Cell Therapy News Volume 19.12 | Apr 9 2018

TOP STORY

A Bioengineered Retinal Pigment Epithelial Monolayer for Advanced, Dry Age-Related Macular Degeneration

A prospective, interventional, FDA-cleared, Phase I/IIa study is being conducted to assess the safety and efficacy of a composite subretinal implant in subjects with advanced non-neovascular age-related macular degeneration. The authors report an interim analysis of the Phase I cohort consisting of five subjects. [Sci Transl Med] Abstract | Press Release 1 | Press Release 2

(Ranked by impact factor of the journal)

Administration of Mesenchymal Stem Cells during ECMO Results in a Rapid Decline in Oxygenator Performance

Researchers provide evidence from an ex vivo model of extracorporeal membrane oxygenation (ECMO) to suggest that the intravascular administration of mesenchymal stem cells during ECMO may adversely impact the function of a membrane oxygenator. [Thorax] Abstract

Human CD19-Targeted Mouse T-Cells Induce B-Cell Aplasia and Toxicity in Human CD19 Transgenic Mice

To elucidate the underlying toxicity mechanisms and test potentially safer CAR T-cells, scientists developed a mouse model in which human CD19 (hCD19)-specific mouse CAR T-cells were adoptively transferred into mice whose normal B-cells expressed a hCD19 transgene at hemizygous levels. [Mol Ther] Abstract

Vitamin A-Coupled Liposomes Carrying TLR4-Silencing shRNA Induce Apoptosis of Pancreatic Stellate Cells and Resolution of Pancreatic Fibrosis

To ensure the efficient delivery of RNAi therapeutic agents to pancreatic stellate cells, vitamin A-coupled liposomes were used as drug carriers to deliver plasmids expressing Toll-like receptor (TLR)4-specific short hairpin RNA (shRNA) to treat pancreatic fibrosis. [J Mol Med] Abstract

Longterm Reversal of Severe Visual Loss by Mitochondrial Gene Transfer in a Mouse Model of Leber Hereditary Optic Neuropathy

Researchers showed reversal of visual dysfunction by a mitochondrially targeted adeno associated virus in transgenic mice harboring a G11778A mutation in the ND4 subunit of complex I persists longterm and it is associated with reduced loss of retinal ganglion cells and their axons, improved oxidative phosphorylation, persistence of transferred ND4 DNA and transcription of ND4 mRNA. [Sci Rep] Full Article

Lentiviral Vector Delivery of Short Hairpin RNA to NgR1 Promotes Nerve Regeneration and Locomotor Recovery in Injured Rat Spinal Cord

Rats were randomly divided into three groups: including the lentiviral vector (LV)-Nogo receptor 1 (NgR1) short-hairpin RNA (shRNA) injection, LV-control shRNA injection and sham. Eight weeks post-injection of LV, spinal cords were examined by histology for changes in cavity size and by immunohistochemistry for changes in expression of NgR1, cell apoptosis, astrocytes, neurons and myelination. [Sci Rep] Full Article

Intramuscular Transplantation of Bone Marrow Cells Prolongs the Lifespan of SOD1^G93A Mice and Modulates Expression of Prognosis Biomarkers of the Disease

Bone marrow intramuscular transplants resulted in increased longevity with improved motor function and decreased motoneuron degeneration in the spinal cord. [Stem Cell Res Ther] Full Article

Excessive Activated T-Cell Proliferation after Anti-CD19 CAR T-Cell Therapy

Excessive activated T-cell proliferation was observed in vivo in one patient after an anti-CD19-CAR T-cell infusion. The patient, who had chemotherapy refractory and CD19⁺ diffuse large B-cell lymphoma, received an anti-CD19 CAR T-cell infusion following conditioning chemotherapy. [Gene Ther] Abstract

Nivolumab Maintenance after Salvage Autologous Stem Cell Transplantation Results in Long Term Remission in Multiple Relapsed Primary CNS Lymphoma

Investigators report the first documented case of a successful third autologous stem cell transplantation in second relapse of primary central nervous system lymphoma. Whole-exome sequencing identified a hypermutated tumor genotype. [Eur J Haematol] Abstract

Influence of Stem Cell Therapy on Thyroid Function and Reactive Oxygen Species Production in Diabetic Rats

Researchers investigated the effect of the treatment with mesenchymal stem cells on thyroid function and reactive oxygen species generation in an experimental model of type 1 diabetes mellitus. [Horm Metab Res] Abstract

Gene Therapy Strategies to Restore ER Proteostasis in Disease

The delivery of endoplasmic reticulum (ER) chaperones or active unfolded protein response components using adeno-associated virus have demonstrated outstanding beneficial effects in several disease models. Scientists discuss efforts to design and optimize gene therapy strategies to improve ER proteostasis in different disease contexts. [Mol Ther] Abstract

Hydrogel-Based Scaffolds to Support Intrathecal Stem Cell Transplantation as a Gateway to the Spinal Cord: Clinical Needs, Biomaterials, and Imaging Technologies

The authors provide an overview of imaging modalities for in vivo monitoring and characterization of biomaterials and stem cells. This comprehensive review serves as a guide for those planning preclinical and clinical studies on intrathecal stem cell transplantation. [Gene Ther] Full Article

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

Data from BioTime’s OpRegen^® Program to Be Presented

BioTime, Inc. announced that data from the ongoing OpRegen^® trial will be presented. [Press release from BioTime, Inc. discussing research to be presented at the 5^th Annual Retinal Cell and Gene Therapy Innovation Summit, Honolulu] Press Release

Lilly and Sigilon Therapeutics Announce Strategic Collaboration to Develop Encapsulated Cell Therapies for the Treatment of Type 1 Diabetes

Eli Lilly and Company and Sigilon Therapeutics announced a global collaboration to develop encapsulated cell therapies for the potential treatment of type 1 diabetes. [Eli Lilly and Company] Press Release

Pfizer and Allogene Therapeutics Enter into Asset Contribution Agreement for Pfizer’s Allogeneic CAR T Immuno-Oncology Portfolio

Pfizer Inc. and Allogene Therapeutics, Inc. announced that the two companies have entered into an asset contribution agreement for Pfizer’s portfolio of assets related to allogeneic CAR T therapy, an investigational immune cell therapy approach to treating cancer. [Pfizer Inc.] Press Release

SpinalCyte Announces MRI Evidence of Disc Regeneration following Phase I/Phase II Clinical Trial of CybroCell™ Dermal Fibroblasts

MRI analysis of patients who received intradiscal injections of human dermal fibroblasts showed significant improvement in disc height after six months. [SpinalCyte, LLC] Press Release

Magenta Therapeutics Announces First Patient Transplanted with MGTA-456 in Phase II Study in Inherited Metabolic Disorders

Magenta Therapeutics announced treatment of the first patient with an inherited metabolic disorder in a Phase II study of MGTA-456, an expanded cord blood stem cell product. [Magenta Therapeutics] Press Release

Be The Match BioTherapies Strengthens Partnership with Magenta Therapeutics

Be The Match BioTherapies^® announced that it has made its second equity investment in Magenta Therapeutics. [Be The Match BioTherapies^®] Press Release

Novartis Enters Agreement to Acquire AveXis Inc. for USD 8.7 Billion to Transform Care in SMA and Expand Position as a Gene Therapy and Neuroscience Leader

Novartis announced that it has entered into an agreement and plan of merger with AveXis, Inc. to acquire the US-based Nasdaq-listed clinical stage gene therapy company for USD 218 per share or a total of USD 8.7 billion in cash. [Novartis AG] Press Release

French Say ‘No Deal’ to Springer as Journal Fight Spreads

Research institutions, first in Germany and now in France, are finding that publishers do not cut off access to journals–even when they stop paying. [Times Higher Education] Editorial

Cutting-Edge Cancer Drug Hobbled by Diagnostic Test Confusion

A landmark cancer drug approved last year seemed to herald a long-anticipated change in the treatment of some tumors: with medicines selected on the basis of molecular markers, rather than the tissue in which the cancer first took root. But clinicians and researchers are struggling to put that theory into practice. [Nature News] Editorial

China Asserts Firm Grip on Research Data

In a move few scientists anticipated, the Chinese government has decreed that all scientific data generated in China must be submitted to government-sanctioned data centers before appearing in publications. At the same time, the regulations call for open access and data sharing. [ScienceInsider] Editorial

UK Survey Reveals Widespread Sexual Misconduct by Academic Staff

A survey of current and former UK students has found that 41% experienced sexual misconduct – such as inappropriate comments, unwanted touching or assault – by staff at university. [Nature News] Editorial

NIH

National Human Genome Research Institute; Notice of Closed Meeting (FR Doc. No:2018-07115) Notice

NEW Annual Meeting of the Israel Stem Cell Society 2018
May 31, 2018
Tel Aviv, Israel

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

NEW 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)

Postdoctoral Fellowship – Functional Genomics of Human Hematopoiesis (Boston Children’s Hospital)

Postdoctoral Fellow – Cancer Research (Indiana University School of Medicine)

Postdoctoral Associate – Cardiovascular Science (Duke University)

Recruit Top Talent: Reach potential candidates by posting your organization’s career opportunities on the Connexon Creative Job Board at no cost.

Have we missed an important article or publication in Cell Therapy News? Click here to submit!

Comments or suggestions? Submit your feedback here.