Volume 15.05 | Feb 10

Issue 15.05 February 10, 2014
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Building the Tools that Fight Type 1 Diabetes
A cure for type 1 diabetes has long eluded even the top experts. Not because they do not know what must be done – but because the tools did not exist to do it. But now scientists, harnessing the power of regenerative medicine, have developed a technique in animal models that could replenish the very cells destroyed by the disease. [Press release from the Gladstone Institute discussing online prepublication in Cell Stem Cell]
Press Release | Full Article | Graphical Abstract
Directed Differentiation of Pluripotent Stem Cells - A Cell Stem Cell Poster
PUBLICATIONS (Ranked by impact factor of the journal)
Platelets Are Efficient and Protective Depots for Storage, Distribution, and Delivery of Lysosomal Enzyme in Mice with Hurler Syndrome
Using a murine mucopolysaccharidosis type I model, scientists demonstrated that megakaryocyte/platelets were capable of producing, packaging, and storing large amounts of α-L-iduronidase (IDUA) with proper catalytic activity, lysosomal trafficking, and receptor-mediated uptake. IDUA can be released directly into extracellular space or within microparticles during megakaryocyte maturation or platelet activation, while retaining the capacity for cross-correction in patient’s cells. [Proc Natl Acad Sci USA] Abstract | Press Release

Transplantation of Human Menstrual Blood Progenitor Cells Improves Hyperglycemia by Promoting Endogenous Progenitor Differentiation in Type 1 Diabetic Mice
Scientists studied therapeutic effect of menstrual blood progenitor cells (MBPCs) and mechanism of β cell regeneration after MBPCs transplantation in type 1 diabetes mellitus model. Intravenous injection of MBPCs can reverse hyperglycemia and weight loss, prolong lifespan, and increase insulin production in diabetic mice. [Stem Cell Dev] Abstract

Elimination of Contaminating Cap Genes in AAV Vector Virions Reduces Immune Responses and Improves Transgene Expression in a Canine Gene Therapy Model
Researchers investigated the effects of elimination of cap gene transfer and of vector purification by CsCl gradient centrifugation on AAV vector immunogenicity and expression following intramuscular injection in dogs. [Gene Ther] Abstract

Mesenchymal Stem Cells for Bronchopulmonary Dysplasia: Phase I Dose-Escalation Clinical Trial
In a Phase I dose-escalation trial, investigators assessed the safety and feasibility of a single, intratracheal transplantation of human umbilical cord blood-derived mesenchymal stem cells in preterm infants at high risk for bronchopulmonary dysplasia. [J Pediatr] Full Article | Press Release

Chitosan Scaffolds Containing Calcium Phosphate Salts and rhBMP-2: In Vitro and In Vivo Testing for Bone Tissue Regeneration
The authors immobilized calcium phosphate salts and bone morphogenetic protein 2 (BMP-2) – combined or alone – into chitosan scaffolds using ISISA process. They analyzed whether the immobilized bone morphogenetic protein preserved its osteoinductive capability after manufacturing process as well as BMP-2 in vitro release kinetic. They also studied both the in vitro and in vivo biocompatibility of the resulting scaffolds using a rabbit model. [PLoS One] Full Article

Mesenchymal Stem Cells Support Hepatocyte Function in Engineered Liver Grafts
Researchers describe enhanced hepatic function achieved using a strategy of sequential infusion of cells and illustrate the advantages of co-cultivating bone marrow-derived mesenchymal stem cells with primary hepatocytes in the engineered whole-liver scaffold. [Organogenesis] Full Article

Transplantation of Mesenchymal Cells Improves Peripheral Limb Ischemia in Diabetic Rats
Investigators tested whether transplantation of adipose tissue-derived mesenchymal stromal cells into the ischemic muscular tissue of diabetic animals would attenuate impaired cell metabolism and microcirculatory function. [Mol Biotechnol] Abstract

Transplantation of Photoreceptors Derived from Human Müller Glia Restore Rod Function in the P23H Rat
Scientists aimed to differentiate human Müller glia with stem cell characteristics into photoreceptor cells by stimulation with growth and differentiation factors in vitro to upregulate gene and protein expression of CRX, NR2E3, and rhodopsin and various phototransduction markers associated with rod photoreceptor development and function and to examine the effect of subretinal transplantation of these cells into the P23H rat, a model of primary photoreceptor degeneration. [Stem Cell Transl Med] Abstract | Press Release

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Culture and Characterize Human Mammary Epithelial Progenitors with Serum-Free EpiCult-B (Human) Free Protocols
Advances in Tissue Engineering through Stem Cell-Based Co-Culture
The advantages of stem cell co-culture strategies and their applications in tissue engineering and regenerative medicine are portrayed through specific examples for several tissues, including orthopedic soft tissues, bone, heart, vasculature, lung, kidney, liver and nerve. A concise review of the progress and the lessons learned are provided, with a focus on recent developments and their implications. [J Tissue Eng Regen Med] Abstract

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

New TeSR™-E8™ is Here For Feeder-Free Culture of Human ES Cells and iPS Cells
Targovax TG01 Immunotherapy Reaches Phase IIa in Operable Pancreatic Cancer
Targovax announced that the clinical trial with TG01 in operable pancreatic cancer now has reached Phase IIa. Promising Phase I-results triggered a 12.5 MNOK milestone, committed in 2012, from current owners. TG01, a RAS mutation-targeted medicine, is used in combination with chemotherapy. [Targovax] Press Release

Aduro BioTech Announces Initiation of Phase IIb ECLIPSE Trial to Evaluate Treatment of Patients with Pancreatic Cancer with the Company’s Innovative Immunotherapies
Aduro BioTech, Inc. announced the initiation of a Phase IIb clinical trial of the company’s immunotherapies GVAX Pancreas and CRS-207. The ECLIPSE trial (Efficacy of Combination Listeria/GVAX Immunotherapy in the Pancreatic Cancer Setting) will enroll approximately 240 adults with previously-treated metastatic pancreatic cancer and will involve over 20 clinical trial sites in the U.S. and Canada. [Business Wire] Press Release

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Judges Side with FDA on Stem Cells
The D.C. Circuit Court of Appeals ruled that culturing a patient’s stem cells for therapeutic use falls under the aegis of the US Food and Drug Administration (FDA). FDA has said therapeutic stem cells should be regulated as drugs. [The Scientist] Editorial | Court Ruling

Stem Cell Lines Not Fit for Clinic
Many of the stem cell lines used by academics and registered with the U.S. National Institutes of Health would not be eligible for commercialization because they don’t pass muster with the Food and Drug Administration, according to a report published in Cell Stem Cell. The lack of harmonization between the agencies’ standards throws up a potential roadblock on the path from the laboratory to the clinic. [The Scientist] Editorial | Report

UK Politicians Demand Action over Dearth of Female Scientists
An influential group of UK politicians has demanded that more be done to increase the number of women in scientific careers. The House of Commons Science and Technology Select Committee points the finger at universities. [Nature News] Editorial

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Merck Enters Strategic Collaborations with Amgen, Incyte and Pfizer to Evaluate Novel Combination Anti-Cancer Regimens with MK-3475
Merck announced it has signed three separate clinical collaboration agreements, through subsidiaries, with Amgen Inc., Incyte Corporation and Pfizer Inc. to evaluate novel combination regimens with MK-3475, Merck’s investigational anti-PD-1 immunotherapy. [Merck] Press Release

Advaxis Expands Collaboration with GRU Cancer Center at Georgia Regents University to Conduct Four Cancer Immunotherapy Clinical Trials
Advaxis, Inc., a biotechnology company developing the next generation of cancer immunotherapies, announced it has expanded its relationship by entering into a master clinical trial agreement with GRU Cancer Center to conduct four Phase I/II clinical trials. [Advaxis, Inc.] Press Release

Audentes Therapeutics and Genethon Announce Agreement to Develop Treatment for Severe Genetic Disease X-Linked Myotubular Myopathy
Audentes Therapeutics, Inc. and Genethon announced that they have entered into an agreement to develop AT001 for the treatment of X-Linked Myotubular Myopathy, a rare, inherited disorder characterized by severe muscle weakness and respiratory impairment. AT001 is a novel drug candidate based on adeno-associated virus gene therapy technology. [Audentes Therapeutics, Inc.] Press Release

JDRF to Provide Additional Support for Upcoming Clinical Trial of ViaCyte’s Encapsulated Cell Therapy for Type 1 Diabetes
JDRF and ViaCyte, Inc. jointly announced that JDRF is providing additional milestone-based funding for the continued development of ViaCyte’s VC-01™ encapsulated cell therapy product candidate for the treatment of type 1 diabetes. [ViaCyte, Inc.] Press Release

EBiSC – The First European Bank for Induced Pluripotent Stem Cells
Pharmaceutical companies who are members of the European Federation of Pharmaceutical Industries and Associations have joined forces with small and medium-sized enterprises and academia in an Innovative Medicines Initiative supported public private partnership project to establish the leading facility for the storage and distribution of induced pluripotent stem cells in Europe. [European Bank for Induced Pluripotent Stem Cells (EBiSC)]
Press Release

Grants Support Immunotherapy-Based Treatment for Childhood Cancer
Two Baylor College of Medicine researchers have received grants from the nonprofit organization Alex’s Lemonade Stand Foundation to advance their immunotherapy-based treatment approaches for childhood cancer from the bench to bedside. [Baylor College of Medicine] Press Release

iTeos Therapeutics SA Files Patent for Immunomodulators in Cancer Immunotherapy
iTeos Therapeutics SA announced the filing of the company’s first patent applications protecting proprietary inhibitors of the intracellular enzyme TDO2, which is expressed at high levels in specific cancers. [iTeos Therapeutics SA] Press Release

BrainStorm Receives US Notice of Allowance for Its Key Stem Cell Technology Patent
BrainStorm Cell Therapeutics announced that it has received a Notice of Allowance from the U.S. Patent Office for its “Mesenchymal Stem Cells for the Treatment of CNS Diseases” (serial number 12/994,761) patent application. [BrainStorm Cell Therapeutics] Press Release
Government-Owned Inventions; Availability for Licensing (FR Doc No. 2014-02490)

Government-Owned Inventions; Availability for Licensing (FR Doc No. 2014-02252)
Guidance Agenda: Guidance Documents CBER Is Planning to Publish During Calendar Year 2014
Food and Drug Administration (United States)

Science Board to the Food and Drug Administration: Request for Nominations (FR Doc No. 2014-02155)
NEW Annual Conference of the British Society for Gene and Cell Therapy (BSGCT)
March 28, 2014
London, United Kingdom

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Postdoctoral Fellow – Hemostasis Gene Therapy (Children’s Hospital of Philadelphia)

Postdoctoral Fellowships – Tissue Regeneration and Molecular Cell Engineering (MD Anderson Cancer Center)

Project Manager – Cell Therapy (University of California – Irvine)

Postdoctoral Position – Epigenetic Enzymes in Breast Cancer (University of Wisconsin – Madison)

Postdoctoral Position – Tissue Engineering and Regenerative Medicine (RUNMC)

Postdoctoral Fellow – Morphogenetic Signaling Pathways in Development and Disease (University of Rochester Medical Center)

Scientific Affairs Coordinator (American Society of Hematology)

Postdoctoral Research Fellow (Brigham & Women’s Hospital and Harvard Medical School)

Stem Cell Manufacturing Scientist (Paramount Recruitment)

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