Volume 15.01 | Jan 13

Issue 15.01 January 13, 2014
Cell Therapy News - Your Industry in an Instant
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Gene Therapy for Parkinson’s Disease Yields Promising Results in First Patients
A new gene therapy for Parkinson’s disease has achieved promising results in its first human tests, involving 15 patients. The treatment, called ProSavin, uses a modified virus to deliver three genes into the striatum, a part of the brain that controls movement. The genes are intended to boost the production of dopamine, a chemical that becomes deficient in patients with Parkinson’s. [Press release from Imperial College London discussing online prepublication in The Lancet] Press Release | Abstract
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PUBLICATIONS (Ranked by impact factor of the journal)
Mesenchymal Stem Cell Therapy in Proteoglycan Induced Arthritis
Investigators explored the immunosuppressive effect and mechanism of action of intraperitoneal and intra-articular mesenchymal stem cell injection in proteoglycan induced arthritis. [Ann Rheum Dis] Abstract

Engineering Cells with Intracellular Agent-Loaded Microparticles to Control Cell Phenotype
Scientists developed an approach to control cell phenotype in vitro and after transplantation by engineering cells with intracellular depots that continuously release phenotype-altering agents for days to weeks. [Nat Protoc] Abstract | Press Release

Application of Stem Cells Derived from the Periodontal Ligament or Gingival Tissue Sources for Tendon Tissue Regeneration
A co-delivery system based on TGF-β3-loaded RGD-coupled alginate microspheres was developed for encapsulating periodontal ligament stem cells or gingival mesenchymal stem cells (MSCs). The capacity of encapsulated dental MSCs to differentiate into tendon tissue was investigated in vitro and in vivo. [Biomaterials] Abstract

The Effect of Mesenchymal Stem Cell Sheets on Structural Allograft Healing of Critical Sized Femoral Defects in Mice
Researchers generated revitalized structural allografts wrapped with mesenchymal stem/progenitor cell sheets, which were produced by expanding primary syngenic bone marrow derived cells on temperature-responsive plates, as a tissue-engineered periosteum. [Biomaterials] Abstract

Scaffoldless Tissue-Engineered Dental Pulp Cell Constructs for Endodontic Therapy
Self-assembled, scaffoldless, three-dimensional tissues were engineered from dental pulp cells and assessed as a device for pulp regeneration. These engineered tissues were placed into the canal space of human tooth root segments that were capped on one end with calcium phosphate cement, and the entire system was implanted subcutaneously into mice. [J Dent Res] Abstract

Bone Tissue Engineering by Using a Combination of Polymer/Bioglass Composites with Human Adipose-Derived Stem Cells
Researchers investigated the osteogenic capacity of a combination of poly(DL-lactic acid) porous foams containing 5 wt% and 40 wt% of Bioglass particles with human adipose-derived stem cells in vitro and in vivo. [Cell Tissue Res] Abstract

Autologous Mesenchymal Stromal Cell Infusion as Adjunct Treatment in Patients with Multidrug and Extensively Drug-Resistant Tuberculosis: An Open-Label Phase I Safety Trial
30 patients with microbiologically confirmed multidrug-resistant or extensively drug-resistant tuberculosis were treated with single-dose autologous bone marrow-derived mesenchymal stromal cells (aimed for 1×106 cells per kg), within 4 weeks of the start of antituberculosis-drug treatment in a specialist center in Minsk, Belarus. [Lancet Resp Med] Abstract | Press Release

Human RPE Stem Cells Grown into Polarized RPE Monolayers on a Polyester Matrix Are Maintained after Grafting into Rabbit Subretinal Space
Trephined pieces of retinal pigment epithelium (RPE) monolayers on polyester were transplanted subretinally in the rabbit, a large-eyed animal model. After 4 days, retinal edema was observed above the implant, detected by spectral domain optical coherence tomography and fundoscopy. [Stem Cell Rep]
Full Article | Graphical Abstract | Press Release

Your Free Copy of the Nature Neuroscience Poster "Neural Stem Cells" Awaits
Molecular, Genetic and Stem Cell-Mediated Therapeutic Strategies for Spinal Muscular Atrophy (SMA)
The authors outline the most interesting therapeutic strategies that are currently developing, which are represented by molecular, gene and stem cell-mediated approaches for the treatment of SMA. [J Cell Mol Med] Full Article

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

Take the "if" out of "diff": Reduce variability when differentiating to hematopoietic cells
Tengion Announces Continued Clinical Progress in Its Two Lead Programs
Tengion, Inc. announced continued clinical progress in its two lead programs, the Neo-Kidney Augment and the Neo-Urinary Conduit. The Neo-Kidney Augment, which is intended to prevent or delay dialysis and transplantation by increasing renal function in patients with advanced chronic kidney disease (CKD), was implanted in five CKD patients in 2013 in the ongoing Phase I clinical trial in Sweden. The Neo-Kidney Augment is safe and well tolerated in the five patients implanted to date. [Tengion, Inc.] Press Release

Cytori to Initiate U.S. Clinical Trial of Adipose-Derived Regenerative Cells in Hamstring Injuries
Cytori Therapeutics has received investigational device exemption approval from the FDA to begin a prospective clinical trial to evaluate the safety and feasibility of Cytori Cell Therapy as a potential treatment for hamstring injuries. The trial, referred to as RECOVER, will begin as a ten-patient, open label study in 2014. [Cytori Therapeutics] Press Release

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Leaked Files Slam Stem-Cell Therapy
A series of damning documents seen by Nature expose deep concerns over the safety and efficacy of the controversial stem-cell therapy promoted by Italy’s Stamina Foundation. The leaked papers reveal the true nature of the processes involved, long withheld by Stamina’s president, Davide Vannoni. Other disclosures show that the successes claimed by Stamina for its treatments have been over-stated. [Nature News] Editorial

U.K. Researchers Ask the Public to Share Medical Data
U.K. medical research charities urged residents to allow scientists to access patient data from the country’s National Health Service. Making their plea through colorful advertisements in the national press, the charities weighed in on a raging national debate. [ScienceInsider] Editorial
MD Anderson Teams Up with Pfizer to Advance Cancer Immunotherapy
The University of Texas MD Anderson Cancer Center and Pfizer will collaborate in the development of immune-based approaches to cancer treatment, the first such agreement made through MD Anderson’s Moon Shots Program immunotherapy platform. [The University of Texas MD Anderson Cancer Center] Press Release

Alnylam and Genzyme Form Transformational Alliance for RNAi Therapeutics as Genetic Medicines
Alnylam Pharmaceuticals, Inc. and Genzyme, a Sanofi company, announced that they have formed a transformational alliance for the development and commercialization of RNAi therapeutics as genetic medicines. [Alnylam Pharmaceuticals, Inc.] Press Release

NeoStem’s Subsidiary, Progenitor Cell Therapy (PCT), to Provide Process Development and Manufacturing Services to Kite Pharma
NeoStem, Inc. and its subsidiary, PCT, announced the execution of a Services Agreement with Kite Pharma, Inc., under which PCT will provide cell therapy process development and manufacturing services for Kite Pharma’s lead engineered Autologous T Cell Therapy clinical development program. [NeoStem, Inc] Press Release

Bellicum Pharmaceuticals Raises $34.4 Million in a Series B Financing
Bellicum Pharmaceuticals, Inc. announced that it has completed a second and final closing of its Series B financing, securing an additional $14.7 million and bringing the total raised in the round to $34.4 million. Current investors provided the additional funding, including AVG Ventures and Remeditex Ventures. The funding will be used to expand clinical development of Bellicum’s lead cellular immunotherapy product candidates. [Bellicum Pharmaceuticals, Inc.] Press Release

The Leukemia & Lymphoma Society Awards $12.6 Million in Research Grants
The Leukemia & Lymphoma Society (LLS) has awarded 21 new grants representing a total investment of $12.6 million to tackle six areas of high unmet medical need in the blood cancers. In response to requests for proposals from researchers in these six critical areas, LLS has awarded these grants under its Translational Research Program, an initiative designed to help accelerate the movement of promising discoveries from the lab to the clinic. [The Leukemia & Lymphoma Society] Press Release

Athersys Announces New Patents in Japan for Stem Cell and Regenerative Medicine Technology
Athersys, Inc. announced that it has been granted patents from the Japan Patent Office for several inventions involving its proprietary cell therapy technology. Patent No. 5398941 covers non-embryonic, multipotent stem cells, such as MultiStem, and applies to all therapeutic applications. Patent No. 5399709 covers the use of such stem cell therapies for the treatment of immune dysfunctions, such as graft-versus-host disease, and inflammatory diseases and autoimmune disorders. [Athersys, Inc.] Press Release

TxCell Enters Col-Treg into Full Development for the Rare Disease Autoimmune Uveitis
TxCell SA announced it has initiated a full development program with Col-Treg for the treatment of Autoimmune Uveitis. Col-Treg is TxCell’s second therapeutic candidate from its ASTrIA platform after Ovasave®, TxCell’s lead autologous Ag-Treg cell-based immunotherapy. [TxCell SA] Press Release

StemCells, Inc. Expands Phase I/II Spinal Cord Injury Trial to North America
StemCells, Inc. announced that a team at the University of Calgary successfully transplanted its first subject in the company’s Phase I/II clinical trial in chronic spinal cord injury, with the company’s proprietary HuCNS-SC® human neural stem cells. [StemCells Inc.] Press Release

CSU Vets Continue Study of Stem-Cell Therapy for Feline Kidney Disease in Fifth Clinical Trial
Chronic kidney disease in older cats is the focus of a fifth clinical trial under way at Colorado State University’s James L. Voss Veterinary Teaching Hospital, where veterinarians are exploring novel stem-cell therapy that could, for the first time, hold promise for treating one of the most perplexing feline diseases. [Colorado State University] Press Release

Cell Medica Announces Orphan Drug Designation in the European Union for Cytovir ADV
Cell Medica Limited announced that the European Medicines Agency’s Committee for Orphan Medicinal Products has issued a positive opinion on an application for orphan designation of a novel T cell immunotherapy under development by the company. [Cell Medica Limited] Press Release
Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2014-00162)

Clinical Center; Notice of Closed Meeting (FR Doc No: 2014-00309)
NEW Strategies to Inhibit Graft-versus-Host Disease While Sparing the Graft-versus-Tumor Response: Finding the Sweet Spot
February 18, 2014
New York City, United States

NEW Stem Cell Summit 2014
April 23-25, 2014
Boston, United States

NEW Cell Line Development Asia 2014
May 20-23, 2014
Shanghai, China

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Scientific Affairs Coordinator (American Society of Hematology)

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

Postdoctoral Associate – Hematopoietic Differentiation (Northwestern University Feinberg School of Medicine)

Stem Cell Manufacturing Scientist (Paramount Recruitment)

Postdoctoral Position – Stem Cells and Bioengineering (Queensland University of Technology/Translational Research Institute)

Clinical MD – Clinical Development Program for Oncology and Autoimmune Diseases (Immunomedics, Inc.)

Chief Medical Officer (Immunomedics, Inc.)

Postdoctoral Fellow – Wound Healing (University of Connecticut Health Center)

Postdoctoral Fellow – Cardiovascular Tissue Engineering (Stanford University School of Medicine)

Postdoctoral Fellow – Liver Tissue Engineering (Harvard Medical School)

Research Technologist – Human Pluripotent Stem Cell Products (STEMCELL Technologies Inc.)

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