Cell Therapy News Volume 16.19 | May 18 2015

X-Shaped DNA Potentiates Therapeutic Efficacy in Colitis-Associated Colon Cancer through Dual Activation of TLR9 and Inflammasomes
Scientists developed two kinds of X-shaped double-stranded oligodeoxynucleotides (X-DNA), a single unit of X-DNA (X_S-DNA) composed of four strands of DNA and a ligated X-DNA complex formed by crosslinking each X_S-DNA to the other, and investigated if they had immunostimulatory activity and could be applied to anti-cancer immunotherapy. [Mol Cancer] Full Article

Lentivirus-Induced ‘Smart’ Dendritic Cells: Pharmacodynamics and GMP-Compliant Production for Immunotherapy against TRP2 Positive Melanoma
Efficacy studies were hampered by difficulties in conventional dendritic cell (DC) manufacturing and low potency. Overcoming these issues, researchers demonstrated higher potency of lentiviral vector-programmed DCs. [Gene Ther] Abstract | Full Article

Human Endometrial Mesenchymal Stem Cells Restore Ovarian Function through Improving the Renewal of Germline Stem Cells in a Mouse Model of Premature Ovarian Failure
Scientists hypothesized that endometrial mesenchymal stem cells (EnSCs) may offer promise for restoration of ovarian dysfunction associated with premature ovarian failure/insufficiency. Transplanted EnSCs were injected into the tail vein of sterilized mice, or culture medium was injected into the sterilized mice via the tail vein as chemoablated group. [J Transl Med] Abstract | Full Article

Phase I Study of the Anti-CD74 Monoclonal Antibody Milatuzumab (hLL1) in Patients with Previously Treated B-Cell Lymphomas
hLL1, a humanized anti-CD74 monoclonal antibody, has activity in preclinical non-Hodgkin lymphoma models. Investigators conducted a Phase I trial in previously treated B-cell malignancies. [Leuk Lymphoma] Abstract

Ex Vivo and In Vivo Modulatory Effects of Umbilical Cord Wharton’s Jelly Stem Cells on Human Oral Mucosa Stroma Substitutes
Researchers generated human oral mucosa substitutes with oral mucosa keratinocytes and human umbilical cord Wharton’s jelly stem cells to determine the influence of these cell sources on stromal differentiation. [Histol Histopathol] Abstract

A Hyaluronan-Based Injectable Hydrogel Improves the Survival and Integration of Stem Cell Progeny following Transplantation
Investigators designed an injectable and bioresorbable hydrogel blend of hyaluronan and methylcellulose and tested it with two cell types in two animal models, thereby gaining an understanding of its general applicability for enhanced cell distribution, survival, integration, and functional repair relative to conventional cell delivery in saline. [Stem Cell Reports] Full Article | Press Release

Adenovirus Improves the Efficacy of Adoptive T-Cell Therapy by Recruiting Immune Cells to and Promoting Their Activity at the Tumor
Scientists studied the capacity of adenovirus to overcome resistance of chicken ovalbumin-expressing B16.OVA murine melanoma tumors to adoptive ovalbumin-specific CD8⁺ T-cell therapy. [Cancer Immunol Res] Abstract

Neurogenetics and Gene Therapy for Reward Deficiency Syndrome: Are We Going to the Promised Land?
The results of selective studies that include the role of epigenetics, noncoding microRNAs in reward deficiency syndrome behaviors especially drug abuse involving alcohol, opioids, cocaine, nicotine, pain and feeding are reviewed. [Expert Opin Biol Ther] Abstract

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

Roche’s Investigational Immunotherapy MPDL3280A Doubled the Likelihood of Survival Compared with Chemotherapy in People with a Specific Type of Lung Cancer
Roche announced interim results from a global, randomized Phase II study in people with previously treated non-small cell lung cancer. The study showed the investigational cancer immunotherapy MPDL3280A doubled the likelihood of survival in people whose cancer expressed the highest levels of programmed death ligand-1 compared with docetaxel chemotherapy. [Press release from F. Hoffmann-La Roche Ltd discussing research to be presented at the 51^st Annual Meeting of the American Society of Clinical Oncology (ASCO), Chicago] Press Release

Experimental Immunotherapy Shows High Response Rate in Advanced Lung Cancer
An early phase study testing an anti-PDL1 agent in combination with standard chemotherapy in the treatment of advanced non-small cell lung cancer has provided promising early results, prompting multiple Phase III studies in lung cancer. [Press release from Georgetown University Medical Center discussing research to be presented at the 51^st Annual Meeting of the American Society of Clinical Oncology (ASCO), Chicago] Press Release

Avalanche Biotechnologies Presents Three Posters at American Society of Gene & Cell Therapy (ASGCT) Annual Meeting
Avalanche Biotechnologies, Inc. announced that three posters, including one providing baseline demographics and characteristics from its Phase IIIa clinical trial for AVA-101, will be presented. [Press release from Avalanche Biotechnologies, Inc. discussing research presented at the ASGCT 18^th Annual Meeting, New Orleans] Press Release

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House Spending Panel Calls for Cuts at NASA, NOAA, and NIST, Small Increase for NSF
The National Science Foundation (NSF) would get a small increase, and NASA’s science programs would see a tiny cut, under a draft 2016 appropriations bill released today by a U.S. House of Representatives spending panel. [ScienceInsider] Editorial

Lilly and Sanford-Burnham Announce Collaboration to Investigate Immunological Therapies
Eli Lilly and Company and Sanford-Burnham Medical Research Institute announced that they will collaborate to discover and develop immunological therapies. Lilly and Sanford-Burnham will investigate potential therapeutics using biotechnology approaches in targeting multiple immune checkpoint modulators for the treatment of immunological diseases such as lupus, Sjögren’s Syndrome, inflammatory bowel disease and other autoimmune disorders. [Eli Lilly and Company] Press Release

Lilly and BioNTech Announce Research Collaboration on Novel Cancer Immunotherapies
Eli Lilly and Company and BioNTech AG announced they have entered into a research collaboration to discover novel cancer immunotherapies. The companies will seek to use the power of the body’s own immune system to attack cancer cells and create possible new treatment options for cancer patients. [Eli Lilly and Company] Press Release

Selecta and Généthon Collaborate to Create Next Generation Gene Therapies Using Selecta’s Synthetic Vaccine Particle Platform
Selecta Biosciences, Inc. and Généthon announced an ongoing research collaboration with the goal of enabling repeat dosing for gene therapies. Based on preliminary results, the companies have identified three applications that might benefit from combining Généthon’s expertise in the development of gene therapy vectors and Selecta’s Synthetic Vaccine Particle platform to prevent undesired immune responses. [Selecta Biosciences, Inc.] Press Release

Sanofi Exercises Option on Second Therapeutic Program with Selecta Biosciences to Develop an Antigen-Specific Immunotherapy Based on Synthetic Vaccine Particle Technology
Selecta Biosciences, Inc. announced that, under the terms of an existing strategic global collaboration, Sanofi has exercised its option to an exclusive license to develop an immunotherapy for the treatment of celiac disease. [Selecta Biosciences, Inc.] Press Release

Argos Therapeutics Provides Update on Enrollment Progress for Ongoing Pivotal Phase III ADAPT Clinical Trial
Argos Therapeutics Inc. announced that more than 1,000 tumor samples have been collected and approximately 400 eligible patients have been randomized to the company’s ongoing, pivotal Phase III ADAPT clinical trial of AGS-003 for the treatment of metastatic renal cell carcinoma. [Argos Therapeutics Inc.] Press Release

Bavarian Nordic Announces Positive Results from Two Pivotal Clinical Studies of IMVAMUNE^® Smallpox Vaccine
Bavarian Nordic A/S announced results from the first of two pivotal Phase III studies of the liquid-frozen formulation of IMVAMUNE^® supporting a Biologics License Application for U.S. approval of the vaccine. [Bavarian Nordic A/S] Press Release

NewLink Genetics’ IMPRESS Phase III Pancreatic Cancer Trial with Algenpantucel-L to Continue following Second Interim Analysis
NewLink Genetics Corporation announced the continuation without any modification or sample size adjustment for its pivotal, Phase III IMmunotherapy for Pancreatic RESectable cancer Study, called “IMPRESS,” of algenpantucel-L for patients with surgically resected pancreatic cancer following the second planned interim data analysis. [NewLink Genetics Corporation] Press Release

Alnylam Files Clinical Trial Application (CTA) for ALN-AAT, an Investigational RNAi Therapeutic for the Treatment of Alpha-1 Antitrypsin Deficiency-Associated Liver Disease (Alpha-1 Liver Disease)
Alnylam Pharmaceuticals, Inc. announced that it has filed a CTA with the U.K. Medicines and Healthcare products Regulatory Agency to initiate a Phase I/II clinical trial with ALN-AAT, a subcutaneously administered investigational RNAi therapeutic targeting alpha-1 liver disease. [Alnylam Pharmaceuticals, Inc.] Press Release

Pluristem’s PLX Cells One Step Closer to Entering Japan’s Accelerated Pathway for Regenerative Medicine
Pluristem Therapeutics Inc. announced that Japan’s Pharmaceuticals and Medical Devices Agency agreed with the proposed quality and large-scale manufacturing methods for PLX-PAD cells for use in clinical trials. [Pluristem Therapeutics Inc.] Press Release

Investigational New Drug Applications Prepared and Submitted by Sponsor-Investigators; Draft Guidance for Industry; Availability (FR Doc. No: 2015-11685)

Biosimilars: Additional Questions and Answers Regarding Implementation of the Biologics Price Competition and Innovation Act of 2009; Draft Guidance for Industry; Availability (FR Doc. No: 2015-11528)

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.

NEW Postdoctoral Fellow – Immune Tolerance (Stanford University)

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

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

In Vivo Biologist – Novel Cell and Gene Therapies (GlaxoSmithKline)

Process Facilitator (Opexa Therapeutics)

Cell Therapy Production Scientist (Paramount Recruitment)

Research Assistant – Exosome-Based Gene Therapy for Huntington’s Disease (University of Oxford)

Postdoctoral Fellow – Neural Tissue Engineering (Nanyang Technological University)

Professor – Gene Therapy (University of Massachusetts)

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