Volume 12.17 | May 2

Cell Therapy News 12.17, May 2, 2011
     In this issue: Science | Policy | Business | NIH | CBER | Regulatory | Events
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Scientists Develop Compound that Effectively Halts Progression of Multiple Sclerosis
Scientists have developed the first of a new class of highly selective compounds that effectively suppresses the severity of multiple sclerosis in animal models. [Press release from The Scripps Research Institute discussing online prepublication in Nature]



Blocking Crucial Molecule Could Help Treat Multiple Sclerosis, Neuroscientists Say
Neuroscientists have identified a driving force behind autoimmune diseases such as multiple sclerosis, and suggest that blocking this cell-signaling molecule is the first step in developing new treatments to eradicate these diseases. [Press release from Thomas Jefferson University Hospital discussing online prepublication in Nature Immunology]

Liver-Cell Transplants Show Promise in Reversing Genetic Disease Affecting Liver and Lungs
Transplanting cells from healthy adult livers may work in treating a genetic liver-lung disorder that affects millions of people worldwide, according to an animal study. [Press release from the Albert Einstein College of Medicine discussing online prepublication in the Journal of Clinical Investigation]

Researchers Discover Mechanism that Could Convert Certain Cells into Insulin-Making Cells
Researchers report that they may have discovered the underlying mechanism that could convert other cell types into pancreatic beta cells. [Press release from the University of California, Los Angeles discussing online prepublication in Developmental Cell]

Scientist Makes Key Innovations in Stem-Cell Technology
A scientist has made two significant stem-cell discoveries that advance medicine and human health by creating powerful new approaches for using stem cells and stem-cell-like technology. [Press release from Gladstone Institute discussing online prepublication in the Proceedings of the National Academy of Science]

Scientists Create Stable, Self-Renewing Neural Stem Cells
Researchers report a game-changing advance in stem cell science: the creation of long-term, self-renewing, primitive neural precursor cells from human embryonic stem cells that can be directed to become many types of neuron without increased risk of tumor formation. [Press release from the University of California, San Diego discussing online prepublication in the Proceedings of the National Academy of Science]

Scientists Discover New Drug Target for Kidney Disease
Two discoveries point to potential new drug therapies for patients with kidney disease. First, researchers discovered a molecular mechanism that sheds light on the disease. Second, the team discovered that STAT3 is strongly, and aberrantly, activated in polycystic kidneys. [Press release from the University of California, Santa Barbara discussing online prepublication in the Proceedings of the National Academy of Science]

Removable ‘Cloak’ for Nanoparticles Helps Them Target Tumors
Like most other drug-delivering nanoparticles, the new particles are cloaked in a polymer layer that protects them from being degraded by the bloodstream. However, the research team designed this outer layer to fall off after entering the slightly more acidic environment near a tumor and reveals another layer that is able to penetrate individual tumor cells. [Press release from the Massachusetts Institute of Technology discussing online prepublication in ACS Nano]

Tekmira and the National Cancer Institute Publish Promising Data Demonstrating the Anti-Tumor Activity of a Novel Cancer Target
Tekmira Pharmaceuticals Corporation announced that promising preclinical data from its collaboration with the National Cancer Institute has been published and presents encouraging pre-clinical data related to the treatment of liver cancer using small interfering RNA enabled by Tekmira’s lipid nanoparticle delivery technology. [Press release from Tekmira Pharmaceuticals Corporation discussing online prepublication in Oncogene]

Finding Molecular Targets of an HIV Drug Used in Cancer Therapy
Researchers have identified potential human molecular targets of the anti-HIV drug Nelfinavir, which may explain why the drug is also effective as a cancer therapy. [Press release from the University of California, San Diego discussing online prepublication in PLoS Computational Biology]

Study Shows Promise for Developing Protein Therapies for Disease Prevention
A computer analysis by researchers shows promise for helping develop therapies for some major diseases by rescuing proteins that have stopped performing normally. [Press release from the University of Michigan Health System discussing online prepublication in Biophysical Journal]

Gene Therapy Shows Promise Against Age-Related Macular Degeneration
A gene therapy approach using a protein called CD59, or protectin, shows promise in slowing the signs of age-related macular degeneration, according to a new in vivo study. [Press release from Tufts University discussing online prepublication in PLoS ONE]

New Technique Extends Cancer-Fighting Cells’ Potency in Melanoma Patients
Scientists have developed a technique that can cause white blood cells to survive in patients’ bloodstreams for well over a year, in some cases, without the need of other, highly toxic treatments. [Press release from Dana-Farber Cancer Institute discussing online prepublication in Science Translational Medicine]

Juventas Therapeutics Completes Successful Phase I Clinical Trial for JVS-100 in Treatment of Patients with Heart Failure
Juventas Therapeutics announced that it has successfully completed its Phase I clinical trial evaluating the safety and preliminary efficacy of JVS-100 for treatment of patients with heart failure. [Press release from Juventas Therapeutics discussing research presented at the Annual Meeting for the American College of Cardiology]

Penn Scientists Develop a New Way to Re-Grow Cartilage
A group of scientists working across disciplines has developed a novel way to allow patients to re-grow cartilage in their own bodies, using their own cells, directly in the site that has been damaged through injury or disease. [Penn State University Press Release]


(Ranked by Impact Factor of the Journal)

Suppression of TH17 Differentiation and Autoimmunity by a Synthetic ROR Ligand
Scientists present SR1001, a high-affinity synthetic ligand that is specific to both retinoic-acid-receptor-related orphan receptors (ROR) α and γt and which inhibits interleukin-17 (TH17) cell differentiation and function. [Nature]

The Encephalitogenicity of TH17 Cells Is Dependent on IL-1- and IL-23-Induced Production of the Cytokine GM-CSF
Researchers found that IL-23 induced production of the cytokine granulocyte-macrophage colony-stimulating factor (GM-CSF) in TH17 cells and that GM-CSF had an essential role in their encephalitogenicity. [Nat Immunol]

Spontaneous Hepatic Repopulation in Transgenic Mice Expressing Mutant Human α1-Antitrypsin by Wild-Type Donor Hepatocytes
Because mutant protein accumulation increases hepatocyte cell stress, scientists investigated whether transplanted hepatocytes expressing wild-type α1-antitrypsin might have a competitive advantage relative to α1-antitrypsin protein (AAT-Z)–expressing hepatocytes, using transgenic mice expressing human AAT-Z. [J Clin Invest]

Pancreatic β Cell Identity Is Maintained by DNA Methylation-Mediated Repression of Arx
Investigators report that β cells deficient in Dnmt1, an enzyme that propagates DNA methylation patterns during cell division, were converted to α cells. [Dev Cell]

Rapid Induction and Long-Term Self-Renewal of Primitive Neural Precursors from Human Embryonic Stem Cells by Small Molecule Inhibitors
Investigators report synergistic inhibition of glycogen synthase kinase 3, transforming growth factor β, and Notch signaling pathways by small molecules can efficiently convert monolayer cultured human embryonic stem cells into homogenous primitive neuroepithelium within 1 week under chemically defined condition. [Proc Natl Acad Sci U S A]

Direct Reprogramming of Mouse Fibroblasts to Neural Progenitors
Scientist show that transient induction of the four reprogramming factors (Oct4, Sox2, Klf4, and c-Myc) can efficiently transdifferentiate fibroblasts into functional neural stem/progenitor cells (NPCs) with appropriate signaling inputs. [Proc Natl Acad Sci U S A]

Polycystin-1 Regulates STAT Activity by a Dual Mechanism
Scientists show that membrane-anchored polycystin-1 activates STAT3 in a JAK2-dependent manner, leading to tyrosine phosphorylation and transcriptional activity. [Proc Natl Acad Sci U S A]

Layer-by-Layer Nanoparticles with a pH-Sheddable Layer for In Vivo Targeting of Tumor Hypoxia
Inspired by the simplicity and versatility of layer-by-layer assembly, researchers applied multilayered polyelectrolyte assemblies on nanoparticles to create viable systemic delivery systems. [ACS Nano]

Molecular Targeting of CSN5 in Human Hepatocellular Carcinoma: A Mechanism of Therapeutic Response
Investigators explored the possibility of the fifth subunit of COP9 signalosome (CSN5) being a potential therapeutic target for hepatocellular carcinoma. [Oncogene]

A Non Membrane-Targeted Human Soluble CD59 Attenuates Choroidal Neovascularization in a Model of Age Related Macular Degeneration
Scientists demonstrate that subretinal delivery of an adenovirus vector expressing a transgene for a soluble non-membrane binding form of human CD59 can attenuate the formation of laser-induced choroidal neovascularization and murine membrane attack complex formation in mice even when the region of vector delivery is distal to the site of laser induced choroidal neovascularization. [PLoS One]



California Ponders Cell-Banking Venture
Investigators creating a wealth of adult-derived stem-cell lines may soon get new banks for storing them. [California Institute for Regenerative Medicine, United States]

NIH Wins Appeal of Stem Cell Injunction
A federal appeals court has ruled in favor of the NIH in a high-stakes law suit challenging the legality of the Obama Administration’s policy on human embryonic stem cells. [U.S. Court of Appeals, United States]

Spending Bill Prohibits U.S.-China Collaborations
A little-noticed clause in the 2011 spending bill signed into law last week cuts off funding for a host of scientific exchanges between the United States and China. [White House, United States]

Drug Industry: President Reneges on Health-Reform Deal
The White House’s new deficit plan has the drug industry crying foul, and that means Big Pharma may now join Big Insurers to push back on health reform. [White House, United States]

Hopes Brighten for Embattled Australian Medical Researchers
Australian Prime Minister Julia Gillard on 19 April sought to reassure scientists that her government was rethinking a rumored $423 million (AUD $400 million) cut to medical research over 3 years, starting in the 2011-12 budget. [Australian Prime Minister, Australia]

Scientists Oppose European Stem Cell Patent Ban
Research scientists hit out at a European Court of Justice case they say could block development of embryonic stem cell-based therapies in Europe. [European Court of Justice, Luxembourg]


Paul Sanberg Receives Everfront Award at Stem Cell Conference in Taiwan
Dr. Paul Sanberg received the Everfront Award at the 4th Pan Pacific Symposium on Stem Cell and Cancer Research. [University of South Florida Press Release]

OncoMed Pharmaceuticals and Bayer HealthCare Expand Cancer Stem Cell Collaboration with Clinical Manufacturing Agreement
OncoMed Pharmaceuticals, Inc. announced it has expanded its collaboration with Bayer HealthCare. [OncoMed Pharmaceuticals, Inc. Press Release]

UC Berkeley Launches Synthetic Biology Institute to Advance Research in Biological Engineering
An alliance of top researchers at the University of California (UC), Berkeley, has formed the UC Berkeley Synthetic Biology Institute, advancing efforts to engineer cells and biological systems in ways that promise to transform technology in health and medicine, energy, the environment, new materials, and a host of other critical arenas. [University of California, Berkeley Press Release]

USC Invests in Biomedical Engineering Innovations
The University of Southern California (USC) Viterbi School of Engineering, the Keck School of Medicine of USC, the USC Stevens Institute for Innovation and the Los Angeles Basin Clinical Translational Science Institute have been selected to participate in the exclusive Coulter Translational Research Partnership Program. [University of Southern California Press Release]

A. Alfred Taubman Increases His Support for Medical Science Institute at U-M to $100 Million
A. Alfred Taubman has become University of Michigan (U-M)’s largest individual donor, with total giving of more than $142 million. [University of Michigan Health System Press Release]

Whittier Foundation Awards $3 Million Nanotechnology Grant to Keck School
The L.K. Whittier Foundation is awarding another $3 million to the nanobiotechnology initiative it established four years ago at the Keck School of Medicine. [University of Southern California Press Release]

NIH Grant Launches Center for Reproductive and Stem Cell Biology
The Center for Reproductive and Stem Cell Biology has been tapped to become one of a select group of National Institutes of Health (NIH) research centers, as a part of a grant of about $10 million awarded earlier. [Stanford University School of Medicine Press Release]

NIH-Funded Centers to Assist Rehabilitation Researchers
The National Institutes of Health (NIH) has provided approximately $30 million over a five year period to fund a network of centers to advance medical rehabilitation research. The centers provide researchers with access to new technologies and resources. [National Institutes of Health Press Release]

RXi Pharmaceuticals Awarded $580,000 in NIH Grants to Advance RNAi Therapeutics
RXi Pharmaceuticals Corporation announced that it was awarded two Small Business Innovation Research (SBIR) grants from the National Institutes of Health (NIH). [RXi Pharmaceuticals Corporation Press Release]

Rice Wins $1.2 Million for Heart-Valve Tissue Research
A team of bioengineers from Rice University is bringing a promising new strategy for growing replacement heart valves closer to reality, thanks to a four-year, $1.2 million grant from the National Institutes of Health. [Rice University Press Release]

Celsense, Inc. Announces Patent Issued for New MRI Reporter Gene
Celsense, Inc. announced that it has received a Notice of Allowance from the U.S. Patent and Trademark Office for its patent application 11/080,732 with claims covering its MRI reporter gene product, Gene Sense. [Celsense, Inc. Press Release]

International Stem Cell Successfully Completes First Series of Preclinical Testing of Hepatocytes Derived from Human Parthenogenetic Stem Cells
International Stem Cell Corporation announces successful completion of the first series of preclinical testing of hepatocytes derived in the lab from human parthenogenetic stem cells. [International Stem Cell Corporation Press Release]

BCM Enrolling Patients for Parkinson’s Study Evaluating CERE-120
Restoring brain function and protecting brain cells of people suffering from Parkinson’s disease is the goal of a new double blind Phase IIb study evaluating CERE-120. Baylor College of Medicine (BCM) is one of 11 major U.S. medical centers currently enrolling patients. [Baylor College of Medicine Press Release]

Tissue Genesis Begins FDA‐Approved Clinical Trial
Tissue Genesis, Inc. announced that it has begun clinical trials for peripheral vascular disease with
adipose derived adult stem cell‐coated vascular grafts at the University of Louisville Hospital. [Tissue Genesis, Inc. Press Release]

Tarix Pharmaceuticals Enrolls First Patient in Phase I Clinical Study of TXA127 in Adult Patients Following Double Cord Blood Stem Cell Transplant
Tarix Pharmaceuticals announced enrollment of the first patient in a Phase I clinical study of TXA127, a pharmaceutical grade formulation of a naturally occurring peptide known to stimulate early hematopoietic precursor cells in the bone marrow. [PR Newswire]

OncoMed Pharmaceuticals Announces FDA Clearance to Commence Phase I Testing of Anti-Cancer Stem Cell Therapeutic OMP-18R5
OncoMed Pharmaceuticals, Inc. announced that the FDA has accepted an IND filing for OMP-18R5, which allows OncoMed to commence Phase I clinical testing. [OncoMed Pharmaceuticals, Inc. Press Release]

Novelos Therapeutics Welcomes Five New Directors
Novelos Therapeutics, Inc. announced that Dr. Rock Mackie, Mr. John Neis, Dr. John Niederhuber, Dr. Michael Tweedle and Dr. Jamey Weichert joined Novelos’ board of directors concurrently with the closing of the recently announced acquisition of Cellectar, Inc. [Novelos Therapeutics, Inc. Press Release]

Arrowhead Research Announces RNAi Leader John J. Rossi, Ph.D. to Join Calando Scientific Advisory Board
Arrowhead Research Corporation announced that John J. Rossi, Ph.D., a pioneer and one of the world leaders in the field of RNAi discovery and development, has joined majority owned subsidiary Calando Pharmaceuticals, Inc. as a senior advisor. [Arrowhead Research Corporation Press Release]

Stanford Creates First PhD Program in Stem Cell Science
Stanford University’s Faculty Senate approved the creation of what officials believe is the first stem cell science PhD program in the nation and, perhaps, the world. The new doctoral program in stem cell biology and regenerative medicine is also the first interdisciplinary doctoral program created by the School of Medicine in recent years. [Stanford University School of Medicine Press Release]


NIH Trims Grants in Wake of Budget Cuts
U.S. biomedical researchers will need to tighten their belts as a result of the 1% drop in the National Institutes of Health’s 2011 budget that became law 2 weeks ago.

New NIH Council of Councils Members Named
The National Institutes of Health today announced the appointments of 20 individuals to the NIH Council of Councils.

NIH Establishes Working Group on the Future Biomedical Research Workforce
A new working group at the National Institutes of Health will examine the future of the biomedical research workforce in the United States. The group will recommend actions to the Advisory Committee to the Director to ensure a diverse and sustainable biomedical and behavioral research workforce.

NIGMS Issues Its First Strategic Plan for Research Training
The National Institute of General Medical Sciences (NIGMS), part of the National Institutes of Health, issued Investing in the Future, a strategic plan for its biomedical and behavioral research training programs.

Proposed Collection; Comment Request; Health Information National Trends Survey 4 (HINTS 4) (FR Doc. 2011-9827)

Center for Scientific Review; Notice of Closed Meetings (FR Doc. 2011-9822)

Center for Scientific Review; Notice of Closed Meetings (FR Doc. 2011-9823)

Center for Scientific Review; Notice of Closed Meetings (FR Doc. 2011-9936)

Center for Scientific Review; Notice of Closed Meetings (FR Doc. 2011-10292)

Center for Scientific Review; Notice of Closed Meetings (FR Doc. 2011-10297)

Center for Scientific Review; Amended Notice of Meeting (FR Doc. 2011-10497

National Center for Research Resources; Notice of Closed Meeting (FR Doc. 2011-10471)

National Institute of General Medical Sciences; Notice of Meeting (FR Doc. 2011-10480)

National Institute of Biomedical Imaging and Bioengineering; Notice of Closed Meeting (FR Doc. 2011-10009)

National Heart, Lung, and Blood Institute; Notice of Meeting (FR Doc. 2011-10304)

Additional Extension of Comment Period on Proposed Adoption and Implementation of the Guide for the Care and Use of Laboratory Animals: Eighth Edition and Removal of Character Limit for Comments (NOT-OD-11-066)

Request for Information (RFI) Related to a Planned Funding Opportunity for Support of Basic and Translational Research in the Area of Tumor Glycomics (NOT-CA-11-012)

Clarification of the Scope and Requirements of PAR-11-150, “Quantitative Imaging for Evaluation of Responses to Cancer Therapies (U01)” (NOT-CA-11-011 )

Notice of Cancellation of Integrated Preclinical/Clinical AIDS Vaccine Development Program (IPCAVD) (U19) (NOT-AI-11-043)

RFP Announcement: An Integrated Approach to Understanding Host-Pathogens Interactions – RFP NIAID-DMID-NIHAI2010100 (NOT-AI-11-038 )


Guidance for Industry: “Computer Crossmatch” (Computerized Analysis of the Compatibility between the Donor’s Cell Type and the Recipient’s Serum or Plasma Type)

April 6, 2011: Vaccines and Related Biological Products Advisory Committee Meeting Transcript

April 7, 2011: Vaccines and Related Biological Products Advisory Committee Meeting Transcript

May 12, 2011: Allergenic Products Advisory Committee Meeting Draft Agenda

May 12, 2011: Allergenic Products Advisory Committee Meeting Public Roster

May 12, 2011: Allergenic Products Advisory Committee Meeting Document: ISO 17025 Accreditation of the Laboratory of Immunobiochemistry

May 12, 2011: Allergenic Products Advisory Committee Meeting Bibliography



FDA Drug Safety Communication: Safety Update on Progressive Multifocal Leukoencephalopathy (PML) Associated with Tysabri (natalizumab)
The U.S. Food and Drug Administration (FDA) continues to evaluate the risk of PML, a rare but serious brain infection, associated with use of Tysabri (natalizumab) for the treatment of multiple sclerosis and Crohn’s disease.

Withdrawal of Approval of New Animal Drug Applications; Phenylbutazone; Pyrantel; Tylosin; Sulfamethazine; Correction [Docket No. FDA-2011-N-0033]

Endocrinologic and Metabolic Drugs Advisory Committee; Notice of Meeting [FR Doc. 2011-10003]

Periodic Review of Existing Regulations; Retrospective Review Under E.O. 13563 [FR Doc. 2011-10131]

Request for Application: Advancing Regulatory Science through Novel Biomarker Research and Science-Based Technologies (U01) [RFA-FD-11-016]

NLM Express Research Grants in Biomedical Informatics (R01) [PAR-11-208]

Corrections to RFA-HL-12-025 Data Coordinating Center for the Cardiovascular Cell Therapy Research Network (CCTRN) (UM1) [NOT-HL-11-140]

Corrections to RFA-HL-12-026 Regional Clinical Centers for the Cardiovascular Cell Therapy Research Network (CCTRN) (UM1) [NOT-HL-11-141]

Notice of Intent to Publish a Funding Opportunity Announcement for a H3Africa Bioinformatics Network [NOT-RM-11-014]


Scheduling of Medicines and Poisons: Delegate’s Decisions
Reasons for delegate’s interim decisions and invitation for further comment.

Scheduling of Medicines and Poisons: Reasons for Delegate’s Interim Decisions, April 2011


NEW The Jackson Laboratory JAX Colony Management System (JCMS) Database Live Tutorial
May 12th, 2011

 The Institute of Cancer Research Integrative Network Biology and Cancer 2.0
May 13-14, 2011
London, United Kingdom

NEW Cold Spring Harbor Asia Conferences Translational Approaches to Cancer
May 24-28, 2011
Suzhou, China

NEW Keystone Symposia: Changing Landscape of the Cancer Genome
June 20-25, 2011
Boston, United States

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.


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

Quality Assurance Manager – Program in Children’s Regenerative Medicine (The University of Texas Medical School at Houston)

Implementation Coordinator – Maternity Leave Contract (STEMSOFT Software Inc.) 

Tenure Track, Assistant/Associate Professor (University of Missouri-Kansas City School of Medicine)

Postdoc – Laboratory of Molecular Tumor Genetics (Duke-NUS Graduate Medical School)

Postdoctoral Research Position – Mucosal Immunology (Department of Microbiology and Immunology, Columbia University)

Postdoctoral Position – Vascular Smooth Muscle Biology (Boston University Medical School)

Supervisor Cell Therapy Laboratory – Cord Blood Bank (The University of Texas MD Anderson Cancer Center)

Supervisor Cell Therapy Lab (The University of Texas MD Anderson Cancer Center) 

Postdoctoral Positions in Immune Cell Activation (Institut de Recherches Cliniques de Montréal)

Postdoctoral Position in Developmental Neurobiology (Institut de Recherches Cliniques de Montréal)

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