Cell Therapy News Volume 18.36 | Oct 30 2017

TOP STORY

Transplantation of Wild-Type Mouse Hematopoietic Stem and Progenitor Cells Ameliorates Deficits in a Mouse Model of Friedreich’s Ataxia

Investigators report the therapeutic efficacy of transplanting wild-type mouse hematopoietic stem and progenitor cells into the YG8R mouse model of Friedreich’s ataxia. [Sci Transl Med] Abstract | Press Release

(Ranked by impact factor of the journal)

Reconstitution of UCP1 Using CRISPR/Cas9 in the White Adipose Tissue of Pigs Decreases Fat Deposition and Improves Thermogenic Capacity

Researchers report application of a CRISPR/Cas9-mediated, homologous recombination-independent approach to efficiently insert mouse adiponectin-UCP1 into the porcine endogenous UCP1 locus. The resultant UCP1 knock-in pigs showed an improved ability to maintain body temperature during acute cold exposure, but they did not have alterations in physical activity levels or total daily energy expenditure. [Proc Natl Acad Sci USA] Abstract | Editorial

Catheter-Directed Intraportal Delivery of Endothelial Cell Therapy for Liver Regeneration: A Feasibility Study in a Large-Animal Model of Cirrhosis

Scientists demonstrate the feasibility of imaging-guided catheter-directed delivery of endothelial cell therapy in a porcine model of cirrhosis for liver regeneration. Swine treated with endothelial cell therapy showed mean levels of surrogate markers of hepatobiliary injury that were consistent with decreases in hepatic fibrosis and biliary ductal damage relative to the control animals, although statistical significance was not met in this pilot study. [Radiology] Abstract | Press Release

Human Periodontal Ligament- and Gingiva-Derived Mesenchymal Stem Cells Promote Nerve Regeneration When Encapsulated in Alginate/Hyaluronic Acid 3D Scaffold

Investigators demonstrated that periodontal ligament stem cells and gingival mesenchymal stem cells isolated from adult human periodontal and gingival tissues assume neuronal phenotype in vitro and in vivo via a subcutaneous transplantation model in nude mice. [Adv Healthc Mater] Abstract

Outcomes of Unrelated Donor Stem Cell Transplant with Post-Transplant Cyclophosphamide for Graft-versus-Host Disease Prophylaxis in Patients with Severe Sickle Cell Disease

The authors report on the first four patients with severe sickle cell disease who underwent unrelated donor hematopoietic cell transplants using a novel myeloablative and immunosuppressive regimen comprising of busulfan, fludarabine and anti-thymocyte globulin with a single dose of post-transplant cyclophosphamide along with tacrolimus and mycophenolate mofetil for graft-versus-host-disease prophylaxis. [Biol Blood Marrow Transplant] Abstract

Effect of Autologous Cord Blood Infusion on Motor Function and Brain Connectivity in Young Children with Cerebral Palsy: A Randomized, Placebo-Controlled Trial

Researchers conducted a Phase II trial of autologous cord blood (ACB) infusion in children with cerebral palsy to test whether ACB could improve function. [Stem Cells Transl Med] Full Article | Press Release

Micromolar Affinity CAR T Cells to ICAM-1 Achieves Rapid Tumor Elimination while Avoiding Systemic Toxicity

A panel of affinity-variant CARs were constructed targeting overexpressed intercellular adhesion molecule (ICAM)-1, a broad tumor biomarker, using its physiological ligand, lymphocyte function-associated antigen-1. Anti-tumor T cell potency in vitro was directly proportional to CAR affinity and ICAM-1 density. [Sci Rep] Full Article

In Vivo Implantation of a Tissue Engineered Stem Cell Seeded Hemi-Laryngeal Replacement Maintains Airway, Phonation and Swallowing in Pigs

Porcine hemi-larynx were de-cellularized under negative pressure. The resultant acellular scaffold was seeded with human bone marrow derived mesenchymal stem cells and primary human epithelial cells. Seeded scaffolds were implanted orthotopically into a defect created in the thyroid cartilage in eight pigs and monitored in vivo for two months. [J Tissue Eng Regen Med] Abstract

Efficacy and Safety of AAV2 Gene Therapy in Children with Aromatic L-Amino Acid Decarboxylase Deficiency: An Open-Label, Phase I/II Trial

Scientists performed an open-label, Phase I/II trial at the National Taiwan University Hospital. They included patients who had a definitive diagnosis and clinical symptoms of aromatic l-amino acid decarboxylase deficiency, who were older than 24 months or had skull bones suitable for stereotactic surgery, and who had an anti-AAV2 antibody titre lower than 1·0 optical density. [Lancet Child Adolesc Health] Full Article

Therapeutic Application of T Regulatory Cells in Composite Tissue Allotransplantation

For future application of T regulatory (Treg) cells as therapeutics in composite tissue allotransplantation (CTA), molecular and cellular characteristics of CTA and immune rejection, Treg cell development and phenotypes, Treg cell plasticity and stability, immune tolerant functions of Treg cells in CTA in preclinical studies, and protocols for therapeutic application of Treg cells in clinical settings are addressed in this review. [J Transl Med] Full Article

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

Gene Therapy Trial Led by UMN Medical School’s Dr. Clark Chen Shows Promise in Treatment of Recurrent Brain Cancer

New data from a Phase I clinical trial led by Clark Chen, M.D., Ph.D., Lyle French Chair in Neurosurgery and Head of the University of Minnesota Medical School Department of Neurosurgery, showed more than a quarter of patients with recurrent high-grade glioma, a form of brain cancer, were alive more than three years after treatment. [Press release from the University of Minnesota Medical School discussing research presented at the 2017 AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics, Philadelphia] Press Release

Unum Therapeutics to Present New Data on Its Antibody-Coupled T Cell Receptor (ACTR) Platform

Unum Therapeutics Inc. announced that the company presented on its ACTR platform. The first poster presentation will highlighted data from non-clinical studies on effective targeting of HER2-amplified cancers with trastuzumab used in combination with ACTR707, a novel ACTR. [Press release from the Unum Therapeutics Inc. discussing research presented at the 2017 AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics, Philadelphia] Press Release

Institut Curie and Pierre Fabre Initiate a Novel Partnership in Immuno-Oncology

Institut Curie and the Institut de Recherche Pierre Fabre will collaborate on new therapeutic strategies to advance cancer treatment for patients. The objective of this novel partnership is to dissect the biology of novel therapeutically tractable targets and evaluate the impact of innovative pharmacological approaches leveraging the Institut Curie’s biological models. [Pierre Fabre] Press Release

Astellas and Universal Cells, Inc. Announce a Collaboration Utilizing Universal Donor Cell Technology

Astellas Pharma Inc. and Universal Cells, Inc. announced that Astellas Institute for Regenerative Medicine and Universal Cells have entered into an exclusive worldwide license agreement to research, develop and commercialize a novel cell therapy for an undisclosed indication. [Astellas Pharma Inc.] Press Release

Poseida Awarded $19.8 Million CIRM Grant to Support Clinical Trial of P-BCMA-101, a T Stem Cell Memory CAR-T Therapy for Multiple Myeloma

Poseida Therapeutics Inc. announced that the California Institute for Regenerative Medicine (CIRM) awarded a $19.8 million grant to support the clinical development of Poseida’s P-BCMA-101 product candidate. [Poseida Therapeutics Inc.] Press Release

Mustang Bio Establishes CAR T Cell Therapy Manufacturing Facility in Massachusetts

Mustang Bio, Inc. announced that it has entered into a lease agreement with the University of Massachusetts Medicine Science Park for a manufacturing facility to support the clinical development and commercialization of the company’s CAR T product candidates. [Mustang Bio, Inc.] Press Release

Following Approval of Israel’s Ministry of Health, Pluristem Extends Its Trial of PLX-R18 to Treat Insufficient Hematopoietic Recovery after Bone Marrow Transplant and Opens Clinical Centers in Israel

Pluristem Therapeutics Inc. announced that it received approval from Israel’s Ministry of Health to initiate a Phase I trial studying the company’s PLX-R18 cell therapy as a treatment for insufficient hematopoietic recovery following hematopoietic cell transplantation. [Pluristem Therapeutics Inc.] Press Release

Shire Receives Orphan Drug Designation for Gene Therapy Candidate SHP654 (BAX 888) for the Treatment of Hemophilia A

Shire plc announced the FDA awarded Orphan Drug Designation to Shire’s gene therapy candidate SHP654, an investigational factor VIII gene therapy for the treatment of hemophilia A. [Shire plc] Press Release

NICE Approves Gene Therapy for Rare ‘Bubble Baby Syndrome’

Strimvelis, a treatment for an ultra-rare inherited immune deficiency condition that has been dubbed ‘bubble baby syndrome’, has been approved by NICE in draft guidance. [National Institute for Health and Care Excellence (NICE)] Press Release

Bitter CRISPR Patent War Intensifies

The long-running battle over US patents for CRISPR–Cas9 gene editing continues. The Broad Institute of Cambridge, Massachusetts, filed a fresh set of arguments with the US government to defend a key patent. [Nature News] Editorial

US March for Science Group Faces Growing Pains

The US group that sparked the global March for Science movement is facing complaints about its management practices as it files for non-profit status and signals its intent to continue as “a movement to advance science and its role in public life”. [Nature News] Editorial

Key Legislator Tells Trump Officials to Back Off on Proposed Overhead Spending Cap for NIH

An influential legislator wants President Donald Trump’s administration and fellow Republicans to drop the notion of capping overhead costs on grants funded by the National Institutes of Health (NIH). [ScienceInsider] Editorial

China Announces Plans to Fast-Track Drug Approval

China is overhauling its drug-approval system to let companies bring their treatments to market quicker and more easily. The Communist Party of China and the State Council, two of the country’s most authoritative bodies, announced plans to reduce the backlog of medicines awaiting approval by the China Food and Drug Administration. Policies will also be introduced to boost the productivity of Chinese drugmakers and spur innovation in health care. [Nature News] Editorial

FDA

De Novo Classification Process (Evaluation of Automatic Class III Designation); Guidance for Industry and Food and Drug Administration Staff; Availability (FR Doc. No:2017-23492) Notice

NEW Keystone Symposia: Rigor in Science – The Challenge of Reproducibility in Biomedical Research
November 8, 2017
Online

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

NEW Postdoctoral Fellows – ImmunoGenomics (San Raffaele-Telethon Institute for Gene Therapy)

NEW Professorship – Gene Therapy of Eye Diseases (Ludwig-Maximilians-Universität München)

Research Technologist – Immunotherapy (STEMCELL Technologies Inc.)

Medical Director – Cell-Based Therapies (Celyad)

Postdoctoral Fellowship – CART-Cell Research (National Institutes of Health)

Postdoctoral Fellow – Cardiac Regeneration (Kyoto University)

Postdoctorate Research Associate – Cell Therapy (Nova Southeastern University)

Postdoctoral Position – Human PSC-Based Cell Therapy in Type 1 Diabetes (University of Copenhagen)

Postdoctoral Position – Development of Gene Therapy (Vitalité Health Network, Université de Moncton)

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

Postdoctoral Fellow – Various Projects (University of Oklahoma)

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