Cell Therapy News Volume 13.09 | Mar 12 2012

Functional Engraftment of Colon Epithelium Expanded In Vitro from a Single Adult Lgr5⁺ Stem Cell
Here investigators describe methods for long-term expansion of colonic stem cells positive for leucine-rich repeat containing G protein-coupled receptor 5 (Lgr5⁺ cells) in culture. To test the transplantability of these cells, the researchers reintroduced cultured GFP⁺ colon organoids into superficially damaged mouse colon. [Nat Med] Abstract

Transferred Melanoma-Specific CD8⁺ T Cells Persist, Mediate Tumor Regression, and Acquire Central Memory Phenotype
As a means to enhance persistence of infused T cells in vivo and limit toxicity, 11 patients with refractory, progressive metastatic melanoma received cyclophosphamide alone as conditioning before the infusion of peripheral blood mononuclear cell-derived, antigen-specific, CD8⁺ cytotoxic T-lymphocyte clones followed by low-dose or high-dose IL-2. [Proc Natl Acad Sci USA] Abstract | Press Release

Improvement of Islet Function in a Bioartificial Pancreas by Enhanced Oxygen Supply and Growth Hormone Releasing Hormone Agonist
Critical shortage of donor organs, gradual loss in graft function over time, and chronic need for immunosuppression limit the indication for islet transplantation to a small group of patients. Here researchers present a promising approach to address these limitations by utilization of a macrochamber specially engineered for islet transplantation. [Proc Natl Acad Sci USA] Abstract | Press Release

Scanning Probe-Enabled Nanocombinatorics Define the Relationship between Fibronectin Feature Size and Stem Cell Fate
In a proof-of-concept investigation of mesenchymal stem cell (MSC) differentiation, combinatorial patterns first enabled a rapid and systematic screening of MSC adhesion, as a function of feature size, while uniform patterns were used to study differentiation with statistically significant sample sizes. [Proc Natl Acad Sci USA]
Abstract | Press Release

The Cartilage Matrix Molecule Components Produced by Human Fetal Cartilage Rudiment Cells within Scaffolds and the Role of Exogenous Growth Factors
This study examined the matrix components elaborated by cultured fetal cartilage rudiment cells, a mixture of chondroblasts/chondroprogenitor cells and committed chondrocytes, in monolayer, cell pellet cultures and in the synthetic scaffolds sodium alginate and polyglycolic acid. The ability of fibroblast growth factor (FGF)-2 and FGF-18 to promote chondrogenesis in pellet cultures was also examined. [Biomaterials] Abstract

Modulation of Mesenchymal Stem Cell Chondrogenesis in a Tunable Hyaluronic Acid Hydrogel Microenvironment
In this study, hyaluronic acid-tyramine hydrogels were explored as biomimetic matrices for caprine mesenchymal stem cells in cartilage tissue engineering. [Biomaterials] Abstract

Functional Characterization of TPO-Expanded CD34⁺ Cord Blood Cells Identifies CD34^–CD61^– Cells as Platelet Producing Cells Early after Transplantation in NOD/SCID Mice and rCD34⁺ Cells as CAFC Colony Forming Cells
It is unknown which sub-populations of TPO expanded cord blood CD34⁺ cells mediate accelerated platelet recovery and bone marrow (BM) engraftment. In this study, the contribution of these sub-populations to human platelet appearance in the blood and BM engraftment was studied in NOD/SCID mice. [Stem Cells] Abstract

Neonatal Gene Therapy with a Gamma Retroviral Vector in Mucopolysaccharidosis VI Cats
Mucopolysaccharidosis VI cats were injected intravenously with a gamma retroviral vector (RV)-expressing feline N-acetylgalactosamine 4-sulfatase, resulting in 5 ± 3 copies of RV per 100 cells in liver. [Mol Ther] Abstract

Long-Term Correction of Very Long-Chain Acyl-CoA Dehydrogenase Deficiency in Mice Using AAV9 Gene Therapy
Very long-chain acyl-coA dehydrogenase (VLCAD)-deficient mice were treated systemically with 1 × 10¹² vector genomes of recombinant adeno-associated virus 9 (rAAV9)-VLCAD. [Mol Ther] Abstract

Therapeutic Angiogenesis due to Balanced Single-Vector Delivery of VEGF and PDGF-BB
Researchers tested the hypothesis that codelivery of platelet-derived growth factor-BB (PDGF-BB) could induce normal angiogenesis in skeletal muscle irrespective of vascular endothelial growth factor (VEGF) levels. [FASEB J] Abstract | Press Release

Three-Dimensional Multipotent Progenitor Cell Aggregates for Expansion, Osteogenic Differentiation and ‘In Vivo’ Tracing with AAV Vector Serotype 6
In this study, researchers tested whether self-complementary adeno-associated viral vectors (AAV) can potentially be used as a gene delivery system in an osteogenically differentiated multipotent adult progenitor cell-based ‘in vivo’ bone formation model in the craniofacial region. [Gene Ther] Abstract

Chimerism and Tolerance without GVHD or Engraftment Syndrome in HLA-Mismatched Combined Kidney and Hematopoietic Stem Cell Transplantation
Researchers developed an approach to induce immune tolerance for organ transplants using a bioengineered mobilized cellular product enriched for hematopoietic stem cells and tolerogenic graft facilitating cells combined with nonmyeloablative conditioning; this approach resulted in engraftment, durable chimerism, and tolerance induction in recipients with highly mismatched related and unrelated donors. [Sci Transl Med]
Abstract | Press Release

Human Müller Glia with Stem Cell Characteristics Differentiate into Retinal Ganglion Cell (RGC) Precursors In Vitro and Partially Restore RGC Function In Vivo following Transplantation
Researchers’ results suggest that transplanted RGC precursors may be capable of establishing local interneuron synapses and possibly release neurotrophic factors that facilitate recovery of RGC function. [Stem Cells Transl Med] Abstract | Press Release

Sangamo Presents New Clinical Data Demonstrating Persistent Positive Effects of ZFN Therapeutic^® for “Functional Cure” of HIV/AIDS
Sangamo BioSciences, Inc. announced that new data from its program to develop a ‘functional cure’ for HIV/AIDS were presented. [Press release from Sangamo BioSciences, Inc. discussing research presented at the 19th Conference on Retroviruses and Opportunistic Infections, Seattle] Press Release

Merck’s Investigational Allergy Immunotherapy Tablet (AIT) Significantly Reduced the Combination of Ragweed Allergy Symptoms and Medication Use in Phase III Trial
Merck announced the results from a Phase III clinical study of its investigational AIT for ragweed pollen. [Press release from Merck & Co., Inc. discussing research presented at the American Academy of Allergy, Asthma & Immunology (AAAAI) Annual Meeting, Orlando] Press Release

Circassia’s Ragweed Allergy Therapy Achieves Positive Phase II Clinical Results
Circassia Ltd announced that its ragweed allergy therapy has achieved positive results in a key phase II clinical trial. In the study, patients with more severe symptoms achieved a significantly greater improvement following treatment with the ToleroMune® T-cell vaccine than those on placebo. [Circassia Ltd] Press Release

Fourteenth Patient Dosed in Neuralstem ALS Stem Cell Trial
Neuralstem, Inc. announced that the second patient to receive stem cells in the cervical region of the spine was dosed in the ongoing Phase I trial of its spinal cord neural stem cells in amyotrophic lateral sclerosis (ALS). The first twelve patients in the trial received stem cell transplants in the lumbar region of the spinal cord only. [Neuralstem, Inc.] Press Release

Genetics Policy Institute (GPI) Files Additional Legal Arguments in Sherley v. Sebelius Stem Cell Case
The GPI joined the Coalition for Advancement of Medical Research in filing an amicus curiae (“friend of the court”) brief in the United States Court of Appeals for the District of Columbia Circuit, supporting federal funding for human embryonic stem cell research. [Genetics Policy Institute] Press Release

Despite Legal Warnings, UNESCO Finally Approves African Science Prize
Overruling its own legal experts, U.N. Educational, Scientific and Cultural Organization’s (UNESCO’s) executive board finally approved a new prize for African scientists. [U.N. Educational, Scientific and Cultural Organization] Press Release

Gene Therapists Ask to Be Released from the RAC
The main U.S. professional society representing gene therapists argued that clinical trials in their maturing field should no longer be required to undergo review by a special federal advisory committee. [Recombinant DNA Advisory Committee (RAC), United States] Press Release

Funding Uncertainty Strands Spain’s Young Scientists
Spanish researchers are feeling the budget squeeze as the country scrambles to negotiate a 2012 budget. [Spanish Government, Spain] Press Release

Brazilian Researchers Protest Budget Cuts
Science agencies in Brazil have voiced concern at the government’s decision to cut the science, technology and innovation budget by nearly a fifth. [The Brazilian Society for the Advancement of Science, Brazil] Press Release

AACR Calls for Letters of Intent for a New Stand Up To Cancer-Cancer Research Institute Cancer Immunology Translational Research Dream Team
Stand Up To Cancer (SU2C) and the Cancer Research Institute (CRI), along with the American Association for Cancer Research (AACR), call upon the cancer research community to submit Letters of Intent for a new Dream Team – the SU2C-CRI Cancer Immunology Translational Research Dream Team. [American Association for Cancer Research] Press Release

Pediatric Research Institute Launched by Children’s Medical Center, UT Southwestern
Children’s Medical Center is partnering with the University of Texas (UT) Southwestern Medical Center to establish a unique $150 million venture that will bring together the finest scientists to pursue research with the potential to change scientific fields, as well as the way disease is treated in children and adults. [University of Texas Southwestern Medical Center] Press Release

Opexa Therapeutics Executes Strategic Service Agreement with Pharmaceutical Research Associates to Manage Clinical Trial
Opexa Therapeutics, Inc. announced that the Company has executed an agreement with Pharmaceutical Research Associates, Inc. to provide contract research organization services to support Opexa’s upcoming Phase IIb clinical trial for Tovaxin, a novel T-cell therapy for multiple sclerosis (MS), in patients with secondary progressive MS. [Opexa Therapeutics, Inc.] Press Release

Medistem Signs Exclusive Worldwide License with Yale University for Treatment of Type 1 Diabetes Using Stem Cells
Medistem Inc. and Yale University have signed an exclusive worldwide licensing agreement covering the generation of pancreatic islets from stem cells such as the Endometrial Regenerative Cell. [Medistem Inc.]
Press Release

Student Innovation at Rensselaer Polytechnic Institute Seeks to Mend Previously Untreatable Tissue Injuries
Christopher Rivet has successfully married two powerful bioengineering technologies to develop a new method for delivering drugs directly to an injury site and jumpstarting the process of tissue regeneration. Rivet is one of three finalists for the 2012 $30,000 Lemelson-MIT Rensselaer Student Prize. [Rensselaer Polytechnic Institute] Press Release

Bellicum Closes $20 Million Series B Financing
Bellicum Pharmaceuticals, Inc. announced that it has raised $20 million in a Series B financing. The funding will be used to advance its lead CaspaCIDe™ and DeCIDe™ programs through Phase II clinical development. [Bellicum Pharmaceuticals, Inc.] Press Release

Medicyte and PRIMACYT Receive Grant to Develop Human Liver Cells for Cell-Based Therapies
Medicyte and PRIMACYT have received a 300,000 Euro grant from the German Federal Ministry of Education and Research to develop culturing methods of hepatocytes for use in cell-based therapies. [Medicyte GmbH] Press Release

BrainStorm Cell Therapeutics Expands Pipeline with the Initiation of a Study for Multiple Sclerosis
BrainStorm Cell Therapeutics Inc. announced plans to initiate a preclinical study assessing the efficacy of its NurOwn™ stem cell technology in patients with Multiple Sclerosis. [BrainStorm Cell Therapeutics Inc.]
Press Release

Cartilage Repair Gel Gives Injuries a Sporting Chance
A cartilage gel being developed by tissue engineers and biochemists could bring increased mobility to people living with debilitating sports injuries. [University of Sydney] Press Release

Medistem Inc. to Add Kidney and Lung Failure to Clinical Trials of Endometrial Regenerative Cells (ERC) Stem Cells in Russia
Medistem Inc., in partnership with its Russian licensee, ERCell, announced the signing of a letter of intent to begin clinical trials using Medistem’s ERC stem cells for renal, lung and peripheral artery disease. [Medistem Inc.] Press Release

Intellect Neurosciences Receives Notice of Allowance from United States Patent and Trademark Office for its ANTISENILIN® Alzheimer’s Disease Immunotherapy Technology Platform
Intellect Neurosciences, Inc. announced that it received a Notice of Allowance from the United States Patent and Trademark Office for patent claims covering the company’s ANTISENILIN® monoclonal antibody platform technology for the treatment and prevention of Alzheimer’s disease. [Intellect Neurosciences, Inc.] Press Release

American Society of Hematology Announces 2012 Translational Research Training in Hematology Program Participants
The American Society of Hematology and the European Hematology Association announce the names of 20 early-career hematologists chosen to participate in the 2012 Translational Research Training in Hematology joint program. [American Society of Hematology] Press Release

Notice of Change in Participation of NIH Institutes and Centers in PAR-11-001 “Animal and Biological Material Resource Centers (P40)” (NOT-OD-12-074)

Notice of Intent to Publish a Funding Opportunity Announcement for Genomic Medicine Pilot Demonstration Project (U01) (NOT-HG-12-011)

Request for Information (RFI): Enhancing the Clinical and Translational Science Awards (CTSA) Program (NOT-TR-12-003)

Partnerships for Sustainable Research and Dissemination of Evidence-Based Medicine (R24) (RFA-HS-12-005)

The Human Islet Distribution Coordinating Center (UC4) (RFA-DK-12-001)

Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2012-5827)

Center for Scientific Review: Notice of Closed Meetings (FR Doc No: 2012-5930)

National Cancer Institute; Notice of Closed Meetings (FR Doc No: 2012-5695)

National Heart, Lung, and Blood Institute; Notice of Closed Meeting (FR Doc No: 2012-5678)

Government-Owned Inventions; Availability for Licensing (FR Doc No: 2012-5356)

Food and Drug Administration (United States)

FDA Approves First Cell-Based Product to Treat Oral Mucogingival Conditions in Adults
The Food and Drug Administration approved GINTUIT, the first cell-based product made from allogeneic human cells and bovine collagen. Press Release

Guidance for the Public, Food and Drug Administration (FDA) Advisory Committee Members, and FDA Staff: Public Availability of Advisory Committee Members’ Financial Interest Information and Waivers; Availability (FR Doc No: 2012-5776)

Agency Information Collection Activities; Proposed Collection; Comment Request; Emergency Shortages Data Collection System (FR Doc No: 2012-5633)

Modernizing the Regulation of Clinical Trials and Approaches to Good Clinical Practice; Public Hearing; Request for Comments (FR Doc No: 2012-5476)

Report on the Performance of Drug and Biologics Firms in Conducting Postmarketing Requirements and Commitments; Availability (FR Doc No: 2012-5302)

European Medicines Agency (European Union)

Committee for Advanced Therapies Re-Elects Christian Schneider as Chair
The European Medicines Agency’s Committee for Advanced Therapies has re-elected Dr Christian Schneider as its chair for a second three-year term. Press Release

Therapeutic Goods Administration (Australia)

Australian Regulatory Guidelines for Complementary Medicines (ARGCM)

Guidelines for Levels and Kinds of Evidence to Support Indications and Claims

NEW International Cord Blood Symposium – 10^th Anniversary Conference: Milestones in Cord Blood Transplantation
June 7-9, 2012
San Francisco, United States

NEW Stem Cell Society Singapore (SCSS) Symposium 2012 – Stem Cell Based Therapy
September 6-7, 2012
Biopolis, Singapore

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

Scientist – Pluripotent Stem Cells (STEMCELL Technologies, Inc.)

Scientist – hPSC Bioengineering (STEMCELL Technologies, Inc.)

Scientist – Antibody and Hybridoma Technology Platforms (STEMCELL Technologies, Inc.)

Business Analyst – Product Management (STEMSOFT Software, Inc.)

Senior Development Associate (Genzyme Corporation)

Scientist II, Innovation – Cell Processing Technologies (Lonza Walkersville, Inc.)

Senior Manager, Innovation – Cell Processing Technologies (Lonza Walkersville, Inc.)

Postdoctoral Fellow and Technician Positions (North Carolina Central University)

Quality Assurance Manager, GMP (Cellular Dynamics International)

Research Technologist III (Medical College of Wisconsin)

Postdoctoral Position: Manager/Chair in the Beta- Cell Biology and Therapy Unit (Kocaeli University)

Postdoctoral Position in Stem Cell Biology/Neuroscience/Cancer (The Jackson Laboratory)


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