Volume 11.30 | Aug 9

Cell Therapy News 11.30, August 9, 2010.
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Scientists Discover New Method for Regenerating Heart Muscle by Direct Reprogramming
Scientists directly reprogrammed fibroblasts in the heart to become cardiomyocytes. In doing so, they also found the first evidence that unrelated adult cells can be reprogrammed from one cell type to another without having to go all the way back to a stem cell state. [Press release from the Gladstone Institute of Cardiovascular Disease discussing online prepublication in Cell]



NIH Genomic Mapping Study Finds Largest Set of Genes Related to Major Risk Factor for Heart Disease
Scanning the genomes of more than 100,000 people from all over the world, scientists report the largest set of genes discovered underlying high cholesterol and high triglycerides. Taken together, the gene variants explain between a quarter and a third of the inherited portions of cholesterol and triglyceride measured in the blood. [Press release from the National Institutes of Health discussing online prepublication in Nature]

Molecular Mechanism Triggering Parkinson's Disease Identified in Study
Scientists have identified a molecular pathway responsible for the death of key nerve cells whose loss causes Parkinson’s disease. [Press release from Stanford University School of Medicine discussing online prepublication in Nature]

Scientists Discover Protein that Shuttles RNA into Mitochondria
Researchers have uncovered a role for an essential cell protein in shuttling RNA into the mitochondria. [Press release from the University of California, Los Angeles Jonsson Comprehensive Cancer Center discussing online prepublication in Cell]

Scientists Find Gas Pedal – and Brake – for Uncontrolled Cell Growth
Researchers have identified a new way to regulate the uncontrolled growth of blood vessels, a major problem in a broad range of diseases and conditions. [Press release from the University of California, San Diego discussing online prepublication in Nature Medicine]

Newts' Ability to Regenerate Tissue Replicated in Mouse Cells by Scientists
Scientists have taken a big step toward being able to confer the regenerative capacity of newts on mammalian muscle cells; they accomplished this feat in experiments with laboratory mice in which they blocked the expression of two tumor-suppressing proteins. [Press release from Stanford University School of Medicine discussing online prepublication in Cell Stem Cell]

Alphavirus-Based Vaccine May Slow Some Cancers
An experimental vaccine based on a virus that causes encephalitis in the wild appears to block tumor growth in some cases of advanced cancer. Scientists say the vaccine is able to stimulate an immune response, even in the face of profound immune system suppression. [Press release from Duke University Medical Center discussing online prepublication in the Journal of Clinical Investigation]

Epileptic Seizures May Be Linked to an Ancient Gene Family
Researchers have identified an ancient gene family that plays a role in regulating the excitability of nerves within the brain. [Press release from the Pennsylvania State University discussing online prepublication in Nature Neuroscience]

Cellular Suicide Spurs Cancer
Two studies looked at the role of a protein called PUMA, which triggers cell death in response to damaged DNA. Both research teams observed the same phenomenon: the absence of the suicide switch protected the mice, not harmed them. [Press release from NatureNews discussing online prepublication in Genes and Development]

Researchers Find Key Step in Body's Ability to Make Red Blood Cells
Researchers found that a tiny fragment of RNA prompts stem cells to mature into red blood cells. They also created an artificial RNA inhibitor to block this process. [Press release from the University of Texas Southwestern Medical Center discussing online prepublication in Genes and Development]

Researchers Identify a Gene Affecting Chronic Pain; Open Way for New Treatment Approach
Researchers have succeeded in identifying a gene associated with susceptibility to chronic pain caused by nerve injury in humans, signaling a significant step toward better understanding and treating of the condition. [Press release from the Hebrew University of Jerusalem discussing online prepublication in Genome Research]

Taiwan Doctors Control Tumor Using Gene Therapy
Doctors have succeeded in controlling a tumor in rodents using gene therapy, and plan to conduct clinical tests on human beings soon. [Press release from Earth Times discussing online prepublication in the Proceedings of the National Academy of Sciences USA]

Scientists Target Possible Cause of One Form of Bowel Disease
A possible cause of irritable bowel syndrome has been traced to a small piece of RNA that blocks a substance protecting the colon membrane, leading to hostile conditions that can produce diarrhea, bloating and chronic abdominal pain. [Press release from Ohio State University discussing online prepublication in Gut]

Gene Brings Teeth in Shape
No healthy teeth without this gene: if during tooth formation the Jagged2 gene is inactivated, and hence the Notch signaling pathway interrupted, tooth crowns will be malformed and enamel will be lacking. [Press release from ScienceDaily discussing online prepublication in Development]

Human Embryonic Stem Cells Purified in New, Rapid Technique
Researchers are reporting the first success in very rapidly purifying one type of embryonic stem cell from a mix of many different types of embryonic stem cells in the culture dish. The technique avoids the need to genetically alter the cells to distinguish them. [Press release from the University of California, San Francisco discussing online prepublication in Stem Cells and Development]

“Genome Mining” May Yield New Drugs
Researchers have discovered a promising new antibiotic using the “genome mining” approach. The drug was extracted from the soil bug Streptomyces after the scientists found and activated a particular group of dormant genes in the microbe. [Press release from the Press Association discussing online prepublication in Microbiology]

Scientists Develop “Designer Protein,” Opening New Door in Cancer Research
Scientists have identified a way to target and destroy Glioblastoma multiforme cells without harming healthy cells. [Press release from Wake Forest University Baptist Medical Center discussing online prepublication in Genes and Cancer]

World's First Demonstration of Successful Adult Brain Cell Re-Growth Confirmed at Canadian Centre for Behavioural Neuroscience
A research team has confirmed — through extensive research and peer-reviewed testing — the world's first successful example of brain cell re-growth in an adult mammal. [University of Lethbridge Press Release]

Herpes Virus Treats Head and Neck Cancer Patients
A genetically engineered cold sore virus has been used to treat head and neck cancer patients in a Phase I/II clinical trial. [Institute of Cancer Research Press Release]

Invention’s Bit of Gold Goes a Long Way
A researcher has combined nanoscale-sized-particles of gold with two powerful tools in molecular biology to make DNA analysis more than 10 times faster at room temperature, rather than previously required higher temperatures. [Simon Fraser University Press Release]

CURRENT PUBLICATIONS (Ranked by Impact Factor of the Journal)

Biological, Clinical and Population Relevance of 95 Loci for Blood Lipids
Researchers screened the genome for common variants associated with plasma lipids in more than 100,000 individuals of European ancestry. Here they report 95 significantly associated loci, with 59 showing genome-wide significant association with lipid traits for the first time. [Nature]

Pathogenic LRRK2 Negatively Regulates MicroRNA-Mediated Translational Repression
Results implicate deregulated synthesis of E2F1/DP caused by the miRNA pathway impairment as a key event in LRRK2 pathogenesis and suggest novel miRNA-based therapeutic strategies. [Nature]

Direct Reprogramming of Fibroblasts into Functional Cardiomyocytes by Defined Factors
Researchers report that a combination of three developmental transcription factors (Gata4, Mef2c, and Tbx5) rapidly and efficiently reprogrammed postnatal cardiac or dermal fibroblasts directly into differentiated cardiomyocyte-like cells. [Cell]

PNPASE Regulates RNA Import into Mitochondria
Researchers show a new role for PNPASE in regulating the import of nuclear-encoded RNAs into the mitochondrial matrix. [Cell]

MicroRNA-132-Mediated Loss of p120RasGAP Activates the Endothelium to Facilitate Pathological Angiogenesis
Researchers show that the microRNA miR-132 acts as an angiogenic switch by targeting p120RasGAP in the endothelium and thereby inducing neovascularization. [Nat Med]

Transient Inactivation of Rb and ARF Yields Regenerative Cells from Postmitotic Mammalian Muscle
Results show that differentiation of mammalian cells is reversed by inactivation of Arf and Rb and support the hypothesis that Arf evolved at the expense of regeneration. [Cell Stem Cell]

An Alphavirus Vector Overcomes the Presence of Neutralizing Antibodies and Elevated Numbers of Tregs to Induce Immune Responses in Humans with Advanced Cancer
Data suggest that virus-like replicon particle-based vectors can overcome the presence of neutralizing antibodies to break tolerance to self antigen and may be clinically useful for immunotherapy in the setting of tumor-induced immunosuppression. [J Clin Invest]

Deletion of the Potassium Channel Kv12.2 Causes Hippocampal Hyperexcitability and Epilepsy
Genetic deletion and pharmacologic block of Kv12.2 substantially reduced the firing threshold of hippocampal pyramidal neurons. [Nature Neurosci]

Apoptosis of Leukocytes Triggered by Acute DNA Damage Promotes Lymphoma Formation
Mice defective in p53-induced apoptosis due to loss of its proapoptotic target gene, puma, resist gamma-irradiation-induced lymphomagenesis. [Genes Dev]

Apoptosis-Promoted Tumorigenesis: Gamma-Irradiation-Induced Thymic Lymphomagenesis Requires Puma-Driven Leukocyte Death
Tumor suppression by Puma deficiency reflected its protection of leukocytes from gamma-irradiation-induced death, because their glucocorticoid-mediated decimation in Puma-deficient mice activated cycling of stem/progenitor cells and restored thymic lymphomagenesis. [Genes Dev]

Defective Erythroid Differentiation in miR-451 Mutant Mice Mediated By 14-3-3Zeta
Researchers show that microRNA-451 (miR-451) regulates erythropoiesis in vivo. Mice lacking miR-451 display a reduction in hematrocrit, an erythroid differentiation defect, and ineffective erythropoiesis in response to oxidative stress. [Genes Dev]



Scientists Decry Proposed Ban on Chimp Research
In the latest twist in the protracted debate over the use of chimpanzees in biomedical research, U.S. senators introduced the Great Ape Protection Act, an identical bill to one that has been stuck in a House of Representatives committee for more than a year. [Senate, United States]

Senate Bill Would Encourage Drugs Targeting Rare Kids' Diseases
A group of bipartisan senators introduced legislation to entice drug makers to focus more intently on cures for uncommon children's diseases. [Senate, United States]

Decision Memo for Allogeneic Hematopoietic Stem Cell Transplantation for Myelodysplastic Syndrome (CAG-00415N)
The Centers for Medicare and Medicaid Services has determined that the evidence does not demonstrate that the use of allogeneic hematopoietic stem cell transplantation improves health outcomes in Medicare beneficiaries with myelodysplastic syndrome. [Centers for Medicare and Medicaid Services, United States]

Solicitation for Nominations: New Clinical Preventive Health for the United States Preventive Services Task Force, [2010–19117] [Agency for Healthcare Research and Quality, United States]

Advisory Commission on Childhood Vaccines; Notice of Meeting [2010–19120] [Health Resources and Services Administration, United States]



Five Prime Therapeutics, Inc. Announces Strategic Alliance with GlaxoSmithKline to Discover Innovative Biologics for Skeletal Muscle Disorders
Five Prime Therapeutics, Inc. announced that it has formed a focused strategic drug discovery alliance with GlaxoSmithKline. [Press Release]

Axiogenesis In-Licenses Yamanaka Patent Portfolio from iPS Academia Japan Covering Induced Pluripotent Stem Cell Technology
Axiogenesis AG and iPS Academia Japan Inc. announced that they have executed a non-exclusive agreement with iPS Academia through which Axiogenesis obtains worldwide access to the extensive portfolio of induced pluripotent stem cell (iPSC) technology for iPSC-derived cardiomyocytes and other cell types, pioneered by Professor Shinya Yamanaka, Center for iPS Cell Research and Application at the University of Kyoto, Japan. [BusinessWire]

Seattle Genetics Expands Antibody-Drug Conjugate Collaboration with Genentech
Seattle Genetics, Inc. announced that it has expanded its antibody-drug conjugate collaboration agreement with Genentech, Inc. [Seattle Genetics, Inc. Press Release]

GlaxoSmithKline and Amplimmune Form Global Strategic Collaboration
Amplimmune, Inc. announced that it has entered into a broad strategic alliance with GlaxoSmithKline (GSK) to further develop PD-1 targeting therapies that may be effective in the treatment of cancer and other diseases. GSK will obtain exclusive worldwide rights to AMP-224 as well as other potential next generation fusion proteins that target PD-1. [Amplimmune, Inc. Press Release]

Movetis Receives a Tender Offer from Shire of EUR19 Cash Per Share for All Outstanding Shares and Warrants
Movetis NV announced that it has received a conditional offer from a Luxembourg incorporated wholly-owned subsidiary of Shire plc to acquire all outstanding shares and warrants of Movetis NV at an offer price of EUR19.00 in cash per share, valuing the Company’s equity, on a fully diluted basis, at EUR428 million. [Movetis NV Press Release]

Tekmira Pharmaceuticals Files IND Application with U.S. FDA for PLK1 SNALP
Tekmira Pharmaceuticals Corporation announced it has filed an Investigational New Drug (IND) application seeking approval from the United States Food and Drug Administration (FDA) to begin a Phase I human clinical trial to evaluate PLK1 SNALP as a treatment for solid tumor cancers. [Tekmira Pharmaceuticals Corporation Press Release]

ACT Files Documentation with FDA for Clinical Trials Using ES Cells to Treat Eye Disease
Advanced Cell Technology, Inc. (ACT) announced that it has submitted documentation and a complete response to substantively address the issues raised by the U.S. Food and Drug Administration (FDA) in connection with the Company’s plans to initiate a Phase I/II multicenter study using embryonic stem (ES) cell derived retinal cells to treat patients with Stargardt’s Macular Dystrophy. [Advanced Cell Technology, Inc. Press Release]

Panel Recommends Defining Select Agents by DNA Sequence
The way to bring synthetically designed genomes that could be potentially as deadly as known pathogens under regulation, according to a new National Academies report, is to develop a new system of defining select agents based on DNA sequences. [ScienceInsider]

Free Access to U.S. Research Papers Could Yield $1 Billion in Benefits
A new economic analysis finds that making taxpayer-funded scientific papers freely available would yield more than $1 billion in benefits to the U.S. economy over 30 years–five times the costs of archiving the papers. [ScienceInsider]


Advanced Technologies for Detection of Perturbation-Induced Cellular Signatures (U01) (RFA-RM-10-004)

Technical Assistance Videocast for RFA-RR-10-007: Clinical and Translational Science Coordinating Center (U54) (NOT-RR-10-012)

NIH Common Fund Transformative Research Projects Program (R01) (RFA-RM-10-010)

Chimpanzee Biomedical Research Program Limited Competition (U42) (RFA-RR-10-008)

Notice of Intent to Publish a Request for Applications for a Parkinson Disease Biomarkers Identification Network Coordinating Center (U01) (NOT-NS-10-024)

Proposed Collection; Comment Request; Assessing the Long-Term Impacts of the John E. Fogarty International Center's Research and Training Programs (FR Doc. 2010-19160)

Center for Scientific Review; Notice of Closed Meeting (FR Doc. 2010-19167)

National Human Genome Research Institute; Notice of Meeting (FR Doc. 2010-19165)


2010 Biological License Application Approvals

2010 Biological License Application Supplement Noteworthy Approvals

2010 Biological Device Application Approvals



FDA Lifts Hold on First-Ever Embryonic Stem Cell Trial
Regulators have given a green light to the world's first approved experiment using embryonic stem cells to treat a human disease. In the Phase I clinical trial, Geron Corp. will use stem cells to treat spinal cord injury.

FDA Issues Assessments of the 510(k) Program and Use of Science in Decision-Making
The U.S. Food and Drug Administration issued two comprehensive evaluations containing recommendations that address three key objectives of the agency’s public health mission as it relates to medical devices — foster device innovation, create a more predictable regulatory environment, and enhance device safety.

Agency Information Collection Activities; Proposed Collection; Comment Request; Generic Food and Drug Administration Rapid Response Surveys [Docket No. FDA-2010-N-0380]

Agency Information Collection Activities; Submission for Office of Management and Budget Review; Comment Request; Administrative Procedures for the Clinical Laboratory Improvement Amendments of 1988 Categorization [Docket No. FDA-2010-N-0199]

Clinical Studies of Safety and Effectiveness of Orphan Products Research Project Grant (R01) [Docket No. FDA-2010-N-0394]

Draft Guidance for Industry and Food and Drug Administration Staff; Medical Devices; Neurological and Physical Medicine Device Guidance Document; Reopening of Comment Period; Correction [Docket No. FDA-2009-D-0495]

Guidance for Industry on Drug Substance Chemistry, Manufacturing, and Controls Information; Availability [Docket No. FDA-2003-D-0243]

Vaccines and Related Biological Products Advisory Committee; Notice of Meeting [Docket No. FDA-2010-N-0001]


Information Sent to Healthcare Professionals in July 2010 About the Safety of Medicines
A list of letters was sent to healthcare professionals in July 2010 to inform of new safety information and advice about medicines.

Adverse Drug Reactions: Update on Anonymized Single Patient Reports (ASPRs): July 2010
The MHRA has implemented a series of changes to the logic which defines which marketing authorization holders receive ASPRs/Individual Case Safety Reports.

EVENTS (Listed by Date)

NEW Securing European Funding for Biomaterials in Medicine and Healthcare: Opportunities and Approach
August 12, 2010
Runcorn, United Kingdom

XXIII International Congress of the Transplantation Society
August 15-19, 2010
Vancouver, Canada

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

European Workshop on Skin Stem Cells
August 31-September 2, 2010
Cambridge, United Kingdom

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

Third Annual Limbal Stem Cell Meeting
September 10, 2010
Reading, United Kingdom

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

First Annual BIO India International Partnering Conference
September 21-22, 2010
Hyderabad, India

Cellular Therapy of Cancer Symposium – ATTACK Project Meeting
September 24-27, 2010
Montpellier, France

International Society for Cellular Therapy 2nd Annual Symposium on Stem Cell Translation: Best Practices & Regulatory Considerations
September 27-28, 2010
San Francisco, United States

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

NEW 9th Annual BIO Investor Forum
October 5-6, 2010
San Francisco, 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

18th Annual BioPartnering Europe
October 10-12, 2010
London, United Kingdom

New York Stem Cell Foundation Fifth Annual Translational Stem Cell Research Meeting
October 12-13, 2010
New York, United States

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

2010 NanoMedicine Summit
October 18-19, 2010
Cleveland, United States

NEW BIO Intellectual Property Counsels Committee Fall Conference and Committee Meeting
October 18-20, 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

NEW BIO-Europe International Partnering Conference
November 15-17, 2010
Munich, Germany

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

8th Annual Commercial Translation of Regenerative Medicine Conference
November 18-19, 2010
London, United Kingdom

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

HealthTech and Medicines Knowledge Transfer Network 7th Annual BioProcessUK Conference
November 24-25, 2010
Manchester, United Kingdom

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

Select Biosciences 5th Annual Stem Cells World Congress
January 24-25, 2011
San Diego, United States

NEW BIO Asia International Partnering Conference
January 24-25, 2011
Tokyo, Japan

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.



Cell Therapy Technologist (Opexa Therapeutics)

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