treatment with Apelin-13 Enhances the Therapeutic Efficacy of a Preconditioned Cell-Based Therapy for Peripheral Ischemia
Investigators examined whether apelin-13, a regulator of vessel maturation, could be an effective promoter of therapeutic angiogenesis, following severe limb ischemia. Combinatorial treatment of hypoxic preconditioned peripheral blood mononuclear cells with apelin-13 resulted in increased blood perfusion and vascular reactivity in ischemic mouse hindlimbs compared with a monotherapy comprising each factor. [Sci Rep] Full Article

Control of B Cell Lymphoma by Therapeutic Vaccination and Acquisition of Immune Resistance Is Independent of Direct Tumor IFN-Gamma Signaling
The authors demonstrated that anti-tumor immunity elicited by vaccination requires IFNγ-signaling within host cells, but not tumor cells. IFNγ receptor deficient mice failed to mount an effective anti-tumor immune response following vaccination despite elevated IFNγ levels. [Immunol Cell Biol] Abstract

Phase I Study of Random, Healthy Donor-Derived Allogeneic Natural Killer Cell Therapy in Patients with Malignant Lymphoma or Advanced Solid Tumors
The current Phase I clinical trial was designed to evaluate safety and possible clinical efficacy of repetitive administrations of MG4101 derived from random, unrelated healthy donors into patients with malignant lymphoma or advanced, recurrent solid tumors. [Cancer Immunol Res] Abstract

Improved Outcome of Children Transplanted for High-Risk Leukemia by Using a New Strategy of Cyclosporine-Based GvHD Prophylaxis
The authors report the outcome of high-risk leukemia children transplanted with a new way of managing cyclosporine (CsA)-based GvHD prophylaxis. A total of 110 hematopoietic stem cell transplantation in 109 ALL or AML children who received CsA without mycophenolate or methotrexate in matched related as well as in matched or mismatched unrelated stem cell transplantation were included. [Bone Marrow Transplant] Abstract

In Vivo RNAi Efficacy of Palmitic Acid-Conjugated Dicer-Substrate siRNA in a Subcutaneous Tumor Mouse Model
Scientists synthesized Dicer-substrate short interfering RNAs consisting of 27-nt double-stranded RNAs conjugated with palmitic acid at the 5′-end of the sense strand and investigated their RNA interference (RNAi) efficacies in vitro and in vivo. [Chem Biol Drug Des] Abstract

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Hurdles to the Introduction of New Therapies for Immune-Mediated Kidney Diseases
The authors discuss seven hurdles that must be addressed in order to appropriately assess and introduce immunologic therapies for immune-mediated kidney disease. [Nat Rev Immunol] Abstract

Megakaryocyte and Megakaryocyte Precursor Related Gene Therapies
Recent advances in genetic manipulation of megakaryocytes could lead to new and improved therapies for treating a variety of disorders. Genetic manipulation of megakaryocytes has progressed to the point where clinically relevant strategies are being developed for human trials for genetic disorders affecting platelets. [Blood] Abstract

Gene Therapy Approaches against Cancer Using In Vivo and Ex Vivo Gene Transfer of Interleukin-12
IL-12 is an immunostimulatory cytokine with strong antitumor properties. Systemic administration of IL-12 in cancer patients led to severe toxic effects, prompting the development of gene therapy vectors able to express this cytokine locally in tumors. [Immunotherapy] Abstract

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

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Novocure Presents Phase II PANOVA Results Suggesting Tumor Treating Fields Therapy plus Chemotherapy May Be Safe as First-Line Treatment and Improve Survival of Patients with Advanced Pancreatic Cancer
Novocure presented data from its ongoing Phase II PANOVA clinical trial showing that Tumor Treating Fields therapy plus first-line chemotherapy gemcitabine is tolerable and safe in patients with advanced pancreatic cancer. [Press release from Novocure discussing research presented at the 2016 ASCO Gastrointestinal Cancers Symposium (ASCO GI), San Francisco] Press Release

Cancer MoonShot 2020 Reaches First Clinical Trial Milestone: Immunotherapy Trial Demonstrates Encouraging Survival Rates in Patients with Metastatic Colorectal Cancer Who Failed All Standard Therapy
Precision Biologics and the Cancer MoonShot 2020 Program ‎announced promising data from its Phase II clinical trial of the first neo-epitope1‎ targeting antibody, ensituximab, studied in patients with refractory metastatic colorectal cancer who failed all standards of care. [Press release from Precision Biologics, Inc. discussing research presented at the 2016 ASCO Gastrointestinal Cancers Symposium (ASCO GI), San Francisco] Press Release

Data Presented Highlight the Feasibility of Second-Line Treatment in Patients with Metastatic Pancreatic Cancer Who Have Received First-Line Abraxane plus Gemcitabine
Celgene Corporation announced that results from multiple analyses evaluated the outcomes of second-line treatments following ABRAXANE® and gemcitabine in first-line metastatic pancreatic cancer patients. [Press release from Celgene Corporation discussing research presented at the 2016 ASCO Gastrointestinal Cancers Symposium (ASCO GI), San Francisco] Press Release

Verastem to Present Scientific Data Supporting FAK Inhibition in Combination with Immune Checkpoint Inhibitors
Verastem, Inc. announced the presentation of preclinical data and participation in an expert panel. [Press release from Verastem, Inc. discussing research to be presented at Immunotherapy World 2016, Washington, DC] Press Release

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Researchers Question Design of Fatal French Clinical Trial
Scientists are voicing concerns over the design of a French drug trial that left one participant dead and several others with severe health problems — and they are calling for more information to be released. [Nature News] Editorial

Money Donated to Stockpile Leading Ebola Vaccine
A public-private partnership will pay Merck & Co. $5 million to stockpile 300,000 doses of an Ebola vaccine that appeared to work in a Guinea trial last year. [ScienceInsider] Editorial
Cambridge Joins Consortium to Launch £40 Million Apollo Therapeutics Fund
Three global pharmaceutical companies and the technology transfer offices of three universities – Imperial College London, University College London and the University of Cambridge – have joined forces with a combined £40 million to create the Apollo Therapeutics Fund. This joint venture will support the translation of ground-breaking academic science from within these universities into innovative new medicines for a broad range of diseases. [University of Cambridge] Press Release

CEVEC and Beckman Research Institute of City of Hope Enter into a Cooperation and License Agreement on CEVEC’s Proprietary CAP®GT Technology
CEVEC Pharmaceuticals GmbH announced a collaboration agreement with Beckman Research Institute of City of Hope on CEVEC’s proprietary CAP®GT technology. [CEVEC Pharmaceuticals GmbH] Press Release

MD Anderson, AbbVie Connect to Advance Cancer Immunotherapy
The immunotherapy platform at The University of Texas MD Anderson Cancer Center and AbbVie will join forces to find new ways to unleash the immune system’s potential to fight cancer. The three-year collaboration agreement provides a framework for MD Anderson and AbbVie to efficiently choose and carry out preclinical and clinical studies evaluating new ideas in the cutting-edge area of immuno-oncology. [AbbVie Inc.] Press Release

Cellectis Enters into New Agreement with CELLforCURE for the cGMP Manufacturing of UCART123 for Hematological Malignancies
Cellectis announced that it entered into a new agreement for the cGMP manufacturing of UCART123 clinical batches, Cellectis’ lead product candidate, with CELLforCURE, an LFB group company and the largest industrial facility for clinical and commercial production of innovative cell therapies in Europe. [Cellectis] Press Release

Enrollment Begun on Phase II Clinical Trial in Crohn’s Disease
Cytori Therapeutics, Inc. announced enrollment of the first two patients in an investigator-initiated clinical trial of Cytori Cell Therapy™ for the treatment of the orphan indication, fistula-in-ano associated with Crohn’s Disease. [Cytori Therapeutics, Inc.] Press Release

Center Will Develop Consistent Manufacturing Processes for Cell-Based Therapies
A $15.7 million grant from the Marcus Foundation has helped launch a new Georgia Institute of Technology research center that will develop processes and techniques for ensuring the consistent, low-cost, large-scale manufacture of high-quality living cells used in cell-based therapies. [Georgia Institute of Technology] Press Release

PCRF Launches World’s First National Tissue Bank for Pancreatic Cancer Research
The world’s first national tissue bank for pancreatic cancer has launched in the UK. The Pancreatic Cancer Research Fund Tissue Bank brings together surgeons, pathologists, oncologists, researchers and database experts to co-ordinate a national – and ultimately international -­ resource that will help to develop new treatments and bring these to patients much faster. [Pancreatic Cancer Research Fund (PCRF)] Press Release

European Commission Approves Label Variation for ADCETRIS® (Brentuximab Vedotin) to Include Data on Retreatment of Adult Patients with Relapsed or Refractory Hodgkin Lymphoma and Systemic Anaplastic Large Cell Lymphoma (sALCL)
Takeda Pharmaceutical Company Limited announced that the European Commission has approved a Type II variation for ADCETRIS® to include data on the retreatment of adult patients with relapsed or refractory (R/R) Hodgkin lymphoma or R/R sALCL who previously responded to ADCETRIS and who later relapse. [Takeda Pharmaceutical Company Limited] Press Release

Agenus Announces Clearance of Investigational New Drug Applications by the FDA for Anti-CTLA-4 and Anti-GITR Antibodies
Agenus Inc. announced that the U.S. Food and Drug Administration (FDA) cleared the company’s investigational new drug application for AGEN1884, an immune checkpoint modulator antibody that binds to cytotoxic T-lymphocyte antigen-4 (CTLA-4). [Agenus Inc.] Press Release

Kadimastem Receives Patent Allowance in the United States for Its Technology in the Field of Diabetes Treatment
Kadimastem announced it has received patent allowance from the United States Patent and Trademark Office, for its innovative technology in the field of diabetes treatment. The patent is for a method of producing insulin-secreting cells from stem cells, and it is likely to constitute the foundation for the company’s products in the field of stem-cell based treatment of diabetes. [Kadimastem] Press Release
Center for Scientific Review; Notice of Closed Meetings (FR Doc. No: 2016-01069)

National Human Genome Research Institute; Notice of Meetings (FR Doc. No: 2016-01066)
NEW ESMO Symposium on Signaling Pathways in Cancer 2016: Focusing on the HER/EGFR Family Signaling
March 4-5, 2016
Sitges-Barcelona, Spain

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Research Associate – Rheumatology (Newcastle University)

NEW Professorship – Transfusion Medicine and Cell Therapeutics (Ludwig-Maximilians-Universität München)

NEW Postdoctoral Fellow/Research Associate – Cancer Therapy (Memorial Sloan Kettering Cancer Center )

NEW Translational Research Scientist – Hematology/Oncology (University of Chicago)

Postdoctoral Position – Cancer Immunotherapy (German Cancer Research Center)

Staff Scientist – Cell Therapy Process Development (Fred Hutchinson Cancer Research Center)

Postdoctoral Position – CAR T Cell Engineering & Cancer Immunotherapy (University Hospital Würzburg)

Postdoctoral Researcher – Prostate Cancer Biology (Roswell Park Cancer Institute)

Postdoctoral Research Scientist – Follicular Lymphoma (Herbert Irving Comprehensive Cancer Center)

Postdoctoral Research Fellow – Cell Cycle Control and Tumorigenesis (Fred Hutchinson Cancer Research Center)

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