Cell Therapy News Volume 18.37 | Nov 6 2017

TOP STORY

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

Spinal muscular atrophy type 1 is a progressive, monogenic motor neuron disease with an onset during infancy that results in failure to achieve motor milestones and in death or the need for mechanical ventilation by two years of age. Researchers studied functional replacement of the mutated gene encoding survival motor neuron 1 in this disease. [N Engl J Med] Abstract | Press Release 1 | Press Release 2

(Ranked by impact factor of the journal)

Secreted αKlotho Isoform Protects against Age-Dependent Memory Deficits

Scientists studied the functional relevance at behavioral level of modifying secreted isoform Klotho (s-KL) levels in the aging brain. They used AAVrh10 vectors to deliver and sustained expression of s-KL in 6- and 12-month-old wild-type C57BL/6J males. [Mol Psychiatry] Full Article | Press Release | Editorial

HIF2α Targeted RNAi Therapeutic Inhibits Clear Cell Renal Cell Carcinoma

Investigators describe an RNAi therapeutic for hypoxia-inducible factor 2α (HIF2α) that utilizes a targeting ligand that selectively binds to integrins αvβ3 and αvβ5 frequently over-expressed in clear cell renal cell carcinoma (ccRCC). They demonstrated that functional delivery of a HIF2α specific RNAi trigger resulted in HIF2α gene silencing and subsequent tumor growth inhibition and degeneration in an established orthotopic ccRCC xenograft model. [Mol Cancer Ther] Abstract | Full Article

Results of a Phase I Study of Quizartinib as Maintenance Therapy in Subjects with Acute Myeloid Leukemia in Remission following Allogeneic Hematopoietic Stem Cell Transplant

This two-part, Phase I, multicenter, open-label, sequential-group, dose-escalation study determined dose-limiting toxicities, maximum tolerated dose, and safety/tolerability of quizartinib, a selective and highly potent FLT3 inhibitor, when administered as maintenance therapy after allogeneic hematopoietic-cell transplant. [Am J Hematol] Abstract

BRAF Peptide Vaccine Facilitates Therapy of Murine BRAF-Mutant Melanoma

Scientists introduced lipid-coated calcium phosphate nanoparticles as a carrier to efficiently deliver a tumor-specific antigen, the BRAF^V600E peptide, to drive dendritic cell maturation and antigen presentation in C57BL6 mice. [Cancer Immunol Immunother] Abstract

A Phase I Vaccination Study with Dendritic Cells Loaded with NY-ESO-1 and α-Galactosylceramide: Induction of Polyfunctional T Cells in High-Risk Melanoma Patients

In this Phase I dose escalation study, researchers treated eight patients with high-risk surgically resected stage II–IV melanoma with intravenous autologous monocyte-derived dendritic cells loaded with the natural killer T cell agonist α-galactosylceramide and peptides derived from the cancer testis antigen New York esophageal squamous cell carcinoma-1 (NY-ESO-1). [Cancer Immunol Immunother] Abstract

Stimulation of a Calcified Cartilage Connecting Zone by GDF-5-Augmented Fibrin Hydrogel in a Novel Layered Ectopic In Vivo Model

The authors investigated whether growth and differentiation factor-5-(GDF-5)-augmented fibrin hydrogel can induce a calcified cartilage-layer in vitro that seamlessly connects cartilage-relevant biomaterials with bone tissue. Human bone marrow stromal cells were embedded in fibrin hydrogel and subjected to chondrogenesis with TGF-β with or without GDF-5 before constructs were implanted subcutaneously into SCID mice. [J Biomed Mater Res B Appl Biomater] Abstract

Hematopoietic Stem Cell Transplantation in Niemann–Pick Disease Type B Monitored by Chitotriosidase Activity

Scientists report a patient with Niemann–Pick disease type B, with early severe onset of disease and pulmonary involvement, treated with hematopoietic stem cell transplant (HSCT) from a bone marrow matched unrelated donor. They confirmed that HSCT is feasible and potentially beneficial for patients with severe phenotype. [Pediatr Blood Cancer] Abstract

Hepatic Intra-Arterial Delivery of a “Trojan-Horses” Gene Therapy: A Pilot Study on Rabbit VX2 Hepatic Tumor Model

Investigators assessed the feasibility and efficacy of intra-arterial administration of mesenchymal stem cells transduced with an optimized gene followed by intravenous administration of cyclophosphamide into the VX2 rabbit liver tumor. [Cardiovasc Intervent Radiol] Abstract

Cellular Therapy for Traumatic Neurological Injury

The authors characterize cell therapy strategies: cell replacement and tissue integration vs. immunomodulation/enhanced endogenous tissue repair. [Pediatr Res] Abstract

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

Servier and Pfizer Announce Preliminary Results of the First-in-Human Trials of UCART19 Will Be Presented

Servier, Pfizer Inc. and Cellectis announced that preliminary results from two Phase I trials with UCART19, the allogeneic anti-CD19 CAR T-cell product being developed by Servier and Pfizer, will be presented. [Press release from Servier discussing research to be presented at the 59^th American Society of Hematology (ASH) Annual Meeting and Exposition, Atlanta] Press Release

Cellectis Announces Two Oral Presentations and One Poster Presentation

Cellectis announced that abstracts regarding the company’s allogeneic, off-the-shelf, CAR T product candidates have been accepted for presentation. [Press release from Cellectis discussing research to be presented at the 59^th American Society of Hematology (ASH) Annual Meeting and Exposition, Atlanta] Press Release

bluebird bio to Present New Data from Clinical Studies of LentiGlobin™ Gene Therapy in Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease and bb2121 in Relapsed/Refractory Multiple Myeloma

bluebird bio, Inc. announced that four oral and seven poster presentations will feature data from bluebird programs. The data will highlight bluebird’s advancement of its LentiGlobin product candidate in patients with transfusion-dependent β-thalassemia and severe sickle cell disease, and its bb2121 product candidate in patients with relapsed/refractory multiple myeloma. [Press release from bluebird bio, Inc. discussing research to be presented at the 59^th American Society of Hematology (ASH) Annual Meeting and Exposition, Atlanta] Press Release

Juno Therapeutics to Highlight New Advances in CD19- and BCMA-Targeted CAR T Therapy

Juno Therapeutics, Inc. announced that 15 abstracts detailing updated clinical and preclinical results from the company and its collaborators will be presented. [Press release from Juno Therapeutics, Inc. discussing research to be presented at the 59^th American Society of Hematology (ASH) Annual Meeting and Exposition, Atlanta] Press Release

Novartis Submits Application to FDA for Kymriah™ (Tisagenlecleucel) in Adult Patients with r/r DLBCL, Seeking Second Indication for First-Ever FDA Approved CAR-T Therapy

Novartis announced that the company has submitted a supplemental Biologics License Application to the FDA for Kymriah™ suspension for intravenous infusion, formerly CTL019, for the treatment of adult patients with relapsed or refractory (r/r) diffuse large B-cell lymphoma (DLBCL) who are ineligible for autologous stem cell transplant. [Novartis AG] Press Release

Mayo Clinic to Offer CAR T-Cell Therapy for Relapsed Non-Hodgkin Lymphoma

Mayo Clinic announced that its Rochester campus is one of 16 cancer centers nationally selected to provide chimeric antigen receptor T-cell therapy for adults with B-cell non-Hodgkin lymphoma who have not responded to, or have relapsed, after two or more lines of treatment. [Mayo Clinic] Press Release

Voyager Therapeutics Announces Update to Parkinson’s Disease Program

Voyager Therapeutics, Inc. announced an update to its VY-AADC program for advanced Parkinson’s disease. [Voyager Therapeutics, Inc.] Press Release

Wave Life Sciences Initiates Clinical Trial for Lead Program in Duchenne Muscular Dystrophy (DMD)

Wave Life Sciences Ltd. announced the initiation of a global Phase I clinical trial for WVE-210201 in DMD patients amenable to exon 51 skipping. [WAVE Life Sciences] Press Release

TCR² Therapeutics Appoints Cell Therapy Pioneer Dr. Stephan Grupp to Advisory Board

TCR² Therapeutics Inc. announced the appointment of Stephan Grupp, MD, PhD, the Yetta Deitch Novotny Professor of Pediatrics at the Perelman School of Medicine at the University of Pennsylvania, and Chief of the Section of Cellular Therapy and Transplant at Children’s Hospital of Philadelphia to its Advisory Board. [TCR² Therapeutics Inc.] Press Release

Lamar Smith, the Departing Head of the House Science Panel, Will Leave a Controversial and Complicated Legacy

When Representative Lamar Smith announced that he would not run for re-election in 2018, after 32 years in Congress, many scientists reacted with glee. [ScienceInsider] Editorial

Pay for US Postdocs Varies Wildly by Institution

The salary data are incomplete and — in some cases — appear to be incorrect. Some researchers are listed as earning nothing, and another study underway suggests a higher overall rate of pay for US postdocs. But the latest analysis underscores the challenges of getting basic information about an under-recognized and misunderstood segment of the academic workforce. [Nature News] Editorial

Leading Western Publisher Bows to Chinese Censorship

Springer Nature, whose publications include Nature and Scientific American, acknowledged that at the government’s request, it had removed articles from its mainland site that touch on topics the ruling Communist Party considers sensitive, including Taiwan, Tibet, human rights and elite politics. [The New York Times] Editorial

FDA

Abbreviated New Drug Applications: Pre-Submission of Facility Information Related to Prioritized Generic Drug Applications (Pre-Submission Facility Correspondence); Draft Guidance for Industry; Availability (FR Doc. No:2017-24099) Notice

Medical Devices; Immunology and Microbiology Devices; Classification of the Newborn Screening Test for Severe Combined Immunodeficiency Disorder (FR Doc. No:2017-23496) Notice

NEW Keystone Symposia: Emerging Cellular Therapies: T Cells and Beyond
February 11-15, 2018
Keystone, Colorado, United States

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NEW Postdoctoral Position – Stem Cell Therapy (University of Southern Denmark)

Research Technologist – Immunotherapy (STEMCELL Technologies Inc.)

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

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

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 – 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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