Volume 11.39 | Oct 11

Cell Therapy News 11.39, October 11, 2010.
In this issue:  Science  |  Policy  |  Business  |  NIH  |  CBER  |  Regulatory  |  Events Subscribe  |  Unsubscribe



Gene Therapy Reveals Unexpected Immunity to Dystrophin in Patients with Duchenne Muscular Dystrophy
An immune reaction to dystrophin, the muscle protein that is defective in patients with Duchenne muscular dystrophy, may pose a new challenge to strengthening muscles of patients with this disease, suggests a new study. [Press release from Nationwide Children’s Hospital discussing online prepublication in the New England Journal of Medicine]

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Addition of Immunotherapy Boosts Pediatric Cancer Survival in Children with Neuroblastoma
Administering a new form of immunotherapy to children with neuroblastoma, a nervous system cancer, increased the percentage of those who were alive and free of disease progression after two years, according to researchers. [Press release from the University of California, San Diego Medical Center discussing online prepublication in the New England Journal of Medicine]

Newly Discovered DNA Repair Mechanism
Researchers have discovered a fundamentally new way that DNA-repair enzymes detect and fix damage to the chemical bases that form the letters in the genetic code. [Press release from Vanderbilt University discussing online prepublication in Nature]

Catalyst Sandwich
Researchers have taken another step towards realizing a new class of polymerase chain reaction enzyme mimics, opening the door for the development of highly sensitive chemical detection systems that go beyond nucleic acid targets. [Press release from Northwestern University discussing online prepublication in Science]

Study Details Structure of Potential Target for HIV and Cancer Drugs
In a technical tour de force, structural biologists have determined the three-dimensional structure of a molecule involved in HIV infection and in many forms of cancer. [Press release from the National Institutes of Health discussing online prepublication in Science]

Elasticity Found to Stretch Stem Cell Growth to Higher Levels
One of the major challenges in stem cell transplants is how to obtain sufficient numbers of these remarkably rare cells to put into patients. To help overcome this issue, researchers have found a way to increase the number of blood-forming stem cells when growing them outside of the body. [Press release from the Centenary Institute discussing online prepublication in Nature Biotechnology]

Safe, Efficient Method Reported for Creating and Differentiating Human Pluripotent Stem Cells
Researchers report the development of a safe and efficient technology to create human induced pluripotent stem cells. [Press release from Children's Hospital Boston discussing online prepublication in Cell Stem Cell]

Complex Coordination: Three-Way Control of Fetal Heart-Cell Proliferation Could Help Regenerate Cardiac Cells
A team of researchers describe the interconnections between three-molecules that control fetal, heart-muscle-cell proliferation in a mouse model that will help cardiologists better understand the natural repair process after heart attacks and help scientists learn how to expand cardiac stem cells for regenerative therapies. [Press release from the University of Pennsylvania School of Medicine discussing online prepublication in Developmental Cell]

Ticking of a Cellular Clock Promotes Seismic Changes in the Chromatin Landscape Associated with Aging
A new study reports that as cells countdown to senescence and telomeres wear down, their DNA undergoes massive changes in the way it is packaged. These changes likely trigger what we call “aging”. [Press release from the Salk Institute for Biological Studies discussing online prepublication in Nature Structural and Molecular Biology]
T Cell Discovery Shows Promise for Type 1 Diabetes Treatment: UBC-CFRI Study
A research team from the University of British Columbia (UBC) and the Child & Family Research Institute (CFRI) at BC Children’s Hospital has identified the role of a type of T cell in type 1 diabetes that may lead to new treatment options for young patients. [Press release from the University of British Colombia discussing online prepublication in the Journal of Immunology]

'Firefly' Stem Cells May Repair Damaged Hearts
Stem cells that glow like fireflies could someday help doctors heal damaged hearts without cutting into patients' chests. The “firefly” stem cells, engineered with the same enzyme that makes fireflies glow, become brighter and brighter as they develop into healthy heart muscle, allowing doctors to track whether and where the stem cells are working. [Press release from the University of Central Florida discussing online prepublication in Stem Cells and Development]

Chemists Design ‘Tunable,’ Cloaked, Toxin Delivery System to Kill Tumors from Within
Researchers have demonstrated that they can deliver a dormant toxin into a specific site such as a tumor for anti-cancer therapy, then chemically trigger the toxin to de-cloak and attack from within. [Press release from the University of Massachusetts Amherst discussing online prepublication in Nature Chemistry]
Crohn’s Disease Trial Uses Stem Cells Taken from Placentas
Patients with Crohn's disease have shown improvement with placenta-derived stem cells. [Press release from The Detroit News discussing research presented at the World Stem Cell Summit]

Surgeons Create Functional Artificial Pancreatic Tissue
Surgeons from Massachusetts General Hospital, Boston, are reporting on a whole new strategy for controlling insulin dependent diabetes without daily injections of insulin. They have bio-engineered a novel matrix that serves as a scaffold for seeding supportive stem cells as well as pancreatic islets. [Press release from Newswise discussing research presented at the 2010 Annual Clinical Congress of the American College of Surgeons]
Novel Cellular-Based Therapy Minimizes the Risks of Immunosuppression After Upper Extremity Transplantation
Three patients are the first in the world to have a novel cell-based treatment for modulating the immune system after upper extremity transplantation. The therapy known as “The Pittsburgh Protocol” is unique in utilizing bone marrow cells to enable reduction of the need for multiple anti-rejection drugs after transplantation. [Press release from Newswise discussing research presented at the 2010 Annual Clinical Congress of the American College of Surgeons]

CURRENT PUBLICATIONS (Ranked by Impact Factor of the Journal)

Dystrophin Immunity in Duchenne's Muscular Dystrophy
Researchers report on delivery of a functional dystrophin transgene to skeletal muscle in six patients with Duchenne's muscular dystrophy. Dystrophin-specific T cells were detected after treatment, providing evidence of transgene expression even when the functional protein was not visualized in skeletal muscle. [N Engl J Med]

Anti-GD2 Antibody with GM-CSF, Interleukin-2, and Isotretinoin for Neuroblastoma
Immunotherapy with ch14.18, GM-CSF, and interleukin-2 was associated with a significantly improved outcome as compared with standard therapy in patients with high-risk neuroblastoma. [N Engl J Med]
An Unprecedented Nucleic Acid Capture Mechanism for Excision of DNA Damage
The structures and supporting biochemical analysis of base flipping and catalysis reveal how the HEAT repeats of AlkD distort the DNA backbone to detect non-Watson-Crick base pairs without duplex intercalation. [Nature]
Allosteric Supramolecular Triple-Layer Catalysts
Researchers report an allosteric supramolecular structure in which a monometallic catalytic site has been buried in the middle layer of a triple-layer complex. [Science]

Structures of the CXCR4 Chemokine Receptor with Small Molecule and Cyclic Peptide Antagonists
Researchers report five independent crystal structures of CXCR4 bound to an antagonist small molecule IT1t and a cyclic peptide CVX15 at 2.5 to 3.2 angstrom resolution. [Science]
Substrate Elasticity Provides Mechanical Signals for the Expansion of Hemopoietic Stem and Progenitor Cells
Data suggest that substrate elasticity and tensegrity are important mechanisms influencing hemopoietic stem and progenitor cell subsets and could be exploited to facilitate cell culture. [Nat Biotechnol]

Highly Efficient Reprogramming to Pluripotency and Directed Differentiation of Human Cells with Synthetic Modified mRNA
Researchers describe a simple, nonintegrating strategy for reprogramming cell fate based on administration of synthetic mRNA modified to overcome innate antiviral responses. [Cell Stem Cell]
Hopx and Hdac2 Interact to Modulate Gata4 Acetylation and Embryonic Cardiac Myocyte Proliferation
Results suggest that Gata4 is a nonhistone target of Hdac2-mediated deacetylation and that Hdac2, Hopx, and Gata4 coordinately regulate cardiac myocyte proliferation during embryonic development. [Dev Cell]
Reduced Histone Biosynthesis and Chromatin Changes Arising from a Damage Signal at Telomeres
Researchers propose a mechanism in which changes in the structural and epigenetic integrity of telomeres affect core histones and their chaperones, enforcing a self-perpetuating pathway of global epigenetic changes that ultimately leads to senescence. [Nat Struct Mol Biol]
Cutting Edge: Increased IL-17-Secreting T Cells in Children with New-Onset Type 1 Diabetes
Recently, distinct subsets of CD4(+)FOXP3(+) T cells with differing functions were identified. Notably, CD45RA(-)CD25(int)FOXP3(low) T cells lack suppressive function and secrete the proinflammatory cytokine IL-17. Therefore, researchers evaluated whether the relative fractions of CD4(+)FOXP3(+) subsets are altered in new-onset type 1 diabetes subjects. [J Immunol]



Court Schedule Suggests Stem Cell Lawsuit Won't Be Resolved Until Late in Year
The court dates for the lawsuit tying up stem cell research suggest the case won't be decided until at least Thanksgiving and could take until the end of the year. [Court of Appeals, United States]

European Medicines Agency Management Board Adopt New Policies on Handling of Conflicts of Interests and on Access to Documents
The European Medicines Agency’s Management Board adopted a number of important policies at its 68th meeting. [European Medicines Agency, European Union]

Spain's Science Budget Proposal: Not as Bad as Feared, But Not Good
After weeks during which Spanish researchers fretted nervously about possibly significant cuts to their country's science budget, Spanish Science and Innovation Minister Cristina Garmendia tried in a press conference to give a reassuring message to the scientific community: the ministry will have about the same amount of money next year as proposed in 2010 with which to fund competitive research projects and scholarships, support national research institutes, and give loans to R&D companies. [Ministry of Science, Spain]

Advisory Commission on Childhood Vaccines; Notice of Meeting (FR Doc. 2010-25115) [Health Resources and Services Administration, United States]



STEMSOFT® Software Inc. Licenses StemLab® Software to The Blood Connection
STEMSOFT Software Inc., the leading provider of software solutions for the cell therapy and transplant community, is pleased to announce the licensing of StemLab, the leading cell therapy transplant and cord blood banking laboratory software, to The Blood Connection in Piedmont, South Carolina. [STEMSOFT Software Inc. Press Release]

STEMSOFT Connect – A Simplified Solution to a Complex Problem
STEMSOFT is pleased to announce the release of their new standard interfaces, STEMSOFT Connect™.  STEMSOFT Connect offers standard interface tools that easily connect commonly used applications and devices to STEMSOFT's applications. [STEMSOFT Software Inc. Press Release]

Vivo Biosciences, ACEA Biosciences, and Roche to Develop New xCELLigence System Assays for Stem Cell and Cancer Research
ACEA Biosciences Inc. and Vivo Biosciences Inc. announced that they have entered into a collaboration for the further development of label-free and real-time cell based assays for the xCELLigence System, which is co-developed by Roche and ACEA and marketed by Roche Applied Science. [Roche Applied Science Press Release]

Dainippon Sumitomo Pharma and SanBio Collaborate on Cell Therapy for Stroke Recovery
Dainippon Sumitomo Pharma Co. Ltd. and SanBio, Inc. announced that they have entered into an option agreement to co-develop a new cell therapy for stroke recovery. [Dainippon Sumitomo Pharma Co. Ltd. Press Release]

Genzyme Board Unanimously Rejects Sanofi-Aventis Tender Offer
Genzyme Corporation announced that its board of directors has voted unanimously to reject the unsolicited $69.00 per share tender offer from Sanofi-Aventis, and the board recommends that Genzyme shareholders not tender their shares to Sanofi-Aventis pursuant to the offer. [Genzyme Corporation Press Release]

Novartis Announces Agreement to Develop Influenza Vaccines Using Revolutionary “Synthetic Genomics” Technology
Novartis announced an agreement with Synthetic Genomics Vaccines Inc. to apply “synthetic genomics” technologies to accelerate the production of the influenza seed strains required for vaccine manufacturing. [Novartis Press Release]

Study to Investigate Menstrual Blood-Derived Stem Cells as Potential Stroke Therapy
The potential for stem cells derived from menstrual blood to benefit stroke sufferers will be jointly investigated by researchers at the University of South Florida, Cryo-Cell International, Inc. a global stem cell company based in Oldsmar, FL, and Saneron CCEL Therapeutics, Inc. [University of South Florida Press Release]

NIH Awards $14.6 Million Translational Cardiovascular Nanomedicine Center
Georgia Tech and Emory University have received a five-year $14.6 million contract from the National Institutes of Health (NIH) to continue the development of nanotechnology and biomolecular engineering tools and methodologies for detecting and treating atherosclerosis. [Georgia Institute of Technology Press Release]

Defense Department Funds Next Phase of Stratatech Clinical Trial
Stratatech Corporation, a regenerative medicine company focused on the commercialization of cell-based, tissue-engineered skin substitute products, announced that the Defense Department’s Armed Forces Institute of Regenerative Medicine  will fund the Phase IIb clinical trial of the company’s flagship StrataGraft® living human skin substitute tissue. [Stratatech Corporation Press Release]

USF, Saneron Get $2.6 Million NIH grant to Develop Umbilical Cord Blood Cell Therapy for Alzheimer’s Disease
The National Institutes of Health has awarded a three-year, $2.6-million grant to the University of South Florida (USF) and Saneron-CCEL Therapeutics, Inc. to establish dosing and safety guidelines for transplanting human umbilical cord blood cells into animal models of Alzheimer’s disease. [University of South Florida Press Release]

Catapult and Mercia Invest in Staffordshire-Based nanoTherics
Catapult Venture Managers and Mercia Technology Seed Fund are key investors in a £1.1m funding round into Staffordshire-based nanoTherics, a company at the cutting edge of the growing gene therapy replacement market. [Catapult Venture Managers Press Release]
University of Arizona Bioengineer Awarded $1.5 Million to Research Tissue Regeneration
Pak Kin Wong aims to discover the rules that govern how biological tissues are formed from individual cells. He is investigating how to grow new tissue to replace that destroyed by disease. [University of Arizona Press Release]

University of Michigan Creates the State’s First Human Embryonic Stem Cell Line
Researchers have created the state’s first human embryonic stem cell line, known as UM4-6. [University of Michigan Press Release]
Epigenome Effort Makes Its Mark
The Roadmap Epigenomics Project, a US$170-million effort to identify and map a vast suite of epigenetic marks — known collectively as the human epigenome — began its first comprehensive data release. [NatureNews] 

Tekmira Intellectual Property Portfolio Solidified with Granting of Key Patents by U.S. Patent & Trademark Office
Tekmira Pharmaceuticals Corporation, a leading developer of RNA interference therapeutics, announced that the United States Patent & Trademark Office has issued three key patents covering portions of Tekmira's technology. [Tekmira Pharmaceuticals Corporation Press Release]
Proteonomix Announces Critical Patent Filing
Proteonomix, Inc. announced that it has filed a utility patent application for the Preparation and Use of Stromal Cells for Treatment of Cardiac Diseases. [Marketwire]
Argos Therapeutics´ Arcelis™ Immunotherapy for Renal Cell Carcinoma Demonstrates Encouraging Results in Phase II Combination Study with Sunitinib
Argos Therapeutics announced that its Arcelis immunotherapy for renal cell carcinoma, AGS-003, demonstrated favorable progression-free survival and safety in combination with sunitinib, based upon updated results from an ongoing Phase II trial. [Argos Therapeutics Press Release]

BioCardia Announces Issuance of Patents Related to Cardiac Regenerative Medicine Using Cell Therapy
BioCardia, Inc. announced the issuance of two U.S. Patents related to cardiac regenerative medicine. Both patents are assigned to the University of California and exclusively licensed to BioCardia. [BioCardia, Inc. Press Release]

Shrinking U.S. Biotechnology Sector Lost 25% of Companies in Past 3 Years
One hundred publicly traded biotechnology companies in the U.S. have been acquired or ceased operations since the end of 2007, a 25 percent drop in the number of active companies, a report said. [Bloomberg]


NIH Announces New Program to Accelerate Research Independence
The National Institutes of Health intends to invest approximately $60 million over the next five years in the NIH Director’s Early Independence Award program to help junior investigators leapfrog over traditional post-doctoral training and move into independent academic positions at U.S. institutions, directly upon completion of their graduate research degrees.

NIH Launches Genotype-Tissue Expression Project
The National Institutes of Health (NIH) announced awards to support an initiative to understand how genetic variation may control gene activity and its relationship to disease. Launched as a pilot phase, the Genotype-Tissue Expression (GTEx) project will create a resource researchers can use to study inherited susceptibility to illness and will establish a tissue bank for future biological studies.
NIH Funds Advanced Development of Three Biodefense Vaccines
The National Institute of Allergy and Infectious Diseases, part of the National Institutes of Health (NIH), announced three new contracts to fund research on vaccines to protect against emerging infectious diseases and biological threats that could be used in a terror attack.

Plan to Develop a Genetic Testing Registry at the National Institutes of Health; Public Meeting; Request for Comments (FR Doc. 2010-25411)

Center for Scientific Review; Amended Notice of Meeting (FR Doc. 2010-25171) 

Center for Scientific Review; Notice of Closed Meetings (FR Doc. 2010-25180) 

Center for Scientific Review; Notice of Closed Meetings (FR Doc. 2010-25185)

National Center for Research Resources; Notice of Closed Meeting (FR Doc. 2010-25176) 

National Institute of Biomedical Imaging and Bioengineering; Notice of Closed Meeting (FR Doc. 2010-25172)

BioMalaysia 2010 645x110 


November 16-17, 2010: Vaccines and Related Biological Products Advisory Committee Meeting Announcement

Waivers for Conflict of Interest for the October 28 – 29, 2010 Transmissible Spongiform Encephalopathies Advisory Committee Meeting




FDA's $25 Million Pitch for Improving Drug Regulation
The U.S. Food and Drug Administration (FDA) is pressing for a big funding boost for “regulatory science”—research that can help it evaluate new treatments better and faster.

FDA to Push for More Investment in Science
Health regulators plan to spend millions of dollars to step up their scientific prowess in a move that officials say will help quickly get new treatments to patients and protect the public against possible health threats.

FDA Issues Regulatory Science Report
The U.S. Food and Drug Administration unveiled a report outlining the agency’s plans to advance regulatory science through its Regulatory Science Initiative.


Information Sent to Healthcare Professionals in September About the Safety of Medicines

EVENTS (Listed by Date)

New York Stem Cell Foundation 5th Annual Translational Stem Cell Research Meeting
October 12-13, 2010
New York, United States

4th International Symposium: Stem Cells, Development and Regulation
October 12-14, 2010
Amsterdam, Netherlands

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

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

BIO Intellectual Property Counsels Committee Fall Conference and Committee Meeting
October 18-20, 2010
Boston, United States

Stem Cells and Regenerative Medicine – New Solutions to Old Problems
October 18-20, 2010
San Antonio, United States

6th Modern Drug Discovery & Development Summit (M3D)
October 20-22, 2010
San Francisco, 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

International Consensus Conference on Risk-Based Decision Making for Blood Safety
October 26-28, 2010
Toronto, Canada

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

Public Workshop on Cell and Gene Therapy Clinical Trials in Pediatric Populations
November 2, 2010
Bethesda, United States

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

BioMalaysia 2010 Conference and Exhibition
November 2-4, 2010
Kuala Lumpur, Malaysia

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

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

The 16th International Conference of the ISD (International Society of Differentiation)
November 15-18, 2010
Nara, Japan

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

Berlin-Brandenburg School for Regenerative Therapies (BSRT) PhD Symposium: Stem Cells — Hopes, Fears and Realities
December 1-3, 2010
Berlin, Germany

2nd European Association for Cardio-Thoracic Surgery (EACTS) Meeting on Cardiac and Pulmonary Regeneration
December 2-3, 2010
Vienna, Austria

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

NEW 5th Annual Stem Cell Meeting on the Mesa
December 7, 2010
La Jolla, United States

American Society for Cell Biology 50th Anniversary Annual Meeting
December 11-15, 2010
Philadelphia, United States

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

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

Keystone Symposia-Stem Cells in Development, Tissue Homeostasis and Disease
January 30-February 4, 2011
Santa Fe, 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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