Cell Therapy News Volume 18.23 | Jul 10 2017

TOP STORY

Reconstruction of the Mouse Extrahepatic Biliary Tree Using Primary Human Extrahepatic Cholangiocyte Organoids

Scientists report a new method for the isolation and propagation of human cholangiocytes from the extrahepatic biliary tree in the form of extrahepatic cholangiocyte organoids (ECOs) for regenerative medicine applications. They explored the regenerative potential of these organoids in vivo and demonstrated that the ECOs self-organize into bile duct-like tubes expressing biliary markers following transplantation under the kidney capsule of immunocompromised mice. [Nat Med] Abstract | Press Release

(Ranked by impact factor of the journal)

Transplantation of Enteric Nervous System Stem Cells Rescues Nitric Oxide Synthase Deficient Mouse Colon

Researchers showed rescue of gut motility after enteric neural stem cells (ENSC) transplantation in a mouse model of human enteric neuropathy, the neuronal nitric oxide synthase deficient mouse model, which displays slow transit in the colon. They further showed that transplantation of ENSC into the colon rescues impaired colonic motility with formation of extensive networks of transplanted cells, including the development of neuronal nitric oxide synthase neurons and subsequent restoration of nitrergic responses. [Nat Commun] Full Article

Polyclonal Regulatory T cell Therapy for Control of Inflammation in Kidney Transplants

The authors conducted a pilot safety and feasibility trial of autologous regulatory T cell (Treg) cell therapy in three kidney transplant recipients with subclinical inflammation noted on six-month surveillance biopsies. The infused cells demonstrated patterns of persistence and stability similar to those observed in non-immunosuppressed subjects receiving the same dose of Tregs. Isolation and expansion of Tregs was feasible in kidney transplant patients on immunosuppression. [Am J Transplant] Abstract

The Clinical Efficacy of First-Generation Carcinoembryonic Antigen (CEACAM5)-Specific CAR T Cells Is Limited by Poor Persistence and Transient Pre-Conditioning-Dependent Respiratory Toxicity

Scientists determined the feasibility of delivering first-generation CAR T cell therapy to patients with advanced, CEACAM5+ malignancy. Patients in cohort 4 received increased intensity pre-conditioning, systemic IL2 support and CAR T cells. No objective clinical responses were observed. CAR T cell engraftment in patients within cohort four was significantly higher. [Cancer Immunol Immunother] Full Article

Adoptive Transfer of Murine T Cells Expressing a Chimeric-PD1-Dap10 Receptor as an Immunotherapy for Lymphoma

Researchers developed a murine chimeric PD1 receptor (chPD1) consisting of the PD1 extracellular domain fused to the cytoplasmic domain of CD3 zeta. The inclusion of a Dap10 or CD28 costimulatory domain in the chPD1 receptor was compared to determine which domain induced optimal anti-tumor immunity in a mouse model of lymphoma. [Immunology] Abstract

Genetic Adjuvantation of a Cell-Based Therapeutic Vaccine for Amelioration of Chagasic Cardiomyopathy

Investigators developed a genetically-adjuvanted, dendritic cell-based immunotherapeutic for acute Chagas disease in an attempt to delay or prevent the cardiac complications that eventually result from chronic Trypanosoma cruzi infection. A vaccine platform transduced with the adenoviral vector and loaded in tandem with recombinant protein reduced parasite burdens by 76% to > 99% in comparison to a variety of different controls and significantly reduced cardiac pathology in a BALB/c mouse model of live Chagas infection. [Infect Immun] Abstract

Sciatic Nerve Regeneration by Transplantation of Schwann Cells via Erythropoietin Controlled-Releasing Polylactic Acid/Multiwalled Carbon Nanotubes/Gelatin Nanofibrils Neural Guidance Conduit

The authors aimed to enhance the efficacy of peripheral nerve regeneration using an electrically conductive biodegradable porous neural guidance conduit for transplantation of allogeneic Schwann cells (SCs). The produced conduit was able to release recombinant human erythropoietin for at least two weeks and exhibited favorable cytocompatibility towards SCs. [J Biomed Mater Res B Appl Biomater] Abstract

Adoptively Transferred vγ9Vδ2 T Cells Show Potent Antitumor Effects in a Preclinical B Cell Lymphomagenesis Model

Researchers used a powerful preclinical model where EBV infection drives the de novo generation of human B cell lymphomas in vivo, and autologous T lymphocytes are held in check by PD-1/CTLA-4–mediated inhibition. They showed that a single dose of adoptively transferred Vδ2⁺ T cells has potent antitumor effects, even in the absence of checkpoint blockade or activating compounds. [JCI Insight] Full Article

HLA-DP in Unrelated Hematopoietic Cell Transplantation Revisited: Challenges and Opportunities

Scientists detail the special features of HLA-DP as model antigens for clinically permissive mismatches mediating limited T-cell alloreactivity with minimal toxicity, and describe future avenues for their exploitation in cellular immunotherapy of malignant blood disorders. [Blood] Abstract

Adeno-Associated Viral Gene Therapy for Mucopolysaccharidoses Exhibiting Neurodegeneration

Researchers focus on the recent outcomes and planned viral vector-mediated gene therapy clinical trials, and the pre-clinical data that supported these studies, for MPS-I (Hurler/Scheie syndrome), MPS-II (Hunter syndrome), and MPS-IIIA and -IIIB (Sanfilippo syndrome). [J Mol Med (Berl)] Abstract

Cultured Epidermal Stem Cells in Regenerative Medicine

The authors highlight the role of epidermal stem cells in the current treatment of skin injury and disease, as well as their potential in novel regenerative medicine applications involving other epithelia. [Stem Cell Res Ther] Full Article

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ARMO BioSciences Presents Clinical Data on Immunotherapy AM0010 in Advanced Pancreatic Cancer

ARMO BioSciences, Inc. announced clinical data on its lead investigational immuno-oncology drug AM0010. AM0010 is being evaluated in an ongoing Phase I/Ib clinical trial that has enrolled 352 advanced cancer patients and in a Phase III clinical trial that is enrolling patients with advanced pancreatic cancer. [Press release from ARMO BioSciences, Inc. discussing research presented at the ESMO 19^th World Congress on Gastrointestinal Cancer, Barcelona] Press Release

Six-Month Results from Capricor’s HOPE-Duchenne Trial to Be Presented

Capricor Therapeutics, Inc. presented interim six-month safety and efficacy results from its Phase I/II HOPE-Duchenne clinical trial of CAP-1002 (allogeneic cardiosphere-derived cells) on a poster. [Press release from Capricor Therapeutics, Inc. discussing research presented at the 2017 Parent Project Muscular Dystrophy (PPMD) Annual Connect Conference, Chicago] Press Release | Poster

CRISPR Therapeutics and Neon Therapeutics Enter Research Collaboration

CRISPR Therapeutics and Neon Therapeutics announced a research collaboration to explore the combination of each company’s proprietary technologies to develop novel T cell therapies. [CRISPR Therapeutics] Press Release

SanBio Receives $20 Million Grant from CIRM for Stroke Clinical Trial

SanBio, Inc. announced it has been awarded a $20 million grant from the California Institute of Regenerative Medicine (CIRM) in support of its Phase IIb clinical trial for the treatment of chronic stroke, jointly sponsored with Sunovion Pharmaceuticals Inc. [SanBio, Inc.] Press Release

ViraCyte Awarded $3.75M from NIH and FDA to Advance T Cell Immunotherapies

ViraCyte, LLC announced that it has been awarded $3.75M from National Institutes of Health (NIH) and the Food and Drug Administration (FDA) to advance its T cell immunotherapies. [ViraCyte, LLC.] Press Release

TissueGene Awarded $750,000 Maryland Stem Cell Grant for Invossa™ Clinical Study

TissueGene, Inc. announced that the Maryland Stem Cell Research Fund has awarded TissueGene a clinical grant for Invossa™, the world’s first cell and gene therapy for degenerative arthritis. [TissueGene, Inc. (PR Newswire Association LLC.)] Press Release

Evotec Received Funding from IFB Hamburg to Identify Antibody-Mediated T-Cell Immunotherapies

Evotec AG announced that the company has been awarded a ‘Programm für Innovation’ grant for a period of two years from the Hamburgische Investitions- und Förderbank (IFB Hamburg), the central development institution of the Free and Hanseatic city of Hamburg, to identify and develop therapeutic antibodies directed against novel immune-checkpoints on T-cells to improve future cancer treatments. [Evotec AG] Press Release

Pluristem Therapeutics (PSTI) Advances Multinational Phase III Critical Limb Ischemia Study, Targeting Initiation at 40 Active Sites by the End of 2017

Pluristem Therapeutics Inc. announced that Austria’s regulatory health agency, the Austrian Agency for Health and Food Safety, has cleared Pluristem to begin enrolment in Austria for its pivotal Phase III trial of PLX-PAD cells to treat Critical Limb Ischemia. [Pluristem Therapeutics Inc.] Press Release

uniQure Announces Updated, Long-Term Clinical Data from Ongoing Phase I/II Trial of AMT-060 In Patients with Severe Hemophilia B

uniQure N.V. announced updated results from its ongoing, dose-ranging Phase I/II trial of AMT-060, its proprietary, investigational gene therapy in patients with severe hemophilia B. The data includes up to 18 months of follow-up from the low-dose cohort and up to one year of follow-up from the second dose cohort. [uniQure N.V.] Press Release

Medigene Submits CTA for Its First Clinical Trial with Proprietary TCR Cellular Immunotherapy MDG1011

Medigene AG has filed a clinical trial authorization application (CTA) to begin the company’s first clinical trial with its proprietary T-cell receptor- (TCR) modified T cells as an immunotherapy to treat a range of blood cancer indications. [Medigene AG] Press Release

Cytori Receives Key Cell Therapy Patent for Scleroderma

Cytori Therapeutics, Inc. announced the issuance of key patents that strengthen its intellectual property portfolio in the U.S. and Europe. Several of Cytori’s recently issued patents contain claims covering the use of Habeo™ Cell Therapy for patients with scleroderma affecting the hands. [Cytori Therapeutics, Inc.] Press Release

American Medical Association Approves New Category III CPT Codes Describing Cytori’s Scleroderma Therapy

Cytori Therapeutics, Inc. announced that the American Medical Association’s (AMA) Current Procedural Terminology (CPT^®) Editorial Panel has approved the company’s request to add two new Category III CPT codes that describe a cell-based therapy for patients with scleroderma of the hands. [Cytori Therapeutics, Inc.] Press Release

Shire Submits Investigational New Drug Application to FDA for Gene Therapy Candidate SHP654 for Treatment of Hemophilia A

Shire plc announced the submission of an investigational new drug application to the U.S. Food and Drug Administration (FDA) for SHP654, also designated as BAX 888, an investigational factor VIII gene therapy for the treatment of hemophilia A. [Shire plc] Press Release

Trump Administration Chooses Georgia Physician to Lead US Public-Health Agency

Obstetrician and gynecologist Brenda Fitzgerald will direct the US Centers for Disease Control and Prevention, Secretary of Health and Human Services Tom Price announced. [Nature News] Editorial

French Scientist Fined for Failure to Disclose Industry Ties

In an unprecedented court case in Paris, an eminent French lung specialist has been fined €50,000 and given a six-month suspended jail sentence because he didn’t disclose his ties to the oil industry during a Senate air-pollution inquiry. [Nature News] Editorial

House Lawmakers Balk at Most Trump Science Cuts in Early Bills

No. That’s the first official answer from lawmakers in the U.S. House of Representatives to President Donald Trump’s request to make deep budget cuts at several key science funding agencies. [ScienceInsider] Editorial

Europe’s Next Big Science-Funding Program Urged to Double Its Budget

Midway through the European Union’s sprawling seven-year, €75-billion research-funding program known as Horizon 2020, scientists are already angling for more money and less red tape in its successor. [Nature News] Editorial

Germany and Poland Launch Research ‘Twinning’ Effort

Germany and Poland are to jointly fund top scientists to start research groups at Polish institutes, in a scheme that could provide a new blueprint for east–west research collaboration in the European Union. [Nature News] Editorial

NIH

Center for Scientific Review; Notice of Closed Meetings (FR Doc. No:2017-14227) Notice

NEW CAR-T Summit 2017
September 5-8, 2017
Boston, United States

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NEW Senior Research Associate – Cellular Therapy Core (University of Texas Health Science Center at Houston)

NEW Postdoctoral Fellow – Single Cell Genomics (BC Cancer Agency Research Center)

NEW Research Associate – Cell Engineering (Caribou Biosciences, Inc.)

PhD Scholarship – Virology/Gene Therapy (University of New South Wales)

Postdoctoral Scientist – Systems Oncology Group (CRUK Manchester Institute)

Quality Improvement Coordinator – Cellular Therapy Core (University of Texas Health Science Center at Houston)

Postdoctoral Position – Tumor Immunology and Cancer Immunotherapy (VIB)

Professor – Stem Cell Biology (University College London)

Specialist – Cell Procurement (Celgene Corporation)

Scientist – Analytical Development (KBI Biopharma)

GMP Manufacturing Director – Cell and Gene Therapy (Fred Hutchinson Cancer Research Center)

Research Fellow – Functional Roles of Stem Cell Derived Extracellular Vesicles (Mayo Clinic)

Professor and Faculty Positions – Various Projects (Academy of Medical Sciences, Zhengzhou University)

Postdoctoral Fellow – Various Projects (University of Oklahoma)

GMP Manufacturing Director – Cell and Gene Therapy (Fred Hutchinson Cancer Research Center)

Assistant or Associate Member – Stem CellGene Therapy (Fred Hutchinson Cancer Research Center)

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