Volume 12.32 | Aug 15

Cell Therapy News 12.32, August 15, 2011
     In this issue: Science | Policy | Business | NIH | CBER | Regulatory | Events
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Genetically Modified “Serial Killer” T Cells Obliterate Tumors in Patients with Chronic Lymphocytic Leukemia, Researchers Report
Researchers have shown sustained remissions of up to a year among a small group of advanced chronic lymphocytic leukemia patients treated with genetically engineered versions of their own T cells. [Press release from Penn Medicine discussing online prepublication in the New England Journal of Medicine and Science Translational Medicine]



Gene-Therapy Enzymes Make Unpredicted Errors
Two papers published show that although zinc-finger nucleases are highly specific, the methods previously used to predict where they might go wrong missed the mark. [Press release from NatureNews discussing online prepublication in Nature Biotechnology and Nature Methods]

Discovery May Eliminate Potentially Lethal Side Effect of Stem Cell Therapy
Researchers have developed a way to remove pluripotent human embryonic stem cells from their progeny before the differentiated cells are used in humans. [Press release from Stanford Medicine discussing online prepublication in Nature Biotechnology]

Newts and Salamanders Can Regrow Their Damaged Hearts, So Why Can’t We?
Stem cell researchers have uncovered for the first time why adult human cardiac myocytes have lost their ability to proliferate, perhaps explaining why the human heart has little regenerative capacity. [Press release from the University of California, Los Angeles discussing online prepublication in the Journal of Cell Biology]

Building New Faces from Stem Cells in the Ear
People in need of surgery to repair or reconstruct damaged cartilage could soon find help in an unlikely place – their ears. Stem cells from human ears have successfully been grown into chunks of cartilage that could replace the synthetic materials currently used in surgery. [Press release from New Scientist discussing online prepublication in the Proceedings of the National Academy of Sciences, United States]

Growing a Heart in the Lab: Researchers Turned Human Embryonic Stem Cells into Heart Cells
Researchers have shown that human embryonic stem cells can be transformed into heart cells using a ‘decellularized’ heart as a scaffold. [Press release from the Institute of Bioengineering and Nanotechnology discussing online prepublication in Biomaterials]

GeT-ting Genes Delivered
Scientists have mimicked the ways viruses infect human cells and deliver their genetic material. The research hopes to apply the approach to gene therapy. [Press release from National Physical Laboratory discussing online prepublication in Chemical Communications]

Genetically-Engineered Spider Silk for Gene Therapy
Genetically engineered spider silk could help overcome a major barrier to the use of gene therapy in everyday medicine, according to a new study that reported development and successful initial laboratory tests of such a material. [Press release from the American Chemical Society discussing online prepublication in Bioconjugate Chemistry]

Scientist Develops Virus That Targets HIV
In what represents an important step toward curing HIV, a scientist has created a virus that hunts down HIV-infected cells. [Press release from the University of Southern California discussing online prepublication in Virus Research]


(Ranked by Impact Factor of the Journal)

Chimeric Antigen Receptor–Modified T Cells in Chronic Lymphoid Leukemia
Scientists designed a lentiviral vector expressing a chimeric antigen receptor with specificity for the B-cell antigen CD19, coupled with CD137 and CD3-zeta signaling domains. [New Engl J Med]

An Unbiased Genome-Wide Analysis of Zinc-Finger Nuclease Specificity
Scientists show that nonhomologous end-joining captures integrase-defective lentiviral vectors at double-strand breaks, tagging these transient events. [Nat Biotechnol]

An Antibody Against SSEA-5 Glycan on Human Pluripotent Stem Cells Enables Removal of Teratoma-Forming Cells
Scientists raised a monoclonal antibody against human embryonic stem cells, designated anti–stage-specific embryonic antigen (SSEA)-5, which binds a previously unidentified antigen highly and specifically expressed on human pluripotent stem cells—the H type-1 glycan. [Nat Biotechnol]

Revealing Off-Target Cleavage Specificities of Zinc-Finger Nucleases by In Vitro Selection
Researchers developed an in vitro selection method that interrogates 1011 DNA sequences for cleavage by active, dimeric zinc-finger nucleases. [Nat Method]

Rb and p130 Control Cell Cycle Gene Silencing to Maintain the Postmitotic Phenotype in Cardiac Myocytes
Investigators hypothesize that genes relevant for cardiac proliferation are targeted to heterochromatin by retinoblastoma (Rb) family members interacting with E2F transcription factors and recruiting heterochromatin protein 1 proteins. [J Cell Biol]

GeT Peptides: A Single-Domain Approach to Gene Delivery
A generic peptide sequence for gene delivery is described. The sequence penetrates eukaryotic cells and promotes active DNA transport into mammalian cells by undergoing differential membrane-induced folding, which renders it both endosomolytic and antibacterial. [Chem Commun (Camb)]

Spider Silk-Based Gene Carriers for Tumor Cell-Specific Delivery
The present study demonstrates pDNA complexes of recombinant silk proteins containing poly(l-lysine) and tumor-homing peptides (THPs), which are globular and approximately 150–250 nm in diameter, show significant enhancement of target specificity to tumor cells by additions of F3 and CGKRK THPs. [Bioconjug Chem]

T Cells with Chimeric Antigen Receptors Have Potent Antitumor Effects and Can Establish Memory in Patients with Advanced Leukemia
Researchers report that chimeric antigen receptors T cells that target CD19 and contain a costimulatory domain from CD137 and the T cell receptor ζ chain have potent non–cross-resistant clinical activity after infusion in three of three patients treated with advanced chronic lymphocytic leukemia. [Sci Transl Med]



Debt Deal Sets Day of Reckoning
Scalpel or guillotine? Those are the possible fates in store for U.S. science funding after Congress and the White House reached a deal to cut federal spending and raise the nation’s self-imposed debt limit. [U.S. Congress, United States]

Embryonic Stem-Cell Approvals Rise
Research using human embryonic stem-cell lines approved under President Barack Obama is accelerating, boosting a scientific field that’s been dogged by legal and political threats. [Obama Administration, United States]

Mass Exodus Roils Brazilian Neuroscience Institute
A rebellion in the scientific ranks has created some recent turmoil at Brazil’s most famous brain research center, the Edmond and Lily Safra International Institute of Neuroscience of Natal. [Edmond and Lily Safra International Institute of Neuroscience of Natal, Brazil]

U.K. Needs to Stop ‘Muddling’ on Gene Patents, Says Report
The Human Genetics Commission issued a plea to health and research institutions to develop a coherent policy on intellectual property, particularly patents on diagnostic tools. [The Human Genetics Commission, United Kingdom]

U.K. Slashes Science and Engineering Ph.D.s
The United Kingdom’s Engineering and Physical Science Research Council has revealed that it may fund 1000 fewer new Ph.D.s in the upcoming academic year than in 2010-11. [Engineering and Physical Science Research Council, United Kingdom]


MolMed Signs an Agreement with GlaxoSmithKline to Develop a Production Process for a Gene Therapy
MolMed S.p.A. announced that it has signed an agreement with GlaxoSmithKline under which MolMed will develop a production process for an investigational gene therapy for Adenosine Deaminase Deficiency – Severe Combined Immune Deficiency. [MolMed S.p.A. Press Release]

Novel Method for Treating Ovarian Cancer
The Fraunhofer Institute for Cell Therapy and Immunology IZI supports Prima BioMed Ltd. in tests and approvals regarding a novel therapeutic method for treating ovarian cancer. [Fraunhofer Institute for Cell Therapy and Immunology IZI Press Release]

Genesis Biopharma Signs Cooperative Research and Development Agreement with the National Cancer Institute to Develop Cancer Immunotherapies
Genesis Biopharma, Inc. announced it has signed a Cooperative Research and Development Agreement with the National Cancer Institute. [PR Newswire]

CI Awarded $1.86 Million Stem Cell Research Training Grant
California State University Channel Islands (CI) recently won a $1.86 million grant from the California Institute for Regenerative Medicine to help prepare graduate students for careers in stem cell research. [California State University Channel Islands Press Release]

YCR Invests £2.15 Million in York Stem Cell Research
Yorkshire Cancer Research (YCR) has awarded £2.15 million to the University of York to further its internationally award-winning progress into prostate cancer stem cell research. [The University of York Press Release]

Cellular Dynamics Launches Innovative Research Grant Program
Cellular Dynamics International announced the recent launch of its Innovative Research Grant Program. [Cellular Dynamics International Press Release]

iCo Therapeutics Announces Key iCo-007 Clinical Development Milestone
iCo Therapeutics Inc. announced the initiation of a US physician-sponsored Phase II clinical trial involving iCo-007, titled the iDEAL study, which  will be conducted across multiple sites throughout the United States. [iCo Therapeutics Inc. Press Release]

apceth Is Granted a Manufacturer’s License for Somatic Cell Therapy
apceth is among the first companies in Germany to be granted a manufacturer’s license for the production of somatic cell therapeutics pursuant to Section 13, German Medicines Act. [B3C Newswire]

Kyoto University Granted Its First iPS Cell Patent by the United States Patent and Trademark Office
Kyoto University announced that it was granted its first patent associated with induced pluripotent stem cell (iPSC) technology, by the United States Patent and Trademark Office. [Kyoto University Center for iPS Cell Research and Application Press Release]

Silence Therapeutics Receives Notice of Allowance for New U.S. Patent Covering Novel Small Interfering RNA Delivery Technology
Silence Therapeutics plc announced that the United States Patent and Trademark Office has issued the Company a notice of allowance on a patent application directed to the fundamental technology involved in AtuPLEX™, one of Silence’s proprietary small interfering RNA delivery systems. [Silence Therapeutics plc Press Release]

La Jolla Institute Opens Major RNAi Center for Identifying Genetic Triggers of Disease
A major Center that will propel scientific efforts to pinpoint the specific genes involved in causing immune diseases, cancer and other diseases was opened at the La Jolla Institute for Allergy & Immunology. [La Jolla Institute for Allergy & Immunology Press Release]

GHSU Launches New Institute for Regenerative and Reparative Medicine
A new Institute for Regenerative and Reparative Medicine is working to help the body repair itself as it grows synergy between Georgia Health Sciences University (GHSU) and the community. [Georgia Health Sciences University Press Release]

Former NIH Director Bernadine Healy Dies
Bernadine Healy, a cardiologist who was the first woman to direct the National Institutes of Health (NIH) from 1991 to 1993, died from brain cancer.

Notice of Correction for PA-11-148 Nanoscience and Nanotechnology in Biology and Medicine (R01) (NOT-EB-11-007)

National Cancer Institute; Notice of Meeting (FR Doc No: 2011-20325)

National Institute of General Medical Sciences Amended Notice of Meeting (FR Doc No: 2011-20323)

September 22-23, 2011: Cellular, Tissue and Gene Therapies Advisory Committee Meeting Announcement



Agency Information Collection Activities; Proposed Collection; Comment Request; Blood Establishment Registration and Product Listing, Form FDA 2830 [FR Doc No: 2011-19955]

Agency Information Collection Activities; Submission for Office of Management and Budget Review; Comment Request; Cooperative Manufacturing Arrangements for Licensed Biologics [FR Doc No: 2011-19958]

Cellular, Tissue and Gene Therapies Advisory Committee; Notice of Meeting [FR Doc No: 2011-20399]


TGA Advisory Committee Guidelines
The purpose of these guidelines is to provide guidance for members, chairs and secretaries of the TGA’s statutory expert advisory committees (and their sub-committees)


NEW 33rd Annual International Conference of the IEEE Engineering in Medicine and Biology Society
August 30 – September 3, 2011
Boston, United States

NEW 15th Congress of the European Society for Organ Transplantation
September 4-7, 2011
Glasgow, United Kingdom

California Institute for Regenerative Medicine/Regenerative Medicine Consortium: Scaffolding Webinar
September 12, 2011

NEW International Society for Cellular Therapy: BMCOGS & Reimbursement Roundtable
September 14, 2011
Minneapolis, United States

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


Lab Technologist – Tissue Culture (STEMCELL Technologies)

Research and Development Technologist, hPSC Media (STEMCELL Technologies)

Scientist (iPSC) (STEMCELL Technologies)

Quality Officer (Senior Hospital Scientist) (Royal Prince Alfred Hospital)

Project Manager/Senior Hospital Scientist (Royal Prince Alfred Hospital)

Postdoctoral Position (Michigan State University)

Postdoctoral Scientists (North Carolina Central University)

Postdoctoral Position (Nanyang Technological University)

Medical Officer (California Institute for Regenerative Medicine)

Postdoctoral Position (University of California, Davis)

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