Volume 11.44 | Nov 15

Cell Therapy News 11.44, November 15, 2010.
In this issue:  Science  |  Policy  |  Business  |  NIH  |  CBER  |  Regulatory  |  Events Subscribe  


Specialized Blood Vessels Jumpstart and Sustain Organ Regeneration 
Researchers showed that sinusoidal endothelial cells in the liver can be manipulated so that they prompt hepatocytes to expand and regenerate lost tissue. [Press release from Howard Hughes Medical Institute discussing online prepublication in Nature]



Launching ISCT's Inaugural Commercial Development Focus Group on Peripheral Vascular Disease
Read more on how ISCT is pursuing a greater strategic alignment with its industry members in the free ISCT Industry White Paper. [International Society for Cellular Therapy]

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Rare Syndrome Aided by Stem Cell Gene Therapy
Stem cell gene therapy resulted in marked clinical improvement for two patients with the rare Wiskott-Aldrich syndrome, researchers reported. [Press release from MedPage Today discussing online prepublication in the New England Journal of Medicine]
Researchers Discover Key Mutation in Acute Myeloid Leukemia
Researchers have discovered mutations in a particular gene that affects the treatment prognosis for some patients with acute myeloid leukemia. [Press release from the National Institutes of Health discussing online prepublication in the New England Journal of Medicine]
Researchers Identify New Aspect of Cell Reprogramming
An interdisciplinary group of leading geneticists and stem cell researchers has found a new genetic aspect of cell reprogramming that may ultimately help in the fine-tuning of induced pluripotent stem cells into specific cell types. [Press release from Harvard University discussing online prepublication in Nature Genetics]
Modeling Autism in a Dish
Researchers successfully used human induced pluripotent stem cells derived from patients with Rett syndrome to replicate autism in the lab and study the molecular pathogenesis of the disease. [Press release from the Salk Institute for Biological Studies discussing online prepublication in Cell]
Gene Identified for Spread of Deadly Melanoma
Researchers have identified a gene linked to the spread of eye melanoma. [Press release from Washington University School of Medicine in St. Louis discussing online prepublication in Science]
Researcher Discovers Potential New Target for Therapy for Aggressive Prostate Cancer
A monoclonal antibody targeting a well-known cell surface protein inhibited prostate cancer growth and metastasis in an aggressive form of the disease that did not respond to hormone therapy. [Press release from the University of California, Los Angeles Jonsson Comprehensive Cancer Center discussing online prepublication in Nature Medicine]
Scientists Find Nerve Cell Activity Drains Stem Cell Pool in Developing Brain
Researchers have shown that as newly formed nerve cells start firing electrical signals, this activity slows down stem cell division, emptying out the stem cell pool in favor of nerve cell formation. [Press release from The Scripps Research Institute discussing online prepublication in Neuron]
Rogue Gene Hijacks Stem Cells to Jumpstart Human Cancer
Researchers have shown that a gene called FOXM1 exploits the inherent self-renewal property of stem cells causing excessive cell proliferation. [Press release from Queen Mary, University of London discussing online prepublication in Cancer Research]

Geron Announces Publications Demonstrating That Its Telomerase Inhibitor Drug Targets Cancer Stem Cells
Researchers demonstrate the inhibitory effect of imetelstat on cancer stem cells from breast and pancreatic tumor cell lines in vitro and in animal models of the human disease. [Press release from Geron Corporation discussing online prepublication in Cancer Research]
Scientists Uncover a Genetic Switch That Turns Immune Responses On and Off
Scientists have identified a “cellular switch,” called eye transformer, that controls the flow of information from chemical signals outside of the cell to genes in the cell nucleus. [Press release from EurekAlert! discussing online prepublication in the FASEB Journal]
Growth Factor Regenerates Tooth Supporting Structures: Results of a Large Randomized Clinical Trial
Researchers found that the topical application of fibroblast growth factor-2 can be efficacious in the regeneration of human periodontal tissue that has been destroyed by periodontitis. [Press release from EurekAlert! discussing online prepublication in the Journal of Dental Research]
Stem Cell Transplants in Mice Produce Lifelong Enhancement of Muscle Mass
A study shows that specific types of stem cells transplanted into the leg muscles of mice prevented the loss of muscle function and mass that normally occurs with aging, a finding with potential uses in treating humans with chronic, degenerative muscle diseases. [Press release from the University of Colorado at Boulder discussing online prepublication in Science Translational Medicine]
Shire Presents Positive New Data at the 60th Annual American Society of Human Genetics for Patients with Type 1 Gaucher Disease
Shire plc presented positive new data from a Phase III clinical trial (study 039) designed to evaluate the efficacy of VPRIV® (velaglucerase alfa for injection) compared with imiglucerase in patients with type 1 Gaucher disease. [Press release from Shire plc discussing research presented at the 2010 Annual American Society of Human Genetics]

CURRENT PUBLICATIONS (Ranked by Impact Factor of the Journal)

Stem Cell Gene Therapy for the Wiskott-Aldrich Syndrome
Researchers treated two patients who had Wiskott-Aldrich (WAS) syndrome with a transfusion of autologous, genetically modified hematopoietic stem cells (HSC). They found sustained expression of WAS protein expression in HSC, lymphoid and myeloid cells, and platelets after gene therapy. [N Engl J Med]
DNMT3A Mutations in Acute Myeloid Leukemia
DNMT3A mutations are highly recurrent in patients with de novo acute myeloid leukemia with an intermediate-risk cytogenetic profile and are independently associated with a poor outcome. [N Engl J Med]
Inductive Angiocrine Signals from Sinusoidal Endothelium Are Required for Liver Regeneration
Researchers demonstrate that liver sinusoidal endothelial cells constitute a unique population of phenotypically and functionally defined VEGFR3+CD34-VEGFR2+VE-cadherin+FactorVIII+CD45- endothelial cells, which through the release of angiocrine trophogens initiate and sustain liver regeneration induced by 70% partial hepatectomy. [Nature]
Large Intergenic Non-Coding RNA-RoR Modulates Reprogramming of Human Induced Pluripotent Stem Cells
Researchers characterize the transcriptional reorganization of large intergenic non-coding RNAs (lincRNAs) that occurs upon derivation of human induced pluripotent stem cells and identify numerous lincRNAs whose expression is linked to pluripotency. [Nat Genet]
A Model for Neural Development and Treatment of Rett Syndrome Using Human Induced Pluripotent Stem Cells
Using Rett syndrome (RTT) as an autism spectrum disorders genetic model, researchers developed a culture system using induced pluripotent stem cells from RTT patients' fibroblasts. [Cell]
Frequent Mutation of BAP1 in Metastasizing Uveal Melanomas
Findings implicate loss of BAP1 in uveal melanoma metastasis and suggest that the BAP1 pathway may be a valuable therapeutic target. [Science]
Monoclonal Antibody Targeting of N-Cadherin Inhibits Prostate Cancer Growth, Metastasis and Castration Resistance
Comparing gene expression in isogenic androgen-dependent and castration-resistant prostate cancer (CRPC) xenografts, researchers found a reproducible increase in N-cadherin expression, which was also elevated in primary and metastatic tumors of individuals with CRPC. [Nat Med]

Visual Activity Regulates Neural Progenitor Cells in Developing Xenopus CNS Through Musashi1
While proliferation of neural progenitors predominates during early central nervous system (CNS) development, progenitor cell fate shifts toward differentiation as CNS circuits develop, suggesting that signals from developing circuits may regulate proliferation and differentiation. Researchers tested whether activity regulates neurogenesis in vivo in the developing visual system of Xenopus tadpoles. [Neuron]
Induction of Human Epithelial Stem/Progenitor Expansion by FOXM1
This study provides the first evidence that FOXM1 participates in an early oncogenic pathway that predisposes cells to tumorigenesis by expanding the stem/progenitor compartment and deregulating subsequent keratinocyte terminal differentiation. [Cancer Res]
Prevention of Muscle Aging by Myofiber-Associated Satellite Cell Transplantation
Researchers demonstrate that engraftment of myofiber-associated satellite cells, coupled with an induced muscle injury, markedly alters the environment of young adult host muscle, eliciting a near-lifelong enhancement in muscle mass, stem cell number, and force generation. [Sci Transl Med]



New Stem Cell Action Coalition Launches Campaign Supporting Embryonic Stem Cell Research
The Genetics Policy Institute announced the launch of a new advocacy coalition of 30 organizations to foster public awareness and advance the cause of embryonic stem cell research in the United States. [Stem Cell Action Coalition, United States]



UCB and Plasticell Partner to Identify Compounds for Stem Cell Research and Cell Therapeutics
UK stem cell technologies firm Plasticell is to provide UCB with services based on its CombiCult™ screening technology to help identify new indications for compounds in stem cell biology, differentiation, and, potentially, tissue regeneration. [Genetic Engineering and Biotechnology News]

The Maryland Stem Cell Research Commission Announces Opportunity for Collaborative Research Grants with State of California Researchers
The Maryland Technology Development Corporation, which administers the Maryland Stem Cell Research Fund, announced a pilot program with the California Institute for Regenerative Medicine (CIRM). This pilot program provides an opportunity for Maryland scientists to conduct collaborative research with the recipients of CIRM Early Translational II Grant Awards. [Maryland Stem Cell Research Commission Press Release]
Clavis Pharma and Clovis Oncology Extend Partnership and Sign New $205 Million Deal for the Development and Commercialization of Anti-Cancer Agent CP-4126 in Asia and Rest of the World
Clavis Pharma ASA and Clovis Oncology, Inc. announced a new agreement for the further development and commercialization of CP-4126 (also known as CO-101), which is currently in Phase II development in pancreatic cancer. [Clavis Pharma Press Release]
Watson's Eden Biodesign and Crucell Sign Vendor Network Agreement Facilitating Access to cGMP Manufacturing Services for Vaccines and Gene Therapies Using PER.C6® Cell Line Technology
Eden Biodesign Limited announced the execution of a non-exclusive Vendor Network Agreement with Crucell, under which Eden Biodesign has become a pre-approved authorized provider of process development and cGMP manufacturing services using Crucell’s proprietary PER.C6® cell line technology. [Eden Biodesign Limited Press Release]
Jefferson Awarded Multi-Million Dollar NIH Grant to Create a Center of Excellence for Autoimmune Diseases
The Department of Neurology at Jefferson Medical College of Thomas Jefferson University has been awarded a multi-million dollar grant from the National Institutes of Health (NIH) for the creation of a center of excellence to study autoimmune diseases from basic science research to its translation into clinical applications. [Thomas Jefferson University Hospital Press Release]
Ceregene Raises $11.5 Million in Series D Financing
Ceregene, Inc. announced that it has closed its Series D preferred financing in the amount of $11.5 million. The funds will be used to help support Ceregene’s currently enrolling Phase IIb clinical study of CERE-120 for the treatment of Parkinson’s disease. [Ceregene, Inc. Press Release]
Delenex Secures Series A Financing
Delenex Therapeutics AG has announced the closing of its Series A venture equity investment round securing CHF 13.5 million (USD 14 million, EUR 10 million). [Delenex Therapeutics Press Release]

Intercytex Reforms as U.S. Department of Defense Funds Vavelta® Scar Therapy Trials for Injured Soldiers
Intercytex, the regenerative medicine company, announces that it has reformed as a private enterprise and that its lead product, the cell therapy (VAVELTA®) will be evaluated by the U.S. military to help treat injured soldiers. [Intercytex Press Release]

Boost for Regenerative Medicine Research in Ireland
In a major boost for Regenerative Medicine research in Ireland, the Minister for Enterprise, Trade and Innovation announced funding of almost EUR10 million for the Regenerative Medicine Institute (REMEDI) at NUI Galway. [Science Foundation Ireland Press Release]
Athersys Announces FDA Authorization of Phase II Clinical Trial for Inflammatory Bowel Disease with Pfizer Regenerative Medicine
Athersys, Inc. announced that the U.S. Food and Drug Administration (FDA) has authorized commencement of a Phase II clinical trial evaluating the safety and efficacy of administration of MultiStem®, Athersys' allogeneic investigational cell therapy product, for the potential treatment of ulcerative colitis. [Athersys, Inc. Press Release]
Oxford BioMedica Announces U.S. IND Approval for Novel Ocular Product in Wet AMD
Oxford BioMedica plc announced that the U.S. Food and Drug Administration has approved its Investigational New Drug (IND) application for the Phase I/II clinical development of RetinoStat®, a novel gene-based treatment for neovascular “wet” age-related macular degeneration (AMD). [Oxford BioMedica plc Press Release]
Prostate Cancer Drug Gets Lukewarm Review
There is only “moderate’’ evidence that the newly approved prostate cancer drug Provenge helps patients, according to an analysis done for Medicare that was made public. [The New York Times]
USC Opens Eli and Edythe Broad CIRM Center
California Governor Arnold Schwarzenegger joined University of Southern California (USC) administrators, trustees, elected public officials and more than 150 invited guests in the dedication and ribbon-cutting ceremony for the Eli and Edythe Broad California Institute for Regenerative Medicine Center for Regenerative Medicine and Stem Cell Research at USC. [University of Southern California Press Release]


Federal Budget Rollback Could Slash NIH Grant Success Rates
Funding rates for biomedical researchers could drop by half, to a historical low of 10%, if Republicans follow through on their vow to cut overall federal discretionary civilian spending to 2008 levels.
New Format Change Announced for National Institutes of Health, Agency for Healthcare Research and Quality, Centers for Disease Control and Prevention, and Food and Drug Administration Funding Opportunity Announcements (NOT-OD-11-019)

Notice of Clarification of the Project Period for Translational Scholar Awards in Pharmacogenomics and Personalized Medicine (K23) (PA-11-009) (NOT-RR-11-001)

Notice to Extend the Expiration Date for PA-07-458, Technology and Methods Development for Genomics, Population Genomics and ELSI (R01), and PA-07-459, Feasibility Studies to Develop Technology and Methods for Genomics, Population Genomics and ELSI (R21) (NOT-HG-11-005) 

Molecular Genetics of Drug Addiction and Related Co-Morbidities (R01) (PA-11-026)

Office of the Director, Office of Biotechnology Activities; Notice of Meeting (FR Doc. 2010-28373)
Center for Scientific Review; Notice of Closed Meetings (FR Doc. 2010-27743)

Center for Scientific Review; Amended Notice of Meeting (FR Doc. 2010-28467)
National Heart, Lung, and Blood Institute; Notice of Closed Meeting (FR Doc. 2010-28069)

Government-Owned Inventions; Availability for Licensing (FR Doc. 2010-27912)



PDA/FDA Adventitious Viruses in Biologics: Detection and Mitigation Strategies Workshop
The PDA/FDA Adventitious Viruses in Biologics: Detection and Mitigation Strategies Workshop is being organized currently as a result of recent viral contamination events. This workshop is intended to encourage modernization in industry with respect to viral detection and control measures. 
FY 2009 Annual Report: Innovative Technology Advancing Public Health




Majority of Drug and Biological Product Makers Meeting Postmarketing Requirements and Commitments
Most makers of approved drug and biological products are meeting their regulatory obligations and meeting targets for postmarketing studies/clinical trials in a timely manner, according to a study released by the U.S. Food and Drug Administration.
Guidance for Industry: Cellular Therapy for Cardiac Disease; Availability [Docket No. FDA-2009-D-0132]


European Medicines Agency and Massachusetts Institute of Technology Launch Joint Project on Regulatory Science
The European Medicines Agency and the Massachusetts Institute of Technology’s Center for Biomedical Innovation and Center for International Studies are launching a collaborative research project with a focus on enhancing regulatory science in pharmaceuticals.


Drugs Designated as Orphan Drugs
Added Afamelanotide (SCENESSE), ex vivo cultured adult human mesenchymal stem cells (PROCHYMAL) and Drug: Tobramycin (TOBI).

Consultation: Proposed Amendments to the Therapeutic Goods Regulations 1990 to Implement the Biologicals Framework

EVENTS (Listed by Date)

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

NEW London Regenerative Medicine Network Meeting
November 17, 2010
London, United Kingdom

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, Canada

Berlin-Brandenburg School for Regenerative Therapies (BSRT) PhD Symposium: Stem Cells – Hopes, Fears and Realities
November 24-25, 2010
Manchester, United Kingdom

American Association for Cancer Research (AACR) 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 (TERMIS) – 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

NEW Partnering for Cures
December 14-15, 2010
New York, United States

Pharmacology in Stem Cells Research and Regenerative Medicine
December 17, 2010
London, United Kingdom

NEW The 3rd Guangzhou International Conference on Stem Cell Biology and Regenerative Medicine
December 17-19, 2010
Guangzhou, China

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

NEW 4th Annual OneMedForum
January 11-13, 2011
San Francisco, 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, D.C., United States

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

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

16th Proteomics Symposium
February 3-6, 2011
Lorne, Australia

36th Lorne Conference on Protein Structure and Function
February 6-10, 2011
Lorne, Australia

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

NEW Stem Cell Research Forum of India 2nd Annual Meeting
February 8-11, 2011
Bangalore, India

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

32nd Lorne Genome Conference
February 13-15, 2011
Lorne, Australia

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

1st Annual Clinical Meeting of the Cell Society
February 18-19, 2011
La Jolla, 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

American Association for Cancer Research (AACR) 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

6th Annual Stem Cell Summit
March 1, 2011
New York, United States

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

Keystone Symposia: Stem Cells, Cancer and Metastasis
March 6-11, 2011
Dillon, United States

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

NEW 15th Annual Hilton Head Workshop – Regenerative Medicine, Innovations for Clinical Applications
March 16-19, 2011
Hilton Head Island, United States

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

March 22, 2011
Farmington, United States

United Kingdom National Stem Cell Network 2011 Annual Scientific Conference
March 30-April 1, 2011
York, United Kingdom

10th Annual New England Science Symposium
April 1, 2011
Boston, United States

American Association for Cancer Research (AACR) 102nd Annual Meeting 2011
April 2-6, 2011
Orlando, United States

5th RNAi & miRNA World Congress
April 26-27, 2011
Boston, United States

3rd Epigenetics World Congress
April 26-27, 2011
Boston, United States

Inaugural Next-Gen Sequencing Congress
April 26-27, 2011
Boston, United States

GTCbio 2nd International Conference on Cancer Immunotherapy & Immunomonitoring
May 2-5, 2011
Budapest, Hungary

Australasian Gene Therapy Society (AGTS) 7th Meeting
May 4-6, 2011
Melbourne, Australia

BioPartnering India
May 4-6, 2011
Bangalore, India

2nd European Biomarkers Summit
May 18-19, 2011
London, United Kingdom

Inaugural Theranostics for Personalised Medicine
May 18-19, 2011
London, United Kingdom

International Society for Cellular Therapy (ISCT) 17th Annual Meeting
May 18-21, 2011
Rotterdam, The Netherlands

American Society of Gene and Cell Therapy (ASGCT) 14th Annual Meeting
May 18-21, 2011
Seattle, United States

13th Annual C21 BioVentures
May 24-26, 2011
Napa, United States

Gordon Research Conference: Viruses & Cells
May 29-June 3, 2011
Lucca (Barga), Italy

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


Lab Technologist – Human Embryonic and Induced Pluripotent Stem Cells (STEMCELL Technologies)

Lab Technologist – Tissue Culture (STEMCELL Technologies)

Product Manager – Pluripotent Stem Cells (STEMCELL Technologies)

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