Cell Therapy News Volume 11.25 | Jul 5 2010

Caltech Biologists Discover How T Cells Make a Commitment
When does a cell decide its particular identity? According to researchers, in the case of T cells, the answer is when the cells begin expressing a particular gene called Bcl11b. [Press release from the California Institute of Technology discussing online prepublication in Science]

Melanoma-Initiating Cells Identified by Study
Scientists have identified a cancer-initiating cell in human melanomas. [Press release from Stanford University School of Medicine discussing online prepublication in Nature]

Study Explores Genetics of Extreme Longevity
In a genome-wide association study that included almost 1,000 people aged 100 or older, a set of 150 single-letter variants in DNA — involving about 70 different genes — was able to distinguish between the centenarians and controls 77% of the time. [Press release from MedPage Today discussing online prepublication in Science]

Tel Aviv University Develops Method for Tracking Adult Stem Cells as They Regress
Scientists have succeeded in tracking the progression of reprogrammed cells through live imaging to learn more about how they are reprogrammed, and how the new cells evolve over time. [Press release from Tel Aviv University discussing online prepublication in Nature Biotechnology]

Gene Regulating Human Brain Development Identified
A team of scientists has identified a single gene that seems to be a master regulator of human brain development, guiding undifferentiated stem cells down tightly defined pathways to becoming all of the many types of cells that make up the brain. [Press release from the University of Wisconsin-Madison discussing online prepublication in Cell Stem Cell]

Reprogrammed Human Blood Cells Show Promise for Disease Research
Cells from frozen human blood samples can be reprogrammed to an embryonic-stem-cell-like state, according to researchers. [Press release from the Whitehead Institute for Biomedical Research discussing online prepublication in Cell Stem Cell]

Chromosomal Abnormality Found for Inherited Clubfoot
By performing a routine genetic screening on 66 patients with an inherited form of clubfoot, researchers found abnormalities in a region of chromosome 17 in four patients. [Press release from Washington University School of Medicine discussing online prepublication in the American Journal of Human Genetics]

Ronin Recruits Protein Allies to Sustain Embryonic Stem Cell Growth
Ronin, crucial to the self-renewal of embryonic stem cells, and a co-regulator called Hcf-1 bind to a small strand of DNA called a hyperconserved enhancer element to control a gene “program” that stimulates growth of stem cells and may even play a role in cancer. [Press release from the Baylor College of Medicine discussing online prepublication in Genes and Development]

Stem Cell Therapy May Provide New Approach to Fight Infection
A new study suggests that a commonly used type of bone marrow stem cell may be able to help treat sepsis. [Press release from NewsWise discussing online prepublication in the American Journal of Respiratory and Critical Care Medicine]

Patients with Treatment-Resistant Chronic Leukemia Respond Positively to Stem Cell Transplants
Allergenic stem cell transplant may be a promising option for patients with treatment-resistant chronic lymphocytic leukemia, regardless of the patient's underlying genetic abnormalities. [Press release from PR Newswire discussing online prepublication in Blood]

Theraclone Sciences Technology Uncovers Rare Anti-Influenza Antibodies
Scientists have identified a new, highly conserved molecular target on the influenza A virus and demonstrated that human antibodies against this target are protective in animal models of seasonal and highly-pathogenic avian influenza. [Press release from Theraclone Sciences discussing online prepublication in the Proceedings of the National Academy of Sciences USA]

Inovio Pharmaceuticals' Novel Gene Immunoadjuvant Significantly Improves DNA Vaccine Immune Responses in Non-Human Primates
Researchers note that co-delivery of Inovio's immunoadjuvant, a DNA plasmid encoding interleukin-28B, with an Inovio optimized SynCon™ DNA vaccine using its proprietary electroporation technology significantly enhanced antigen-specific killer T cell responses in rhesus monkeys. [Press release from Inovio Pharmaceuticals, Inc. discussing online prepublication in Molecular Therapy]

Mechanism That May Trigger Degenerative Disease Identified
A mechanism that regulates stem cell differentiation in mice testes suggests a similar process that may trigger degenerative disease in humans. [Press release from Pennsylvania State University discussing online prepublication in Biology of Reproduction]

Reversal of Fortune for Parkinson's Disease Transplant Treatment
Researchers have overcome a major obstacle in the development of a transplant treatment which could relieve the symptoms of Parkinson's disease. [Press release from Imperial College London discussing online prepublication in Science Translational Medicine]

Cardio3 BioSciences Reports Positive Three-Month Data from Its Clinical Trial of C-Cure® in Heart Failure
Cardio3 BioSciences announced positive safety data and preliminary efficacy results from its clinical trial of C-Cure®, a breakthrough stem cell therapy for heart failure. [Cardio3 BioSciences Press Release]

Neurologix Announces Successful Phase II Trial of Gene Therapy for Parkinson's Disease
Study participants who received the investigational gene therapy NLX-P101 experienced statistically significant and clinically meaningful improvements in off-medication motor scores compared to control subjects who received sham surgery. [Neurologix Inc. Press Release]

AFREZZA™ Demonstrates Long-Term Efficacy in Controlling Blood Sugar Levels in Patients with Type 2 Diabetes
AFREZZA™ [insulin human (rDNA origin)] Inhalation Powder provides long-term glucose control comparable to usual insulin therapy but with a significantly reduced incidence of hypoglycemia and less weight gain in patients with type 2 diabetes. [Press release from MannKind Corporation discussing research presented at the 70th Scientific Sessions of the American Diabetes Association]

CURRENT PUBLICATIONS (Ranked by Impact Factor of the Journal)

Human Melanoma-Initiating Cells Express Neural Crest Nerve Growth Factor Receptor CD271
Researchers show that in melanomas, tumor stem cells can be isolated prospectively as a highly enriched CD271+ melanoma tumor stem cell population using a process that maximizes viable cell transplantation [Nature]

An Early T Cell Lineage Commitment Checkpoint Dependent on the Transcription Factor Bcl11b
Researchers found that Bcl11b is necessary for T lineage commitment in mice and is specifically required both to repress natural killer cell-associated genes and to down-regulate a battery of stem cell or progenitor cell genes at the pivotal stage of commitment. [Science]

Genetic Signatures of Exceptional Longevity in Humans
Researchers built a genetic model that includes 150 single-nucleotide polymorphisms (SNPs) and found that it could predict exceptional longevity with 77% accuracy in an independent set of centenarians and controls. Further in silico analysis revealed that 90% of centenarians can be grouped into 19 clusters characterized by different combinations of SNP genotypes of varying predictive value. [Science]

Dynamic Single-Cell Imaging of Direct Reprogramming Reveals an Early Specifying Event
Researchers used high-resolution, time-lapse imaging to trace the reprogramming process over two weeks from single mouse embryonic fibroblasts to pluripotency factor-positive colonies. [Nat Biotechnol]

Pax6 Is a Human Neuroectoderm Cell Fate Determinant
Researchers show that Pax6 is uniformly expressed in early neuroectoderm cells of human fetuses and those differentiated from human embryonic stem cells. [Cell Stem Cell]

Reprogramming of Human Peripheral Blood Cells to Induced Pluripotent Stem Cells
Researchers derived induced pluripotent stem cells (iPSCs) from frozen human peripheral blood samples. Some of the iPSCs had rearrangements of the T cell receptor, indicating that T cells can be reprogrammed to pluripotency. [Cell Stem Cell]

Familial Isolated Clubfoot Is Associated with Recurrent Chromosome 17q23.1q23.2 Microduplications Containing TBX4
Result suggests that chromosome 17q23.1q23.2 microduplication is a relatively common cause of familial isolated clubfoot and provides strong evidence linking clubfoot etiology to abnormal early limb development. [Am J Hum Genet]

Ronin/Hcf-1 Binds to a Hyperconserved Enhancer Element and Regulates Genes Involved in the Growth of Embryonic Stem Cells
Researchers report that Ronin (Thap11), which is essential for the self-renewal of embryonic stem cells, binds with its transcriptional coregulator, Hcf-1, to a highly conserved enhancer element that previously lacked a recognized binding factor. [Genes Dev]

Mesenchymal Stem Cells Reduce Inflammation while Enhancing Bacterial Clearance and Improving Survival in Sepsis
Data demonstrate the efficacy of mesenchymal stem cell therapy for experimental sepsis, and suggest that immunomodulatory cell therapy may be an effective adjunctive treatment to reduce sepsis-related morbidity and mortality. [Am J Respir Crit Care Med]

Allergenic Stem Cell Transplantation Provides Durable Disease Control in Poor-Risk Chronic Lymphocytic Leukemia: Long-Term Clinical and MRD Results of the GCLLSG CLL3X Trial
Allergenic stem cell transplantation for poor-risk chronic lymphocytic leukemia can result in long-term minimal residual disease (MRD)-negative survival in up to half of the patients independent of the underlying genomic risk profile. [Blood]

Human Antibodies Reveal a Protective Epitope that Is Highly Conserved Among Human and Nonhuman Influenza A Viruses
Researchers describe the isolation of a panel of monoclonal antibodies derived from the IgG+ memory B cells of healthy, human subjects that recognize a previously unknown conformational epitope within the ectodomain of the influenza matrix 2 protein, M2e. [Proc Natl Acad Sci U S A]

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U.S. Appeals Court Reinstates Stem Cell Suit
A U.S. appeals court reinstated a lawsuit that challenges an Obama administration policy for federal funding of some human embryonic stem cell research. [Court of Appeals, United States]

Research Budgets Face Austerity in 2012
The National Institutes of Health and other life sciences and biomedical research funding agencies will need to trim their budgets for 2012, which are to be planned over the summer, to satisfy a White House that is now stressing deficit reduction and belt-tightening after two years of stimulus funding-boosted research budgets. [The Office of Management and Budget, United States]

The Reality of Human Stem Cell Research in Europe
A new report from the European Science Foundation examines the key scientific questions for human stem cell research in the context of the rapidly emerging field of regenerative medicine. [European Science Foundation, European Union]

AstraZeneca Loses Generic Drug Case versus EU
A European Union (EU) court backed an EU antitrust decision against Anglo-Swedish drug maker AstraZeneca, lending weight to a regulatory crackdown on firms blocking cheaper drugs coming to market. [The General Court, European Union]

Strong Medicine for French Research
The medical-research adviser to France's president aims to shift power and money to universities. [National Research Agency, France]

Meeting of the National Advisory Council for Healthcare Research and Quality [Agency for Healthcare Research and Quality, United States]

Maryland Stem Cell Research Commission – Public Meeting Notice [Maryland Stem Cell Research Commission, United States]

Celgene to Acquire Abraxis BioScience Inc.
Celgene Corporation and Abraxis BioScience Inc. jointly announced the signing of a definitive merger agreement in which Celgene has agreed to acquire Abraxis BioScience. [Celgene Corporation Press Release]

Sanofi-aventis to Acquire TargeGen Inc.
Sanofi-aventis announced that it has signed an agreement for the acquisition of TargeGen Inc., a privately held U.S. biopharmaceutical company developing small molecule kinase inhibitors for the treatment of certain forms of leukemia, lymphoma and other hematological malignancies and blood disorders. [Sanofi-Aventis Press Release]

Lilly to Acquire Alnara Pharmaceuticals
Eli Lilly and Company and Alnara Pharmaceuticals, Inc. announced they have signed a definitive merger agreement whereby Lilly will acquire Alnara. [Alnara Pharmaceuticals Press Release]

Families of Spinal Muscular Atrophy (SMA) Collaboration with Nationwide Children’s for Gene Therapy Development
Families of Spinal Muscular Atrophy awarded a grant to the Nationwide Children’s Hospital at Ohio State University toward their Gene Therapy Development Program. This program works toward replacement of the entire SMN1 gene, and has the potential to be groundbreaking in the treatment of SMA patients. [Families of Spinal Muscular Atrophy Press Release]

Calistoga Pharmaceuticals Raises $40 Million in Series C Financing
Calistoga Pharmaceuticals, Inc. announced it has closed a $40 million Series C financing. The funds will be used to advance Calistoga Pharmaceuticals’ drug development pipeline of isoform-selective phosphatidylinositol 3 kinase inhibitors. [Calistoga Pharmaceuticals Press Release]

‘Next Generation’ Gene Sequencer to Determine the Genetic Links of Heart Disease
Funded by the National Institute for Health Research, the Next Generation Genetic Sequencer will initially be used for research purposes to enable clinicians to better understand heart disease by carefully categorizing patients into disease types and discovering the genetic factors that cause the disease. Over the next 10 years, researchers will look at 10,000 patients above the age of 16 years. [Royal Brompton Hospital Press Release]

Cyclacel Pharmaceuticals Announces FDA Orphan Drug Designation for Sapacitabine in Both AML And MDS
Cyclacel Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to the company’s sapacitabine (CYC682) product candidate for the treatment of both acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS). [Cyclacel Pharmaceuticals, Inc. Press Release]

Tarix Pharmaceuticals' TXA127 Receives FDA Orphan Drug Designation for Stem Cell Engraftment
Tarix Pharmaceuticals announced that its drug, TXA127, has been granted Orphan Drug designation from the U.S. Food and Drug Administration (FDA) as a treatment to enhance engraftment in patients receiving a stem cell transplant. [PR Newswire]

Pluristem is Advancing Toward Two Phase IIb Clinical Trials for Treatment of Critical Limb Ischemia and Moderate-Severe Claudication
Pluristem Therapeuitcs Inc. announced that following its meeting with the Paul Ehrlich Institute the company is advancing toward designing two Phase IIb clinical trials for its placenta-derived cell therapy product, PLX-PAD, for the treatment of Critical Limb Ischemia and Moderate-severe Claudication. [Pluristem Therapeuitcs Inc. Press Release]

Cutting Drug Prices Hampers New Development
A report by the European School of Management and Technology Competition Analysis, and commissioned by the drugmaker Novartis, said there was a direct link between strict regulation and low innovation in the sector. [Reuters]

NIH Director Formalizes Rejection of Diseased Stem Cell Lines
The director of the National Institutes of Health (NIH) approved eight more human embryonic stem cell lines for federal funding. His decision brings to 75 those lines now available to NIH-supported researchers under President Barack Obama's liberalized, 2009 policy.

Center for Scientific Review; Notice of Closed Meetings

Exceptions and Alternative Procedures Approved Under 21 CFR 640.120

Charter of the Allergenic Products Advisory Committee

FOOD AND DRUG ADMINISTRATION (United States)

Science Board Advisory Committee; Notice of Meeting [Docket No. FDA-2010-N-0001]

Guidance for Industry: Adaptive Design Clinical Trials for Drugs and Biologics

Medical Devices; Availability of Safety and Effectiveness Summaries for Premarket Approval Applications [Docket Nos. FDA-2010-M-0068, FDA-2010-M-0078, FDA-2010-M-0063, FDA-2010-M-0135, FDA-2010-M-0158]

EVENTS

Worldwide Innovative Networking (WIN) Symposium in Personalized Cancer Medicine
July 7-9, 2010
Paris, France

Translational Cancer Medicine 2010 – USA
July 11-14, 2010
San Francisco, United States

United Kingdom National Stem Cell Network (UKNSCN) Annual Scientific Conference
July 12-14, 2010
Nottingham, United Kingdom

2nd International Conference on Cellular and Molecular Bioengineering (ICCMB2)
August 2-4, 2010
Singapore City, Singapore

CHI's 5th Annual Immunotherapeutics and Vaccine Summit (ImVacS)
August 17-19, 2010
Cambridge, United States

Select Biosciences 3rd Annual Stem Cells Europe Conference
August 24-25, 2010
Edinburgh, Scotland

Select Biosciences 2nd Annual World Biobanking Summit
August 24-25, 2010
Edinburgh, Scotland

Select Biosciences Inaugural Cellular Therapy Summit
August 24-25, 2010
Edinburgh, Scotland

Viral Vectors in Gene Therapy: Applications and Novel Production Methods
August 26-September 4, 2010
Kuopio, Finland

Clinical Trials International Summit 2010
September 1-2, 2010
Shanghai, China

International Society for Cellular Therapy (ISCT) – Europe 2nd Regional Meeting
September 11-14, 2010
Belgirate, Italy

Hydra VI: The European Summer School on Stem Cells & Regenerative Medicine
September 11-17, 2010
Hydra, Greece

Joint Metastasis Research Society – American Association of Cancer Research Conference on Metastasis and the Tumor Microenvironment
September 12-15, 2010
Philadelphia, United States

Stem Cells USA & Regenerative Medicine Congress 2010
September 13-15, 2010
Philadelphia, United States

Select Biosciences 7th Annual RNAi & miRNA Europe
September 14-15, 2010
Dublin, Ireland

Select Biosciences Inaugural Epigenetics Europe Conference
September 14-15, 2010
Dublin, Ireland

Select Biosciences Inaugural Genomics Automation Europe Conference
September 14-15, 2010
Dublin, Ireland

Tissue Engineering & Regenerative Medicine International Society (TERMIS) – Asia Pacific 2010
September 15-17, 2010
Sydney, Australia

International Society for Cell and Gene Therapy of Cancer (ISCGT) Congress
September 27-29, 2010
Doha, Qatar

4th American Association of Cancer Research International Conference on Molecular Diagnostics in Cancer Therapeutic Development
September 27-30, 2010
Denver, United States

Select Biosciences 6th Annual Ion Channel Targets Conference
September 28-29, 2010
Washington, DC, United States

Cytokines 2010: Cytokines in Infectious Diseases, Autoimmune Disorders and Cancer
October 3-7, 2010
Chicago, United States

2010 World Stem Cell Summit
October 4-6, 2010
Detroit, United States

5th World Congress on Preventive & Regenerative Medicine (WCRM)
October 5-7, 2010
Hannover, Germany

17th Annual Meeting German Society for Gene Therapy (DG-GT e.V.)
October 6-9, 2010
Munich, Germany

COGEM Symposium: GM Viruses as Medicine: Panacea or Pandora's Box?
October 7, 2010
Amsterdam, The Netherlands

American Association of Blood Banks (AABB) Annual Meeting & Cellular Therapy and Transfusion Medicine Expo (CTTXPO) 2010
October 9-12, 2010
Baltimore, United States

Translational Cancer Research for Basic Scientists
October 17-22, 2010
Boston, United States

European Society of Gene and Cell Therapy (ESGCT) 18th Annual Congress
October 22-25, 2010
Milan, Italy

3rd Annual Dubai Congress on Anti-Aging and Aesthetic Medicine (DCAAAM)
October 26-27, 2010
Dubai, United Arab Emirates

World Stem Cells & Regenerative Medicine Congress Asia 2010
October 26-29, 2010
Singapore City, Singapore

Select Biosciences 3rd Annual Molecular Diagnostics World Congress
October 28-29, 2010
San Diego, United States

Fraunhofer Life Science Symposium 2010
October 29-30, 2010
Leipzig, Germany

Stem Cell Society Singapore Symposium 2010: Translating Science to Therapy
November 2-3, 2010
Singapore City, Singapore

World Cord Blood Congress
November 4-7, 2010
Marseille, France

Select Biosciences 5th Annual European Biomarkers Summit
November 9-10, 2010
Florence, Italy

Select Biosciences International Forum on Stem Cells
November 12-13, 2010
Tianjin, China

Select Biosciences 2nd Annual RNAi Asia Conference
November 15-16, 2010
Singapore City, Singapore

9th Annual Gene Therapy Symposium for Heart, Lung, and Blood Diseases
November 17-19, 2010
Sonoma, United States

Stem Cell Network 10th Annual Scientific Meeting
November 22-24, 2010
Calgary, Canada

Tissue Engineering & Regenerative Medicine International Society (TERMIS) – North America 2010 Annual Conference
December 5-8, 2010
Orlando, United States

Phacilitate 7th Annual Cell & Gene Therapy Forum 2011
January 24-26, 2011
Washington DC, United States

Visit our events page to stay up to date with the latest events in the cell, gene and immunotherapy community.

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