Volume 16.08 | Mar 2

Issue 16.08 March 2, 2015
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Successful Function of Autologous iPSC-Derived Dopamine Neurons following Transplantation in a Non-Human Primate Model of Parkinson’s Disease
Scientists analyzed cynomolgus monkey induced pluripotent stem cell (iPSC)-derived midbrain dopamine neurons for up to two years following autologous transplantation in a Parkinson’s disease model. [Cell Stem Cell]
Abstract | Graphical Abstract
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PUBLICATIONS (Ranked by impact factor of the journal)
A Small-Molecule Screen for Enhanced Homing of Systemically Infused Cells
Pretreatment of mesenchymal stromal cells (MSCs) with Ro-31-8425 increased MSC firm adhesion to an intercellular adhesion molecule 1-coated substrate in vitro and enabled targeted delivery of systemically administered MSCs to inflamed sites in vivo in a CD11a-dependent manner. [Cell Rep]
Full Article | Graphical Abstract | Press Release

Protective Effects of Human iPS-Derived Retinal Pigmented Epithelial Cells in Comparison with Human Mesenchymal Stromal Cells and Human Neural Stem Cells on the Degenerating Retina in rd1 Mice
Scientists investigated the rescue effects of induced pluripotent stem (iPS)-retinal pigmented epithelium cells in comparison with those cells in a clinical trial on photoreceptor degeneration in rd1 mice. Cells were injected into the subretinal space of immune-suppressed two-week-old rd1 mice. [Stem Cells] Abstract

In Vivo Targeted Delivery of ANGPTL8 Gene for Beta Cell Regeneration in Rats
Researchers employed ultrasound-targeted microbubble destruction to deliver human ANGPTL8 gene plasmids to the pancreas, liver and skeletal muscle of normal adult rats. [Diabetologia] Abstract

Tonsil-Derived Mesenchymal Stem Cells Ameliorate CCl4-Induced Liver Fibrosis in Mice via Autophagy Activation
Scientists used human palatine tonsil as a novel source of mesenchymal stem cells (T-MSCs) and examined their ability to differentiate into hepatocyte-like cells in vivo and in vitro. A carbon tetrachloride (CCl4) mouse model was used to investigate the ability of T-MSCs to home to the site of liver injury. [Sci Rep] Full Article

The Chromatin-Modifying Protein HMGA2 Promotes Atypical Teratoid/Rhabdoid Cell Tumorigenicity
Scientists used lentiviral short-hairpin RNA to suppress high-mobility group atypical teratoid (AT)-hook 2 (HMGA2) in AT/rhabdoid tumor cell lines and found that loss of HMGA2 led to decreased cell growth, proliferation, and colony formation and increased apoptosis. They also found that suppression of HMGA2 negatively affected in vivo orthotopic xenograft tumor growth, more than doubling median survival of mice from 58 days to 153 days. [J Neuropathol Exp Neurol] Abstract | Press Release

The Impact of HIV-1 Genetic Diversity on the Efficacy of a Combinatorial RNAi-Based Gene Therapy
To determine the breadth of the RNA interference (RNAi) gene therapy approach, researchers studied the susceptibility of HIV-1 subtypes A-E and drug-resistant variants. In addition, they monitored the evolution of HIV-1 escape variants. They demonstrated that the combinatorial RNAi therapy is highly effective against most isolates. [Gene Ther] Abstract

High-Dose Therapy with Auto-SCT Is Feasible in High-Risk Cardiac Amyloidosis
Cardiac involvement in light-chain amyloidosis predicts poor prognosis and is associated with higher TRM and morbidity during high-dose therapy and auto-SCT (HDT-ASCT). Scientists studied the outcomes of 30 patients with cardiac amyloidosis undergoing HDT-ASCT at their center between January 1998 and March 2012. [Bone Marrow Transplant] Abstract

A Phase II Study of Inotuzumab Ozogamicin and Rituximab, followed by Autologous Stem Cell Transplantation in Patients with Relapsed/Refractory Diffuse Large B-Cell Lymphoma
Researchers evaluated the safety and efficacy of inotuzumab ozogamicin, a targeted humanized anti-CD22 antibody conjugated to calicheamicin, plus rituximab every three weeks, up to six cycles, followed by high dose therapy and autologous stem cell transplantation in high-risk relapsed/refractory diffuse large B-cell patients. [Leuk Lymphoma] Abstract

Long-Term Expansion, Enhanced Chondrogenic Potential, and Suppression of Endochondral Ossification of Adult Human MSCs via WNT Signaling Modulation
Researchers report that the signaling protein WNT3A, in combination with FGF2, supports long-term expansion of human bone marrow-derived mesenchymal stem cells (MSCs). Inhibition of WNT signals during differentiation prevented calcification and maintained cartilage properties following implantation in a mouse model. [Stem Cell Rep] Full Article

Generation of Scaffoldless Hyaline Cartilaginous Tissue from Human iPSCs
Scientists developed an approach to generate scaffoldless hyaline cartilage from human induced pluripotent stem cells (hiPSCs). Subcutaneous transplantation of the hiPSC-derived particles generated hyaline cartilage that expressed type II collagen, but not type I collagen, in immunodeficiency mice. [Stem Cell Rep]
Full Article | Graphical Abstract

Learn More: UM729, a Novel Small Molecule for Ex Vivo Expansion of Human HSCs
Manufacture of Tumor- and Virus-Specific T Lymphocytes for Adoptive Cell Therapies
The authors review current manufacturing methods for the large-scale production of clinical-grade tumor-infiltrating lymphocytes, virus-specific and genetically modified chimeric antigen receptor or T-cell receptor transduced T cells in the context of Phase I/II clinical trials as well as the regulatory pathway to get these complex personalized cellular products to the clinic. [Cancer Gene Ther] Abstract

Gene Therapy for Radioprotection
Although much early research focused on small-molecule radioprotectors, there has been a growing interest in gene therapy for radioprotection. The amenability of gene therapy vectors to targeting, as well as the flexibility of gene therapy to accomplish ablation or augmentation of biologically relevant genes, makes gene therapy an excellent strategy for radioprotection. [Cancer Gene Ther] Abstract

B-Cell Activating Factor in the Pathophysiology of Multiple Myeloma: A Target for Therapy?
The authors discuss the role of B-cell activating factor (BAFF) in the pathophysiology of multiple myeloma (MM), and the potential of BAFF-inhibitory therapy for the treatment of MM. Multiple studies have shown that BAFF functions as a survival factor for malignant myeloma cells. [Blood Cancer J] Full Article

Visit our reviews page to see a complete list of reviews in the cell, gene and immunotherapy research field.

Free Nature Reviews Immunology Poster: The Immune Response to HIV
PROVENGE® (Sipuleucel-T) Demonstrates Sustained Immune Response Two Years after Treatment in Biochemically-Recurrent Prostate Cancer
Valeant Pharmaceuticals International, Inc. announced the presentation of new long-term, preliminary results from the Phase II STAND trial demonstrating a robust immune response with PROVENGE® that continues two years after completing treatment in men with biochemically-recurrent prostate cancer. [Press release from Valeant Pharmaceuticals International, Inc. discussing research presented at the 2015 Genitourinary Cancers Symposium, Orlando] Press Release

Bavarian Nordic Announces Updated Overall Survival Data from Combination Immunotherapy Data of PROSTVAC plus Ipilimumab
Bavarian Nordic A/S announced that updated overall survival data from an NCI sponsored combination study of Bavarian Nordic’s active prostate cancer immunotherapy candidate PROSTVAC and ipilimumab, an immune checkpoint inhibitor will be presented. [Press release from Bavarian Nordic A/S discussing research be presented at the 2015 Genitourinary Cancers Symposium, Orlando] Press Release

4th Annual Cell Line Development & Engineering Asia
India’s Budget Disappoints Scientists
If India’s scientists had hoped that Prime Minister Narendra Modi’s sweeping victory in last year’s general election heralded a step change for research funding, they have now been twice disappointed. In the country’s 2015–16 budget, announced on 28 February, funding for science remained flat in real terms, and has nosedived for some departments. [Nature News] Editorial

Therapeutic Cancer Vaccine Survives Biotech Bust
The first therapeutic cancer vaccine to be approved in the United States will stay on the market despite the financial collapse of the trailblazing biotechnology company that developed it. The vaccine, Provenge, was purchased on 23 February by Valeant Pharmaceuticals of Laval, Canada, which paid US$415 million for the prostate-cancer treatment and other assets of the bankrupt Dendreon Corporation. [Nature News] Editorial

Lone Physicist in Congress Joins Science Panel
The U.S. House of Representative’s one-man physics caucus is joining its science committee—with the goal of restoring science to its rightful place in legislative discourse. [ScienceInsider] Editorial

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Stanley Center at the Broad Institute and New York Stem Cell Foundation Partner to Develop Stem Cell Resource for Schizophrenia and Psychiatric Diseases
The New York Stem Cell Foundation and the Stanley Center at the Broad Institute of MIT and Harvard are partnering to create a foundational stem cell resource to study psychiatric disorders through the production of induced pluripotent stem cell lines from individuals with schizophrenia and other psychiatric disorders. [The Broad Institute] Press Release

Frederick Alt of Harvard University and Boston Children’s Hospital Awarded 2015 Szent-Gyorgyi Prize for Progress in Cancer Research
The National Foundation for Cancer Research announced that Frederick Alt, Ph.D., Professor of Genetics at Harvard Medical School, Director of the Program in Cellular and Molecular Medicine at Boston Children’s Hospital, and Howard Hughes Medical Institute Investigator has been awarded the 2015 Szent-Györgyi Prize for Progress in Cancer Research. [National Foundation for Cancer Research] Press Release

Dr. Julie Brahmer, MD, Named March LUNGevity Hero for Research in Groundbreaking Field of Immunotherapy
To kick off Women in History month, LUNGevity Foundation announced Dr. Julie Brahmer, MD, as the March LUNGevity Hero for her work in the field of immunotherapy for lung cancer patients. [LUNGevity Foundation (PR Newswire Association LLC)] Press Release | Blog Post

Ocata Therapeutics Receives Two New Patents for Immune-Modulatory Cell Technology
Ocata Therapeutics, Inc. announced that the United States Patent and Trademark Office issued two U.S. patents directed to the company’s hemangio-derived mesenchymal cells (HMCs). Ocata’s HMC technology introduces a new era of cell therapy for ophthalmic, autoimmune and inflammatory diseases. This development enables the generation of a potentially unlimited supply of potent HMCs from renewable pluripotent stem cell sources. [Ocata Therapeutics, Inc.] Press Release

Rowan Researcher Targets Stem Cell-Based Therapy for Rare Childhood Disease
Paola Leone, PhD, the director of the Cell and Gene Therapy Center and a professor of Cell Biology at the Rowan University School of Osteopathic Medicine, has been awarded a three-year, $477,000 grant from the National Institute of Neurological Disorders and Stroke to develop a stem cell-based therapy for Canavan disease, a rare but devastating neurological disorder in children that typically takes a child’s life by age 10. [Rowan University] Press Release

T-Cell Therapy Clinical Trial Now Offered to Cancer Patients at C.S. Mott Children’s Hospital
A clinical trial using T-cell therapy that uses the patients’ own immune cells to hunt down cancer cells is now being offered at the University of Michigan’s C.S. Mott Children’s Hospital. “For patients for whom we’ve exhausted all other options, this therapy has provided hope against a highly aggressive form of ALL, in situations where nothing else has been successful,” says John Levine, M.D., clinical director of the Pediatric Blood and Marrow Transplantation Program at C.S. Mott Children’s Hospital. [University of Michigan] Press Release

Aduro Biotech Collaborates with Leaders in Cancer Research on Investigator-Sponsored Phase II Trial Combining Aduro’s Innovative Immunotherapies with Anti-PD-1 Technology to Treat Patients with Advanced Pancreatic Cancer
Aduro Biotech, Inc. announced the initiation of an investigator-sponsored Phase II clinical trial of the company’s immuno-oncology product candidates GVAX Pancreas and CRS-207 in combination with Bristol-Myers Squibb’s Opdivo, a monoclonal antibody against programmed death-1 receptor. [Aduro Biotech, Inc.] Press Release

Arrowhead Begins Phase I Trial of ARC-AAT for Treatment of Liver Disease Associated with Alpha-1 Antitrypsin Deficiency
Arrowhead Research Corporation announced that it has initiated dosing in a Phase I clinical trial of ARC-AAT, the company’s candidate for the treatment of liver disease associated with alpha-1 antitrypsin deficiency, a rare genetic disorder that severely damages the liver and lungs of affected individuals. [Arrowhead Research Corporation] Press Release

U.S. Food and Drug Administration Accepts Supplemental Biologics License Application for Yervoy® (Ipilimumab) as Adjuvant Treatment for Patients with Stage 3 Melanoma Who Are at High Risk of Recurrence
Bristol-Myers Squibb Company announced that the U.S. Food and Drug Administration has accepted for filing and review the supplemental biologics license application for Yervoy for the adjuvant treatment of patients with stage 3 melanoma who are at high risk of recurrence following complete surgical resection. [Bristol-Myers Squibb Company] Press Release

Celimmune Founded to Develop Novel Therapeutics for Celiac and Other Serious Autoimmune Diseases
Celimmune LLC announced that it will initially focus its distinctive core competence in translational medicine, immunotherapy clinical development and commercialization on combating celiac disease. [Celimmune LLC (PR Newswire Association LLC)] Press Release

ABIVAX Recruits First Patient in a Pivotal Phase IIb/III Clinical Trial with ABX203, a Novel Immunotherapy against Chronic Hepatitis B
ABIVAX announced that it has dosed in New Zealand the first patient in a Phase IIb/III clinical trial of ABX203 which is taking place in several countries of the Asia-Pacific region. [ABIVAX] Press Release
Center for Scientific Review; Notice of Closed Meetings (FR Doc. No: 2015-03778)

Center for Scientific Review; Notice of Closed Meeting (FR Doc. No: 2015-03631)

Center for Scientific Review; Notice of Closed Meetings (FR Doc. No: 2015-03628)
NEW CAR-T CELLS Stem Memory versus Mature T Cells
March 18, 2015 11:00 AM PST

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Postdoctoral Position – Gene Therapy of Lysosomal Storage Diseases (Cincinnati Children’s Hospital Medical Center)

NEW Associate Director, Center for Gene Therapy (City of Hope)

Research Associate – Pluripotent Stem Cell Biology (STEMCELL Technologies Inc.)

Scientist – Reprogramming and Pluripotent Stem Cell Biology (STEMCELL Technologies Inc.)

Scientist – PSC Biology and Bioengineering (STEMCELL Technologies Inc.)

Scientist – Pluripotent Stem Cell Media Development, High Throughput Screening (STEMCELL Technologies Inc.)

Scientist – Cell Culture Support Products (STEMCELL Technologies Inc.)

Scientist – Liver Cell Biology (STEMCELL Technologies Inc.)

Assistant Professor – Neuroscience (Georgia State University)

Research Technician in AAV Gene Therapy (Icahn School of Medicine at Mount Sinai)

T Cell Process Development Scientist/Senior Scientist (Adaptimmune Ltd.)

Senior Process Development Engineer (Sangamo BioSciences, Inc.)

Director – Vector Production (Sangamo BioSciences, Inc.)

Assistant/Associate Professor – Gastroenterology (North Carolina State University College of Veterinary Medicine)

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