Volume 11.33 August 30, 2010

Targeted Drug Leads to Rapid Regression of Metastatic Melanoma in Patients with Mutated BRAF Gene
Use of an experimental targeted drug to treat metastatic melanoma tumors with a specific genetic signature was successful in more than 80 percent of patients in a Phase I clinical trial. [Press release from Massachusetts General Hospital discussing online prepublication in the New England Journal of Medicine]
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Gene Involved in Fuchs Corneal Dystrophy Is Found
A research team has found a gene likely responsible for Fuchs corneal dystrophy. [Press release from the University of Oregon discussing online prepublication in the New England Journal of Medicine]
Article

Gene Scan Finds Link Across Array of Childhood Brain Disorders
Mutations in a single gene can cause several types of developmental brain abnormalities that experts have traditionally considered different disorders. Researchers found those mutations through whole exome sequencing -- a new gene scanning technology that cuts the cost and time of searching for rare mutations. [Press release from the National Institutes of Health discussing online prepublication in Nature]
Article

Team Finds a Genetic Rarity: A Mutation That Restores Health
Scientists have unraveled the secrets of a rare phenomenon with potential therapeutic implications -- disease-causing genes that show a high frequency of self-repair. [Press release from Yale University discussing online prepublication in Science]
Article

Scientists Unveil Structure of Adenovirus, the Largest High-Resolution Complex Ever Found
Scientists have pieced together the structure of a human adenovirus -- the largest complex ever determined at atomic resolution. The new findings about the virus may lead to more effective gene therapy and to new anti-viral drugs. [Press release from the Scripps Research Institute discussing online prepublication in Science]
Article

A Better Way to Grow Stem Cells
Researchers have devised a synthetic surface that includes no foreign animal material and allows stem cells to stay alive and continue reproducing themselves for at least three months. [Press release from the Massachusetts Institute of Technology discussing online prepublication in Nature Materials]
Article

Liver Cells Created from Patients’ Skin Cells
By creating diseased liver cells from a small sample of human skin, scientists have for the first time shown that stem cells can be used to model a diverse range of inherited disorders. [Press release from the University of Cambridge discussing online prepublication in the Journal of Clinical Investigation]
Article

Gene Therapy to Treat Epilepsy a Step Closer
Researchers introduced genes that increase the expression of certain receptors for neuropeptides in the brain. [Press release from Lund University discussing online prepublication in Brain]
Article

Double-Therapy Approach Effectively Inhibited Brain Cancer Recurrence
Researchers have identified a novel approach of combining chemotherapy with a targeted therapy to decrease the recurrence of glioblastoma multiforme. [Press release from the University of Massachusetts Medical School discussing online prepublication in Cancer Research]
Article

Human Umbilical Cord Blood Cells Aid Lab Animal Brain Cell Survival After Simulated Stroke
Human umbilical cord blood cells used to treat cultured rat brain cells deprived of oxygen appear to protect astrocytes from cell death after stroke-like damage. [Press release from EurekAlert! discussing online prepublication in Stem Cell Reviews and Reports]
Article

Seeing the World with New Eyes: Biosynthetic Corneas Restore Vision in Humans
A new study has shown that biosynthetic corneas can help regenerate and repair damaged eye tissue and improve vision in humans. [Press release from Linköping University discussing online prepublication in Science Translational Medicine]
Article

CURRENT PUBLICATIONS

Inhibition of Mutated, Activated BRAF in Metastatic Melanoma
Treatment of metastatic melanoma with PLX4032 in patients with tumors that carry the V600E BRAF mutation resulted in complete or partial tumor regression in the majority of patients. [N Engl J Med]
Article

E2-2 Protein and Fuchs's Corneal Dystrophy
Genetic variation in transcription factor 4 gene (TCF4) contributes to the development of Fuchs's corneal dystrophy. [N Engl J Med]
Article

Non-Canonical Inhibition of DNA Damage-Dependent Ubiquitination by OTUB1
Researchers report that OTUB1, a deubiquitinating enzyme, is an inhibitor of double-strand break-induced chromatin ubiquitination. [Nature]
Article

Whole Exome Sequencing Identifies WDR62 Mutations in Severe Brain Cortical Malformations
Researchers demonstrate the use of whole-exome sequencing to identify recessive mutations in WD repeat domain 62 (WDR62) as the cause of a wide spectrum of severe cerebral cortical malformations including microcephaly, pachygyria with cortical thickening as well as hypoplasia of the corpus callosum. [Nature]
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Mitotic Recombination in Patients with Ichthyosis Causes Reversion of Dominant Mutations in KRT10
The high frequency of somatic reversion in ichthyosis with confetti suggests that revertant stem cell clones are under strong positive selection and/or that the rate of mitotic recombination is elevated in individuals with this disorder. [Science]
Article

Crystal Structure of Human Adenovirus at 3.5 Angstrom Resolution
Researchers report the x-ray structure at 3.5 angstrom resolution of the 150-megadalton adenovirus capsid containing nearly 1 million amino acids. [Science]
Article

Combinatorial Development of Biomaterials for Clonal Growth of Human Pluripotent Stem Cells
Researchers developed the first chemically defined, xeno-free, feeder-free synthetic substrates to support robust self-renewal of fully dissociated human embryonic stem and induced pluripotent stem cells. [Nat Mater]
Article

Modeling Inherited Metabolic Disorders of the Liver Using Human Induced Pluripotent Stem Cells
Researchers report a simple and effective platform for hepatocyte generation from patient-specific human induced pluripotent stem cells. [J Clin Invest]
Article

A Biosynthetic Alternative to Human Donor Tissue for Inducing Corneal Regeneration: 24-Month Follow-Up of a Phase I Clinical Study
With further optimization, biosynthetic corneal implants could offer a safe and effective alternative to the implantation of human tissue to help address the current donor cornea shortage. [Sci Transl Med]
Article

Dynamic MicroRNA Expression Programs During Cardiac Differentiation of Human Embryonic Stem Cells: Role for miR-499
Data give significant insight into the regulatory networks that govern human embryonic stem cells (hESC) differentiation, and highlights the ability of microRNAs to perturb, and even control, the genes that are involved in cardiac specification of hESCs. [Circ Cardiovasc Genet]
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Federal Judge: Obama Stem Cell Policy Halted
Research on embryonic stem cells funded by the U.S. government must stop immediately for a court-ordered review, Judge Royce Lamberth of the U.S. District Court for the District of Columbia ruled. [United States District Court for the District of Columbia]
Article

Request for Comments on Synthetic Biology (FR Doc. 2010-21359) [Department of Health and Human Services, United States]
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Roche Enters $1.1 Billion Drug Deal With U.S.'s Aileron
Roche Holding AG entered into a partnership deal with U.S.-based biopharmaceutical firm Aileron Therapeutics to produce a novel class of drugs to treat diseases such as inflammation and metabolic ailments, speeding up recent efforts to broaden its R&D pipeline by entering into licensing agreements and acquisitions. [The Wall Street Journal]
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Wake Forest Institute for Regenerative Medicine Signs a Memorandum of Understanding with NUI Galway
The Network of Excellence for Functional Biomaterials and the Regenerative Medicine Institute at National University of Ireland (NUI) Galway have formally signed a Memorandum of Understanding with Wake Forest Institute for Regenerative Medicine. [National University of Ireland, Galway Press Release]
Article

Soligenix Announces Award of $1.2 Million FDA Orphan Products Development Grant
Soligenix, Inc. announced that it has been awarded a three year $1.2 million clinical research grant by the Office of Orphan Products Development of the U.S. Food and Drug Administration (FDA) pursuant to its Orphan Products Development Program. [Soligenix, Inc. Press Release]
Article

Fox Center Receives Donation to Further Research on Stem Cell Therapy for Corneal Scarring
The Louis J. Fox Center for Vision Restoration of University of Pittsburgh Medical Center (UPMC) and the University of Pittsburgh received a $244,000 donation from the Western Pennsylvania Medical Eye Bank Foundation. The donation, to be matched by UPMC, will be used to further research on stem cell therapy for corneal scarring. [University of Pittsburgh Medical Center Press Release]
Article

Neuralstem Files FDA Application for First Chronic Spinal Cord Injury Stem Cell Trial
Neuralstem, Inc. announced that it has filed an Investigational New Drug application with the United States Food and Drug Administration (FDA) to begin a Phase I safety clinical trial for chronic spinal cord injury with its spinal cord stem cells. [Neuralstem, Inc. Press Release]
Article

Shire Announces European Approval of VPRIV® (velaglucerase alfa) for the Treatment of Type 1 Gaucher Disease
Shire plc announced that the European Commission has granted marketing authorization for VPRIV® (velaglucerase alfa), a human cell line derived enzyme replacement therapy for the long-term treatment of type 1 Gaucher disease. [Shire plc Press Release]
Article

Celera Announces Issuance of United States Patent Relating to LPA Gene Variant Associated with Increased Risk for Myocardial Infarction
Celera Corporation announced that the United States Patent and Trademark Office has issued United States Patent 7,781,168 relating to methods of determining heart attack risk by detecting the Ile4399Met genetic polymorphism in the protease-like domain of LPA. [Celera Corporation Press Release]
Article

European Patent Office Allows Advaxis/University of Pennsylvania Modified Listeria Protein Listeriolysin O Patent
Advaxis, Inc. has been notified by the European Patent Office that patent application WO2001/072329, for the use of Prokaryotic PEST-Like Peptides for Enhancing Immunogenicity of Antigen has been allowed for issuance. [Advaxis, Inc. Press Release]
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NIH Director Collins Says Cell Ruling Won't Halt Most Ongoing Grants
National Institutes of Health (NIH) Director Francis Collins said that the court injunction blocking federally funded research on human embryonic stem cells will not affect grant payments that have already gone out the door this year.
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NIH Director's Response to Stem Cell Injunction
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Office of the Director, National Institutes of Health; Notice of Meeting (FR Doc. 2010-20861)
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Center for Scientific Review; Notice of Closed Meetings (FR Doc. 2010-21357)
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National Cancer Institute; Notice of Closed Meeting (FR Doc. 2010-21282)
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National Human Genome Research Institute; Notice of Closed Meeting (FR Doc. 2010-21317)
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National Human Genome Research Institute; Notice of Meetings (FR Doc. 2010-20858)
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National Center for Research Resources; Notice of Closed Meeting (FR Doc. 2010-21280)
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National Cancer Institute's Best Practices for Biospecimen Resources (FR Doc. 2010-20872)
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Revolutionary Genome Sequencing Technologies -- The $1000 Genome (SBIR [R43/R44]) (RFA-HG-10-014)
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Public Workshop on Cell and Gene Therapy Clinical Trials in Pediatric Populations
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Guidance for Industry: Electronic Submission of Lot Distribution Reports for Biological Products
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FOOD AND DRUG ADMINISTRATION (United States)

Roche, Genentech Hit with Refuse-to-File Letter
The FDA has handed Roche and Genentech a refuse-to-file letter for the developers' BLA for trastuzumab-DM1.
Article

Food and Drug Administration Clinical Trial Requirements, Regulations, Compliance, and Good Clinical Practice; Public Workshop [FR Doc. 2010-20834]
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Public Workshop on Medical Devices and Nanotechnology: Manufacturing, Characterization, and Biocompatibility Considerations [FR Doc. 2010-20837]
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