Cell Therapy News Volume 17.35 | Sep 26 2016

TOP STORY

Pretreatment with a γ-Secretase Inhibitor Prevents Tumor-Like Overgrowth in Human iPSC-Derived Transplants for Spinal Cord Injury

Because Notch signaling plays a role in maintaining neural stem/progenitor cells (NS/PCs), scientists evaluated the effects of γ-secretase inhibitor (GSI) and found that pretreating human induced pluripotent stem cell (hiPSC)-NS/PCs with GSI promoted neuronal differentiation and maturation in vitro, and GSI pretreatment also reduced the overgrowth of transplanted hiPSC-NS/PCs and inhibited the deterioration of motor function in vivo. [Stem Cell Reports] Full Article | Graphical Abstract

(Ranked by impact factor of the journal)

Tumor-Specific Gene Therapy for Pancreatic Cancer Using Human Neural Stem Cells Encoding Carboxylesterase

Scientists confirmed in vivo that in mice injected with BxPC3 on their skin, there was significant reduction of tumor size in those treated with both F3.CE and BxPC3 adjacent to the cancer mass. [Oncotarget] Abstract

Autologous Menstrual Blood-Derived Stromal Cells Transplantation for Severe Asherman’s Syndrome

In this experimental, non-controlled and prospective three-year clinical study involving seven patients with Asherman’s syndrome, autologous menstrual blood-derived stromal cells were isolated and cultured from menstrual blood of each patient within ~two weeks and then transplanted back into their uterus. [Hum Reprod] Abstract

Hyaluronic Acid-Laminin Hydrogels Increase Neural Stem Cell Transplant Retention and Migratory Response to SDF-1α

The authors investigated the capacity of the hyaluronic acid-laminin based hydrogel (HA-Lm gel) to promote neural progenitor/stem cell (NPSC) response to exogenous SDF-1α in vivo. They observed the HA-Lm gel to significantly increase NPSC transplant retention and migration in response to SDF-1α in a manner critically dependent on signaling via the SDF-1α-CXCR4 axis. [Matrix Biol] Abstract

Clinical-Grade Isolated Human Kidney Perivascular Stromal Cells as an Organotypic Cell Source for Kidney Regenerative Medicine

In vivo, in a neonatal kidney injection model, human kidney-derived perivascular stromal cells (hkPSCs) reintegrated and survived in the interstitial compartment, whereas bone marrow-derived mesenchymal stromal cells did not show this potential. Moreover, hkPSCs gave protection against the development of acute kidney injury in vivo in a model of rhabdomyolysis-mediated nephrotoxicity. [Stem Cells Transl Med] Abstract

Pathological Classification of Human iPSC-Derived Neural Stem/Progenitor Cells towards Safety Assessment of Transplantation Therapy for CNS Diseases

Researchers evaluated the histology of neural stem/progenitor cells generated from integration-free human peripheral blood mononuclear cell-derived induced pluripotent stem cells (iPSC-NSPCs) following transplantation into central nervous system (CNS) of immunodeficient mice. [Mol Brain] Abstract

Adult-Derived Human Liver Stem/Progenitor Cells Infused 3 Days Post-Surgery Improve Liver Regeneration in a Mouse Model of Extended Hepatectomy

Investigators examined the influence of delivering adult human liver mesenchymal stem/progenitor cells at two different early time points in an immunodeficient mouse model that had undergone a 70%-hepatectomy procedure. [Cell Transplant] Full Article

Development of an Optimized AAV2/5 Gene Therapy Vector for Leber Congenital Amaurosis Owing to Defects in RPE65

In animal models of RPE65 deficiency, subretinal injection of recombinant adeno-associated virus 2/2 vectors carrying RPE65 cDNA improves rod photoreceptor function, and intervention at an early stage of disease provides sustained benefit by protecting cone photoreceptors against retinal degeneration. [Gene Ther] Full Article

Total Body Irradiation Must Be Delivered at High Dose for Efficient Engraftment and Tolerance in a Rhesus Stem Cell Gene Therapy Model

Investigators evaluated both engraftment and tolerance for gene-modified cells using a rhesus hematopoietic stem cell gene therapy model following reduced intensity conditioning. [Mol Ther Methods Clin Dev] Full Article

Noninvasive In-Vivo Tracing and Imaging of Transplanted Stem Cells for Liver Regeneration

The authors provide a descriptive overview of imaging modalities and discuss recent advances in the field of molecular imaging of intrahepatic stem cell grafts. [Stem Cell Res Ther] Abstract

Hematopoietic Stem Cell Transplantation in Inborn Errors of Metabolism

This review summarizes the main results of hematopoietic stem cell transplantation in selected inborn errors of metabolism. [Curr Opin Hematol] Abstract

Hyaluronic Acid (HA) Scaffolds and Multipotent Stromal Cells (MSCs) in Regenerative Medicine

The authors discuss the use of hyaluronic acid-based scaffolds, together with multipotent stromal cells, as a very promising tool in regenerative medicine. [Stem Cell Rev] Abstract

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

Alnylam Reports Positive Initial Results from Ongoing Phase I/II Study of ALN-GO1, an Investigational RNAi Therapeutic for the Treatment of Primary Hyperoxaluria Type 1

Alnylam Pharmaceuticals, Inc. announced initial data from its ongoing Phase I/II study with ALN-GO1, an investigational RNAi therapeutic targeting glycolate oxidase for the treatment of Primary Hyperoxaluria Type 1. [Press release from Alnylam Pharmaceuticals, Inc. discussing research presented at the 17^th Congress of the International Pediatric Nephrology Association (IPNA), Iguaçu] Press Release

NW Bio Presents Updated Data From DCVax^®-Direct Phase I Trial

Northwest Biotherapeutics announced that Dr. Marnix Bosch, Chief Technical Officer, presented an updated and more detailed analysis of the Phase I DCVax^®-Direct Trial data. [Press release from Northwest Biotherapeutics discussing research presented at the 5^th Annual SMi Cancer Vaccines Conference, London] Press Release

LentiGlobin™ Investigational Gene Therapy for Transfusion-Dependent Beta-Thalassemia Accepted into European Medicines Agency’s PRIME Program

bluebird bio, Inc. announced that the European Medicines Agency has granted access to its Priority Medicines (PRIME) scheme for LentiGlobin drug product in the treatment of patients with transfusion-dependent beta-thalassemia. [bluebird bio, Inc.] Press Release

Kerastem Completes Enrollment in US Phase II Hair Growth Trial

Kerastem has completed enrollment of STYLE, the company’s US Phase II Clinical Trial. STYLE is Kerastem’s Phase II randomized, blinded, and controlled investigation of Kerastem therapy in early stage female and male pattern baldness. [Kerastem] Press Release

Dicerna Prioritizes Resources to Advance GalXC™ Product Candidates

Dicerna Pharmaceuticals, Inc. announced that the company will focus its resources on its proprietary GalXCTM technology platform to advance development of product candidates in its core therapeutic areas of rare diseases, chronic liver diseases, cardiovascular disease and viral infectious diseases. [Dicerna Pharmaceuticals, Inc.] Press Release

First Patient Enrolled in Toca 6, Tocagen’s Trial for Metastatic Cancers Evaluating Intravenously Delivered Toca 511 Virus

Tocagen, Inc. announced the first patient has been enrolled in Toca 6, a Phase 1b study evaluating intravenously delivered Toca 511 in combination with Toca FC in patients with metastatic cancers. [Tocagen, Inc.] Press Release

Regen BioPharma Responds to FDA Comments on Its Application for Orphan Drug Designation on Its Aplastic Anemia Therapy

Regen BioPharma Inc. has submitted additional data and other responses to the United States Food and Drug Administration (FDA) supporting its application requesting Orphan Drug status for the use of HemaXellerate in aplastic anemia. [Regen BioPharma Inc. (PR Newswire Association LLC.)] Press Release

Cellular Dynamics Announces Launch of iCell^® Hepatoblasts to Enable Research into Therapies that Stimulate Liver Regeneration

Cellular Dynamics International, Inc. announced the launch of iCell^® Hepatoblasts, liver progenitor cells generated from human induced pluripotent stem cells. [Cellular Dynamics International, Inc.] Press Release

Capricor Therapeutics Awarded $2.4 Million from U.S. Department of Defense to Support Manufacturing Process Development for Therapeutic Exosomes

Capricor Therapeutics, Inc. announced that the U.S. Department of Defense has awarded the company a grant in the amount of approximately $2.4 million to be used toward establishing a scalable, commercially-ready process to manufacture CAP-2003. [Capricor Therapeutics, Inc.] Press Release

Four Men and Two Women are Running for the Top Slot at the World Health Organization

The World Health Organization in Geneva, Switzerland, unveiled the names of the six candidates who are vying to succeed Margaret Chan as director-general. [Science Insider] Editorial

Worldwide Brain-Mapping Project Sparks Excitement — and Concern

In recent years, brain-mapping initiatives have been popping up around the world. They have different goals and areas of expertise, but now researchers will attempt to apply their collective knowledge in a global push to more fully understand the brain. [Nature News] Editorial

Titanic Clash over CRISPR Patents Turns Ugly

Much of the focus is on the teams centred at Berkeley and the Broad Institute, whose ‘foundational’ patents cover a wide swathe of CRISPR–Cas9 applications. Although Berkeley’s team filed for a patent first, the Broad opted for an expedited review process, and its patents were granted earlier. [Nature News] Editorial

Spain Joins Bandwagon for ‘Openness’ About Animal Research

The Spanish scientific community has pledged to become more transparent about animal research. Ninety research centers, universities, scientific societies, and companies around Spain have adopted a set of standards, launched by the Confederation of Spanish Scientific Societies, on how research organizations should open up communication channels about their use of laboratory animals. [Science Insider] Editorial

New Fellowship Aims to Increase Diversity in the Life Sciences

A new program aims to launch the careers of life scientists from underrepresented groups by providing up to 8 years of support, covering both the postdoctoral training and junior faculty stages. The Howard Hughes Medical Institute’s Hanna H. Gray Fellows Program will award up to 15 recipients with $60,000 of salary support and $20,000 in flexible funds paid to their institutions for each of up to 4 years of postdoc training. [Science Insider] Editorial

FDA

A List of Biomarkers Used as Outcomes in Development of FDA-Approved New Molecular Entities and New Biological Therapeutics (October 2007 to December 2015); Establishment of a Public Docket; Correction (FR Doc. No:2016-23106) Notice

NIH

Prospective Grant of Exclusive Patent License: Development of Adeno-Associated Virus-Based Vectors for the Treatment of Menkes Disease and Related Copper Transport Disorders (FR Doc. No:2016-23134) Notice

Prospective Grant of Start-Up Exclusive Evaluation Patent License: Development of Autologous Tumor-reactive T Cells Isolated From Peripheral Blood for the Treatment of Metastatic Follicular Thyroid Cancer and Metastatic Soft Tissue Sarcomas (FR Doc. No:2016-23048) Notice

Prospective Grant of Exclusive Patent License: Development of an Antibody-Drug Conjugate for Use in PhotoImmunoTherapy (FR Doc. No:2016-22820) Notice

NIH Policy on the Dissemination of NIH-Funded Clinical Trial Information (FR Doc. No:2016-22379) Notice

NEW Bone & Cartilage: From Development to Human Diseases
October 24-28, 2016
Suzhou, China

NEW Novel Concepts in the Biology and Treatment of Metastatic Cancer Conference
November 5-8, 2016
Pyphos, Cyprus

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

NEW Faculty Position – Stem Cell Biology and Regenerative Medicine (Stanford University)

NEW Research Associate – Hematology (Editas Medicine)

Faculty Position – Stem Cell Research (Children’s Research Institute at UTSW)

Assistant Professorship – Stem Cells and Regenerative Medicine (University of Southern California)

Postdoctoral Positions – Stem Cell Research, Hematopoiesis, Cancer, Genome Editing (Icahn School of Medicine)

Faculty Position – Cancer Research (Children’s Research Institute at UTSW)

Assistant Professor – Molecular and Cellular Biology (Harvard University)

Postdoctoral Fellow – Novel Systems for Treatment of Type 1 Diabetes (Toronto General Research Institute & McEwen Centre for Regenerative Medicine)

Group Leader – Stem Cells and Ageing (University of Queensland)

Assistant Professor – Developmental Biology (Florida State University)

Visiting Research Specialist – Physiology and Biophysics (University of Illinois at Chicago)

Research Associate – Cellular Therapy (California Institute for Biomedical Research)

Research Scientist – Downstream Bioprocess for Vaccine, Gene Therapy and Biosimilars (Univercells)

Postdoctoral Fellow – Tumor Immunology and Head and Neck Cancer (University of Lausanne)

Postdoctoral Fellow – Tumor Immunology and T Cell Differentiation (University of Lausanne)

Postdoctoral Fellow – Pluripotent Stem Cell Technology (University of California Davis)

Associate Member – Stem Cell and Gene Therapy (Fred Hutchinson Cancer Research Center)

Postdoctoral Fellow – Pathogenic Mechanisms of Rare Bone Disorders (University of Connecticut Health)

Postdoctoral Fellow – Oxidative Lung Injury (Temple University)

Postdoctoral Fellow – Lentivirus-Mediated FOXP3 Gene Transfer (Stanford University School of Medicine)

Postdoctoral Fellow – Hematology/Oncology (Boston Children’s Hospital)

Postdoctoral Research Fellow – Cell Cycle Control and Tumorigenesis (Fred Hutchinson Cancer Research Center)

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