Volume 15.16 | April 28

Issue 15.16 April 28, 2014
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TOP STORY
Bionic Ear Technology Used for Gene Therapy
Researchers used electrical pulses delivered from a cochlear implant to deliver gene therapy, thereby successfully regrowing auditory nerves. The research also heralds a possible new way of treating a range of neurological disorders, including Parkinson’s disease, and psychiatric conditions such as depression through this novel way of delivering gene therapy. [Press release from the University of New South Wales discussing online prepublication in Science Translational Medicine] Press Release | Abstract
Enter to Win a Travel Award to ISSCR 2014
 
PUBLICATIONS (Ranked by impact factor of the journal)
Enhancing Adult Nerve Regeneration through the Knockdown of Retinoblastoma Protein
Scientists describe a role for the retinoblastoma (Rb) protein, widely expressed in adult sensory neurons and their axons, during regeneration. In an in vivo regenerative paradigm following complete peripheral nerve trunk transection, direct delivery of Rb short interfering RNA prompts increased outgrowth of axons from proximal stumps and entrains Schwann cells to accompany them for greater distances. [Nat Commun] Abstract | Press Release

Crtc1 Activates a Transcriptional Program Deregulated at Early Alzheimer’s Disease-Related Stages
Adeno-associated viral-mediated cAMP-responsive element binding protein-regulated transcription coactivator-1 (Crtc1) overexpression in the hippocampus efficiently reverses Aβ-induced spatial learning and memory deficits by restoring a specific subset of Crtc1 target genes. [J Neurosci] Abstract | Press Release

Renal Telocytes Contribute to the Repair of Ischemically Injured Renal Tubules
Researchers investigated the paracrine effect of renal telocytes (TCs) on renal tubular epithelial cells in vitro, the regenerative function of renal TCs in renal tubules after ischemia-reperfusion injury in vivo and the possible mechanisms involved. [J Cell Mol Med] Full Article

In Situ Recruitment of Human BMSCs Using Chemokines for Articular Cartilage Regeneration
The chemotaxis of bone marrow-derived mesenchymal stem cells (BMSCs) toward chemokines, which may give rise to a complete regeneration of the articular cartilage, was investigated. CCR2, CCR4, CCR6, CXCR1, and CXCR2 were expressed in normal BMSCs and were increased significantly upon treatment with pro-inflammatory cytokines. [Cell Transplant] Abstract

Adenoviruses Using the Cancer Marker EphA2 as a Receptor In Vitro and In Vivo by Genetic Ligand Insertion into Different Capsid Scaffolds
Investigators established cell type-specific entry of HAdV-5-based vectors by genetic ligand insertion into a chimeric fiber with shaft and knob domains of the short HAdV-41 fiber. This fiber format was reported to ablate transduction in vitro and biodistribution to the liver in vivo. [PLoS One] Full Article

A Preliminary Evaluation of Efficacy and Safety of Wharton’s Jelly Mesenchymal Stem Cell Transplantation in Patients with Type 2 Diabetes Mellitus
No data are available on the efficacy and safety of allogeneic Wharton’s Jelly-derived mesenchymal stem cell (WJ-MSC) transplantation in patients with type 2 diabetes mellitus (T2DM). Investigators performed a non-placebo controlled prospective Phase I/II study to determine efficacy and safety of WJ-MSC transplantation in T2DM. [Stem Cell Res Ther] Abstract | Full Article

Long-Term Fate of Grafted Hippocampal Neural Progenitor Cells following Ischemic Injury
Researchers assessed the long-term survival, migration, differentiation, and functional outcome of neural progenitor cells transplanted into the ischemic murine brain. [J Neurosci Res] Abstract

Controlled Release of BMP-2 Using a Heparin-Conjugated Carrier System Reduces In Vivo Adipose Tissue Formation
Investigators evaluated the induction of mineralized/adipose tissue formation and the bone-healing pattern associated with the controlled release of E. coli-derived recombinant human bone morphogenetic protein-2 (BMP-2) by a heparin-conjugated fibrin system using ectopic and orthotopic in vivo models, respectively. [J Biomed Mater Res A] Abstract

Human Bone Marrow Mesenchymal Stem Cell-Derived Hepatocytes Improve the Mouse Liver after Acute Acetaminophen Intoxication by Preventing Progress of Injury
Mesenchymal stem cells from human bone marrow were differentiated into hepatocyte-like cells in vitro and transplanted into livers of immunodeficient Pfp/Rag2-/- mice treated with a sublethal dose of acetaminophen (APAP) to induce acute liver injury. APAP induced a time- and dose-dependent damage of perivenous areas of the liver lobule. [Int J Mol Sci] Abstract | Download Full Article

Webinar: Modeling Human Neurological Disease with iPS Cells
 
REVIEWS
Potential for Cell Therapy in Parkinson’s Disease Using Genetically-Programmed Human Embryonic Stem Cell-Derived Neural Progenitor Cells
The authors focus their review on current state-of-the-art techniques for harnessing human embryonic stem cells-based strategies towards development of a stem cell therapeutic for Parkinson’s disease. They also briefly describe a novel genetic-programming approach that may address many of the key challenges that remain in the field and that may hasten clinical translation. [J Comp Neurol] Abstract

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

Free Wallchart: Small Molecules, Big Impact in PSC Research
 
SCIENCE NEWS
Alnylam and Collaborators Present New Clinical Data for Patisiran, an RNAi Therapeutic Targeting Transthyretin (TTR) in Development for the Treatment of TTR-Mediated Amyloidosis (ATTR)
Alnylam Pharmaceuticals, Inc. and collaborators announced new clinical data for patisiran, an RNAi TTR in development for the treatment of ATTR. The company presented updated Phase II results in patients with familial amyloidotic polyneuropathy confirming robust TTR knockdown of up to 96% with a mean TTR knockdown of approximately 80%. [Press release from Alnylam Pharmaceuticals, Inc. discussing research presented at the International Symposium on Amyloidosis (ISA), Indianapolis] Press Release

Study Suggests Targeting B Cells May Help with MS
A new study suggests that targeting B cells may be associated with reduced disease activity for people with multiple sclerosis (MS). 231 people with relapsing-remitting MS received either a placebo or one of several low dosages of the drug ofatumumab, which is an anti-B cell antibody, for 24 weeks, with the first 12 weeks making up the placebo-controlled period. [Press release from American Academy of Neurology (AAN) discussing research presented at the AAN’s 66th Annual Meeting, Philadelphia] Press Release
 
POLICY
Investigator of Controversial Stem-Cell Study Resigns
The head of a Japanese committee investigating claims that stem cells could be made using mechanical stress or acid resigned from the committee over anonymous allegations that at least one of his own papers contained problematic data. He says he resigned out of concern that the incident could complicate the current investigation. [Nature News] Editorial

NSF’s Science Board Criticizes Bill to Alter Agency’s Programs
The presidentially appointed oversight body to the National Science Foundation (NSF) has taken the unprecedented step of publicly criticizing pending legislation that it feels would be harmful to the $7 billion research agency. [ScienceInsider] Editorial

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BUSINESS
Medigene Receives New US Patent for TCR-Modified T Cell Immunotherapy
Medigene AG announced that the US Patent Office has issued patent No. 8,697,854 “High affinity T cell receptor and use thereof”, relating to T-cell receptors (TCRs) against the tumor associated antigen tyrosinase. [Medigene AG] Press Release

Avalanche Biotechnologies Secures $55 Million in Oversubscribed Series B Financing
Avalanche Biotechnologies, Inc., a developer of gene therapies for serious eye diseases such as wet age-related macular degeneration, announced the successful completion of a $55 million Series B financing. [Avalanche Biotechnologies, Inc.] Press Release

Orbsen Therapeutics in €6 Million Research Funding Success
Orbsen Therapeutics is a spin-out from NUI Galway’s Regenerative Medicine Institute. MERLIN (MEsynchymal stem cells to Reduce Liver INflammation) will advance Orbsen’s proprietary cell therapy to a Phase IIa clinical trial in patients with inflammatory liver disease. [Orbsen Therapeutics] Press Release

Athersys Announces Results from Phase II Study of MultiStem® Cell Therapy for Ulcerative Colitis
Athersys, Inc. announced interim results from the Phase II clinical study of the administration of Athersys’ MultiStem® cell therapy to treatment refractory ulcerative colitis (UC) patients being conducted by Pfizer, Inc. The cell therapy failed to show meaningful benefit, following a single administration, in patients suffering from chronic, moderate-to-severe UC who have failed other therapies. [Athersys, Inc.] Press Release

FDA Approves Commencement of BrainStorm’s Phase II ALS Clinical Trial in U.S.
BrainStorm Cell Therapeutics announced that the US Food and Drug Administration (FDA) has approved commencement of its Phase II clinical trial with NurOwn™ in patients with Amyotrophic Lateral Sclerosis (ALS). [BrainStorm Cell Therapeutics, Inc.] Press Release

Bioheart Announces Clinical Study for Chronic Obstructive Pulmonary Disease
Bioheart, Inc. announced that it will enroll patients in a new study for chronic obstructive pulmonary disease (COPD). The study will enroll up to 100 patients to determine the safety and efficacy of adipose derived stem cells or AdipoCell™ in patients with COPD. [Bioheart, Inc.] Press Release

Pluristem’s Manufacturing Facility Approved by European Auditors for Phase III Trials
Pluristem Therapeutics Inc. announced that its new manufacturing facility has received the European Union’s Qualified Person Declaration. With this declaration, Pluristem is now approved to use cell therapies manufactured at its facility in all phases of its clinical trials conducted in the European Union, including Phase I, Phase II and Phase III. [Pluristem Therapeutics Inc.] Press Release

Advanced Cell Technology’s Chief Scientific Officer Dr. Robert Lanza Named One of TIME Magazine’s 100 Most Influential People in the World
Advanced Cell Technology, Inc. announced that the company’s Chief Scientific Officer, Robert Lanza, MD, has been selected for the 2014 TIME 100 annual list of the hundred most influential people in the world. [Advanced Cell Technology, Inc.] Press Release
 
NIH
Government-Owned Inventions; Availability for Licensing (FR Doc. No: 2014-09354)
 
REGULATORY
Food and Drug Administration (United States)

Expedited Access for Premarket Approval Medical Devices Intended for Unmet Medical Need for Life Threatening or Irreversibly Debilitating Disease or Conditions; Draft Guidance for Industry and Food and Drug Administration Staff; Availability (FR Doc. No: 2014-09189)
 
EVENTS
NEW International Society for Stem Cell Research (ISSCR) 12th Annual Meeting
June 18-21, 2014
Vancouver, Canada


Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
 
JOB OPPORTUNITIES
NEW Developmental-Stem Cell Biologist (Mayo Clinic – Rochester)

NEW Postdoctoral Position – Functional Characterization of Neurons Derived from Human Pluripotent Stem Cells (Radboud University Nijmegen)

Postdoctoral Fellowship – Hematology, Immunology, Cancer Therapeutics (Nationwide Children’s Hospital)

Business Development Executive (Research Instruments Ltd)

Postdoctoral Fellow – Therapeutic Corneal Endothelial Cell Culture (Lions VisionGift)

Postdoctoral Research Fellow / Research Officer – Stem Cell Research (Bioprocessing Technology Institute)

Stem Cell Research Technician (Van Andel Research Institute)

Postdoctoral Position – Human Stem Cell Biology and Hematopoiesis (Johns Hopkins University School of Medicine)

Director of GMP/GLP Quality Operations (University of Pennsylvania, Perelman School of Medicine)

Postdoctoral Position – Tissue Engineering and Regenerative Medicine (RUNMC)

Research Associate – Cell Separation (STEMCELL Technologies Inc.)

Research Technologist – Pluripotent Stem Cells (STEMCELL Technologies Inc.)

Research Technologist – Particle Chemistry (STEMCELL Technologies Inc.)

Research Technologist – PSC Biology and Bioengineering (STEMCELL Technologies Inc.)


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