Volume 11.22 | Jun 14

Cell Therapy News 11.22, June 14, 2010.
In this issue:  Science  |  Policy  |  Business  |  NIH  |  CBER  |  Regulatory  |  Events Subscribe  |  Unsubscribe


TOP STORY


Stem Cells for First Time Used to Create Abnormal Heart Cells for Study of Heart Condition
Researchers have for the first time differentiated human stem cells to become heart cells with cardiomyopathy. [Press release from the Mount Sinai Medical Center discussing online prepublication in Nature]

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SCIENCE NEWS


Rare Genetic Variations Involving Whole Sections of DNA Implicated in Autism
Losses and duplications of whole chunks of DNA at sites across our genomes are likely to play a role in autism spectrum disorders, suggests a new international study. [Press release from the Wellcome Trust Centre for Human Genetics discussing online prepublication in Nature]

Down's Syndrome Chromosome Yields More Cancer-Blocking Genes
Scientists have discovered cancer-blocking activity for genes carried on chromosome 21 — an extra copy of which is carried by people with Down’s syndrome. [Press release from Cancer Research UK discussing online prepublication in Nature]

Researchers Discover How Mutations in Presenilin Gene Cause Early-Onset Alzheimer's Disease
Researchers have discovered how mutations in the presenilin 1 gene cause early-onset Alzheimer's disease. [Press release from New York University Langone Medical Center discussing online prepublication in Cell]

Scientists’ Finding of an Ancient Viral Invasion that Shaped the Human Genome an Important Step Towards Advancement in Regenerative Medicine
Scientists have discovered that viruses that ‘invaded’ the human genome millions of years ago have changed the way genes get turned on and off in human embryonic stem cells. [Press release from the Genome Institute of Singapore discussing online prepublication in Nature Genetics]

Gene Swaps May Make Cancers Vulnerable
Researchers have identified a set of hybrid genes that may accelerate the growth of prostate cancer, gastric cancer, and melanoma. [Press release from Howard Hughes Medical Institute discussing online prepublication in Nature Medicine]

Scientists Take First Steps in Growing Working Livers
Scientists have created working liver grafts in the lab, and say the research could one day allow the growth of livers for transplant. [Press release from BBC Science News discussing online prepublication in Nature Medicine]

Reprogramming Immune System Cells to Produce Natural Killer Cells for Cancer
Researchers produced a new type of cell by knocking out a single gene essential in the pathways of development of immune cells: the modified cells become a novel type, which the authors call Induced T to Natural Killer Cells. [Press release from the Wellcome Trust Sanger Institute discussing online prepublication in Science]

Novel 'Cell Wires' to Patch up Heart or Nerve Damage
Researchers have discovered a way to align huge arrays of nano-sized filaments and living cells over macroscopic distances. [Press release from the Royal Society of Chemistry discussing online prepublication in Nature Materials]

Researchers Discover Mechanism that Limits Scar Formation
Researchers have discovered that an unexpected cellular response plays an important role in breaking down and inhibiting the formation of excess scar tissue in wound healing. [Press release from the University of Illinois at Chicago discussing online prepublication in Nature Cell Biology]

New Type of Human Stem Cell May Be Easier to Manipulate
Researchers have developed a new type of human pluripotent stem cell that can be manipulated more readily than currently available stem cells. [Press release from the Massachusetts General Hospital Center for Regenerative Medicine discussing online prepublication in Cell Stem Cell]

Ironing Out Inflammation: Substance in Iron Metabolism Displays Life-Saving Potential
In a surprising discovery that someday may lead to new treatments for many inflammatory diseases, scientists found that a hormone involved in iron metabolism can save mice from deadly acute inflammation. [Press release from the University of Utah discussing online prepublication in the Journal of Clinical Investigation]

How the Wrong Genes Are Repressed
The mechanism by which ‘polycomb’ proteins critical for embryonic stem cell function and fate are targeted to DNA has been identified by scientists. [Press release from University College London discussing online prepublication in Molecular Cell]

Obstacles to Stem Cell Therapy Cleared
Researchers have come up with a new technique to prevent tumors developing in connection with stem cell transplantations. [Press release from Lund University discussing online prepublication in the Proceedings of the National Academy of Sciences USA]

Mount Sinai Researchers Approaching Universal Treatment for All Strains of Influenza
Researchers have, for the first time, identified a small-viral RNA, derived from the influenza virus, that is integral to the switch from transcription to replication. [Press release from the Mount Sinai School of Medicine discussing online prepublication in the Proceedings of the National Academy of Sciences USA]

Gene Related to Aging Plays Role in Stem Cell Differentiation
A gene shown to play a role in the aging process appears to play a role in the regulation of the differentiation of embryonic stem cells. [Press release from Thomas Jefferson University Hospital discussing online prepublication in Aging Cell]

Researchers Find Way to Prevent Blindness in Research Model for Retinitis Pigmentosa
Researchers have found a way to use a radical new type of gene therapy to prevent blindness caused by retinitis pigmentosa. [Press release from The University of Oklahoma Health Sciences Center discussing online prepublication in the Journal of the Federation of American Societies for Experimental Biology]

Innovative University of Virginia Technology Could Make Fat the Cure for What Ails You
New technology involving adipose stem cells could one day be used to treat patients with severe wounds and other serious conditions. [Press release from the University of Virginia discussing online prepublication in Tissue Engineering, Part A]

New Therapy Offers Hope to Spinal Muscular Atrophy (SMA) Patients
Research has shown that a novel gene transfer system has the potential to provide an effective therapeutic treatment for SMA patients. [Press release from the University of Sheffield discussing online prepublication in Science Translational Medicine]

Tissue Regeneration Research Could Help Keep Seniors Active
A researcher is currently trying to develop microscopic polymer fibers to help rebuild human tissue and speed the healing process. [Press release from Queen's University discussing research presented at the Advanced Foods and Materials Network 6th Annual Conference]

Argos Therapeutics Presents Positive Phase II Data for Arcelis™ Dendritic Cell-based Immunotherapy in Advanced RCC
Argos Therapeutics announced the presentation of positive interim data from a Phase II trial that evaluated the clinical activity, safety and immune response of AGS-003 treatment, given in combination with sunitinib, in patients with newly diagnosed advanced renal cell carcinoma (RCC). [Press release from Argos Therapeutics, Inc. discussing research presented at the 46th Annual Meeting of the American Society of Clinical Oncology]

Reduced-Dose Velcade-Thalidomide-Dexamethasone Combination Shows Promise as Initial Therapy Prior to Stem Cell Transplantation
The combination treatment of reduced-dose Velcade (bortezomib), thalidomide (Thalomid), and dexamethasone (Decadron), abbreviated vTD, appears to be a safe and effective induction treatment for newly diagnosed multiple myeloma patients prior to stem cell transplantation. [Press release from the Myeloma Beacon discussing research presented at the 46th Annual Meeting of the American Society of Clinical Oncology]

Oxford BioMedica Announces Two-Year Phase I/II Results of ProSavin® In Parkinson's Disease
Oxford BioMedica announced new data from the ongoing Phase I/II trial of ProSavin®, its novel gene therapy for the treatment of Parkinson’s disease. [Press release from Oxford Biomedica plc discussing research presented at the 9th Annual Congress of the French Society of Cell and Gene Therapy]

CURRENT PUBLICATIONS (Ranked by Impact Factor of the Journal)

Patient-Specific Induced Pluripotent Stem-Cell-Derived Models of LEOPARD Syndrome
Researchers show that in vitro-derived cardiomyocytes from LEOPARD syndrome induced pluripotent stem cells (iPSCs) are larger, have a higher degree of sarcomeric organization and preferential localization of NFATC4 in the nucleus when compared with cardiomyocytes derived from human embryonic stem cells or wild-type iPSCs derived from a healthy brother of one of the LEOPARD syndrome patients. [Nature]

Functional Impact of Global Rare Copy Number Variation in Autism Spectrum Disorders
Results reveal many new genetic and functional targets in autism spectrum disorders that may lead to final connected pathways. [Nature]

Tumor Angiogenesis Is Reduced in the Tc1 Mouse Model of Down’s Syndrome
Researchers expect that, in addition to the candidate genes that they show to be involved in the repression of angiogenesis, the Tc1 mouse model of Down’s syndrome will permit the identification of other endothelium-specific anti-angiogenic targets relevant to a broad spectrum of cancer patients. [Nature]

Lysosomal Proteolysis and Autophagy Require Presenilin 1 and Are Disrupted by Alzheimer-Related PS1 Mutations
Researchers show that macroautophagy, a lysosomal degradative pathway essential for neuron survival, requires the Alzheimer's disease-related protein presenilin-1. [Cell]

Transposable Elements Have Rewired the Core Regulatory Network of Human Embryonic Stem Cells
Data indicate that species-specific transposable elements have substantially altered the transcriptional circuitry of pluripotent stem cells. [Nat Genet]

Rearrangements of the RAF Kinase Pathway in Prostate Cancer, Gastric Cancer and Melanoma
Results emphasize the key role of RAF family gene rearrangements in cancer, suggest that RAF and MEK inhibitors may be useful in a subset of gene fusion–harboring solid tumors and demonstrate that sequencing of tumor transcriptomes and genomes may lead to the identification of rare targetable fusions across cancer types. [Nat Med]

Organ Reengineering Through Development of a Transplantable Recellularized Liver Graft Using Decellularized Liver Matrix
Results provide a proof of principle for the generation of a transplantable liver graft as a potential treatment for liver disease. [Nat Med]

Reprogramming of T Cells to Natural Killer-Like Cells upon Bcl11b Deletion
Researchers show that the transcription factor Bcl11b was expressed in all T cell compartments and was indispensable for T-lineage development. When Bcl11b was deleted, T cells from all developmental stages acquired NK cell properties and concomitantly lost or decreased T cell-associated gene expression. [Science]

A Self-Assembly Pathway to Aligned Monodomain Gels
The nature of the self-assembly process and its biocompatibility would allow formation of cellular wires in situ that have any length and customized peptide compositions for use in biological applications. [Nat Mater]

The Matricellular Protein CCN1 Induces Fibroblast Senescence and Restricts Fibrosis in Cutaneous Wound Healing
Researchers show that the matricellular protein CCN1, which is dynamically expressed at sites of wound repair, can induce fibroblast senescence by binding to integrin alpha6beta1 and the heparan sulphate proteoglycans. [Nat Cell Biol]

A Murine ESC-Like State Facilitates Transgenesis and Homologous Recombination in Human Pluripotent Stem Cells
Researchers demonstrate that human induced pluripotent stem cell reprogramming in the presence of LIF yields human stem cells that display morphological, molecular, and functional properties of murine embryonic stem cells (ESCs). [Cell Stem Cell]

MammoCultBreastCancer_645x110_v01




POLICY


Hospital Clinic de Barcelona Obtained the First Accreditation for Cell Therapy Research
The certification, awarded for the first time in Catalonia, allows the Hospital Clinic de Barcelona to produce drugs in gene therapy, cell therapy and tissue engineering. [Spanish Medicines Agency, Spain]

Final Report of The National Academies' Human Embryonic Stem Cell Research Advisory Committee and 2010 Amendments to the National Academies' Guidelines for Human Embryonic Stem Cell Research [National Academy of Sciences, United States]

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BUSINESS


Cellectis Bioresearch and Lonza Enter Development and Commercial Manufacturing Agreement
Cellectis Bioresearch and Lonza announced that the companies have entered into an agreement for the development and commercialization of a bioengineered cell line. [Cellectis Bioresearch Press Release]

Lonza Announces Worldwide, Exclusive Licensing and Supply Agreements with California Stem Cell Inc.
Lonza Inc. has entered worldwide, exclusive licensing and supply agreements with California Stem Cell Inc. This collaboration makes novel pluripotent stem cell products readily available to researchers worldwide. [Lonza, Inc. Press Release]

Connecticut Awards $9.8 Million in Stem Cell Research Grants
Connecticut state has awarded $9.8 million in grants to support nearly two dozen stem cell research projects by scientists at the University of Connecticut and at Yale. [State of Connecticut Press Release]

ISSCR Launches Web Site Providing Information on Stem Cell Treatments
The International Society for Stem Cell Research (ISSCR) announced that it has launched “A Closer Look at Stem Cell Treatments” (www.closerlookatstemcells.org), a web site to arm patients, their families and doctors with information they need to make decisions about stem cell treatments. [International Society for Stem Cell Research Press Release]

International Stem Cell Corporation’s Parthenogenetic Stem Cell Patent is Approved by the United States Patent and Trademark Office
International Stem Cell Corporation announced that the United States Patent and Trademark Office has granted ISCO patent number 7,732,202, the first of several pending patents relating to its development of human parthenogenetic stem cells. [International Stem Cell Corporation Press Release]

NIH Approves Advanced Cell Technology's Stem Cell Line for Federal Funding
Advanced Cell Technology, Inc. announced that human embryonic stem cell line MA135 was unanimously approved for federal funding at the 100th Meeting of the Advisory Committee to the Director National Institutes of Health (NIH). [Advanced Cell Technology, Inc. Press Release]

Human Genome Sciences Announces Submission of Biologics License Application to FDA for Benlysta® (Belimumab)
Human Genome Sciences, Inc. announced that it has submitted a Biologics License Application to the U.S. Food and Drug Administration for approval to market BENLYSTA® (belimumab) for the treatment of systemic lupus erythematosus. [Human Genome Sciences, Inc. Press Release]

Faster Treatment of Heart Patients with a Finger Prick of Blood: New Bedside Tool to Detect Rare Endothelial Progenitor Cells
From sample to answer in just one hour with just a finger prick of blood is what researchers aim to achieve with their novel microfluidic system for rapid and sensitive detection of rare circulating endothelial progenitor cells. [A*STAR Institute of Microelectronics Press Release]

Stem Cell Therapeutics Corp. Announces the Issuance of United States Patent
Stem Cell Therapeutics Corp. announced that it has been granted U.S. Patent 7,704,737 entitled, “Oligodendrocyte Production from Multipotent Neural Stem Cells.” [Stem Cell Therapeutics Corp. Press Release]




NIH


NIH Chief Collins Faces Stem Cell Donation Dilemma
The NIH Advisory Committee to the Director, which advises NIH chief Francis Collins on whether lines meet the Obama ethics guidelines, faced the decision on whether 53 more stem cell lines should be added to that total.

NIH Researchers Explore How Healthy, Young Adults View the Role Genetics Plays in Improving Health
Most healthy young adults place greater emphasis on health habits than on genetic risk factors when considering what causes common diseases, a research team from the National Human Genome Research Institute and the Henry Ford Health System in Detroit has found.

Enhancing Peer Review: Advance Notice on Post-Submission Application Materials for NIH Training and Related Applications (NOT-OD-10-104)

Notice of Availability of NINDS Administrative Supplements for Promoting Optogenetics Applications in Basic and Translational Neuroscience Research (NOT-NS-10-021)

PFINDR: Phenotype Finder IN Data Resources: A Tool to Support Cross-study Data Discovery Among NHLBI Genomic Studies (UH2/UH3) (RFA-HL-11-020)

IMT_645x110


 


CBER


Charter of the Vaccines and Related Biological Products Advisory Committee
The Vaccines and Related Biological Products Advisory Committee advises the Commissioner or designee in discharging responsibilities as they relate to helping to ensure safe and effective drugs for human use and, as required, any other product for which the Food and Drug Administration has regulatory responsibility.

Roster of the Cellular, Tissue and Gene Therapies Advisory Committee

Charter of the Transmissible Spongiform Encephalopathies Advisory Committee

Vaccines and Related Biological Products Advisory Committee Meeting Transcript




REGULATORY


FOOD AND DRUG ADMINISTRATION (United States)

FDA Allows City of Hope to Proceed with the First Human Neural Stem Cell Clinical Trial to Treat Brain Tumors
The Food and Drug Administration (FDA) allows City of Hope researchers to proceed with conducting the first-in-human study of a neural stem cell therapy targeting recurrent high-grade gliomas, the most aggressive type of brain tumor.

MEDICINES AND HEALTHCARE PRODUCTS REGULATORY AGENCY (United Kingdom)

Clinical Trials for Medicines Section Updated
The MHRA has updated the Clinical Trials for Medicines section of its website. 

THERAPEUTIC GOODS ADMINISTRATION (Australia)

Advertising and Supply of Therapeutic Goods to Complementary Healthcare Professionals: Schedule 1 to the Therapeutic Goods Regulations

Increased Transparency of the Prescription Medicine Regulatory Process




EVENTS

SPECIAL FEATURE

ImVacS brings together international vaccine leaders to discuss challenges and current breakthroughs in the world of vaccines. The four vaccine-focused meetings address innovating strategies to meet current health threats, changing technology for vaccine manufacture, top challenges faced during pre-clinical development, and progress underway for adjuvants and novel delivery systems.

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

International Society for Stem Cell Research (ISSCR) 8th Annual Meeting
June 16-19, 2010
San Francisco, United States

Accelerating Anticancer Agent Development and Validation
June 23-25, 2010
North Bethesda, United States

XXXIst International Congress of the International Society of Blood Transfusion (ISBT) in joint cooperation with the 43rd Congress of the German Society for Transfusion Medicine and Immunohematology (DGTI)
June 26-July 1, 2010
Berlin, Germany

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

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

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

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

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

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

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

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

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


 

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