Volume 12.20 | May 23

Cell Therapy News 12.20, May 23, 2011
     In this issue: Science | Policy | Business | NIH | CBER | Regulatory | Events
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Editing Scrambled Genes in Human Stem Cells May Help Realize the Promise of Combined Stem Cell-Gene Therapy
Using mutated versions of the lamin A gene as an example to demonstrate the versatility of their virus-based approach, researchers successfully edited a diseased gene in patient-specific induced pluripotent stem cells as well as adult stem cells. [Press release from the Salk Institute for Biological Studies discussing online prepublication in Cell Stem Cell]



Team Discovers Key to Fighting Drug-Resistant Leukemia
A team of scientists has uncovered the basis for acute lymphoblastic leukemia drug resistance: BCL6, a protein that leukemia cells use to stay alive. [Press release from the University of California, San Francisco discussing online prepublication in Nature]

Leucine Deprivation Proves Deadly to Malignant Melanoma Cells
Researchers have found that depriving human melanoma cells of the essential amino acid leucine can be lethal to the cells, suggesting a possible strategy for therapeutic intervention. [Press release from Whitehead Institute for Biomedical Research discussing online prepublication in Cancer Cell]

Stem Cells Reverse Disease in a Model of Parkinson’s Disease
A team of researchers has now compared the ability of cells derived from different types of human stem cell to reverse disease in a rat model of Parkinson disease and identified a stem cell population that they believe could be clinically relevant. [Press release from ScienceDaily discussing online prepublication in the Journal of Clinical Investigation]

Stopping HIV Transmission with a Molecular Barrier
Using a technique that silences genes promoting infection, researchers have developed a novel, topically-applied molecular microbicide capable of preventing HIV transmission. [Press release from Children’s Hospital Boston discussing online prepublication in the Journal of Clinical Investigation]

Artificial Tissue Promotes Skin Growth in Wounds
Improved tissue grafts designed by scientists that promote vascular growth could hasten healing, encourage healthy skin to invade the wounded area and reduce the need for surgeries. [Press release from Cornell University discussing online prepublication in Biomaterials]

Detecting Lung Cancer Early: Researchers Lay Foundation for Future Blood Test
A person’s blood reveals whether he or she has lung cancer, according to researchers. [Press release from ScienceDaily discussing online prepublication in Clinical Cancer Research]

Rare Tumor’s ‘Fingerprint’ Used to Develop Cheap and Reliable New Test
Researchers have developed a cheap and reliable diagnostic test for a rare form of cancer. The test involves screening tumor samples for a particular molecular fingerprint unique to this cancer. [Press release from Wellcome Trust discussing online prepublication in the Journal of Pathology]

Low-Dose Sorafenib May Improve Therapy For Head And Neck Cancer
Adding low doses of the targeted agent sorafenib to the chemotherapy and radiation used to treat head and neck cancer might significantly improve patient care and quality of life, according to a new study. [Press release from The Ohio State Medical Center discussing online prepublication in Molecular Cancer Therapeutics]

Researchers Create Nanopatch for the Heart
Engineers have created a nanopatch for the heart that tests show restores areas that have been damaged, such as from a heart attack. [Press release from Brown University discussing online prepublication in Acta Biomaterialia]

Aldagen Announces Publication of Phase I/II Results for ALD-301, a Stem Cell Therapy in Development for Critical Limb
Aldagen, Inc. announced that results from the Phase I/II clinical trial of ALD-301 were published. ALD-301 is a stem cell therapy being developed as a treatment for critical limb ischemia, the most advanced form of peripheral arterial disease. [Press release from Reuters discussing online prepublication in Catheterization and Cardiovascular Interventions]

Cell Therapy Aims to Prevent Transplant Rejection
A cell therapy that could prevent transplanted organs being rejected, and remove the need for prolonged use of immunosuppressant drugs, has shown promise in early-stage studies in mice. [Press release from Oxford University discussing online prepublication in Science Translational Medicine]

Cell Therapy to Prevent Organ Rejection
Researchers have used cells found naturally in the body, to re-educate the immune system to prevent rejection of an organ transplant while remaining capable of fighting infections and cancer. [Press release from Kings College London discussing online prepublication in Science Translational Medicine]

Experts Develop Technique to Duplicate Immunity Boosting Cells to Unprecedented Levels
Researchers have discovered a method to quickly and exponentially grow regulatory T-cells. The new process enables replication of the cells by tens of millions in several weeks, a dramatic increase over previous duplication methods. [Press release from the University of Minnesota Academic Health Center discussing online prepublication in Science Translational Medicine]

Queen’s Scientists Teaming Up to Cure Premature Baby Blindness
Two teams are taking different approaches to a condition called Retinopathy of Prematurity (ROP). The first team has the aim of tackling the disease at a very early stage, which will minimize the damaging effects of ROP. The second team is investigating whether stem cells from babies’ own umbilical cords might have the power to repair their damaged eyes and save their sight. [Queen’s University Belfast Press Release]

Jennerex and Partners Present Positive JX-594 Randomized Phase II Clinical Data Showing Promising Survival Benefit in Patients with Advanced Liver Cancer
Jennerex, Inc. presented preliminary data from a randomized dose-ranging Phase II trial of JX-594 in patients with advanced liver cancer showing a benefit in overall survival for the high dose group. [Press release from Jennerex, Inc. discussing research presented at the American Society of Gene and Cell Therapy Annual Meeting]

MetMAb in Combination with Tarceva Doubled the Time People with Lung Cancer Lived without Their Disease Getting Worse
Genentech announced the final results from a randomized, multicenter, double-blind Phase II study with its investigational personalized medicine, MetMAb, in people with previously treated advanced non-small cell lung cancer. [Genentech, Inc. Press Release]


(Ranked by Impact Factor of the Journal)

BCL6 Enables Ph+ Acute Lymphoblastic Leukemia Cells to Survive BCR–ABL1 Kinase Inhibition
Scientists report the discovery of a novel mechanism of drug resistance, which is based on protective feedback signaling of leukemia cells in response to treatment with tyrosine kinase inhibitors. [Nature]

Defective Regulation of Autophagy Upon Leucine Deprivation Reveals a Targetable Liability of Human Melanoma Cells In Vitro and In Vivo
Investigators found that leucine deprivation causes the caspase-dependent apoptotic death of melanoma cells because it fails to appropriately activate autophagy. [Cancer Cell]

Targeted Gene Correction of Laminopathy-Associated LMNA Mutations in Patient-Specific iPSCs
Researchers show that helper-dependent adenoviral vectors provide a highly efficient and safe method for correcting mutations in large genomic regions in human induced pluripotent stem cells (iPSCs) and can also be effective in adult human mesenchymal stem cells. [Cell Stem Cell]

Protein-Based Human iPS Cells Efficiently Generate Functional Dopamine Neurons and Can Treat a Rat Model of Parkinson Disease
Scientists compared multiple human induced pluripotent stem (iPS) cells lines derived by virus- and protein-based reprogramming to human ES cells. [J Clin Invest]

Inhibition of HIV Transmission in Human Cervicovaginal Explants and Humanized Mice Using CD4 Aptamer-siRNA Chimeras
Investigators showed that CD4 aptamer-siRNA chimeras specifically suppress gene expression in CD4+ T cells and macrophages in vitro, in polarized cervicovaginal tissue explants, and in the female genital tract of humanized mice. [J Clin Invest]

Microstructured Templates for Directed Growth and Vascularization of Soft Tissue In Vivo
Researchers present a strategy to control the spatial patterns of cell and vascular in-growth throughout the volume of a bioremodelable and resorbable matrix via well-defined micropores and networks of microchannels. [Biomaterials]

Blood-Based Gene Expression Signatures in Non–Small Cell Lung Cancer
Scientists evaluated the applicability of whole blood–based gene expression profiling for the detection of non–small cell lung cancer. [Clin Cancer Res]

Aberrant Succination of Proteins in Fumarate Hydratase Deficient Mice and Hereditary Leiomyomatosis and Renal Cell Cancer Patients Is a Robust Biomarker of Mutation Status
Using a previously characterized antibody against S-(2-succinyl) cysteine, investigators show that genetic ablation of fumarate hydratase causes high levels of protein succination. [J Pathol]



‘Soap Opera’ Sours Cancer Chief Hunt
A bitter row between a prominent Spanish biologist and the country’s science minister has spiraled into a potentially damaging leadership crisis at the country’s top cancer research institute. [Spanish National Cancer Research Centre, Spain]

Chu Calls NSF’s Decision to Abandon Deep Underground Science and Engineering Laboratory ‘Disappointing’
Energy Secretary Steven Chu says that he’s “very disappointed” with the National Science Foundation (NSF) for pulling out of a planned $875-million underground science lab in South Dakota. [National Science Foundation, United States]

Leopoldina Makes Recommendations to the G8 Heads of State and Government
Together with the national science academies of the other G8 states, the German National Academy of Sciences Leopoldina has prepared two statements for the G8 states in the run-up to the G8 Summit of Heads of State and Government. [German National Academy of Sciences Leopoldina, Germany]

Wellcome Trust Calls for Clearer Vision for NHS Reform
The government’s proposed reforms to the National Health Service lack a clear vision of what a 21st-century health service should deliver, according to the Director of the Wellcome Trust, Sir Mark Walport. Furthermore, he argues, the reforms overlook the potential of medical research to translate into better care for patients and contribute to the nation’s economic recovery. [Wellcome Trust, United Kingdom]


Tekmira and Bristol-Myers Squibb Expand Collaboration to Include Broader Applications of LNP Technology
Tekmira Pharmaceuticals Corporation announced that it has expanded its current multi-year collaboration with global biopharmaceutical company Bristol-Myers Squibb Company to include evaluation of newly developed Tekmira proprietary lipid nanoparticle formulations designed for delivery to tumors and other tissues outside the liver. [Tekmira Pharmaceuticals Corporation Press Release]

Cellzome to Study Epigenetic Factors with Neusentis
Cellzome announced a joint project with Pfizer’s Pain & Sensory Disorders and Regenerative Medicine unit ‘Neusentis’, to characterize epigenetic factors involved in stem cell differentiation using Cellzome’s Episphere™ technology. [Cellzome Press Release]

Shire to Establish New Regenerative Medicine Business Unit through Cash Acquisition of Advanced BioHealing, Inc., Including US Marketed DERMAGRAFT®
Shire plc announced that it has signed an agreement to acquire Advanced BioHealing, Inc. [Shire plc Press Release]

Histogenics Acquires ProChon Biotech
Histogenics Corporation announced that it has acquired ProChon Biotech, Ltd. in a stock-for-stock exchange. [BusinessWire]

Benitec Raises AU $8 Million to Forge Ahead with Its Gene Silencing Therapeutics: Rights Issue Closes Oversubscribed
Benitec Ltd announced its fully underwritten AU$8 million renounceable rights issue, incorporating an entitlement offer and a shortfall offer, had successfully closed oversubscribed. [Benitec Ltd. Press Release]

Cytonet’s Liver Cell Therapy Awarded Orphan Drug Designation for the Treatment of Urea Cycle Disorders
Cytonet announced it has been granted orphan drug designation by the U.S. Food and Drug Administration. [PR Newswire]

Ministry of Health Approves BrainStorm’s NurOwn™ for the First Clinical Trial of Adult Stem Cell Therapy for ALS
BrainStorm Inc. and Hadasit announced that Israel’s Ministry of Health has approved the Phase I/II clinical trial of NurOwn™, BrainStorm’s autologous stem cell therapy for people with amyotrophic lateral sclerosis (ALS or Lou Gehrig’s Disease). [BrainStorm Inc. Press Release]

Cytheris and Cancer Immunotherapy Trials Network (CITN) Announce Selection of Recombinant Interleukin-7 (CYT107) for Initial Studies at Member Institutions
Cytheris SA and CITN announced the selection of CYT107, the investigational multifunctional cytokine under development by Cytheris, as one of the first immunotherapeutic agents proposed for initial clinical trials at network institutions. [Cytheris SA Press Release]

BioInvent and ThromboGenics Announce the Start of New Study of Novel Antibody Anti-Cancer Agent TB-403 (Anti-PlGF) by Partner Roche
BioInvent International AB and co-development partner ThromboGenics NV announce that their partner Roche has dosed the first patient in a phase Ib/II study with the novel antibody anti-cancer agent TB-403 (RG7334). [BioInvent International AB Press Release]

ViaCyte, Inc., Names Anthony Gringeri Chief Development Officer
ViaCyte, Inc. announced the appointment of Anthony Gringeri as Chief Development Officer. [Viacyte, Inc. Press Release]

Genesis Biopharma Forms Scientific and Medical Advisory Board
Genesis Biopharma, Inc. announced the formation of a Scientific and Medical Advisory Board. [Genesis Biopharma, Inc. Press Release]


Office of Biotechnology Activities, Office of Science Policy, Office of the Director; Notice of Meeting (FR Doc No: 2011-12269)

Office of the Director, National Institutes of Health; Notice of Meeting (FR Doc No: 2011-12046)

Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2011-12267)

Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2011-12049)

Center for Scientific Review; Amended Notice of Meeting (FR Doc No: 2011-12465)

National Center for Research Resources; Amended Notice of Meeting (FR Doc No: 2011-12045)

National Center for Research Resources; Notice of Closed Meeting (FR Doc No: 2011-12466)

National Institute of Biomedical Imaging and Bioengineering; Notice of Closed Meeting (FR Doc No: 2011-12230)

Submission for OMB Review; Comment Request; NCI Cancer Genetics Services Directory Web-Based Application Form and Update Mailer (FR Doc No: 2011-12047)

Submission for OMB Review; Comment Request; A Generic Submission for Formative Research, Pre-Testing, Stakeholder Measures and Advocate Forms at NCI (FR Doc No: 2011-12375)

May 31, 2011: Cellular, Tissue and Gene Therapies Advisory Committee Meeting Draft Roster

May 31, 2011: Cellular, Tissue and Gene Therapies Advisory Committee Meeting Draft Agenda

Fatalities Reported to FDA Following Blood Collection and Transfusion: Annual Summary for Fiscal Year 2010



Ultra High Throughput Sequencing for Clinical Diagnostic Applications–Approaches To Assess Analytical Validity; Public Meeting; Request for Comments [FR Doc No: 2011-12310]


European Medicines Agency Discusses Pharmacovigilance with French General Inspectorate of Social Affairs
On Tuesday 17 May, the European Medicines Agency hosted a meeting with the French General Inspectorate of Social Affairs to discuss the current system of the safety monitoring of medicines in the European Union.

Report Published from Workshop on Ethics and GCP in Clinical Trials Outside the European Union
The European Medicines Agency published a meeting report reflecting discussions and comments made on its draft reflection paper on ethical and good-clinical-practice (GCP) aspects of clinical trials of medicinal products for human use conducted in third countries and submitted in marketing authorization applications to the European Medicines Agency.

Meeting Highlights from the Committee for Medicinal Products for Human Use (CHMP) 16-19 May 2011


Guidance on the GMP Clearance of Overseas Medicine Manufacturers
The Australian Regulatory Guidelines GMP Clearance for Overseas Manufacturers (17th edition) will be phased in over the next 3 months.

Call for Input: Review to Improve Transparency of the Therapeutic Goods Administration
Added APVMA discussion on the TGA’s Transparency Review.

Uniform Recall Procedure for Therapeutic Goods (URPTG)
Updated recall co-ordinators’ contact details.

Review to Improve Transparency of the Therapeutic Goods Administration
Added APVMA discussion on the TGA’s Transparency Review.


NEW The Beatson International Cancer Conference 2011
July 3-6, 2011
Glasgow, United Kingdom

NEW Gordon Research Conferences: Cancer Nanotechnology
July 17-22, 2011
Waterville, United States

NEW 70th Annual Meeting of the Society for Developmental Biology
July 21-25, 2011
Chicago, United States

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

Post Doctoral Fellow (University of California, Los Angeles)

Staff Scientist Position (Spanish National Cancer Center)

Postdoctoral Research Fellows (Stem Cells) (A*Star Bioprocessing Technology Institute) 

Quality Assurance Manager – Program in Children’s Regenerative Medicine (The University of Texas Medical School at Houston)

Implementation Coordinator – Maternity Leave Contract (STEMSOFT Software Inc.) 

Tenure Track, Assistant/Associate Professor (University of Missouri-Kansas City School of Medicine)

Postdoc – Laboratory of Molecular Tumor Genetics (Duke-NUS Graduate Medical School)

Postdoctoral Positions in Immune Cell Activation (Institut de Recherches Cliniques de Montréal)

Postdoctoral Position in Developmental Neurobiology (Institut de Recherches Cliniques de Montréal)

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