Volume 11.41 | Oct 25

Cell Therapy News 11.41, October 25, 2010.
In this issue:  Science  |  Policy  |  Business  |  NIH  |  CBER  |  Regulatory  |  Events Subscribe  


Gene Therapy May Be Powerful New Treatment for Major Depression 
Researchers say animal and human data suggest gene therapy to the brain may be able to treat patients with major depression who do not respond to traditional drug treatment. [Press release from the Weill Cornell Medical College discussing online prepublication in Science Translational Medicine]



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Scientists Show TAp63 Suppresses Cancer Metastasis
Long overshadowed by p53, its famous tumor-suppressing sibling, the p63 gene does the tougher, important job of stifling the spread of cancer to other organs. [Press release from The University of Texas MD Anderson Cancer Center discussing online prepublication in Nature]
PiggyBac Joins Armory in Fight Against Cancer
Researchers have developed a genetic tool in mice to speed the discovery of novel genes involved in cancer. The system – called PiggyBac – has already been used by the team to identify novel candidate cancer-causing genes. [Press release from the Wellcome Trust Sanger Institute discussing online prepublication in Science]
Scientists Turn Stem Cells into Cells for Cartilage Repair
Researchers took human embryonic stem cells and developed a culture procedure involving a precise sequential program of conditions to specifically produce chondrocytes. [Press release from The University of Manchester discussing online prepublication in Nature Biotechnology]
Not All Stem Cells the Same, Say Researchers
Until now it's been thought that human stem cell lines are all identical and possess the same ability to differentiate. But new research has shown there are subsets of stem cells that respond differently depending on what stem cell properties are measured and what test is used. [Press release from McMaster University discussing online prepublication in Nature Methods]
Promise of Outpatient Brain Gene Therapy Is One Step Closer
Researchers describe how they can transfer genes into brain neurons intravenously, using a viral gene delivery vehicle that causes no side effects. [Press release from Thomas Jefferson University discussing online prepublication in Nature Methods]
Scientists Uncover Strategy to Effectively Target Drug Resistant Childhood Cancers
Motivated by a pressing need to find new treatments for high-risk childhood sarcomas, scientists used cutting-edge gene silencing technology to identify a specific protein involved in resistance to therapies against a target called IGF1R in childhood sarcomas. [Press release from the British Columbia Cancer Agency discussing online prepublication in Cancer Research]
Promising Preclinical Data Demonstrate Anti-Tumor Activity of a Novel Cancer Target via RNA Interference
Scientists identified a novel oncology target called COP1 that is over-expressed in human liver cancers and can accurately predict patient survival. They designed specific siRNA that, when delivered systemically with Tekmira's lipid nanoparticle technology, silence the COP1 gene. [Press release from Tekmira Pharmaceuticals Corporation discussing online prepublication in Cancer Research]
Somatic Cells Differentiate into Hepatocyte-Like Cells
A research team at the Okayama University Graduate School of Medicine, working with colleagues at the University of Pittsburgh School of Medicine, found that mouse induced pluripotent stem (iPS) cells are able to proliferate in vitro without limits and could be cultured to become hepatocyte-like cells. [Press release from EurekAlert! discussing online prepublication in Cell Transplantation]
DNA Minivectors Provide New Gene Therapy Tool
Tiny wound-up circular bits of DNA that carry a short “hairpin” of RNA could prove a solution to the quest for a non-toxic, long-lived gene therapy vector designed to silence cancer genes in tumor cells, said researchers. [Press release from the Baylor College of Medicine discussing online prepublication in Gene Therapy]

Synthetic FlexBone Could Help Speed Bone Transplant Recovery
Current synthetic substitutes for bone tissue grafts rarely possess the bone-like properties needed for successful grafting and are often difficult for surgeons to manipulate in the operating room. In response to these challenges, researchers have developed an easy-to-produce, inexpensive, synthetic bone material called FlexBone. [Press release from the University of Massachusetts Medical School discussing online prepublication in Tissue Engineering Part A]
Exercise Boosts Muscle Stem Cells
It turns out that endurance exercise, like running, doesn't just tone muscles, it enhances muscle stem cells too. Researchers found that the number of muscle stem cells, called satellite cells, increased after rats spent 13 weeks running on a treadmill for 20 minutes a day. [Press release from ScienceShots discussing online prepublication in PLoS One]

Bioelectrical Signals Turn Stem Cells' Progeny Cancerous
Biologists have discovered that a change in membrane voltage in newly identified “instructor cells” can cause stem cells’ descendants to trigger melanoma-like growth in pigment cells. The team also found that this metastatic transformation is due to changes in serotonin transport. The discovery could aid in the prevention and treatment of diseases like cancer and vitiligo as well as birth defects. [Press release from Tufts University discussing online prepublication in Disease Models and Mechanisms]

CURRENT PUBLICATIONS (Ranked by Impact Factor of the Journal)

TAp63 Suppresses Metastasis Through Coordinate Regulation of Dicer and miRNAs
Data provide a novel understanding of the roles of TAp63 in tumor and metastasis suppression through the coordinate transcriptional regulation of Dicer and miR-130b and may have implications for the many processes regulated by miRNAs. [Nature]
A Genome-Wide RNAi Screen Reveals Determinants of Human Embryonic Stem Cell Identity
The study uncovers a wealth of novel human embryonic stem cell (hESC) regulators wherein PRDM14 exemplifies a key transcription factor required for the maintenance of hESC identity and the reacquisition of pluripotency in human somatic cells. [Nature]

PiggyBac Transposon Mutagenesis: A Tool for Cancer Gene Discovery in Mice
Researchers show that insertional mutagenesis by the PiggyBac transposon can be used for cancer gene discovery in mice. [Science]
Directed Differentiation of Human Embryonic Stem Cells Toward Chondrocytes
Researchers report a chemically defined, efficient, scalable and reproducible protocol for differentiation of human embryonic stem cells toward chondrocytes. [Nat Biotechnol]
Clonal Tracking of hESCs Reveals Differential Contribution to Functional Assays
Using clonal tracking by retroviral integration, researchers analyzed in situ the propensity of individual human embryonic stem cells (hESCs) to contribute to different functional assays. They observed different clonal distributions in teratomas versus in vitro differentiation assays. [Nat Methods]
Efficient CNS Gene Delivery by Intravenous Injection
Intravenous injection of recombinant SV40-derived viral vectors, particularly with mannitol pretreatment, resulted in extensive expression of multiple transgenes throughout the central nervous system (CNS). [Nat Methods]
Synthetic Lethality Screens Reveal RPS6 and MST1R as Modifiers of Insulin-Like Growth Factor-1 Receptor Inhibitor Activity in Childhood Sarcomas
Researchers confirmed macrophage-stimulating 1 receptor tyrosine kinase (MST1R) expression across a broad panel of childhood sarcomas, and found that loss of MST1R by RNA interference blocks downstream ribosomal protein S6 (RPS6) activation when combined with BMS-536924 in vitro. [Cancer Res]

Definition of Ubiquitination Modulator COP1 as a Novel Therapeutic Target in Human Hepatocellular Carcinoma
In an orthotopic mouse xenograft model of hepatocellular carcinoma, systemic delivery of a modified COP1 short interfering RNA by stable nucleic acid-lipid particles suppressed neoplastic growth in liver without unwanted immune responses. [Cancer Res]
Hepatic Differentiation of Mouse iPS Cells In Vitro
Researchers present instant hepatic differentiation of mouse induced pluripotent stem cells using combined 3-day treatments of activin A and basic fibroblast growth factor with subsequent 8-day hepatocyte growth factor. [Cell Transplant]
Reversal of Depressed Behaviors in Mice by p11 Gene Therapy in the Nucleus Accumbens
Results suggest that p11 loss in rodent and human nucleus accumbens may contribute to the pathophysiology of depression. Normalization of p11 expression within this brain region with adeno-associated virus-mediated gene therapy may be of therapeutic value. [Sci Transl Med]
Transfection of Short Hairpin RNA-Encoding Minivector DNA of a Few Hundred Base Pairs to Regulate Gene Expression in Lymphoma Cells
Minivector DNA and siRNA were capable of silencing the gene encoding anaplastic lymphoma kinase, a key pathogenic factor of human anaplastic large cell lymphoma, and this silencing caused inhibition of the lymphoma cells. [Gene Ther]



Just a Flesh Wound? U.K. Science Budget Spared Deep Cuts
The U.K. government's long-awaited Comprehensive Spending Review was released, and it calls for a flat science budget over the next 4 years. While inflationary costs mean that the budget will likely see a cut of about 10% in real terms over that period, this scenario is far less dramatic than rumored cuts of 20% to 30% in real cash. [HM Treasury, United Kingdom]
Secretarial Review and Publication of the Annual Report to Congress Submitted by the Contracted Consensus-Based Entity Regarding Performance Measurement (FR Doc. 2010-26795) [Department of Health and Human Services, United States]
Partnerships to Advance the National Occupational Research Agenda (NORA) (FR Doc. 2010-25973) [Centers for Disease Control and Prevention, United States]

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GSK, Fondazione Telethon and Fondazione San Raffaele to Collaborate on Gene Therapy for Rare Diseases
GlaxoSmithKline PLC (GSK), Fondazione Telethon and Fondazione San Raffaele announced a new strategic alliance to research and develop novel treatments to address rare genetic disorders, using gene therapy carried out on stem cells taken from the patient’s bone marrow (ex vivo). [GlaxoSmithKline Press Release]

Sanofi-aventis Establishes Research Collaboration with Harvard University
Sanofi-aventis announced a research collaboration with Harvard University. The goal of the collaboration is to advance knowledge in the area of human health through basic and applied research and to promote scientific exchange between Harvard University and Sanofi-aventis. [Sanofi-aventis Press Release]
Sanofi-aventis Announces Expiration of Hart-Scott-Rodino Waiting Period for Acquisition of Genzyme
Sanofi-aventis announced that the waiting period under the Hart-Scott Rodino Antitrust Improvements Act of 1976, as amended, applicable to Sanofi-aventis’ proposed acquisition of Genzyme Corporation has expired. [Sanofi-aventis Press Release]
Celgene Completes Acquisition of Abraxis
Celgene Corporation announced it has completed its acquisition of Abraxis BioScience, Inc. [Celgene Corporation Press Release]
Cryo-Cell International Confirms Rejection of Additional Unsolicited Proposals
Cryo-Cell International, Inc. confirmed that it has recently received additional inquiries from third parties interested in acquiring the Company. [Cryo-Cell International, Inc. Press Release]
Mesoblast Acquisition of U.S. Company Approved by Angioblast Stockholders
Stockholders of United States adult stem cell company Angioblast Systems Inc. voted unanimously at a special stockholders’ meeting in favor of becoming a wholly-owned subsidiary of publicly listed global regenerative medicine company, Mesoblast Limited. [Mesoblast Limited Press Release]
CIRM Funds $72 Million to Translate Basic Discoveries into New Therapies, Recruit Stem Cell Leaders to California
The California Institute for Regenerative Medicine (CIRM) approved funding for 19 awards worth $67 million in its second round of awards designed to move good ideas out of the lab and into the clinic. [The California Institute for Regenerative Medicine Press Release]
$3.7 Million to Push Cancer Discoveries Down the ‘Pipeline’
The Cancer Institute New South Wales has awarded the Garvan Institute’s Cancer Research Program $3.7 million, research funding that should lead to improved clinical decision-making for breast, prostate and pancreatic cancer within the next 5 years. [The Garvan Institute Press Release]
Combining Stem Cell and Gene Therapy Techniques to Tackle a Deadly Blood Disease
The National Institutes of Health has awarded a three-year, $3.9 million grant to Children's Hospital Boston researchers and their colleagues to develop a therapy to treat Fanconi anemia. [Children’s Hospital Boston Press Release]
NIH Awards Karmanos Cancer Institute Breast Cancer Researchers Nearly $3 Million for Ground-Breaking Immunotherapy Clinical Study
Scientists at the Barbara Ann Karmanos Cancer Institute in Detroit have received a nearly $3 million, five-year research project (RO1) grant from the National Institutes of Health to study a potentially ground-breaking immunotherapy treatment for women with triple-negative breast cancer. [Barbara Ann Karmanos Cancer Institute Press Release]
Incyte Achieves $19 Million Milestone as Novel JAK1 and JAK2 Inhibitor Advances into Phase IIb Development for Rheumatoid Arthritis
Incyte Corporation announced that it has earned a $19 million dollar milestone payment from Eli Lilly and Company based on the initiation of a Phase IIb clinical trial evaluating INCB28050 for the treatment of rheumatoid arthritis. [Incyte Corporation Press Release]
Dicerna Pharmaceuticals Announces Second Close of Series B Financing
Dicerna Pharmaceuticals, Inc., a second generation RNA interference company developing novel therapeutics utilizing its proprietary Dicer Substrate Technology™ and Dicer Substrate siRNA molecules, announced a second closing of its Series B equity financing, securing an additional $4 million and bringing the total capital raised in this round to $29 million. [Dicerna Pharmaceuticals, Inc. Press Release]
Aastrom Submits Special Protocol Assessment to FDA for Its Phase III CLI Program
Aastrom Biosciences, Inc. announced that it has submitted to the U.S. Food and Drug Administration (FDA) a special protocol assessment describing the company's proposed Phase III clinical development program in critical limb ischemia (CLI). [Aastrom Biosciences, Inc. Press Release]

Aastrom Receives FDA Fast Track Designation for Phase III CLI Program
Aastrom Biosciences, Inc. announced that the U.S. Food & Drug Administration (FDA) has granted fast track designation for the company's critical limb ischemia (CLI) cell therapy development program. [Aastrom Biosciences, Inc. Press Release]
Phase II Study with Ocrelizumab Shows Significant Reduction in Disease Activity for Multiple Sclerosis Patients
Roche and Biogen Idec announced 24-week results from a Phase II study of ocrelizumab in patients with relapsing-remitting multiple sclerosis, the most common form of the disease. [Roche Press Release]
Ceregene Initiates a New Controlled Phase IIb Trial of CERE-120 for Parkinson's Disease
Ceregene, Inc. announced that enrollment has begun in a new double-blind sham surgery-controlled Phase IIb study evaluating CERE-120 in Parkinson’s disease patients. CERE-120 is a gene therapy product that delivers the neurotrophic factor neurturin to degenerating or dying dopamine neurons. [Ceregene, Inc. Press Release]
CEL-SCI Corporation Receives Approval from Seven Hospital Institutional Review Boards in India to Commence Phase III Clinical Trial of Multikine in Head and Neck Cancer
CEL-SCI Corporation announced it has received approval from seven hospital Institutional Review Boards in India to begin enrollment of subjects for a Phase III clinical trial of Multikine®, the Company's flagship immunotherapy developed as a first-line standard of care in treating head and neck cancer. [CEL-SCI Corporation Press Release]


NIH Recovery Act Awards Enable Crucial Health Research Technologies
Thanks to the Recovery Act, more health scientists now will have access to state-of-the-art devices, the National Institutes of Health announced. Through its National Center for Research Resources, NIH has awarded $300 million in shared instrumentation grants. 

Center for Scientific Review; Notice of Closed Meetings (FR Doc. 2010-26362)
Center for Scientific Review; Notice of Closed Meetings (FR Doc. 2010-26363)
Center for Scientific Review; Notice of Closed Meetings (FR Doc. 2010-26317)
Center for Scientific Review; Notice of Closed Meetings (FR Doc. 2010-26497)
Center for Scientific Review; Notice of Closed Meetings (FR Doc. 2010-26499)
Center for Scientific Review; Notice of Closed Meetings (FR Doc. 2010-26028)

National Cancer Institute; Notice of Closed Meeting (FR Doc. 2010-26702)
National Cancer Institute; Notice of Closed Meetings (FR Doc. 2010-26706)
National Cancer Institute; Notice of Closed Meeting (FR Doc. 2010-26390)
Office of the Director, National Institutes of Health; Notice of Meeting (FR Doc. 2010-26036)


Public Workshop: Product Development Program for Interventions in Patients with Severe Bleeding Due to Trauma or Other Causes  

2010 Meeting Materials, Transmissible Spongiform Encephalopathies Advisory Committee



FDA Writes Prescription for IT Innovation
The Food and Drug Administration is prioritizing IT as part of its five-year strategic plan, including accelerating a program to build innovative devices to improve field and laboratory analysis.
Guidance for Industry: Qualification Process for Drug Development Tools

Agency Information Collection Activities; Submission for Office of Management and Budget Review; Comment Request; Current Good Manufacturing Practice Quality System Regulations (Docket No. FDA-2010-N-0273)


Meeting Highlights from the Committee for Medicinal Products for Human Use (CHMP), 18-21 October 2010


Export of Human Blood/Tissue and Related Materials
Public Submissions: Proposed Standards and Code of GMP for Human Blood and Blood Components, Human Tissues and Human Cellular Therapies
Public Submissions: Proposed Standards for Hematopoietic Progenitor Cells Intended for Use in Hematopoietic Reconstitution

EVENTS (Listed by Date)

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

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

2nd Annual Stem Cells Asia and Regenerative Medicine Congress
October 26-28, 2010
Seoul, Korea

Select Biosciences 2nd Annual Lab-on-a-Chip World Congress
October 28-29, 2010
San Diego, United States

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

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

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

5th UK Cancer Stem Cell Symposium
November 2, 2010
London, United Kingdom

BioMalaysia 2010
November 2-3, 2010
Kuala Lumpur, Malaysia

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

2010 National GvHD Symposium
November 4, 2010
Cleveland, United States

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

9th Annual American Association for Cancer Research (AACR) International Conference: Frontiers in Cancer Prevention Research
November 7-10, 2010
Philadelphia, United States

Cambridge Healthtech Institute's Inaugural Stem Cells in Drug Discovery and Development
November 8-9, 2010
San Diego, United States

GTCbio 8th Annual Vaccines: “All Things Considered” Conference
November 8-9, 2010
Washington, DC, United States

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

Select Biosciences 2nd International Forum on Stem Cells (2010 IFSC)
November 12-13, 2010
Tianjin, China

Select Biosciences 2nd Annual RNAi Asia Conference and Exhibition
November 15-16, 2010

The 16th International Conference of the International Society of Differentiation: From Stem Cells to Organisms
November 15-18, 2010
Nara, Japan

3rd Annual Workshop on Genome Engineering
November 16, 2010
Seattle, United States

22nd European Organization for Research and Treatment of Cancer (EORTC)-National Cancer Institute (NCI)-American Association for Cancer Research (AACR) Symposium: Molecular Targets and Cancer Therapeutics
November 16-19, 2010
Berlin, 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

Translational Excellence in Regenerative Medicine (TeRM) – Biophysical Stimuli and Mass Transport for Tissue Development
November 18-19, 2010
Lattrop, The Netherlands

4th Annual Conference of the Korea Society of Gene Therapy (KSGT)
November 19, 2010
Seoul, Korea

Stem Cell Network's 10th Annual Scientific Meeting
November 22-24, 2010
Calgary, Alberta

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

Tumor Immunology: Basic and Clinical Advances
November 30-December 3, 2010
Miami, United States

2ND European Association for Cardio-Thoracic Surgery (EACTS) Meeting on Cardiac and Pulmonary Regeneration
December 3-4, 2010
Vienna, Austria

3rd Annual BioPartnering China
December 5-7, 2010
Shanghai, China

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

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

Genesis 2010
December 9, 2010
London, United Kingdom

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

Functional Consequences of Structural Variation in the Genome
January 8-13, 2011
Steamboat Springs, Colorado

Nano in Cancer: Linking Chemistry, Biology, and Clinical Applications In Vivo
January 12-15, 2011
Miami, United States

COGEM Symposium: GM Viruses as Medicine: Panacea or Pandora's Box?
January 20, 2011
Amsterdam, The Netherlands

Extracellular Matrix and Cardiovascular Remodeling
January 23-28, 2011
Tahoe City, United States

5th Annual Stem Cells and 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

Phacilitate 9th Annual North American Vaccine Forum
January 24-26, 2011
Washington, DC, United States

NEW IQPC’s 4th Biobanking Conference
January 24-26, 2011
Philadelphia, United States

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

CMC Workshop: Translating Science into Successful Regulatory Submissions
February 7-9, 2011
Washington, DC, United States

23rd Lorne Cancer Conference
February 10-12, 2011
Lorne, Australia

BMT Tandem Meetings (ASBMT/CIBMTR)
February 17-21, 2011
Honolulu, Hawaii

18th International Molecular Medicine Tri-Conference (MMTC)
February 23-25, 2011
San Francisco, United States

Molecular Medicine Tri-Conference: Stem Cells – Revolutionizing Regenerative Medicine and Personalized Therapy 2011
February 23-25, 2011
San Francisco, United States

Molecular Medicine Tri-Conference: Inaugural Circulating Tumor Cells for Cancer Detection, Diagnosis, Prognosis and Treatment
February 23-25, 2011
San Francisco, United States

Targeting PI3K/mTOR Signaling in Cancer
February 24-27, 2011
San Francisco, United States

9th Annual BioPartnering North America
February 27-March 1, 2011
Vancouver, Canada

18th Conference on Retroviruses and Opportunistic Infections (CROI 2011)
February 27-March 2, 2011
Boston, United States

American Association for Cancer Research (AACR) Stem Cells, Development, and Cancer
March 3-6, 2011
Vancouver, Canada

Stem Cells, Cancer and Metastasis (C4)
March 6-11, 2011
Dillon, United States

Spring Meeting of the Dutch Society of Gene Therapy (NVGT)
March 10-11, 2010
Heeze, The Netherlands

BIO-Europe Spring 2011
March 14-16, 2011
Milan, Italy

The 6th International Conference on Oncolytic Viruses as Cancer Therapeutics
March 16-19, 2011
Las Vegas, United States

HIV Evolution, Genomics and Pathogenesis
March 20-25, 2011
Whistler, British Columbia

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



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