Volume 15.40 | Oct 13

Issue 15.40 October 13, 2014
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From Stem Cells to Billions of Human Insulin-Producing Cells
With human embryonic stem cells as a starting point, scientists were for the first time able to produce, in the kind of massive quantities needed for cell transplantation and pharmaceutical purposes, human insulin-producing beta cells equivalent in most every way to normally functioning beta cells. The stem cell-derived beta cells are presently undergoing trials in animal models, including non-human primates. [Press release from Harvard Stem Cell Institute discussing publication in Cell] Press Release | Abstract | Graphical Abstract | Video
View Data: UM729, a Novel Small Molecule for Ex Vivo Expansion of Human hSCs
PUBLICATIONS (Ranked by impact factor of the journal)
A Modified γ-Retrovirus Vector for X-Linked Severe Combined Immunodeficiency
Scientists enrolled nine boys with X-linked severe combined immunodeficiency in parallel trials in Europe and the United States to evaluate treatment with a self-inactivating (SIN) γ-retrovirus vector containing deletions in viral enhancer sequences expressing γc (SIN-γc). All patients received bone marrow-derived CD34+ cells transduced with the SIN-γc vector, without preparative conditioning. [N Engl J Med] Abstract | Press Release

The Ion Channel TRPV1 Regulates the Activation and Proinflammatory Properties of CD4+ T Cells
Scientists found that TRPV1 was functionally expressed in CD4+ T cells, where it acted as a non-store-operated Ca2+ channel and contributed to T cell antigen receptor (TCR)-induced Ca2+ influx, TCR signaling and T cell activation. In models of T cell-mediated colitis, TRPV1 promoted colitogenic T cell responses and intestinal inflammation. [Nat Immunol] Abstract | Press Release

Engrafted Human Stem Cell-Derived Hepatocytes Establish an Infectious HCV Murine Model
Hepatocyte-like cells (HLCs) differentiated from human embryonic stem cells and patient-derived induced pluripotent stem cells could be engrafted in the liver parenchyma of immune-deficient transgenic mice carrying the urokinase-type plasminogen activator gene driven by the major urinary protein promoter. The HLCs were maintained for more than three months in the livers of chimeric mice, in which they underwent further maturation and proliferation. [J Clin Invest] Full Article

Conversion of Th17 into IL-17Aneg Regulatory T Cells: A Novel Mechanism in Prolonged Allograft Survival Promoted by Mesenchymal Stem Cell-Supported Minimized Immunosuppressive Therapy
Researchers found that when mesenchymal stem cells were used to induce long-term acceptance of allogeneic heart grafts in mice, the induction of regulatory T cells was preceded by development of a CD11bhiGr1int myeloid-derived immunosuppressive cell-mediated Th17 response. [J Immunol] Abstract

Promoting the Recovery of Injured Liver with PHBVHHx Scaffolds Loaded with Umbilical Cord-Derived Mesenchymal Stem Cells
PHBVHHx scaffolds loaded with mesenchymal stem cells were isolated from umbilical cord were transplanted into liver-injured mice. Liver morphology improved after 30 days of transplantation and looked similar to normal liver. [Tissue Eng Part A] Abstract

Clinical Experience with Autologous M2-Macrophages in Children with Severe Cerebral Palsy
To evaluate the safety and efficacy of autologous M2-macrophages scientists conducted a pilot clinical trial in 21 children with severe cerebral palsy. Intradural injection of M2-cells into the lumbar spinal area did not induce any serious adverse events. [Cell Transplant] Full Article

Human Adenovirus-Specific γ/δ and CD8+ T Cells Generated by T-Cell Receptor Transfection to Treat Adenovirus Infection after Allogeneic Stem Cell Transplantation
Since allo-reactive endogenous T-cell receptor (TCR) of donor T lymphocytes would induce graft-versus-host disease in a mismatched patient, scientists transferred the TCR into γ/δ T cells, which are not allo-reactive. In direct comparison with TCR-transfected α/β T cells, TCR-CD8α-co-transfected γ/δ T cells produced more tumor necrosis factor, and lysed peptide-loaded target cells as efficiently. [PLoS One] Full Article

GvHD Prophylaxis with Sirolimus-Tacrolimus May Overcome the Deleterious Effect on Survival of HLA Mismatch after Reduced-Intensity Conditioning Allo-SCT
The authors indicate favorable outcomes after allogeneic hematopoietic SCT using the combination of sirolimus/tacrolimus combination as GvHD prophylaxis with OS in the range of 55-70%, and non-significant differences in overall outcomes, irrespective of the presence of any mismatches at obligatory loci. [Bone Marrow Transplant] Abstract

Adoptive Therapy with Donor Lymphocyte Infusion after Allogenic Hematopoietic SCT in Pediatric Patients
Researchers analyzed the experience of Polish Pediatric Group for hematopoietic stem cell transplantation in respect to donor lymphocyte infusion procedure. [Bone Marrow Transplant] Abstract

Radiosensitization of Gliomas by Intracellular Generation of 5-Fluorouracil Potentiates Prodrug Activator Gene Therapy with a Retroviral Replicating Vector
Researchers investigated whether, in addition to its direct cytotoxic effects, 5-fluorouracil generated intracellularly by retroviral replicating vector-mediated cytosine deaminase/5-fluorocytosine prodrug activator gene therapy could also act as a radiosensitizing agent. [Cancer Gene Ther] Abstract

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Translational Research in Oncology – 10 Years of Progress and Future Prospects
The authors review selected developments in translational cancer biology, diagnostics, and therapeutics that have occurred over the past decade and offer thoughts on future prospects for the next few years. [Nat Rev Clin Oncol] Abstract

Stem Cell Transplantation for Primary Immunodeficiencies: The European Experience
The authors report on the most recent hematopoietic stem cell transplantation European results and suggest some opportunities for better treatment of certain primary immunodeficiencies (PIDs). Progress on gene therapy is also discussed, as it emerges as an interesting option for PIDs management. [Curr Opin Allergy Clin Immunol] Abstract

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

25% Off First Order: MNCs, CD34+ cells and more! Expires October 31st, 2014
Intensity Therapeutics, Inc.’s Research Selected for Presentation
Intensity Therapeutics, Inc., a privately-held biotechnology company developing proprietary cancer immunotherapy products, announced that preclinical data using the company’s novel drug product, INT230-6, was selected for a poster presentation. [Press release from Intensity Therapeutics, Inc. discussing research presented at the National Cancer Institute’s annual Cancer Immunology and Immunotherapy Meeting, Bethesda] Press Release | Poster

Genocea Presents Complete Phase I/IIa Data for HSV-2 Immunotherapy
Genocea Biosciences, Inc. announced the presentation of final data from the Phase I/IIa study of GEN-003, the company’s immunotherapy candidate for treatment of genital herpes. [Press release from Genocea Biosciences, Inc. discussing research presented at IDWeek 2014™, Philadelphia] Press Release

ViaCyte, Inc. to Present at Upcoming Stem Cell Meeting on the Mesa Conference
ViaCyte, Inc., a privately-held regenerative medicine company developing a stem cell-derived islet replacement therapy for the treatment of diabetes, called VC-01™, will give two presentations. [Press release from ViaCyte, Inc. discussing research presented at Stem Cell Meeting on the Mesa Conference, La Jolla] Press Release

Global Controls in Stem Cells
European Scientists Ask Governments to Boost Basic Research
The economies of Germany and Greece may have little in common. But scientists in those two countries, and across Europe, believe that their national science systems are facing similar assaults from what they view as wrong-headed government policies. [ScienceInsider] Editorial

NIH Makes $32 Million in Awards to Mine Big Data
Hoping to tame the torrent of data churning out of biology labs, the National Institutes of Health (NIH) announced $32 million in awards in 2014 to help researchers develop ways to analyze and use large biological data sets. [ScienceInsider] Editorial

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Bristol-Myers Squibb, Pharmacyclics and Janssen Announce Clinical Collaboration to Evaluate OPDIVO® (nivolumab) and IMBRUVICA® (ibrutinib) in Non-Hodgkin Lymphoma
Bristol-Myers Squibb Company, Pharmacyclics, Inc., and Janssen Research & Development, LLC announced they have entered into a clinical trial collaboration agreement to evaluate the safety, tolerability and preliminary efficacy of Bristol-Myers Squibb’s investigational PD-1 immune checkpoint inhibitor OPDIVO® in combination with IMBRUVICA®, an oral Bruton’s tyrosine kinase inhibitor co-developed and co-marketed by Pharmacyclics and Janssen. [Bristol-Myers Squibb Company] Press Release

Veristat and Molecular Templates Inc. Enter into Preferred Provider Agreement for Refractory Non-Hodgkin’s B-Cell Lymphoma Trial
Veristat announced that Molecular Templates has entered into a Preferred Provider Agreement with Veristat. Veristat will provide a broad spectrum of clinical support given their depth of experience in oncology and work with novel therapeutics. [Veristat] Press Release

Kadimastem Received a Quick Response from the FDA: The Development Process for the Stem Cell-Based Drug for ALS Is Expected to Be Significantly Shortened
Kadimastem announced that only one day after approaching the FDA regarding the cellular treatment that it is developing for ALS, an encouraging response was received. Based on the quality of the data presented, the FDA recommends rapidly moving forward in the approval process towards product development and safety towards clinical trials. [Kadimastem] Press Release

ENZO Biochem Awarded Patent for Novel Vectors Involving Gene Therapy Treatments for Cancer, Virus Infections & Genetic Diseases
Enzo Biochem, Inc. announced that the U.S. Patent and Trademark Office has awarded the company Patent No. 8,846,893, covering novel vectors for gene therapy treatments for cancers, virus infections and genetic diseases. [ENZO Biochem, Inc.] Press Release

BioTime’s Subsidiary Cell Cure Neurosciences Ltd. Files an IND with the FDA for OpRegen® Designed to Treat Patients with Dry-AMD
BioTime, Inc., HBL Hadasit Bio-Holdings Ltd. and Cell Cure Neurosciences Ltd. (Cell Cure) announced that Cell Cure has filed an investigational new drug (IND) application with the United States Food and Drug Administration (FDA) seeking to initiate a Phase I/IIa clinical trial of OpRegen® in patients with geographic atrophy, the severe stage of the dry form of age-related macular degeneration (dry-AMD). [Cell Cure Neurosciences Ltd.]
Press Release

BrainStorm Reports Last Patient Visit in Phase IIa ALS Study
BrainStorm Cell Therapeutics announced that the last patient has completed the last visit in its Phase IIa clinical trial of NurOwn™ in Amyotrophic Lateral Sclerosis (ALS), conducted at the Hadassah Medical Center in Jerusalem. [BrainStorm Cell Therapeutics] Press Release

FDA Grants Fast Track Designation to NurOwn™ for the Treatment of ALS
BrainStorm Cell Therapeutics Inc. announced that the United States Food and Drug Administration (FDA) has designated NurOwn™ as a Fast Track product for the treatment of amyotrophic lateral sclerosis (ALS). [BrainStorm Cell Therapeutics Inc. (PR Newswire Association LLC)] Press Release

Pluristem Wins Cell Therapy Patent Case in Europe; European Patent Office Confirms Validity of Amended Claims
Pluristem Therapeutics Inc., a developer of placenta-based cell therapy products, announced that its European Patent No. EP2200622, titled, “ADHERENT CELLS FROM ADIPOSE OR PLACENTA TISSUES AND USE THEREOF IN THERAPY”, was upheld by the European Patent Office in amended form, affirming its validity. [Pluristem Therapeutics Inc.] Press Release

Agilis Announces Appointment of Dr. Paola Leone to Scientific Advisory Board
Agilis Biotherapeutics, LLC, a synthetic biology-based company focused on developing DNA-based therapeutics for rare genetic diseases, announced the addition of Professor Paola Leone, PhD, Director of the Cell and Gene Therapy Center, Department of Cell Biology, Rowan University, School of Osteopathic Medicine, to its scientific advisory board. [Agilis Biotherapeutics, LLC] Press Release
Center for Scientific Review Notice of Closed Meetings (FR Doc. No: 2014-24059)
Food and Drug Administration (United States)

Flow Cytometric Devices; Draft Guidance for Industry and Food and Drug Administration Staff; Availability (FR Doc. No: 2014-24308)
NEW CIRM Showcase: Accelerating Stem Cell Treatments to Patients
November 5, 2014
San Francisco, United States

NEW 5th International Conference on Stem Cells and Cancer (ICSCC-2014): Proliferation, Differentiation and Apoptosis
November 8-10, 2014
New Delhi, India

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Quality Improvement Coordinator – Regenerative Medicine (The University of Texas Health Science Center at Houston)

Postdoctoral Position – Leukemic Stem Cells (BC Cancer Agency)

Postdoctoral Position – Hepatic Regenerative Medicine (INSERM)

Postdoctoral Fellowship – AAV Gene Therapy (Icahn School of Medicine at Mount Sinai)

Postdoctoral Position – Novel Mediators of Cardiovascular Disease and Novel Therapeutic Strategies (Monzino Cardiology Center)

Postdoctoral Fellow – Cardiac Differentiation of Pluripotent Stem Cells (Stanford University)

Postdoctoral Fellow – Cardiac Animal Surgery (Stanford University)

Postdoctoral Position – Transplantation Aspects of Hepatocytes Derived from Human Pluripotent Stem Cells (INSERM UMR 1064)

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.)

Scientist – Immunology/Cell Separation (STEMCELL Technologies Inc.)

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