Cell Therapy News Volume 14.18 | May 13 2013

Megakaryocytes Promote Murine Osteoblastic HSC Niche Expansion and Stem Cell Engraftment after Radioablative Conditioning
Scientists demonstrated that host megakaryocytes function critically in expansion of the endosteal niche after preparative radioablation and in the engraftment of donor hematopoietic stem cells (HSCs). [Blood] Abstract

Engineering Bone Tissue Substitutes from Human Induced Pluripotent Stem Cells
Researchers induced three human-induced pluripotent stem cell (hiPSC) lines with different tissue and reprogramming backgrounds into the mesenchymal lineages and used a combination of differentiation assays, surface antigen profiling, and global gene expression analysis to identify the lines exhibiting strong osteogenic differentiation potential. They then engineered functional bone substitutes by culturing hiPSC-derived mesenchymal progenitors on osteoconductive scaffolds in perfusion bioreactors and confirmed their phenotype stability in a subcutaneous implantation model for 12 weeks. [Proc Natl Acad Sci USA] Abstract | Press Release

IL-15 in Tumor Microenvironment Causes Rejection of Large Established Tumors by T Cells in a Noncognate T Cell Receptor-Dependent Manner
Investigators report that T cells eradicated two-week-old solid tumors that expressed IL-15, eliminating antigen-negative cells. In contrast, control tumors that lacked IL-15 expression consistently relapsed. Interestingly, even tumors lacking expression of cognate antigen were rejected when expressing IL-15, indicating that rejection after adoptive T-cell transfer was independent of cognate antigen expression. [Proc Natl Acad Sci USA] Abstract

Intracoronary Infusion of Allogeneic Mesenchymal Precursor Cells Directly following Experimental Acute Myocardial Infarction Reduces Infarct Size, Abrogates Adverse Remodeling and Improves Cardiac Function
The authors assessed the safety, feasibility and efficacy of intracoronary delivery of allogeneic mesenchymal precursor cells directly following acute myocardial infarction in sheep. [Circ Res] Abstract

Improved Cell Therapy Protocol for Parkinson’s Disease Based on Differentiation Efficiency and Safety of Hesc-, Hipsc and Non-Human Primate Ipsc-Derived DA Neurons
Researchers aimed to improve the safety of human- and non-human primate-iPSC (PiPSC)-derived dopaminergic (DA) neurons. PiPSC-derived NCAM⁺/CD29^low DA-neurons were able to restore motor function of 6-OHDA lesioned rats 16 weeks after transplantation. [Stem Cells] Abstract

Tissue-Engineered Cardiac Patch for Advanced Functional Maturation of Human ESC-Derived Cardiomyocytes
Scientists investigated the effects of 3-dimensional tissue-engineered culture environment and cardiomyocyte purity on structural and functional maturation of human embryonic stem cell (ESC)-derived cardiomyocytes. [Biomaterials] Abstract | Press Release

Generation of a Hypomorphic Model of Propionic Acidemia Amenable to Gene Therapy Testing
Researchers generated an adult hypomorphic model of Propionic acidemia in Pcca⁻ mice using a transgene bearing an A138T mutant of the human PCCA protein. This adult model allowed gene therapy testing by intravenous injection with adenovirus serotype 5 and adeno-associated virus 2/8 vectors. [Mol Ther] Abstract

Novel Adenoviral Vector Induces T-Cell Responses Despite Anti-Adenoviral Neutralizing Antibodies in Colorectal Cancer Patients
In this phase I/II study, cohorts of patients with advanced colorectal cancer were immunized with escalating doses of E1-deleted adenovirus subtype 5 (Ad5) [E1-, E2b-]-carcinoembryonic antigen (CEA). CEA-specific cell-mediated immune responses were observed despite the presence of preexisting Ad5 immunity in a majority of patients. [Cancer Immunol Immunother] Abstract | Press Release

Cell-Based Immunotherapy against Gliomas: From Bench to Bedside
Cellular immunotherapy is currently being explored as therapeutic approach to treat malignant brain tumors. This review discusses advances in active, passive, and vaccine-based immunotherapeutic strategies for gliomas both at the bench and in the clinic. [Mol Ther] Abstract

Cellular Immunotherapy for Plasma Cell Myeloma
Allogeneic hematopoietic cell transplantation for plasma cell myeloma can lead to graft-vs-myeloma immunity and long-term survivorship, but limited efficacy and associated toxicities have prevented its widespread use. Cellular immunotherapies seek to induce more specific, reliable and potent antimyeloma immune responses with less treatment-related risk than is possible with allogeneic transplantation. [Bone Marrow Transplant] Abstract

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

Preclinical Research Shows PLX Cells May Be Effective in Treating Preeclampsia
Pluristem Therapeutics Inc. announced its PLacental eXpanded (PLX) cells tested in preclinical animal models of preeclampsia effectively improved several parameters of the disease. [Press release from Pluristem Therapeutics Inc. discussing research to be presented at the Society for Gynecological Investigation Summit, Jerusalem] Press Release

Groundbreaking Corneal Transplant with Pre-Loaded Donor Tissue Performed at Massachusetts Eye and Ear
The first successful cornea transplant with donor endothelial tissue preloaded by an eye bank has been performed at Massachusetts Eye and Ear in Boston, Mass. Roberto Pineda II, M.D. recently performed the groundbreaking transplant. [Massachusetts Eye and Ear] Press Release

Pressure Builds on Congress to Kill NSF Bill
Several former top officials at the National Science Foundation (NSF) and the chairs of its oversight body yesterday wrote to Representative Lamar Smith (R-TX) urging him to withdraw a bill proposing changes to grant-making at the agency. That letter is signed by three previous NSF directors and three past chairs of the National Science Board, NSF’s presidentially appointed oversight body. The second letter, from 18 scientists who once headed individual research directorates at the agency, also argues that many of NSF’s most spectacular successes would not have qualified for funding under the terms of the legislation. [National Science Foundation, United States] Press Release

Canada to Convert NRC into ‘Toolbox’ for Industry
After two years of flogging the need to transform Canada’s National Research Council (NRC) into a toolbox for industry, the Conservative government announced that the 97-year-old agency is “open for business” under its new philosophy. Under the new policies, NRC scientists will tackle questions raised by individual businesses, while more basic research will be shuffled off to other departments or abandoned altogether. [National Research Council, Canada] Press Release

Russian Scientists Decry New International Funding Rules
Russian researchers are up in arms over a government decree issued which turns the process of issuing research grants into a bureaucratic nightmare for international foundations. The decree introduces new regulations according to which any organization that wants to award grants to Russian researchers must obtain permission from the Ministry of Education and Science for every grant. [Ministry of Education and Science, Russia]
Press Release

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NeoStem’s Subsidiary, Progenitor Cell Therapy, to Provide Autologous and Allogeneic Cellular Therapy Services to MedStar Georgetown University Hospital
NeoStem, Inc. and its subsidiary, Progenitor Cell Therapy, LLC (PCT) announced that PCT has entered into a Cell Therapy Processing Customer Agreement with MedStar Georgetown University Hospital to provide autologous and allogeneic cellular therapy services for cellular products provided by MedStar Georgetown University Hospital (MGUH) and deliver resulting cellular therapy products to MGUH for patient care. [NeoStem, Inc.]
Press Release

A*STAR’s Genome Institute of Singapore Partners Oxford Biodynamics to Discover and Monitor Cells for Pathologies or Abnormalities
The Genome Institute of Singapore from the Agency for Science, Technology and Research (A*STAR) and Oxford Biodynamics (OBD) will collaborate to identify epigenetic signatures utilizing OBD’s proprietary EpiSwitch^TM biomarker discovery platform that can robustly differentiate or confirm the epigenetic footprint of Induced Pluripotent Stem Cells, Embryonic Stem Cells or Progenitor Cells from representative cell lines. [Oxford Biodynamics Limited] Press Release

Grant to Fund Nanotech Therapies for Traumatic Brain Injuries
The U.S. Defense Advanced Research Projects Agency has awarded $6 million to a team of researchers, including Massachusetts Institute of Technology’s Sangeeta Bhatia, to develop nanotechnology therapies for the treatment of traumatic brain injury and associated infections. [Massachusetts Institute of Technology]
Press Release

USC Norris Comprehensive Cancer Center Studies Target Immune System, Women’s Cancers
The USC Norris Comprehensive Cancer Center, a part of the Keck School of Medicine, has recently begun recruiting female participants for three new clinical trials studying breast, ovarian and cervical cancer immunotherapies. It is scheduled to open recruitment for a second ovarian cancer study over the summer. No other facility in California provides patients with access to all four of the trials. [University of Southern California] Press Release

Cell Medica Announces Treatment of First Pediatric Patient in Early Stage Clinical Trial of Cytovir ADV
Cell Medica announced the treatment of the first patient in the ASPIRE Trial, an early stage Phase I/II clinical study investigating the safety and efficacy of Cytovir^TM ADV for the treatment of adenovirus infections in immunosuppressed pediatric patients following bone marrow transplantation. Cytovir ADV is comprised of naturally occurring T lymphocyte cells which demonstrate immune response functions when exposed to adenovirus antigens. [Cell Medica] Press Release

Arrowhead Receives Patent Allowance for DPC siRNA Delivery System with Peptide Backbone
Arrowhead Research Corporation announced that it has received a Notice of Allowance from the U.S. Patent and Trademark Office for U.S. Patent Application Number 13/326,433 entitled, “Peptide-Based In Vivo siRNA Delivery System.” The allowed claims specifically protect the use of targeted melittin or melittin-like peptides to facilitate delivery of siRNA conjugates to hepatocytes. This new patent protection covers the composition of the Dynamic Polyconjugate, or DPC, delivery system used in Arrowhead’s RNAi-based drug candidate, ARC-520, against chronic hepatitis B virus infection. [Arrowhead Research Corporation] Press Release

Stem Cell Research for New MS Treatment
Modified adult stem cells may hold the key to a new treatment for multiple sclerosis (MS), according to University of Adelaide researchers, speaking during ‘Kiss Goodbye to MS’ month. The researchers have started a three-year research project using adult stem cells from fat tissue to send cells with special anti-inflammatory properties directly to the damaged site in the central nervous system. [The University of Adelaide] Press Release

Loyola Opens New Unit for Cancer Patients Undergoing Stem Cell Transplants
Loyola University Medical Center has opened a new 20-bed unit for patients undergoing stem cell transplants for cancers such as leukemia, lymphoma and multiple myeloma. The unit treats patients with hematologic cancers. Patients receive high-dose chemotherapy and/or radiation to kill cancer cells. Because these treatments also destroy immune system cells, patients are infused with stem cells derived from bone marrow or umbilical cord blood. [Loyola University Health System] Press Release

Pioneering Researchers Recognized for Scientific Achievements in Gene and Cell Therapy
The American Society of Gene & Cell Therapy (ASGCT) is honored to recognize Dr. Sonia Skarlatos, PhD, and Dr. Catherine McKeon, PhD as the recipients of the Distinguished Service Award. This award recognizes an ASGCT Member or group that has consistently fostered and enhanced the field of genetic and cellular therapy. [American Society of Gene & Cell Therapy] Press Release

NIH Details Impact of 2013 Sequester Cuts
After weeks of worrying about how the mandatory across-the-board 2013 budget cuts known as the sequester would play out at the National Institutes of Health (NIH), the biomedical research community now has final figures. The bottom line is as grim as expected: The agency’s overall budget will fall by $1.71 billion compared to 2012, to $29.15 billion, a cut of about 5%, according to an NIH notice. Press Release

Office of Biotechnology Activities; Recombinant DNA Research: Proposed Actions Under the NIH Guidelines for Research Involving Recombinant or Synthetic Nucleic Acid Molecules (NIH Guidelines) (FR Doc No: 2013-11222)

Proposed Collection; 60-Day Comment Request: National Cancer Institute (NCI) Alliance for Nanotechnology in Cancer Platform Partnership Scientific Progress Reports (FR Doc No: 2013-11294)

National Heart, Lung, and Blood Institute; Notice of Closed Meetings (FR Doc No: 2013-11099)

Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2013-10978)

Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2013-10972)

Prospective Grant of Start-Up Exclusive Evaluation Option License Agreement: Gene Therapy and Cell-Based Therapy for Cardiac Arrhythmias (FR Doc No: 2013-10859)

Government-Owned Inventions; Availability for Licensing (FR Doc No: 2013-10857)

Proposed Collection; 60-Day Comment Request: Financial Sustainability of Human Tissue Biobanking (NCI) (FR Doc No: 2013-10772)

Expanded and Improved Approach to Identifying Surface Protein Markers Could Support Development of Therapies with Human Bone Marrow Multipotent Stromal Cells

FDA Discovery that Ad5 Vector Uses Coagulation Factor X to Block Complement Proteins Has Major Implications for Gene Therapy

Food and Drug Administration (United States)

Agency Information Collection Activities; Announcement of Office of Management and Budget Approval; Guidance on Informed Consent for In Vitro Diagnostic Device Studies Using Leftover Human Specimens That Are Not Individually Identifiable (FR Doc No: 2013-11125)

Agency Information Collection Activities; Announcement of Office of Management and Budget Approval; Public Health Service Guideline on Infectious Disease Issues on Xenotransplantation (FR Doc No: 2013-10887)

Guidance for Industry: Implementation of an Acceptable Abbreviated Donor History Questionnaire and Accompanying Materials for Use in Screening Frequent Donors of Blood and Blood Components; Availability (FR Doc No: 2013-10889)

NEW The Jackson Laboratory: Using Mice in Research –  Everything Not in the Literature
May 16, 1:00PM (ET in US)
Webinar

NEW World Conference on Regenerative Medicine
October 23-25, 2013
Leipzig, Germany

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.

Postdoctoral Fellow – Stem Cell and Cancer Biology (Johns Hopkins University School of Medicine)

Postdoctoral Position – Cell Therapy (Goethe University Frankfurt am Main)

Faculty Position – Stem Cell, Vascular and Developmental Biology in Ophthalmology (Zhongshan Ophthalmic Center, Sun Yat-Sen University)

Senior Scientist – Stem Cell Therapy: Metabolic Diseases (Paramount Recruitment)

Postdoctoral Research Fellow – Immunology (Beth Israel Deaconess Medical Center, Harvard Medical School)

Postdoctoral Fellowships – Muscular Dystrophy, Cardiomyopathy, Respiratory Failure, Gene Therapy (University of Pennsylvania)

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