Cell Therapy News Volume 19.13 | Apr 16 2018

TOP STORY

Grafted Brain Organoids Provide Insight into Neurological Disorders

Researchers report a new approach that can develop more sophisticated organoid models by ensuring they receive sufficient oxygen and other nutrients via transplantation into rodents. [Press release from the Salk Institute for Biological Studies discussing online prepublication in Nature Biotechnology] Press Release | Abstract | Editorial 1 | Editorial 2

(Ranked by impact factor of the journal)

Potent Antitumor Efficacy of Anti-GD2 CAR T Cells in H3-K27M⁺ Diffuse Midline Gliomas

Scientists report that patient-derived mutated histone H3 K27M (H3-K27M)-mutant glioma cell cultures exhibit uniform, high expression of the disialoganglioside GD2. In five independent patient-derived H3-K27M⁺ diffuse midline glioma orthotopic xenograft models, systemic administration of GD2-targeted CAR T cells cleared engrafted tumors except for a small number of residual GD2^lo glioma cells. [Nat Med] Abstract | Press Release

Somatic Genome Editing with the RCAS-TVA-CRISPR-Cas9 System for Precision Tumor Modeling

Researchers combined the RCAS-TVA system with the CRISPR-Cas9 genome editing tools for precise modeling of human tumors. They showed that somatic deletion in neural stem cells of a variety of known tumor suppressor genes leads to high-grade glioma formation. [Nat Commun] Full Article | Press Release

Transposon-Modified Antigen-Specific T Lymphocytes for Sustained Therapeutic Protein Delivery In Vivo

Investigators report the utility of antigen-specific T lymphocytes as a regulatable peptide delivery platform for in vivo therapy. piggyBac transposon modification of murine cells with luciferase allowed them to visualize T cells after adoptive transfer. [Nat Commun] Full Article | Press Release

In Vivo CRISPR Screening Unveils Histone Demethylase UTX as an Important Epigenetic Regulator in Lung Tumorigenesis

The authors provided a systematic screening of tumor suppressor genes in vivo and identified UTX as an important epigenetic regulator in lung tumorigenesis. [Proc Natl Acad Sci USA] Abstract

In Vivo Migration of Mesenchymal Stem Cells to Burn Injury Sites and Their Therapeutic Effects in a Living Mouse Model

Investigators non-invasively monitored the tracking of mesenchymal stem cells (MSCs) toward burn injury sites using MSCs expressing firefly luciferase (Fluc) gene in living mice, and evaluated the effects of the MSCs at the injury site. Murine MSCs co-expressing Fluc and green fluorescent protein were established using a retroviral system. [J Control Release] Abstract | Graphical Abstract

Exploring the Potential of Polyurethane-Based Soft Foam as Cell-Free Scaffold for Soft Tissue Regeneration

Scientists developed a biodegradable polyurethane-based scaffold for soft tissue regeneration, characterized by an exceptional combination between softness and resilience. Exploring the potential as a cell-free scaffold required profound understanding of the impact of its intrinsic physico-chemical properties on the biological performance in vivo. [Acta Biomater] Abstract | Graphical Abstract

Compressed Collagen Enhances Stem Cell Therapy for Corneal Scarring

Researchers investigated the efficacy of compressed collagen gel as a vehicle to deliver stem cells from human corneal stroma for corneal therapy. [Stem Cells Transl Med] Full Article

Transplantation of Neural Progenitor Cells Expressing Glial Cell Line‐Derived Neurotrophic Factor into the Motor Cortex as a Strategy to Treat Amyotrophic Lateral Sclerosis

Human cortical‐derived neural progenitor cells engineered to secrete glial cell line‐derived neurotrophic factor (GDNF) were transplanted into the SOD1^G93A amyotrophic lateral sclerosis rat cortex, where they migrated, matured into astrocytes, and released GDNF. [Stem Cells Transl Med] Full Article

Endoscopic Submucosal Injection of Adipose-Derived Mesenchymal Stem Cells Ameliorates TNBS-Induced Colitis in Rats and Prevents Stenosis

Researchers evaluated the feasibility, safety and efficacy of endoscopic administration of adipose-derived mesenchymal stem cells in a colitis model in rats. [Stem Cell Res Ther] Full Article

Current State of Immunotherapy for Glioblastoma

The authors examine the current state of immunotherapy for gliomas, notably glioblastoma, the implications for combining the current standard-of-care treatment modalities with immunotherapies, potential biomarkers of response, and future directions for glioblastoma immuno-oncology. [Nat Rev Clin Oncol] Abstract

Pluripotent Stem Cell-Based Therapy for Parkinson’s Disease: Current Status and Future Prospects

Researchers discuss the prospects and challenges of human pluripotent stem cell-based cell therapy for Parkinson’s disease. [Prog Neurobiol] Abstract

Visit our reviews page to see a complete list of reviews in the cell therapy research field.

CRISPR Therapeutics Presents Positive Data on Allogeneic CRISPR-Based CAR-T Cell Therapies

CRISPR Therapeutics announced the presentation of new data from the company’s allogeneic CAR-T program. [Press release from CRISPR Therapeutics discussing research presented at the American Association for Cancer Research (AACR) Annual Meeting 2018, Chicago] Press Release

Affimed Presents Poster Highlighting Progress toward Novel EGFR-Targeting Therapy

Affimed N.V. presented a poster entitled “Pharmacokinetics and in vitro/in vivo characterization of high-affinity bispecific EGFR/CD16A NK cell engagers for the treatment of EGFR-expressing tumors” in the “Therapeutic Antibodies, Including Engineered Antibodies 2” session. [Press release from Affimed N.V. discussing research presented at the American Association for Cancer Research (AACR) Annual Meeting 2018, Chicago] Press Release

Fate Therapeutics Announces Off-the-Shelf CAR T-Cell Cancer Immunotherapy

Fate Therapeutics, Inc. announced that the company is presenting new preclinical data on FT819, its off-the-shelf CAR T-cell product candidate. [Press release from Fate Therapeutics, Inc. discussing research presented at the American Association for Cancer Research (AACR) Annual Meeting 2018, Chicago] Press Release

Fred Hutch Scientists to Feature Next-Generation T-Cell Therapies, Big Data, Precision Medicine and More

Fred Hutchinson Cancer Research Center’s latest findings will be featured in about 50 presentations. [Press release from Fred Hutchinson Cancer Research Center discussing research presented at the American Association for Cancer Research (AACR) Annual Meeting 2018, Chicago] Press Release

Promethera Biosciences Presents Novel Data on HepaStem’s Ability to Reduce Key Disease Parameters in Advanced Stage NASH In Vivo Model

Promethera Biosciences SA presented novel preclinical data on its development program HepaStem. [Press release from Promethera Biosciences SA discussing research presented at the European Association for the Study of the Liver (EASL) Annual Congress 2018, Paris] Press Release

Baylor University Medical Center Begins Breakthrough CAR-T Cancer Treatment for Patients with Non-Hodgkin’s Lymphoma

Baylor University Medical Center, a part of Baylor Scott & White Health, and Texas Oncology announced that they are now offering CAR-T cancer therapy to treat diffuse large B-cell lymphoma. These providers are working together and are among the first in the nation to treat patients with the only CAR-T cell therapy approved by the FDA to treat this form of cancer. [Baylor Scott and White Health (Newswise, Inc.)] Press Release

GSK Signs Strategic Agreement to Transfer Rare Disease Gene Therapy Portfolio to Orchard Therapeutics

GSK and Orchard Therapeutics announced a strategic agreement, under which GSK will transfer its portfolio of approved and investigational rare disease gene therapies to Orchard, securing the continued development of the programs and access for patients. [Orchard Therapeutics] Press Release

City of Hope Certified to Provide First FDA-Approved CAR T Cell Therapy for Pediatric and Young Adult Patients with Relapsed or Refractory B Cell Acute Lymphoblastic Leukemia

City of Hope announced that it is a certified treatment center to administer tisagenlecleucel CAR T cell therapy – the first CAR T cell therapy approved by the FDA – to patients up to 25 years of age with B cell precursor acute lymphoblastic leukemia that is refractory or in second or later relapse. [City of Hope] Press Release

Nature Cell and Biostar Stem Cell Research Institute Begin World’s First Stem Cell-Based Regenerative Treatment for Alzheimer’s Disease in Japan

South Korea’s Biostar Stem Cell Research Institute announced that Trinity Clinic Fukuoka, a partner hospital in Japan, received approval for the application of regenerative medicine in Alzheimer’s disease and officially began stem cell treatment. [Biostar Stem Cell Research and Development (Business Wire, Inc.)] Press Release

SpinalCyte Applies with FDA to Expand Human Clinical Trial of CybroCell^™ Dermal Fibroblasts as Investigational New Drug (IND)

SpinalCyte, LLC has filed an Investigational New Drug Application with the FDA to expand its study of CybroCell human dermal fibroblasts to treat degenerative disc disease. [SpinalCyte, LLC] Press Release

Pfizer Doses First Patient Using Investigational Mini-Dystrophin Gene Therapy for the Treatment of Duchenne Muscular Dystrophy

Pfizer Inc. has initiated a Phase Ib clinical trial for its mini-dystrophin gene therapy candidate, PF-06939926, in boys with Duchenne muscular dystrophy. [Pfizer Inc.] Press Release

Neuralstem Announces First Surgery Completed in Cervical Cohort of Phase I Clinical Trial in Patients with Chronic Spinal Cord Injury

Neuralstem, Inc. announced that it completed the first surgery in the second cervical injury cohort of a Phase I clinical trial in patients with chronic spinal cord injury. [Neuralstem, Inc.] Press Release

The University of Texas MD Anderson Cancer Center and Nanobiotix Have an Agreement to Run Immunotherapeutic Pre-Clinical Research in Lung Cancer Combining NBTXR3 and Nivolumab

NANOBIOTIX announced that it will cooperate with The University of Texas MD Anderson Cancer Center, Houston TX, to work on NBTXR3, Nanobiotix’s lead product. The project will take place over the course of two years and will evaluate the use of NBTXR3 activated by radiotherapy plus anti-PD1 Nivolumab, provided by Bristol-Myers Squibb in lung cancer models (in vitro and in vivo). [NANOBIOTIX] Press Release

University Declines to Sanction Doctor Who Referred Patient for Deadly Transplant

The doctor who referred a cancer patient for the first-ever artificial trachea implant will not face disciplinary action from the University of Iceland in Reykjavik, where he works. Patient Andemariam Beyene died after the implant, a polymer scaffold seeded with his own stem cells, failed. [ScienceInsider] Editorial

A Final Dash across the United States: Updates from the 2018 March for Science

The March for Science celebrated its anniversary. And although the turnout around the world was significantly smaller than last year, supporters haven’t lost any of their energy. [ScienceInsider] Editorial

FDA

Agency Information Collection Activities; Proposed Collection; Comment Request; Permanent Discontinuation or Interruption in Manufacturing of Certain Drug and Biological Products (FR Doc. No:2018-07684) Notice

NIH

National Center for Advancing Translational Sciences; Notice of Closed Meeting (FR Doc. No:2018-07753) Notice

NEW Gordon Research Conferences: Cardiac Regulatory Mechanisms
June 3-8, 2018
New London, New Hampshire, United States

Visit our events page to see a complete list of events in the community.

NEW Scientist/Senior Scientist – Lentivirus Production (Obsidian Therapeutics)

NEW Scientist/Senior Scientist – Bioinformatics and Data Sciences (Obsidian Therapeutics)

Postdoctoral Associate – Cancer Therapy (St. Jude Children’s Research Hospital)

Scientist – Discovery Biology (Prevail Therapeutics)

Postdoctoral Research Fellow – Cell/Gene Therapy (Stanford University)

Postdoctoral Fellow – CAR T (City of Hope)

Postdoctoral Fellow – Precision Therapy of Aggressive Lymphoma (AstraZeneca)

Postdoctoral Fellow – Leukemia (Necker Institut Enfants Malades)

Assistant Professor – Translation of Preclinical Regenerative Medicine into Advanced Therapy Medicinal Products (Lund University)

Postdoctoral Research Fellow – Hematopoiesis (Fred Hutchinson Cancer Research Center)

Postdoctoral Fellow – Immunology, Immunotherapy, Gene Therapy (City of Hope)

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