Volume 14.27 | Jul 15

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TOP STORY
Gene Therapy Using Lentivirus Promising in Three Youngsters
Researchers developed a new gene therapy approach to treatment of Wiskott-Aldrich Syndrome, a fatal inherited form of immunodeficiency. They took the patients’ own blood stem cells and, in the laboratory, used the lentiviral vector combined with the normal WASP gene to correct the genetic defect in the blood. After a special treatment to eliminate their defective immune system, the patients received their own blood cells that had been altered to contain the normal WASP gene. [Press release from EurekAlert! discussing online prepublication in Science] Press Release | Abstract
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PUBLICATIONS (Ranked by impact factor of the journal)
Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy
The authors used a lentiviral vector to transfer a functional arylsulfatase A (ARSA) gene into hematopoietic stem cells (HSCs) from three presymptomatic patients who showed genetic, biochemical, and neurophysiological evidence of late infantile metachromatic leukodystrophy. After reinfusion of the gene-corrected HSCs, the patients showed extensive and stable ARSA gene replacement, which led to high enzyme expression throughout hematopoietic lineages and in cerebrospinal fluid. [Science] Abstract | Press Release

TCR-Ligand koff Rate Correlates with the Protective Capacity of Antigen-Specific CD8+ T Cells for Adoptive Transfer
Researchers developed a technology where dissociation (koff rate) of truly monomeric peptide-major histocompatibility complex molecules bound to surface-expressed T cell receptors (TCRs) can be monitored by real-time microscopy in a highly reliable manner. In two different murine infection models, they demonstrated that T cell populations with lower koff rates confer significantly better protection than populations with fast koff rates. [Sci Transl Med] Abstract | Press Release

Transient B Cell Depletion Combined with Apoptotic Donor Splenocytes Induces Xeno-Specific T and B Cell Tolerance to Islet Xenografts
Peritransplant infusion of apoptotic donor splenocytes cross-linked with ethylene carbodiimide (ECDI-SPs) has been demonstrated to effectively induce allogeneic donor-specific tolerance. Researchers aimed to determine the effectiveness and additional requirements for tolerance induction for xenogeneic islet transplantation using donor ECDI-SPs. In a rat-to-mouse xenogeneic islet transplant model, they showed that rat ECDI-SPs alone significantly prolonged islet xenograft survival, but failed to induce tolerance. [Diabetes]
Abstract | Press Release

Injectable Laminin-Functionalized Hydrogel for Nucleus Pulposus Regeneration
An injectable, laminin-111 functionalized poly(ethylene glycol) (PEG-LM111) hydrogel was developed as a biomaterial carrier for cell delivery to the intervertebral disc (IVD). Researchers evaluated the mechanical properties of the PEG-LM111 hydrogel, and its ability to retain delivered cells in the IVD space. [Biomaterials] Abstract

Iron Administration Before Stem Cell Harvest Enables MR Imaging Tracking after Transplantation
Investigators aimed to determine whether intravenous ferumoxytol can be used to effectively label mesenchymal stem cells in vivo and can be used for tracking of stem cell transplants. [Radiology]
Abstract | Press Release

Transplantation of Islet-Like Cell Clusters Derived from Human Dental Pulp Stem Cells Restores Normoglycemia in Diabetic Mice
Scientists compared the yield of generated islet-like cell clusters (ICCs) from stem cells from pulps of human exfoliated deciduous teeth (SHED) and dental pulp stem cells from permanent teeth. ICCs derived from SHED were packed in immuno-isolatory biocompatible macro-capsules and transplanted into streptozotocin-induced diabetic mice. Non-diabetic and diabetic controls were transplanted with macro-capsules with or without islets. [Cytotherapy] Abstract

Wnt/β-Catenin Signaling Triggers Neuron Reprogramming and Regeneration in the Mouse Retina
Researchers showed that upon activation of Wnt/β-catenin signaling, mouse retinal neurons can be transiently reprogrammed in vivo back to a precursor stage. This occurs after their spontaneous fusion with transplanted hematopoietic stem and progenitor cells. Moreover, the authors demonstrated that retinal damage is essential for cell-hybrid formation in vivo. Newly formed hybrids can proliferate, commit to differentiation toward a neuroectodermal lineage, and finally develop into terminally differentiated neurons. This results in partial regeneration of the damaged retinal tissue, with functional rescue. [Cell Rep]
Abstract | Press Release | Graphical Abstract

Heart Grafts Tolerized through Third-Party Multipotent Adult Progenitor Cells Can Be Retransplanted to Secondary Hosts with No Immunosuppression
Investigators report that stable allograft survival can be achieved following third-party multipotent adult progenitor cell (MAPC) infusion in a rat model of fully allogeneic, heterotopic heart transplantation. Furthermore, long-term accepted heart grafts recovered from MAPC-treated animals can be successfully retransplanted to naïve animals without additional immunosuppression. [Stem Cells Transl Med] Abstract | Press Release

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REVIEWS
Safety of Intra-Articular Cell-Therapy with Culture-Expanded Stem Cells in Humans: A Systematic Literature Review
The authors aimed to systematically review the literature and provide an overview of reported adverse events of intra-articular treatment with culture-expanded stem cells in humans. [Osteoarthritis Cartilage] Abstract

Cell Therapy, a Novel Remedy for Dilated Cardiomyopathy? A Systematic Review
In this systematic review researchers aimed to provide an overview of preclinical and clinical studies performed on cell therapy for dilated cardiomyopathy. [J Card Fail] Abstract

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

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SCIENCE NEWS
First In-Depth Study of Knee Cartilage Repair Technique Shows Improvement in Patient Outcomes and Regeneration of Knee Cartilage
A natural tissue graft can spur regeneration of cartilage and improve symptoms in patients who have cartilage damage in their knee. De Novo NT is an FDA approved cartilage repair technique. The product is made out of minced cartilage from organ donors under the age of 13. Unlike cartilage in adults, this young tissue has a high proliferative capacity. Cells from the transplanted cartilage escape or migrate into surrounding area tissues, multiply, and form a new cartilage tissue matrix that integrates with the surrounding host tissue. [Press release from Hospital for Special Surgery discussing research presented at the Annual Meeting of the American Orthopaedic Society for Sports Medicine, Chicago] Press Release

Alnylam Reports Positive Top-Line Results for ALN-TTRsc, a Subcutaneously Administered RNAi Therapeutic Targeting Transthyretin (TTR) for the Treatment of TTR-Mediated Amyloidosis
Alnylam Pharmaceuticals, Inc. announced positive top-line results from its ongoing Phase I trial of ALN-TTRsc, a subcutaneously administered RNAi therapeutic targeting the TTR gene for the treatment of TTR-mediated amyloidosis. The company is reporting that ALN-TTRsc achieved robust and statistically significant knockdown of serum TTR protein levels of greater than 80% in healthy volunteer subjects, in line with results for ALN-TTRsc previously reported in non-human primates. [Press release from Alnylam Pharmaceuticals, Inc. discussing research to be presented at the Annual Scientific Meeting of the Heart Failure Society of America, Orlando] Press Release

The European Cancer Congress 2013
 
POLICY
NSF Is on a Budget Roll
According to a final spending plan recently posted by the agency, the National Science Foundation (NSF)’s budget for the current fiscal year ending on 30 September is $149 million lower than in 2012. But that drop of 2.1%, to $6.884 billion, is less than half the 5% decline for the entire civilian government triggered by the sequestration mandated in the 2011 Budget Control Act. [National Science Foundation, United States]
Press Release

E.U. Commission Beefs Up Research Partnerships with Industry
The European Commission wants to double the amount of public money available for five research programs with industry, known as Joint Technology Initiatives. Together, they will receive €6.44 billion from the European Union budget between 2014 and 2020, up from €3.12 billion in 2007 to 2013, research commissioner Máire Geoghegan-Quinn announced. The deal is part of the European Union’s new Innovation Investment Package. [European Commission, European Union] Press Release

Outcry Over Plans for ‘Japanese NIH’
Many people admire the US National Institutes of Health (NIH) as a model of how biomedical research should be funded. Japanese Prime Minister Shinzo Abe has taken that admiration a step further than most, with a plan to copy the NIH’s structure. The plan, which came to light with the publication of two government strategies, one on economic growth and one on health care, would mimic the centralized control of the NIH by consolidating management of research money for a range of research institutes. But the plan also includes a goal to boost clinical applications, and many of the country’s life-sciences societies fear that the institute would not emulate the part of the NIH that they most admire: its commitment to basic research. [Prime Minister, Japan]
Press Release

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BUSINESS
NeoStem Advances Autoimmune Platform through UCSF Collaboration
NeoStem, Inc. announced that the Company has executed agreements with the University of California, San Francisco (UCSF) and the laboratories of Jeffrey Bluestone, PhD, and Qizhi Tang, PhD, to collaborate on the development of human regulatory T cells for the treatment of type 1 diabetes, steroid resistant asthma, and organ transplant rejection. [NeoStem, Inc.] Press Release

uniQure Signs EU Commercialization Agreement with Chiesi Farmaceutici for First Approved Gene Therapy Treatment, and Announces EUR 45 Million (USD 58 Million) in Equity and Collaboration Financing
uniQure B.V. announced it has signed collaboration agreements with Chiesi Farmaceutici SpA for the commercialization of Glybera, the first gene therapy to receive regulatory approval in Europe, as well as the co-development of a gene therapy for hemophilia B. [uniQure B.V.] Press Release

ViaCyte, Inc. Raises $10.6 Million in a Private Financing to Support Clinical Development of its Cell Therapy Product for Insulin Dependent Diabetes
ViaCyte, Inc. announced that it completed a private equity financing transaction, providing the Company $10.6 million through the sale of Series C-1 Preferred Stock, together with warrants to purchase stock. The Company will use the funds to pursue clinical development of VC-01. VC-01 is a development-stage product that consists of pancreatic precursor cells (designated PEC-01) manufactured through directed differentiation of stem cells sourced from a proprietary human embryonic stem cell line, and encapsulated in a proprietary, immune protective medical device (the ENCAPTRA® device). [ViaCyte, Inc.] Press Release

MCW Researcher to Study Main Complication after Bone Marrow Transplantation
The Medical College of Wisconsin (MCW) has received a four year, $1.5 million grant from the National Institute of Health’s National Heart, Lung and Blood Institute to study graft versus host disease (GvHD), the major complication associated with bone marrow transplants. In this study, Dr. Drobyski will define the role of specific regulatory T cell populations in GvHD, in order to develop strategies that will help patients better tolerate transplantation. [Medical College of Wisconsin] Press Release

Calimmune Initiates HIV Stem Cell Study at Two California Research Sites
Calimmune announced that the first patient has begun treatment in a Phase I/II clinical trial designed to determine whether a pioneering genetic medicine approach can help to protect individuals infected with HIV from the effects of the virus. The study, “Safety Study of a Dual Anti-HIV Gene Transfer Construct to Treat HIV-1 Infection,” utilizes a gene medicine called Cal-1, developed in the lab of Nobel Laureate Dr. David Baltimore and by Calimmune. [Calimmune] Press Release

Bellicum Pharmaceuticals Initiates Clinical Trial with Novel Controlled Dendritic Cell Immunotherapy in Metastatic Castrate Resistant Prostate Cancer
Bellicum Pharmaceuticals, Inc. announced it has initiated treatment of the first patient in a study of BPX-201 dendritic cells in combination with activating agent AP1903. The Phase I dose escalation trial in patients with metastatic castrate resistant prostate cancer was designed to evaluate the safety, tolerability and clinical outcomes of BPX-201 and AP1903, which incorporate the Company’s second generation DeCIDe™ immunotherapy technology. [Bellicum Pharmaceuticals, Inc.] Press Release

Pluristem’s South Korean Partner Files IND with Korean FDA for PLX Cells
On the heels of announcing its strategic partnership agreement with Cha Bio&Diostech, Pluristem Therapeutics Inc. announced that Cha Bio&Diostech “Cha” has filed its first investigational new drug (IND) application for Pluristem’s PLacental eXpanded (PLX) cells with the Korean Food and Drug Administration (KFDA). This IND is for the use of PLX-PAD in the treatment of intermittent claudication (IC) and mirrors the clinical protocol implemented by Pluristem in its FDA supervised Phase II IC clinical trial in the United States. [Pluristem Therapeutics Inc.] Press Release

SpinalCyte, LLC Receives Japanese Patent for Spinal Disc Tissue Engineering
SpinalCyte, LLC announced the Japan Patent Office issued a Certificate Of Patent on its patent application “Methods and Compositions For Repair Of Cartilage Using An In Vivo Bioreactor” to regrow the spinal disc, using human dermal fibroblasts. The allowed claims include the use of human dermal fibroblasts from the patient’s own body, to regrow the nucleus of the spinal disc in vivo. [SpinalCyte, LLC] Press Release

CFDRC Developing Non-Invasive Stem Cell Analyzer Technology
CFD Research Corporation (CFDRC) has been awarded a two-year $1 million contract from the DoD’s Defense Health Program to continue their research into the detection and sorting of stem cells according to their differentiation states. [CFD Research Corporation] Press Release

American CryoStem Granted IRB Approval for Adipose Derived Stem Cell Processing Protocols
American CryoStem Corporation announced that its protocols for the processing of Stromal Vascular Fraction and culturing of mesenchymal stem cells from autologous adipose tissue have been approved by the Institutional Review Board (IRB) of the International Cell Surgical Society. [American CryoStem Corporation] Press Release  
 
NIH
Senate Spending Panel Approves $31 Billion for NIH in 2014, Restoring Sequester Cuts
A Senate spending panel approved a bill giving the National Institutes of Health (NIH) $31 billion in 2014, a figure that would restore this year’s painful 5% cut from sequestration and give the agency a small increase over its 2012 budget. Press Release

Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2013-16337)

National Cancer Institute; Notice of Closed Meeting (FR Doc No: 2013-16338)  
 
CBER
HPC, Cord Blood BLA 125432  
 
REGULATORY
Food and Drug Administration (United States)

Agency Information Collection Activities; Proposed Collection; Comment Request; Human Cells, Tissues, and Cellular and Tissue-Based Products: Establishment Registration and Listing; Form FDA 3356; Eligibility Determination for Donors; and Current Good Tissue Practice (FR Doc No: 2013-16692)

Agency Information Collection Activities; Proposed Collection; Comment Request; Human Tissue Intended for Transplantation (FR Doc No: 2013-16556)  
 
EVENTS
NEW The Jackson Laboratory: Beyond Leptin – Emerging Mouse Models of Type 2 Diabetes
July 18, 1:00PM (ET in US)
Webinar


NEW Stem Cell Society Singapore (SCSS) Symposium 2013
November 18-19, 2013
Singapore, Singapore


Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
 
JOB OPPORTUNITIES
NEW Postdoctoral Position – Stem Cell Therapy and Epigenetics of Retina (University of Pittsburgh, Ophthalmology)

Postdoctoral Fellow – Neural Tissue Engineering (School of Chemical & Biomedical Engineering, Nanyang Technological University)

Postdoctoral Fellowship – Immunology (Johns Hopkins University School of Medicine)

PhD Positions – Tissue Engineering (8 European Research Institutes)

Postdoctoral Fellowship – Organic Synthesis and Medicinal Chemistry (Masaryk University)

Research Scientist/Senior Research Officer – Monoclonal Antibodies (Bioprocessing Technology Institute/A*STAR)

Postdoctoral Fellow/Research Scientist – Tissue Engineering (Institute of Bioengineering and Nanotechnology)

Director of Cell Processing Facility (S L Collins Associates, Inc.)

Senior Postdoctoral Research Position – Molecular Cardiovascular and Renal Research (University of Oklahoma Health Sciences Center)

Postdoctoral Researcher – Hematopoietic Stem Cells (Albert Einstein College of Medicine)


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