Volume 12.06 | Feb 14

     In this issue: Science | Policy | Business | NIH | CBER | Regulatory | Events
Skin Cells Help to Develop Possible Heart Defect Treatment in First-of-Its-Kind Study
Using skin cells from young patients who have a severe genetic heart defect, scientists have generated beating heart cells that carry the same genetic mutation. The newly created cardiomyocytes allowed the researchers for the first time to examine and characterize the disorder at the cellular level. [Press release from Stanford University School of Medicine discussing online prepublication in Nature]


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Searching for the Soul of the Genome
Scientists have developed a novel approach to detect long-distance chromosomal interactions and have applied this method to the chromosome 9 gene desert, revealing that the association results from an altered inflammatory signaling response in individuals with increased coronary artery disease risk. [Press release from the University of California, San Diego School of Medicine discussing online prepublication in Nature]

Insight into Major Cause of Age-Related Blindness
A team of researchers has discovered a molecular mechanism implicated in geographic atrophy, the major cause of untreatable blindness in the industrialized world. [Press release from the University of Kentucky discussing online prepublication in Nature]

Boosting Body’s Immune Response May Hold Key to HIV Cure
Australian scientists have successfully cleared an HIV-like infection from mice by boosting the function of cells vital to the immune response. A team has shown that a cell signaling hormone called interleukin-7 reinvigorates the immune response to chronic viral infection, allowing the host to completely clear virus. [Press release from the Walter and Eliza Hall Institute discussing online prepublication in Cell]

Discovery Provides New Insights into Pluripotent Stem Cell Biology
A discovery suggests that a signaling molecule critical for proper embryonic development of a multicellular organism also plays a role in maintaining the fundamental properties of pluripotent stem cells. Researchers have found that a protein called beta-catenin controls the ability of mouse embryonic stem cells to differentiate into various new types of specialized cells such as neurons. [Press release from McMaster University discussing online prepublication in Cell Stem Cell]

Addiction to Life-Saving, Self-Digestion Process Can Aid Cancer Cells in Tumor Growth
Researchers have determined that cancer cells are “addicted” to autophagy.  They also showed that the inhibition of that process could prove to be a valuable treatment approach for aggressive cancers. [Press release from The Cancer Institute of New Jersey discussing online prepublication in Genes and Development]

Early Infusion of Donor T Regulatory Cells Prevents Graft-Versus-Host Disease and Enhances Immune Recovery in High-Risk Blood Cancer Patients
In a trial conducted at the University of Perugia in Italy by a group that has pioneered the use of partially matched donors, researchers explored the use of regulatory T cells that control immune reactions. [Press release from the American Society of Hematology discussing online prepublication in Blood]
Researchers Turn Salmonella into Anti-Viral Gene Therapy Agent
Researchers have reprogrammed Salmonella into a safe transport vehicle for virus-stopping enzymes. Not only did this technique effectively treat mice infected with cytomegalovirus, it worked as an oral solution that was swallowed instead of injected. [Press release from the University of California, Berkeley discussing online prepublication in the Proceedings of the National Academy of Sciences USA]

A New Way to Attack Pathogens
Scientists demonstrate that by stopping bacteria’s ability to degrade RNA — a “housekeeping” process crucial to their ability to thrive — scientists were able to stop methicillin-resistant Staphylococcus aureus or MRSA both in the laboratory and in infected mice. [Press release from the University of Rochester Medical Center discussing online prepublication in PLoS Pathogens]

How Nasal Stem Cells Might Prevent Childhood Deafness
Scientists have shown for the first time in mice that nasal stem cells injected into the inner ear have the potential to reverse or restore hearing during early onset sensorineural hearing loss. [Press release from the Garvan Institute discussing online prepublication in Stem Cells]

Nanoparticles May Enhance Circulating Tumor Cell Detection
Tiny gold particles can help doctors detect tumor cells circulating in the blood of patients with head and neck cancer, researchers have found. [Press release from Emory University discussing online prepublication in Cancer Research]

Dramatic Improvement in Parkinson Disease Symptoms Following Intranasal Delivery of Stem Cells to Rat Brains
Successful intranasal delivery of stem cells to the brains of rats with Parkinson disease yielded significant improvement in motor function and reversed the dopamine deficiency characteristic of the disease. [Press release from ScienceDaily discussing online prepublication in Rejuvenation Research]

Researchers From UTHealth and Athersys Present Preclinical Data Illustrating Potential Benefits of MultiStem® for Treating Stroke
Medical researchers from The University of Texas Health Science Center at Houston (UTHealth) presented new research results that demonstrated how MultiStem®, a novel stem cell therapy being developed by Athersys, Inc., provided multiple benefits when administered in preclinical models of ischemic stroke. [Press release from Athersys, Inc. discussing research presented at the American Heart Association International Stroke Conference 2011]

Tolerx Presents Preclinical Data on Novel Cancer Immunotherapeutic, TRX518, a First-in-Class Anti-GITR Antibody
Tolerx, Inc. presented results from preclinical studies with TRX518, a first-in-class immunomodulatory agent for the treatment of cancer. TRX518 is a monoclonal antibody reactive with the glucocorticoid-induced tumor necrosis factor receptor (GITR) and is designed to enhance the immune system‘s anti-tumor response by enabling T cells to more effectively attack cancer cells. [Press release from Tolerx, Inc. discussing research presented at the Keytsone Symposia Antibodies as Drugs Meeting]

Flu Breakthrough Promises a Vaccine to Kill All Strains
Scientists at Oxford University have successfully tested a universal flu vaccine that could work against all known strains of the illness. The vaccine targets proteins inside the flu virus that are common across all strains, instead of those that sit on the virus’s external coat, which are liable to mutate. While traditional vaccines prompt the body to create antibodies, this vaccine boosts the number of the body’s T-cells. [The Guardian]
CURRENT PUBLICATIONS (Ranked by Impact Factor of the Journal)

Using Induced Pluripotent Stem Cells to Investigate Cardiac Phenotypes in Timothy Syndrome
To explore the effect of the Timothy syndrome mutation on the electrical activity and contraction of human cardiomyocytes, researchers reprogrammed human skin cells from Timothy syndrome patients to generate induced pluripotent stem cells, and differentiated these cells into cardiomyocytes. [Nature]

9p21 DNA Variants Associated with Coronary Artery Disease Impair Interferon-Gamma Signaling Response
Findings establish a link between coronary artery disease genetic susceptibility and the response to inflammatory signaling in a vascular cell type and thus demonstrate the utility of genome-wide association study findings in directing studies to novel genomic loci and biological processes important for disease etiology. [Nature]

DICER1 Deficit Induces Alu RNA Toxicity in Age-Related Macular Degeneration
Researchers show that the microRNA-processing enzyme DICER1 is reduced in the retinal pigmented epithelium (RPE) of humans with geographic atrophy, and that conditional ablation of Dicer1, but not seven other miRNA-processing enzymes, induces RPE degeneration in mice. [Nature]

IL-7 Engages Multiple Mechanisms to Overcome Chronic Viral Infection and Limit Organ Pathology
Mice infected with LCMV clone-13 have persistent high-level viremia and a dysfunctional immune response. Interleukin-7, a cytokine that is critical for immune development and homeostasis, was used here to promote immunity toward clone-13, enabling elucidation of the inhibitory pathways underlying impaired antiviral immune response. [Cell]

Beta-Catenin Enhances Oct-4 Activity and Reinforces Pluripotency Through a TCF-Independent Mechanism
Researchers used mouse embryonic stem cells (ESCs) lacking glycogen synthase kinase-3 (GSK-3) to demonstrate that a single GSK-3 substrate, beta-catenin, controls the ability of ESCs to exit the pluripotent state and to differentiate into neurectoderm. [Cell Stem Cell]

Activated Ras Requires Autophagy to Maintain Oxidative Metabolism and Tumorigenesis
Researchers report that expression of a H-rasV12 or K-rasV12 oncogene up-regulates basal autophagy, which is required for tumor cell survival in starvation and in tumorigenesis. [Genes Dev]

Tregs Prevent GvHD and Promote Immune Reconstitution in HLA-Haploidentical Transplantation
Researchers show for the first time in humans that adoptive transfer of T regulatory cells (Tregs) prevented GvHD in the absence of any post-transplant immunosuppression, promoted lymphoid reconstitution, improved immunity to opportunistic pathogens and did not weaken the Graft vs. Leukemia effect. [Blood]

Oral Delivery of RNase P Ribozymes by Salmonella Inhibits Viral Infection in Mice
Results provide direct evidence that oral delivery of M1GS RNA by Salmonella-based vectors effectively inhibits viral gene expression and replication in mice. Moreover, this study demonstrates the utility of Salmonella-mediated oral delivery of RNase P ribozyme for gene-targeting applications in vivo. [Proc Natl Acad Sci U S A]

Small Molecule Inhibitors of Staphylococcus aureus RnpA Alter Cellular mRNA Turnover, Exhibit Antimicrobial Activity, and Attenuate Pathogenesis
Findings indicate that RnpA, either alone, as a component of the RNase P holoenzyme, and/or as a member of a more elaborate complex, may play a role in S. aureus RNA degradation and provide proof of principle for RNA catabolism-based antimicrobial therapy. [PLoS Pathog]

Functional Effects of Adult Human Olfactory Stem Cells on Early-Onset Sensorineural Hearing Loss
Results indicate that transplantation of adult human olfactory mucosa-derived stem cells can help preserve auditory function during early-onset progressive sensorineural hearing loss. [Stem Cells]
Detection of Circulating Tumor Cells in Human Peripheral Blood Using Surface-Enhanced Raman Scattering Nanoparticles
Researchers report a new method using Surface-Enhanced Raman Spectroscopy to directly measure targeted circulating tumor cells in the presence of white blood cells. [Cancer Res]



House Spending Panel Makes Deep Cuts to Research
The appropriations committee in the House of Representatives has proposed cutting $62 billion from current spending across the government, including large reductions at the three leading agencies funding basic research: the National Institutes of Health, the Department of Energy, and the National Science Foundation. [House of Representatives, United States]

Second Chance for NIH Grant Applicants
Did you miss the funding cutoff despite a stellar score on your U.S. National Institutes of Health (NIH) grant? Don’t give up hope. The National Health Council, a Washington, D.C.-based nonprofit umbrella group for some 100 patient-advocacy groups and companies, unveiled a database on Monday that will hook up rejected projects and potential funders. [National Health Council, United States]

Europe Asks Researchers for Funding Ideas
The European Commission is asking for help designing — and renaming — its next research and innovation funding program. The current program, Framework Programme 7, will spend EUR 53.3 billion over 7 years before it ends in 2013. [European Commission, European Union]

Setting the Record Straight on Neuroscience Funding
The Medical Research Council sets the record straight following recent concerns in the research community about the level of investment in neuroscience research in the UK. [Medical Research Council Press Release]

Public Meeting of the Presidential Commission for the Study of Bioethical Issues; Notice of Meeting (FR Doc. 2011-3023) [Department of Health and Human Services, United States]



Griffith University in Queensland Australia Signs International Affiliation Agreement with the Centre for Drug Research and Development (CDRD)
The Centre for Drug Research and Development announced that Griffith University in Queensland Australia has signed an international affiliation agreement allowing for collaboration and the exchange of information between CDRD and Griffith’s researchers, which includes the Eskitis Institute for Cell and Molecular Therapies and the Griffith Health Institute. [Centre for Drug Research and Development Press Release]

Lilly and the Juvenile Diabetes Research Foundation Partner to Fund Regenerative Medicine Research in Type 1 Diabetes
Eli Lilly and Company and the Juvenile Diabetes Research Foundation announced that they have signed an agreement to fund early-stage research that could enable patients with type 1 diabetes to regenerate insulin-producing cells destroyed by the disease. [Juvenile Diabetes Research Foundation Press Release]

Elan and Boehringer Ingelheim Announce Development and Manufacturing Agreement for Elan’s Antibody-Based Therapeutics Pipeline
Elan plc and Boehringer Ingelheim announced that they have entered into a global technical development and manufacturing agreement for antibody-based therapeutics. [Boehringer Ingelheim GmbH Press Release]

GSK Plots New Approach to Working with Academia
Pharma and biotech companies have long recognized academia as a rich source of innovative early research. In most cases companies cherry-pick the most promising projects and license them from the universities where they originated, ending academic involvement. But GlaxoSmithKline is adopting a new approach. The pharma plans to work closely with medical researchers over the entire decade-plus drug development process. [FierceBiotech]

Mesoblast Issues New Equity to Cephalon, Receives Further Cash Injection
Regenerative medicine company Mesoblast Limited reported that it had issued an additional 24,702,056 ordinary shares to global biopharmaceutical company Cephalon Inc., and received an additional cash injection of $139 million. [Mesoblast Limited Press Release]

Lundbeck Expands Its Commercial Opportunities in Canada and Latin America
H. Lundbeck A/S announced that Lundbeck has been granted commercial rights to several Cephalon products in Canada and Latin America. As part of the agreement, Lundbeck will register and commercialize several key products which are currently available in the U.S. and/or Europe on behalf of Cephalon. [H. Lundbeck A/S Press Release]

UT Southwestern Scientist Awarded $450,000 Grant for Cancer Research
Dr. Benjamin P. Tu, assistant professor of biochemistry at the University of Texas (UT) Southwestern Medical Center, has received a Rachleff Innovation Award from the Damon Runyon Cancer Research Foundation. Dr. Tu, who investigates how cell growth and proliferation are coordinated with metabolism in a cell, is one of five recipients selected to get the three-year, $450,000 grants this year. [University of Texas Southwestern Medical Center Press Release]

Pace Picks Up for Clinical Trials to Evaluate Stem Cell Therapies
Stem cell science remains a fast-moving frontier — that much was made clear by leading scientists discussing their latest discoveries during a scientific symposium honoring the opening of the Ray and Dagmar Dolby Regeneration Medicine Building on the University of California, San Francisco Parnassus campus. [University of California, San Francisco Press Release]

Neuralstem Receives FDA Orphan Designation for Spinal Cord Stem Cells for Treatment of ALS
Neuralstem, Inc. announced that the U.S. Food and Drug Administration’s Office of Orphan Products Development has granted it orphan drug designation for the treatment of Amyotrophic Lateral Sclerosis (ALS) with its human spinal cord derived neural stem cells (NSI-566RSC), currently in a Phase I safety study to evaluate the safety of the product and the surgical route of administration in a wide range of ALS patients. [Neuralstem, Inc. Press Release]

Neuralstem Updates ALS Clinical Trial Progress
Neuralstem, Inc. updated the progress of its ongoing Phase I human clinical trial of the company’s spinal cord stem cells in the treatment of ALS (amyotrophic lateral sclerosis, or Lou Gehrig’s disease) at Emory University in Atlanta, Georgia. The company announced that, after reviewing the safety data from the first nine patients, the trial’s Safety Monitoring Board has unanimously approved moving to the last group of ALS patients in this part of the safety trial. [Neuralstem, Inc. Press Release]

Cell Medica Announces First Patient Treated in Cytomegalovirus~ACE/ASPECT Clinical Trial
Cell Medica announced that the first patient has been treated in the CMV~ACE/ASPECT trial at University College Hospital London. [Cell Medica Ltd. Press Release]

Pervasis Receives FDA Fast Track Status for Vascugel® to Prevent Arteriovenous Access Failure in Patients Undergoing Hemodialysis
Pervasis Therapeutics announced that the U.S. Food and Drug Administration (FDA)  has granted Fast Track review status for Vascugel® for the prevention of hemodialysis access failure in patients with end stage renal disease. [Pervasis Therapeutics, Inc. Press Release]

RegeneRx Receives Notice of Australian Patent to be Granted for the Use of T(Beta)4 to Prevent Scarring and Fibrosis
RegeneRx Biopharmaceuticals, Inc. announced that the Company has received notice from the Australian Patent and Trademark Office that it intends to approve its patent application for the use of thymosin beta 4 (T(beta)4), its analogues, isoforms, and other derivatives, for preventing extracellular matrix buildup that may occur from injuries or diseases of the heart, skin, brain, or eye, among others. [RegeneRx Biopharmaceuticals, Inc. Press Release]
UCSF Stem Cell Building Opens, A Milestone for Pioneering Program
The University of California, San Francisco (UCSF) celebrates the official opening of the Ray and Dagmar Dolby Regeneration Medicine Building, a testament to the power of perseverance in the stem cell field. [University of California, San Francisco Press Release]

Geron Appoints David Greenwood President, Interim CEO and Director
Geron Corporation announced a new leadership structure that reflects the company’s progression into mid-stage clinical development of multiple therapeutic product candidates. [Geron Corporation Press Release]

Scientists Warn Against Stifling Effect of Widespread Patenting in Stem Cell Field
A team of scholars urges the scientific community to act collectively to stem the negative effects of patenting and privatizing of stem cell lines, data and pioneering technologies. This means grappling with the ambiguity of several fundamental distinctions typically made in ethics, law, and common practice, the experts insist. [Johns Hopkins Berman Institute of Bioethics Press Release]

Lilly CEO Says Industry, Public Policy Must Pave Way For Innovation
A new approach to research, along with public policies that support medical innovation, will help biopharmaceutical companies address stubborn diseases with potentially devastating personal and financial consequences, such as cancer and Alzheimer’s disease, according to John Lechleiter, Ph.D., chairman, president and CEO of Eli Lilly and Company. [Eli Lilly and Company Press Release]

Many Breakthrough Drugs Come from Publicly Funded Research: Study
A surprising number of valuable new drugs and vaccines approved in the United States have arisen wholly from research funded by the public sector, new research finds. [HealthDay]


NHGRI Charts Course for the Next Phase of Genomics Research
A new strategic plan from an arm of the National Institutes of Health envisions scientists being able to identify genetic bases of most single-gene disorders and gaining new insights into multi-gene disorders in the next decade. This should lead to more accurate diagnoses, new drug targets and the development of practical treatments for many who today lack therapeutic options, according to the plan from the National Human Genome Research Institute (NHGRI).

NIH Budget: Post-Stimulus Cliff Still Looming, But Not Until Next Year
A budget analysis from the Federation of American Societies for Experimental Biology suggests that the post-stimulus “cliff” — the steep drop in funding for the National Institutes of Health (NIH) when its windfall from the 2009 stimulus act runs out — will not hit researchers this year, as expected. Instead, stimulus funds are actually peaking this year.

Senators Oppose Plan to Dismantle NIH Resources Center
Two senators, including the chair of a powerful spending panel, have added their voices to a flood of complaints from researchers about the National Institutes of Health’s (NIH’s) plan to abolish its center for research resources to make room for a new translational sciences center. [Senate, United States]

National Institutes of Health Statement of Organization, Functions, and Delegations of Authority (FR Doc. 2011-2848)

Proposed Collection; Comment Request; Cancer Biomedical Informatics Grid[supreg] (caBIG[supreg]) Support Service Provider (SSP) Program (NCI) (FR Doc. 2011-3144)

National Cancer Institute; Notice of Meeting (FR Doc. 2011-2992)
Center for Scientific Review; Notice of Closed Meetings (FR Doc. 2011-2723)
Center for Scientific Review; Notice of Closed Meetings (FR Doc. 2011-2722)
Center for Scientific Review; Notice of Closed Meetings (FR Doc. 2011-2850)
Center for Scientific Review; Notice of Closed Meetings (FR Doc. 2011-2998)
Center for Scientific Review; Notice of Closed Meetings (FR Doc. 2011-2990)

Center for Scientific Review; Notice of Closed Meetings (FR Doc. 2011-3105)


Fluvirin (Influenza Virus Vaccine) – Untitled Letter
February 2, 2011 Approval Letter – FEIBA

February 2, 2011 Approval Letter – Prevnar 13



FDA Says Drug Firms Didn’t Finish Follow-Ups
Federal officials questioned companies that make six cancer drugs about their failure to complete follow-up studies they promised to conduct in exchange for accelerated approval.

FDA Launches Medical Device Innovation Initiative
The U.S. Food and Drug Administration proposed the Innovation Pathway, a priority review program for new, breakthrough medical devices and announced the first submission: a brain-controlled, upper-extremity prosthetic that will serve as a pilot for the program.

FDA Finalizes Regulation for Certain Software, Hardware Used with Medical Devices
The FDA announced a final rule that provides a less-burdensome path to market for certain hardware and software products used with medical devices. The rule classifies these products, known as Medical Device Data Systems or MDDS, as Class I or low-risk devices, making them exempt from premarket review but still subject to quality standards.


European Medicines Agency and Swissmedic Extend Confidentiality Arrangement
The European Medicines Agency and Swissmedic have extended their confidentiality arrangement for a year. The arrangement allows the two agencies to continue to exchange confidential information relating to the medicines used in the context of the 2009 (H1N1) influenza pandemic.

European Medicines Agency Launches Web Survey to Gather Feedback on Online User Experience
The European Medicines Agency has launched a new online survey to gather user feedback on the Agency’s website.


Proposed Amendments to the Therapeutic Goods Regulations 1990 to Implement the Biologicals Framework (Added Public Submissions)
The Cost Recovery Impact Statement (CRIS) for the Regulation of Biologicals (Added Public Submissions)


NEW Biorepositories Asia
April 11-13, 2011
San Francisco, United States

NEW Physiology 2011: Cellular & Integrative Neuroscience Themed Meeting
July 11-14, 2011
Oxford, United Kingdom

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


Lab Technologist – Tissue Culture (STEMCELL Technologies)

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

Quality Assurance Specialist – Gene Transfer & Somatic Cell Engineering Facility (Memorial Sloan-Kettering Cancer Center)

Postdoctoral Position – Germline Stem Cells (R. L. Brinster, School of Veterinary Medicine, University of Pennsylvania)

Scientific Director (Adipose Derived Stem Cell Bank)

Process Technical Development Scientist (StemCells, Inc.)

Field Applications Specialist – Cell Therapy (Medical) (Pall Corporation)
Postdoctoral Positions – Stem Cell Biology (Joslin Diabetes Center/Harvard Stem Cell Institute)
Cell Therapy Manufacturing Manager (Opexa Therapeutics)

Assistant Professor (University of Pittsburgh School of Medicine, Center for Cellular and Molecular Engineering)
Post-Doctoral Positions (University of Texas MD Anderson Cancer Center)
PhD Students – Stem Cell Differentiation and Cellular Reprogramming (Ulm University Hospital)

Senior Scientist – Embryonic Stem Cells – Hepatocytes (Hays Pharma)

Research Technician Position in Human Stem Cells (University of Pittsburgh)

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