Cell Therapy News Volume 19.42 | Dec 17 2018

TOP STORY

Infusion of Third-Party Mesenchymal Stromal Cells after Kidney Transplantation: A Phase I-II, Open-Label, Clinical Study

Researchers report on the one-year follow-up of an open-label Phase I-II trial of a single infusion of third-party mesenchymal stromal cell (MSC) post-kidney transplantation, in addition to standard immunosuppression. Ten kidney transplant recipients from deceased donors received third-party bone marrow MSCs on day 3 ± 2 post-transplant and were compared to ten concurrent controls. [Kidney Int] Abstract

(Ranked by impact factor of the journal)

Therapeutic Effect of Hepatocyte Growth Factor-Overexpressing Bone Marrow-Derived Mesenchymal Stem Cells on CCl₄-Induced Hepatocirrhosis

Bone marrow-derived mesenchymal stem cells from host rats were transduced with an adenoviral vector labelled with green fluorescent protein to overexpress hepatocyte growth factor. [Cell Death Dis] Full Article

Gender-Independent Efficacy of Mesenchymal Stem Cell Therapy in Sex Hormone-Deficient Bone Loss via Immunosuppression and Resident Stem Cell Recovery

Scientists investigated the therapeutic efficacy of using an infusion of mesenchymal stem cells (MSCs) to treat sex hormone-deficient bone loss and its underlying mechanisms. In particular, they compared the impacts of MSC cytotherapy in the two genders with the aim of examining potential gender differences. [Exp Mol Med] Full Article

Remission of Hyperglycemia in Spontaneously Diabetic NOD Mice upon Transplant of Microencapsulated Human Umbilical Cord Wharton Jelly‐Derived Mesenchymal Stem Cells (hUCMS)

Researchers transplanted microencapsulated hUCMS into the peritoneal cavity of non obese diabetic (NOD) mice with either severe or mild diabetes mellitus. Blood glucose levels were monitored following xenograft, in either basal or upon glucose stimulation. [Xenotransplantation] Abstract

Mesenchymal Stem Cells Increase Heme Oxygenase 1-Activated Autophagy in Treatment of Acute Liver Failure

The authors explored whether mesenchymal stem cells treat acute liver failure by regulating autophagy and whether heme oxygenase 1 was involved in the same pathway. [Biochem Biophys Res Commun] Abstract

Enhanced Delivery of Oncolytic Adenovirus by Neural Stem Cells for Treatment of Metastatic Ovarian Cancer

Investigators demonstrated the ability of an allogeneic, clonal neural stem cell line to protect oncolytic viral cargo from neutralizing antibodies within patient ascites fluid, and to deliver it to tumors within preclinical peritoneal ovarian metastases models. [Mol Ther Oncolytics] Full Article | Graphical Abstract

Recombinant AAV-CEA Tumor Vaccine in Combination with an Immune Adjuvant Breaks Tolerance and Provides Protective Immunity

Using a transgenic mouse model constitutively expressing human carcinoembryonic antigen (CEA) in a spatiotemporal manner as a self-protein and a syngeneic mouse colon cancer cell line, MC38-CEA, overexpressing CEA, researchers tested the potential of a novel genetic immunotherapy approach against CEA-expressing tumors, using recombinant adeno associated virus vector encoding CEA and appropriately timed immune adjuvant application. [Mol Ther Oncolytics] Full Article

CD19⁺IgM⁺ Cells Demonstrate Enhanced Therapeutic Efficacy in Type 1 Diabetes Mellitus

Investigators describe a protective effect on autoimmune diabetes and reduced destructive insulitis in NOD.scid recipients following splenocyte injections from diabetic NOD donors and sorted CD19⁺ cells compared with NOD.scid recipients receiving splenocytes alone. [JCI Insight] Full Article

AAV CRISPR Editing Rescues Cardiac and Muscle Function for 18 Months in Dystrophic Mice

Adeno-associated virus (AAV)-mediated CRISPR editing is a revolutionary approach for treating inherited diseases. Sustained, often life-long mutation correction is required for treating these diseases. Unfortunately, this has never been demonstrated with AAV CRISPR therapy. Scientists addressed this question in the mdx model of Duchenne muscular dystrophy. [JCI Insight] Full Article

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Exploiting Oncogenic Drivers along the CCNG1 Pathway for Cancer Therapy and Gene Therapy

The authors focus on a pivotal and therapeutically accessible locus of executive cell cycle checkpoint control—focusing specifically on the cyclin G1 protein (CCNG1 proto-oncogene product) and the associated oncogenic drivers arrayed along the aberrant biochemical pathways that promote and ensure uncontrolled cell proliferation, resulting in oncogenesis, increasingly aggressive metastasis, and chemotherapeutic refractoriness. [Mol Ther Oncolytics] Full Article

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Bellicum Reports Safety Results and Promising Activity of Its Controlled CAR-T Candidate BPX-601 in Patients with Advanced Pancreatic Cancer

Bellicum Pharmaceuticals, Inc. reported safety and promising activity of BPX-601 in Part 1 of a Phase I/II dose-escalation study in patients with advanced, metastatic pancreatic cancer expressing prostate stem cell antigen. [Press release from Bellicum Pharmaceuticals, Inc. discussing research presented at the 2018 European Society for Medical Oncology Immuno-Oncology Congress (ESMO-IO), Geneva] Press Release

Gene Therapy Targets Sickle-Cell Disease

Gene therapy might offer a cure for sickle-cell disease, and clinical trials are already under way. “In the long run I think it will be able to cure the disease,” says Vichinsky, a hematologist and oncologist at the University of California, San Francisco Benioff Children’s Hospital in Oakland. [Nature News] Press Release

Voyager Therapeutics Joins Michael J. Fox Foundation Initiatives and Education Campaigns to Accelerate the Development of New Therapies and a Cure for Parkinson’s Disease

Voyager Therapeutics, Inc. announced joining multiple partnerships with The Michael J. Fox Foundation for Parkinson’s Research including the Parkinson’s Progression Markers Initiative and the Parkinson’s Disease Education Consortium. [Voyager Therapeutics, Inc.] Press Release

NHMRC to Fund Major Phase II Clinical Trial of Cynata’s Cymerus™ MSCs in Patients with Osteoarthritis

Cynata Therapeutics Limited announced that the Australian National Health and Medical Research Council (NHMRC) has approved a grant to fund a Phase II clinical trial to evaluate Cynata’s Cymerus™ mesenchymal stem cells (MSCs) as a treatment for osteoarthritis. [Cynata Therapeutics Limited (GlobeNewswire, Inc.)] Press Release

Sangamo Announces Treatment of First Patient in Phase I/II Clinical Trial of In Vivo Genome Editing Therapy for Hemophilia B

Sangamo Therapeutics, Inc. announced treatment of the first patient in the Phase I/II clinical trial evaluating SB-FIX, an investigational in vivo genome editing therapy for patients with hemophilia B. [Sangamo Therapeutics, Inc.] Press Release

AGTC Announces Topline Interim Six-Month Data from Phase I/II X-Linked Retinoschisis Clinical Study; Termination of Biogen Collaboration

Applied Genetic Technologies Corporation reported topline interim six-month data from its Phase I/II clinical trial of rAAV2tYF-CB-hRS1, an investigational AAV-based gene therapy delivered via intravitreal injection for X-linked retinoschisis due to mutations in the RS1 gene. [Applied Genetic Technologies Corporation] Press Release

FDA Accepts BrainStorm’s NurOwn^® IND Application for Progressive Multiple Sclerosis

BrainStorm Cell Therapeutics Inc. announced that the FDA has accepted the Investigational New Drug (IND) application to initiate a Phase II clinical trial of NurOwn^® for the treatment of progressive multiple sclerosis. [BrainStorm Cell Therapeutics Inc.] Press Release

Orchard Therapeutics and SIRION Biotech Announce Licensing Agreement to Enhance Gene Therapy Manufacturing Efficiency

Orchard Therapeutics and SIRION Biotech GmbH announced the entry into a license agreement, pursuant to which Orchard has licensed SIRION’s LentiBOOST™ technology to enhance manufacturing efficiency for certain of Orchard’s ex vivo autologous hematopoietic stem cell gene therapy drug candidates. [Orchard Therapeutics plc.] Press Release

Orchard Therapeutics Announces the Build-Out of New Gene Therapy Manufacturing Facility in California

Orchard Therapeutics announced that it has signed a long-term lease agreement to build-out a gene therapy manufacturing facility in Fremont, California. [Orchard Therapeutics plc.] Press Release

Gilbert Family Foundation Announces $12 Million Gene Therapy Initiative to Address Underlying Cause of NF1

Over the next three years, $12 million in research grants from the Gilbert Family Foundation will fund several renowned multi-disciplinary research teams to identify which gene therapy techniques are most promising for neurofibromatosis type 1 (NF1). [The Gilbert Family Foundation (PR Newswire Association LLC.)] Press Release

Regulating the Gene-Therapy Revolution

For rare genetic diseases that affect the young, such as a neurodegenerative condition called spinal muscular atrophy, gene therapies bring much-needed hope — a chance for the child to live a relatively normal life. But they also raise serious fears about their efficacy and the potential risks that accompany irreversible one-off treatments. [Nature News] Editorial

China Introduces ‘Social’ Punishments for Scientific Misconduct

Researchers in China who commit scientific misconduct could soon be prevented from getting a bank loan, running a company or applying for a public-service job. The government has announced an extensive punishment system that could have significant consequences for offenders — far beyond their academic careers. [Nature News] Editorial

University of California and Elsevier Locked in Negotiations

The University of California (UC) is pushing back against the publishing giant Elsevier over fees and access to research papers. The UC system’s five-year contract expires on December 31 and it has prepared for faculty and students to lose access Elsevier’s journals if they don’t reach an agreement in time. [The Scientist] Editorial

South Korean University Delays Vote on whether to Suspend President

Leaders at the Korea Advanced Institute of Science and Technology decided 6–3 to defer a vote on whether to suspend the university’s president Shin Sung-Chul, who has been accused of making illegal payments to a lab in California — allegations that he denies. The board will take the matter up again at its next meeting. [Nature News] Editorial

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