Volume 12.13 | Apr 4

Cell Therapy News 12.13, April 4, 2011.
     In this issue: Science | Policy | Business | NIH | CBER | Regulatory | Events

Clinical Trial Success for Crohn’s Disease Cell Therapy
Researchers have used patient’s own immune system cells derived from peripheral blood mononuclear cells to treat patients with chronic inflammatory diseases like Crohn’s disease. [Press release from EurekAlert! discussing research presented at the UK National Stem Cell Network 2011 Annual Science Conference]



Newly Discovered Epigenetic Tag Offers Insight into Embryonic Stem Cell Regulation
Scientists have gained a new understanding of how molecular signals and switches control how an embryo develops into an adult. The new research details how a newly discovered form of epigenetic regulation controls the development of embryonic stem cells. [Press release from the Babraham Institute discussing online prepublication in Nature]
Human Embryonic Stem Cells Provide New Insight into Muscular Dystrophy
New research uses human embryonic stem cells to make a clinically-important contribution to the understanding of myotonic dystrophy type 1, and highlights the incredible potential that embryonic stem cells hold for unraveling the complex molecular mechanisms involved in a variety of human conditions. [Press release from EurekAlert! discussing online prepublication in Cell Stem Cell]

Scientists Devise Targeted Therapy Strategy for Rare Form of Childhood Cancer
By “distracting” cancer proteins from their usual activity, scientists at Dana-Farber Cancer Institute and Brigham and Women’s Hospital have caused cells in a rare, lethal form of cancer to begin behaving like normal cells — one of the longest-standing, and most rarely achieved, goals of cancer research. [Press release from EurekAlert! discussing online prepublication in Cancer Research]

New “Nanodrug” Breaks Down Barriers to Attack Breast Cancer Cells from the Inside Out
Throwing stones at castle walls is one way to attack an enemy, but sneaking inside makes the target much more vulnerable. Researchers have employed a similar strategy using a mouse model to target important mechanisms inside the most challenging breast cancer cells. [Press release from Cedars-Sinai Medical Center discussing online prepublication in Cancer Research]

Intelligent Design: Engineered Protein Fragment Blocks the AIDS Virus from Entering Cells
In what could be a potential breakthrough in the battle against AIDS and a major development in the rational design of new drugs, scientists have engineered a new protein that prevents the virus from entering cells. [Press release from EurekAlert! discussing online prepublication in the FASEB Journal]

New Research Holds Promise for Gene Engineering and Therapy
Scientists have discovered a biochemical version of a principle well known among confectioners. Call it the “peanut butter and chocolate” rule: sometimes two things work better together than alone. The researchers have developed a remarkably effective way to combine two methods that scientists use as vehicles to carry DNA into cell nuclei. [Press release from the Polytechnic Institute of New York University discussing online prepublication in the Journal of Controlled Release]

Pathfinder Cells Demonstrate Ability to Regenerate Damaged Tissue and Restore Pancreatic Function in a Diabetic Mouse Model
Pathfinder, LLC announced publication of promising preclinical data demonstrating that the Company’s unique cell-based therapy is able to completely reverse diabetes in a mouse model. [Press release from PR Newswire discussing online prepublication in Rejuvenation Research]

Researcher Creates Personalized Therapy That Causes Cancer Cells to Kill Themselves
A physician-researcher has developed a personalized therapy to treat a wide range of cancers. The treatment is based on a naturally occurring human enzyme that has been genetically modified to fool cancer cells into killing themselves. [Press release from Wayne State University School of Medicine discussing online prepublication in Cancer Gene Therapy]

Viral Replicase Points to Potential Cancer Therapy
New research demonstrates that an artificial plasmid coding for the replicase genes of Sindbis virus causes regression and destruction of lung cancer, or melanoma, cells in mice. [Press release from EurekAlert! discussing online prepublication in BMC Cancer]

New Nanoscale Tool May One Day Build Molecular Structures That Treat Disease
A research team has developed a tool for rapidly and efficiently building molecular structures that could one day be used to treat and diagnose diseases. [Press release from the Wyss Institute for Biologically Inspired Engineering at Harvard University discussing online prepublication in Nature Communications]

3D Lab Experiments Aid Adult Stem Cell Versatility
A researcher’s experiments hope to recreate the microscopic 3D environment that stem cells would normally occupy inside our bodies and so give an accurate approximation of the factors that might influence the ability of mesenchymal stem cells to eventually produce different types of cell for regenerative medicine. [Press release from the University of York discussing research presented at UK National Stem Cell Network 2011 Annual Science Conference]

Why Stem Cells Don’t Just Want to Make Neurons
Research provides another piece in the puzzle of why it can be so hard to produce large numbers of the same type of cell in the lab – a process that is vital for scaling up stem cell production for therapeutic use. [Press release from the Biotechnology and Biological Sciences Research Council discussing research presented at UK National Stem Cell Network 2011 Annual Science Conference]

Attacking Bowel Cancer on Two Fronts
Researchers identified that the stem cells which are crucial to maintaining a healthy intestine are also the site at which bowel cancers first begin, and they hope to use healthy stem cells to regenerate tissues to help patients with Crohn’s disease and some cancers. [Press release from the UK National Stem Cell Network discussing research presented at UK National Stem Cell Network 2011 Annual Science Conference]

New Inhibitor Prevented Lesions, Reduced Tumor Size in Basal Cell Cancer
A new hedgehog pathway inhibitor demonstrated efficacy in preventing and treating basal cell cancer among patients with basal cell nevus syndrome, a rare inheritable disease, according to Phase II data. [Press release from the American Association for Cancer Research (AACR) discussing research presented at the AACR 102nd Annual Meeting 2011]

Immune System May Guide Chemotherapy for Breast Cancer
A study showed how evaluating the immune response in the tumor microenvironment may help researchers better target therapy in breast cancer. [Press release from the American Association for Cancer Research (AACR) discussing research presented at the AACR 102nd Annual Meeting 2011]

Researchers Couple Tumor Cryoablation with Immunotherapy to Improve Tumor Immunity
Scientists at the Barbara Ann Karmanos Cancer Institute in Detroit presented data showing that when tumor cryoablation is coupled with immune system modulation, the procedures have the potential to initiate or amplify tumor immunity. [Press release from PR Newswire discussing research presented at the American Association for Cancer Research 102nd Annual Meeting 2011]

Adding Stem Cells to Common Bypass Surgery May Improve Heart Failure
In a new research study, surgeons are adding a patient’s own stem cells to the heart during cardiac bypass surgery. The goal of this research study is to determine whether the stem cell infusion will generate new blood vessels and improve heart function more than what is seen through bypass surgery alone. [Methodist DeBakey Heart & Vascular Center Press Release]

(Ranked by Impact Factor of the Journal)

Dynamic Regulation of 5-Hydroxymethylcytosine in Mouse Embryonic Stem Cells and During Differentiation
Researchers propose that the balance between hydroxymethylation and methylation in the genome is inextricably linked with the balance between pluripotency and lineage commitment. [Nature]

Mutant Human Embryonic Stem Cells Reveal Neurite and Synapse Formation Defects in Type 1 Myotonic Dystrophy
By using neuronal progeny derived from human embryonic stem cells carrying the causal myotonic dystrophy type 1 mutation, researchers have identified an early developmental defect in genes involved in neurite formation and the establishment of neuromuscular connections. [Cell Stem Cell]

Genome-Wide Analysis of 5-Hydroxymethylcytosine Distribution Reveals Its Dual Function in Transcriptional Regulation in Mouse Embryonic Stem Cells
Researchers present a genome-wide profile of 5-hydroxymethylcytosine in mouse embryonic stem cells. [Genes Dev]

Differentiation of NUT Midline Carcinoma by Epigenomic Reprogramming
The potential therapeutic utility of histone deacetylase inhibitor differentiation therapy was established in three different NUT midline carcinoma xenograft models, where it produced significant growth inhibition and a survival benefit. [Cancer Res]

Polymalic Acid–Based Nanobiopolymer Provides Efficient Systemic Breast Cancer Treatment by Inhibiting both HER2/neu Receptor Synthesis and Activity
Results of the study showed that the lead drug tested significantly inhibited the growth of HER2/neu-positive breast cancer cells in vitro and in vivo by enhanced apoptosis and inhibition of HER2/neu receptor signaling with suppression of Akt phosphorylation. [Cancer Res]

Multidrug Efflux Pumps and Cancer Stem Cells: Insights Into Multidrug Resistance and Therapeutic Development
It is imperative to gain a better understanding of the mechanisms involved in the resistance of stem cells to chemotherapy, which could lead to the discovery of new therapeutic targets and improvement of current anticancer strategies. [Clin Pharmacol Ther]

Molecular Engineering of RANTES Peptide Mimetics with Potent Anti-HIV-1 Activity
Regulated on activation normal T-cell-expressed and secreted peptide mimetics provide new leads for the development of safe and effective HIV-1 entry inhibitors. [FASEB J]

Translating the Lessons from Gene Therapy to the Development of Regenerative Medicine
While there is significant excitement about the study and the therapeutic potential of human embryonic stem cells, the ethical issues surrounding the destruction of embryos that is necessary to generate such cells has slowed scientific investigation into their use. [Mol Ther]



Minnesota Stem Cell Research Ban Could Mean Criminalizing Patients
A bill in the Minnesota Legislature that would ban some forms of stem cell research could have unintended consequences for patients and researchers if it becomes law. [Minnesota Legislature, United States]

House Panel Takes Next Step Toward New Patent Rules
A sweeping overhaul of the U.S. patent system appears to be gaining momentum in Congress. Representative Lamar Smith (R-TX), chair of the House Judiciary Committee, unveiled his panel’s reform plan (H.R. 1249), called the America Invents Act. [Congress, United States]

Medicare Moves to Pay for Prostate Cancer Drug Provenge
The federal health insurance program for the elderly moved to pay for an expensive vaccine recently approved to treat men with advanced prostate cancer. [Centers for Medicare and Medicaid Services, United States]



Regulus Therapeutics Receives Exclusive License for Intellectual Property to microRNA-33a and microRNA-33b to Treat Diseases that Include Atherosclerosis and Metabolic Syndrome
Regulus Therapeutics Inc. announced that it has obtained exclusive rights from New York University for intellectual property covering methods of use in modulating microRNA-33a (miR-33a) and microRNA-33b (miR-33b) for metabolic diseases such as atherosclerosis and metabolic syndrome. [Regulus Therapeutics Inc. Press Release]

Yale and Gilead Sciences Announce Cancer Research Collaboration
Gilead Sciences, Inc. and Yale School of Medicine announced the formation of a multi-year research collaboration focused on the discovery of novel cancer therapies. [Gilead Sciences, Inc. Press Release]

RXi Pharmaceuticals Aggressively Moves into Late-Stage Clinical Development with the Agreement to Acquire Apthera, Inc.
RXi Pharmaceuticals Corporation and Apthera, Inc. announced the signing of a definitive merger agreement under which RXi will acquire Apthera. [RXi Pharmaceuticals Corporation Press Release]

Sanofi-aventis Successfully Completes Exchange Offer for Genzyme Corporation
Sanofi-aventis announced that it has successfully completed its exchange offer for all outstanding shares of common stock of Genzyme Corporation. [Sanofi-aventis Press Release]

Valeant Pharmaceuticals Proposes to Acquire Cephalon, Inc. for $73 Per Share in Cash
Valeant Pharmaceuticals International, Inc. announced that it had made a proposal to the Board of Directors of Cephalon, Inc., to acquire Cephalon for $73.00 per share in cash. [Valeant Pharmaceuticals International, Inc. Press Release]

Alnylam Achieves Key Technology Transfer Objective in Its Takeda Alliance; Earns $10 Million Payment
Alnylam Pharmaceuticals, Inc. announced that it has earned a $10 million technology transfer payment from Takeda Pharmaceutical Company Limited as part of the strategic alliance the companies formed in May 2008. [Alnylam Pharmaceuticals, Inc. Press Release]

Stem Cell Voice Box Transplant Trials Could Begin in 2013
Professor Martin Birchall announced that the first clinical trials for stem cell voice transplants will begin in 2013, thanks to a million pounds provided by the Medical Research Council. [Medical Research Council Press Release]

CPRIT Awards $36.7 Million to UT Southwestern Investigators for Cancer Studies
The Cancer Prevention and Research Institute of Texas (CPRIT) has awarded more than $36.7 million in new grants to investigators at UT Southwestern Medical Center to support cancer-related projects and to recruit pre-eminent cancer investigators. [University of Texas Southwestern Medical Center Press Release]

Duncan Cancer Center Researchers Awarded $15.9 Million in New Round of CPRIT Grants
Researchers in the National Cancer Institute-designated Dan L. Duncan Cancer Center at Baylor College of Medicine have received an additional $15.9 million in the new round of grants announced by the Cancer Prevention Research Institute of Texas (CPRIT). [Baylor College of Medicine Press Release]

MRC Cash Boost to Maximize Discovery of Mouse Models of Human Diseases
The Medical Research Council (MRC) has announced that it is to invest in excess of £60m over the next five years into mouse genetics research at MRC Harwell, Oxfordshire. [Medical Research Council Press Release]

University of Michigan Creates State’s First Disease-Specific Human Embryonic Stem Cell Lines
University of Michigan researchers have created the state’s first human embryonic stem cell lines that carry the genes responsible for inherited disease. [University of Michigan Press Release]

TxCell Receives Approval for a Phase I/II Clinical Trial in Rheumatoid Arthritis
TxCell SA announced the approval by AFSSAPS, the French regulatory agency, of its application to carry out a Phase I/II clinical trial of its second product candidate, TX-RAD.
[TxCell SA Press Release]

University of Pennsylvania Pioneers HIV Gene-Therapy Trial
University of Pennsylvania researchers are still recruiting HIV-positive participants for a trial of a gene therapy approach intended to block HIV from entering human immune cells. [National Prevention Information Network Press Release]

CaridianBCT Revolutionizes Cell Therapy Industry with New Cell Expansion Product
CaridianBCT, a leading global provider of technology, products and services in the blood banking, transfusion medicine and cell therapy industries, announced the launch of a new product, the Quantum Cell Expansion System. [CaridianBCT, Inc. Press Release]

Phase III Study of VX-770 in Children Ages 6 to 11 with a Specific Type of Cystic Fibrosis Showed Profound Improvements in Lung Function (FEV1) and Other Measures of Disease Through 24 Weeks
Vertex Pharmaceuticals Incorporated announced positive results from a 24-week analysis of the ongoing Phase III ENVISION study of VX-770, an oral medicine in development that targets the defective protein that causes cystic fibrosis. ENVISION was designed to evaluate VX-770 among children ages 6 to 11 with the G551D mutation in the CFTR gene. [Vertex Pharmaceuticals Incorporated Press Release]

Glenmark’s Switzerland’s R&D Center Discovers a Monoclonol Antibody Targeting Leukemia and Lymphomas of B-Cell Origin
Glenmark Pharmaceuticals S.A. announced the discovery and initiation of IND enabling studies of a Novel Biological Entity lead candidate, GBR 401, an anti-CD19 monoclonal antibody. [Glenmark Pharmaceuticals S.A. Press Release]

Biogen Idec and Knopp Biosciences Announce Enrollment of the First Patient in a Global Phase III Study of Dexpramipexole for ALS
Biogen Idec and Knopp Biosciences announced enrollment of the first patient in EMPOWER, a multi-national Phase III study evaluating the efficacy, safety and pharmacokinetics of dexpramipexole in patients with amyotrophic lateral sclerosis (ALS). [Biogen Idec Press Release]

Aastrom Announces Key Manufacturing Milestone and New Generic Name for Its Cell Therapy Product
Aastrom Biosciences, Inc. announced that it has completed an important product manufacturing milestone with the transfer of cell cassette manufacturing to ATEK Medical under the strategic partnership announced in October 2010. [Aastrom Biosciences, Inc. Press Release]

Mount Sinai Hospital Opens New Centre for Regenerative Medicine
Mount Sinai Hospital’s internationally respected Samuel Lunenfeld Research Institute announced the opening of a new Centre for Regenerative Medicine and Musculoskeletal Research at 25 Orde Street. [Samuel Lunenfeld Research Institute Press Release]

Pediatric Nanomedicine Center Links Health Care and Engineering
Physicians and engineers within a new center devoted to pediatric nanomedicine will develop targeted, molecular-sized nanoparticles as part of a unique approach to treating pediatric diseases. [Emory University Woodruff Health Sciences Center Press Release]

Dendreon Statement on CMS Proposed Decision Memo for PROVENGE®
Dendreon appreciates the work and diligent scientific review conducted by Centers for Medicare and Medicaid Services (CMS) that resulted in the draft decision memo, in which CMS proposes that the evidence is adequate to conclude that the use of autologous cellular immunotherapy with PROVENGE® improves health outcomes for Medicare beneficiaries with asymptomatic or minimally symptomatic metastatic castrate resistant (hormone refractory) prostate cancer and thus is reasonable and necessary for that indication. [Dendreon Corporation Press Release]

Generex Unveils Strategic Development Plan
Generex Biotechnology Corporation held a press conference at the NASDAQ Marketsite in New York City to unveil management’s strategic development plan for the Company’s future growth. [Generex Biotechnology Corporation Press Release]

Cancer Discovery: Call for Papers
In response to a call from the scientific community, the American Association for Cancer Research is proud to launch Cancer Discovery, a highly visible and unique forum that will bring together the entire span of cancer science. [American Association for Cancer Research Press Release]


Time to Rethink the NIH
A radical restructure is the only way to solve the systemic problems of the world’s biggest funder of biomedical research, argues Michael M. Crow.

NIH Firm on Grant Application Rules
When does no mean no? The answer for researchers applying for funding from the US National Institutes of Health (NIH) is after the second ‘no’. Officials at the biomedical agency stood by that policy despite a 20 February petition from 2,335 scientists urging them to restore a system that allowed three tries per project.

Report to Nation Finds Continued Declines in Many Cancer Rates
Rates of death in the United States from all cancers for men and women continued to decline between 2003 and 2007, the most recent reporting period available, according to the latest Annual Report to the Nation on the Status of Cancer. [National Institutes of Health, United States]

Laboratory Animal Welfare: Proposed Adoption and Implementation of the Eighth Edition of the Guide for the Care and Use of Laboratory Animals (FR Doc. 2011-7316)

National Human Genome Research Institute; Notice of Closed Meeting (FR Doc. 2011-7625)

National Cancer Institute; Notice of Closed Meeting (FR Doc. 2011-7637)

National Cancer Institute; Notice of Closed Meetings (FR Doc. 2011-7634)

Center for Scientific Review; Notice of Closed Meetings (FR Doc. 2011-7641)

Center for Scientific Review; Notice of Closed Meetings (FR Doc. 2011-7617)

Center for Scientific Review; Notice of Closed Meeting (FR Doc. 2011-7818)

Center for Scientific Review; Notice of Closed Meetings (FR Doc. 2011-7923)

April 6-7, 2011: Vaccines and Related Biological Products Advisory Committee Briefing Documents

February 25, 2011: Vaccines and Related Biological Products Advisory Committee Meeting Transcript

Guidance for Industry: Class II Special Controls Guidance Document: In Vitro HIV Drug Resistance Genotype Assay

Guidance for Industry: Class II Special Controls Guidance Document: Cord Blood Processing System and Storage Container

Guidance for Industry and FDA Staff: Class II Special Controls Guidance Document: Automated Blood Cell Separator Device Operating by Centrifugal or Filtration Separation Principle

March 24, 2011 Approval Letter – Zostavax



Postmarketing Studies and Clinical Trials — Implementation of Section 505(O)(3) of the Federal Food, Drug, and Cosmetic Act

Molecular and Clinical Genetics Panel of the Medical Devices Advisory Committee; Notice of Meeting; Reopening of the Comment Period [Docket No. FDA-2011-N-0066]

Food and Drug Administration Clinical Trial Requirements, Regulations, Compliance, and Good Clinical Practice; Public Workshop [Docket No. FDA-2011-N-0002]


Increased Fees Coming into Effect on 1 April 2011
Fees payable to the European Medicines Agency by applicants and marketing-authorization holders are increasing by 2.1% on Friday 1 April.

Deadlines for Submission of Applications for Orphan Medicinal Product Designation to the EMA 2011/2012


Information Sent to Healthcare Professionals in March About the Safety of Medicines


NEW 11th Workshop of the Korean Society of Gene and Cell Therapy (KSGCT) – Gene Delivery for Stem Cell Science
April 28, 2011
Seoul, Korea

NEW Discussion Workshop: Stem Cells
May 6, 2011
London, United Kingdom

NEW American Thoracic Society Annual International Symposium on Pulmonary Care (ATS 2011)
May 13-18, 2011
Denver, United States

NEW 6th International Meeting on Gene Therapy of Arthritis and Related Disorders
May 22-24, 2011
London, United Kingdom

NEW 10th Annual Meeting of the French Society of Cell and Gene Therapy (SFTCG)
June 6-8, 2011
Nantes, France

NEW 2nd Annual Therapeutic Protein Production Meeting
June 10, 2011
London, United Kingdom

NEW Annual Meeting of the Spanish Society of Gene and Cell Therapy (SETGyC)
September 21-23, 2011
Zaragoza, Spain

NEW Induced Pluripotent Stem Cells: Production and Utility in Regenerative Medicine
October 6, 2011
London, United Kingdom

NEW EORTC-NCI-ASCO Annual Meeting on “Molecular Markers in Cancer” (ENASCO)
October 27-29, 2011
Brussels, Belgium

NEW 4th Northwest Genome Engineering Consortium (NGEC) Annual Workshop on Genome Engineering
November 8, 2011
Seattle, United States

NEW 10th Annual Gene Therapy Symposium for Heart, Lung, and Blood Diseases
November 16-18, 2011
Sonoma, United States

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)

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

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 Position (Johns Hopkins University School of Medicine)

Manager Clinical Affairs (Bioheart, Inc.)

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)

Special Assistant to the Director (Center for Biologics Evaluation and Research)

Postdoctoral Fellow in Gene Expression/Cancer and Stem Cell Biology (University of Cincinnati College of Medicine)

Postdoctoral Positions in Immune Cell Activation (Institut de recherches cliniques de Montréal)

Postdoctoral Position in Developmental Neurobiology (Institut de recherches cliniques de Montréal)

Postdoctoral Research Fellows – Transcriptional Control in Hematopoietic Stem Cells and Leukemia (Institut de recherches cliniques de Montréal)

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