Volume 12.22 | Jun 6

Cell Therapy News 12.22, June 6, 2011
     In this issue: Science | Policy | Business | NIH | CBER | Regulatory | Events
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Gene Therapy Flunks Limb Ischemia Test
A large Phase III clinical trial failed to confirm the beneficial effects of a promising gene therapy for critical limb ischemia that had been seen in early studies, investigators reported. [Press release from Medpage Today discussing online prepublication in The Lancet]


Vaccine and IL-2 Improve Response, Survival in Patients with Advanced Melanoma
A vaccine for advanced melanoma, supplemented by the immunotherapy drug interleukin-2, has shown promise in one of the most comprehensive studies of the skin cancer vaccine’s effectiveness to date. [Press release from the University of South Florida discussing online prepublication in the New England Journal of Medicine]

Neuroscientists Map a New Target to Wipe Pain Away
Researchers have discovered a peptide that short circuits a pathway for chronic pain. [Press release from Indiana University School of Medicine discussing online prepublication in Nature]

Stem Cell Treatment to Prevent Leukemia Returning Is a Step Closer
Researchers have identified a way of eliminating leukemic stem cells, which could in the future lead to new treatments that may enable complete remission for leukemia patients. [Press release from King’s College London discussing online prepublication in Cell Stem Cell]

Gene Therapy Flunks Limb Ischemia Test
A large Phase III clinical trial failed to confirm the beneficial effects of a promising gene therapy for critical limb ischemia that had been seen in early studies, investigators reported. [Press release from Medpage Today discussing online prepublication in The Lancet]

Vaccine Increases Disease-Free Survival for Follicular Lymphoma Patients
A lymphoma vaccine uniquely tailored for each patient extends disease-free survival by 14 months, with signs of an even better response for patients with a specific biological marker, a team reported. [Press release from The University of Texas MD Anderson Cancer Center discussing online prepublication in the Journal of Clinical Oncology]

New Drugs Target Delay of Huntington’s Symptoms
Chemists have synthesized a new form of DNA, one that can begin to be used to create new gene regulation therapies, for the prevention or treatment of diseases such as cancer. [Press release from McMaster University discussing online prepublication in Nature Chemical Biology]

Scientists Reactivate Immune Cells Exhausted by Chronic HIV
Scientists have demonstrated why certain immune cells chronically exposed to HIV shut down, and how they can be reactivated. [Press release from the National Institute of Allergy and Infectious Diseases discussing online prepublication in the Journal of Clinical Investigation]

A New Form of DNA
Chemists have synthesized a new form of DNA for the prevention or treatment of diseases such as cancer. [Press release from Northwestern University discussing online prepublication in the Journal of the American Chemical Society]

Minivectors Renew Gene Therapy Hopes for Lung Treatments
Minivector™ DNA survive the stress of aerosolization and can carry gene therapy deep into the lungs, said researchers. [Press release from Baylor College of Medicine discussing online prepublication in Gene Therapy]

Stem Cells From Fat Used To Repair Skull Defects
Stem cells derived from abdominal fat are a potentially valuable new approach to repairing skull defects after brain surgery, according to a study. [Press release from Medical News Today discussing online prepublication in Neurosurgery]

Potential New Drug Candidate Found for Alzheimer’s Disease
Researchers have demonstrated that oral administration of a cysteine protease inhibitor, E64d, not only reduces the build-up of β-amyloid in the brains of animal models for Alzheimer’s disease, but also results in a substantial improvement in memory deficit. [Press release from the University of California, San Diego discussing online prepublication in the Journal of Alzheimer’s Disease]

Stem Cells: Gene Repair Tool for Stem Cells
Researchers engineered an adenovirus that efficiently fixes mutated portions of the lamin A (LMNA) gene in stem cells created by reprogramming patients’ cells. [Nature Press Release]

Argos Therapeutics´ Arcelis™ Immunotherapy for the Treatment of Renal Cell Carcinoma (RCC) in Combination with Sunitinib Demonstrates Strong Immunologic Response that Correlates to Improved Clinical Outcomes
Argos Therapeutics announced that its Arcelis™ immunotherapy for the treatment of RCC, AGS-003, in combination with sunitinib demonstrated a strong immunologic response that correlated to improved clinical outcomes. [Argos Therapeutics discussing research presented at the 2011 Annual Meeting of the American Society of Clinical Oncology]

Study Finds Vaccine Extends Recurrent GBM Survival Rates by Two to Three Times
Cancer researchers found that the brain tumor vaccine HSPPC-96 for treating recurrent gliobastoma (GBM) has a favorable safety profile and extends survival by two to three times more than the current median survival rate. [Press release from University Hospitals discussing research presented at the 2011 Annual Meeting of the American Society of Clinical Oncology]

Combination Antibody Therapy Shows Promise in Metastatic Melanoma
A duo of drugs can be safely administered and are potentially more effective than either drug alone for advanced, inoperable melanomas, according to a Phase I clinical trial. [Press release from ScienceDaily discussing research presented at the 2011 Annual Meeting of the American Society of Clinical Oncology]

Agennix Announces New Data from Talactoferrin Phase II Trials in Non-Small Cell Lung Cancer and in Severe Sepsis
Agennix AG announced that new data from Phase II trials in non-small cell lung cancer and in severe sepsis demonstrating the activity and tolerability of talactoferrin, an oral immunotherapy with antibacterial properties, were presented. [Press release from Agennix AG discussing research presented at the 2011 Annual Meeting of the American Society of Clinical Oncology]

Vical Presents Positive Correlation Between Response and Survival at ASCO for Completed Allovectin® Melanoma Trials
Vical Incorporated announced results from new statistical analyses of data from three previously completed clinical trials of the company’s Allovectin® immunotherapy in patients with metastatic melanoma, showing with strong positive correlation that responders lived significantly longer than nonresponders. [Press release from Vical Incorporated discussing research presented at the 2011 American Society of Clinical Oncology]

Stem Cell Treatment May Offer Option for Broken Bones that Don’t Heal
Researchers have shown in an animal study that transplantation of adult stem cells enriched with a bone-regenerating hormone can help mend bone fractures that are not healing properly. [Press release from the University of North Carolina at Chapel Hill School of Medicine discussing research presented at The Endocrine Society’s 93rd Annual Meeting]

Aastrom Demonstrates Statistically Significant Improvement in Time to First Occurrence of Treatment Failure at 12 Months in RESTORE-CLI Clinical Trial
Aastrom Biosciences, Inc. announced that the final analysis of data from all 86 randomized patients in the RESTORE-CLI trial showed that the trial met its primary safety and efficacy endpoints, demonstrating a statistically significant improvement in the time to first occurrence of treatment failure at 12 months. [Aastrom Biosciences, Inc. Press Release]

Targeted Therapy Promising For Cancer Patients
Customizing targeted therapies to each tumor’s molecular characteristics may be more effective for some types of cancer, according to research. [The University of Texas MD Anderson Cancer Center Press Release]

Diabetic Drug Could Help Prevent the Spread of Cancer
A protein activated by certain drugs already approved for treating Type II diabetes may slow or stop the spread of breast tumors. [Queen’s University Press Release]

(Ranked by Impact Factor of the Journal)

gp100 Peptide Vaccine and Interleukin-2 in Patients with Advanced Melanoma
In patients with advanced melanoma, the response rate was higher and progression-free survival longer with vaccine and interleukin-2 than with interleukin-2 alone. [N Engl J Med]

Suppression of Inflammatory and Neuropathic Pain by Uncoupling Collapsin Response Mediator Protein 2 from the Presynaptic Ca2+ Channel Complex
Investigators reported that inflammatory and neuropathic hypersensitivity can be suppressed by inhibiting the binding of collapsin response mediator protein 2 to N-type voltage-gated calcium channel and thereby reducing channel function. [Nature]

Kynurenine 3-Monooxygenase Inhibition in Blood Ameliorates Neurodegeneration
Researchers described the synthesis and characterization of JM6, a small-molecule prodrug inhibitor of kynurenine 3-monooxygenase. [Cell]

Effect of Fibroblast Growth Factor NV1FGF on Amputation and Death: A Randomized Placebo-Controlled Trial of Gene Therapy in Critical Limb Ischemia
The Phase III trial TAMARIS provided no evidence that non-viral 1 fibroblast growth factor (NV1FGF) is effective in reduction of amputation or death in patients with critical limb ischemia. [Lancet]

Functional Crosstalk Between Bmi1 and MLL/Hoxa9 Axis in Establishment of Normal Hematopoietic and Leukemic Stem Cells
In this study, scientists investigated whether leukemia-associated fusion proteins could initiate leukemia in the absence of Bmi1. [Cell Stem Cell]

Vaccination with Patient-Specific Tumor-Derived Antigen in First Remission Improves Disease-Free Survival in Follicular Lymphoma
Vaccination with patient-specific hybridoma-derived idiotype vaccine after chemotherapy-induced complete response (CR)/ CR unconfirmed may prolong disease-free survival in patients with follicular lymphoma. [J Clin Oncol]


Bracing Tour of China Leaves E.U. Science Chief Impressed
Visiting China for the first time, Europe’s top research official, Máire Geoghegan-Quinn, admitted that she was bowled over by how quickly the rising power is muscling up its R&D. [European Union’s Commissioner for Research, Innovation, and Science, European Union]

NC House Backs Drug-Maker Lawsuit Protections
North Carolina (NC) lawmakers have approved legislation that would shield drug companies from state liability suits. The state’s House voted 86-32 to grant FDA-approved drugs immunity from lawsuits, putting the new law in the hands of the state Senate. [North Carolina House, United States]
House Trims Homeland Security Science Spending
Next year’s budget for science and technology at the U.S. Department of Homeland Security would be drastically downsized under a spending bill passed by the House of Representatives. [House of Representatives, United States]


PR 2011-05-30 Immune Collaboration with Stirling and Zodiac
Immune Network Ltd. advises that it has entered into a conditional agreement with Stirling Products Limited and Zodiac Capital Limited with regard to an improved formulation of Immunoxel/Dzherelo recently developed and clinically tested by the Company. [Immune Network Ltd. Press Release]

MediGene and The Johns Hopkins University Sign Development Collaboration for First Vaccine Candidates from MediGene’s AAVLP Platform
MediGene AG and The Johns Hopkins University have agreed to collaborate in the field of AAVLP vaccine technology. [MediGene AG Press Release]

Bristol, Roche Team Up on Melanoma Study
Bristol-Myers Squibb and Roche Holding AG said they would evaluate their respective cancer drugs as a potential combination therapy for metastatic melanoma. [Reuters]

$1.1 Million Donation for Pioneering Stem Cell MS Trial
Multiple Sclerosis (MS) research is to benefit from a $1.1 million donation to further a pioneering bone marrow stem cell trial, which has been shown to have possible benefits for the treatment of the disease. [University of Bristol Press Release]

Cells Do Talk to One Another, but the Question Remains How
The National Science Foundation’s Advances in Biological Informatics program area has awarded a three-year, $1.12 million research grant to three Virginia Tech researchers with expertise in systems biology and tissue engineering: T.M. Murali of computer science, Padma Rajagopalan of chemical engineering, and Rich Helm of biochemistry. [Virginia Tech Press Release]

MaRS Innovation and GlaxoSmithKline Announce Strategic Partnership to Accelerate Translational Research in Health Sciences
MaRS Innovation and GlaxoSmithKline Inc. announced a new development fund – the GSK-MaRS Innovation Fund – that will support and fast-track the commercialization of some of the country’s most promising translational research coming from 16 leading academic health sciences centers, hospitals and universities derived from the MI membership. [MaRS Innovation Press Release]

UTSA and Southwest Research Institute to Collaborate on Biomedical Research
The University of Texas at San Antonio (UTSA) Office of the Vice President for Research and Southwest Research Institute (SwRI) announce an award of $200,000 in FY 2013 Connect program funding to UTSA Peter T. Flawn Professor of Biomedical Engineering Rena Bizios and SwRI Senior Research Scientists Vicky Poenitzsch and Xingguo Cheng for their collaborative research proposal, “Novel Scaffolds for Tendon-Ligament Regeneration and Tissue Engineering Applications.” [The University of Texas at San Antonio Press Release]

Clavis Pharma Receives Government Grant to Develop hENT1 Biomarker Assay for Targeted Therapy of AML Patients
Clavis Pharma ASA announced that it has been awarded a grant from The Research Council of Norway of up to NOK $14 mill (USD $2.5 mill) for the development of a flow cytometry method for the detection and quantification of human Equilibrative Nucleoside Transporter (hENT1) in patients suffering from acute myeloid leukemia (AML). [Clavis Pharma ASA Press Release]

YM BioSciences Announces Orphan Drug Designation for JAK1/JAK2 Inhibitor CYT387 in Europe
YM BioSciences Inc. announced that it has received positive opinions from the Committee for Orphan Medicinal Products of the European Commission to grant Orphan Medicinal Product Designation to its JAK1/JAK2 inhibitor, CYT387, for the treatment of primary myelofibrosis, post-polycythaemia vera myelofibrosis and post-essential thrombocythaemia myelofibrosis. [YM BioSciences Inc. Press Release]

SymBio Announces Korea NDA Approval of Bendamustine Hydrochloride (SyB L-0501)
SymBio Pharmaceuticals Limited announced that the Korea Food and Drug Administration approved bendamustine HCI (SyB L-0501) for the treatment of patients with chronic lymphocytic leukemia and multiple myeloma on May 31, 2011. [SymBio Pharmaceuticals Limited Press Release]

Consortium Opens Landmark Personalized Medicine Children’s Cancer Trial
The Neuroblastoma and Medulloblastoma Translational Research Consortium announced the opening of a first-of-its-kind genomic-based clinical trial to treat and study pediatric cancer – specifically relapsed and refractory neuroblastoma. [Van Andel Institute Press Relase]

Researcher Leads National Study of Novel Approach to Treat Triple-Negative Breast Cancer
A multicenter clinical trial will evaluate a new approach to treat triple-negative breast cancer. The study will help researchers determine if an experimental drug, entinostat, can reprogram tumor cells to express a protein called estrogen receptor to make them sensitive to hormone therapy. [University of Maryland Press Release]

Pioneering Stem Cell Bandage Receives UK Approval for Clinical Trial
The world’s first clinical trial for the treatment of patients with torn meniscal cartilage has received approval from the UK regulatory agency, the MHRA, to commence. The Phase I trial will treat meniscal tear patients with a cell bandage product, seeded with the patient’s own, expanded, stem cells. [University of Bristol Press Release]

Tekmira Expands Oncology Pipeline with RNAi Therapeutic Targeting Novel Cancer Genes WEE1 and CSN5
Tekmira Pharmaceuticals Corporation announced that it has secured licenses from Alnylam Pharmaceuticals, Inc. under its InterfeRx™ program to develop a new RNAi therapeutic targeting two validated oncology targets: WEE1 and CSN5. [Tekmira Pharmaceuticals Corporation Press Release]


Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2011-13534)

Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2011-13669)

Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2011-13794)

Center for Scientific Review; Amended Notice of Meeting (FR Doc No: 2011-13657)

Center for Scientific Review; Amended Notice of Meeting (FR Doc No: 2011-13792)

Center for Scientific Review; Amended Notice of Meeting (FR Doc No: 2011-13793)

Center for Scientific Review; Cancellation of Meeting (FR Doc No: 2011-13535)

National Cancer Institute; Notice of Closed Meetings (FR Doc No: 2011-13539)

National Cancer Institute; Amended Notice of Meeting (FR Doc No: 2011-13537)

Support of NIGMS Program Project Grants (P01) (PAR-11-220)

Notice of the National Human Genome Research Institute’s Interest in Receiving Applications to Analyze and Develop Methods for X Chromosome Genome-wide Association (GWA) Data (NOT-HG-11-021)

Collaborative Interdisciplinary Team Science in the National Institute of Diabetes and Digestive and Kidney Diseases Research Areas (R24) (PAR-11-221)


Guidance for Industry – Donors of Blood and Blood Components: Notification of Donor Deferral – Small Entity Compliance Guide

FDA Clinical Trial Requirements, Regulations, Compliance and GCP

Roster of the Cellular, Tissue and Gene Therapies Advisory Committee

Letter To Foreign and Domestic Medical Device and Component Establishments That May Be Affected by the March 2011 Japan Earthquake and Tsunami



Draft Guidance for Industry and FDA Staff: Commercially Distributed In Vitro Diagnostic Products Labeled for Research Use Only or Investigational Use Only: Frequently Asked Questions; Availability [Docket No. FDA-2011-D-0305]

Agency Information Collection Activities; Submission for Office of Management and Budget Review; Comment Request; Data To Support Drug Product Communications, as Used by the Food and Drug Administration [Docket No. FDA-2011-N-0067]

Agency Information Collection Activities: Proposed Collection; Reports and Records Under Prescription Drug Marketing Act of 1987 [FR Doc No: 2011-13442]

Collaboration in Regulatory Science and Capacity To Advance Global Access to Safe Vaccines and Biologicals [Docket No. FDA-2011-N-0375]

Draft Guidance for Clinical Investigators, Industry, and FDA Staff: Financial Disclosure by Clinical Investigators; Correction [Docket No. FDA-1999-D-0742 (formerly Docket No. 1999D-4396)]


ENCePP Publishes the ‘Guide on Methodological Standards in Pharmacoepidemiology’
The European Medicines Agency and the European Network of Centers for Pharmacoepidemiology and Pharmacovigilance (ENCePP) have published the ENCePP guide on methodological standards in pharmacoepidemiology.

European Medicines Agency and Heads of Medicines Agencies Propose Measures to Make Information in Application Dossiers More Transparent
The European Medicines Agency and the Heads of Medicines Agencies have released a guidance document on the identification of commercially confidential information and protection of personal data within the structure of the marketing-authorization dossier for public consultation.

European Medicines Agency Marks 40th Anniversary of PIC/S
The European Medicines Agency is marking the 40th anniversary of the Pharmaceutical Inspection Convention and Pharmaceutical Inspection Co-operation Scheme (PIC/S).

Study Provides Recommendations on European Medicines Agency’s Communication on Medicines
A report written by an independent expert has highlighted a number of recommendations to help improve the European Medicines Agency’s communication on the benefits and risks of medicines.


Information Sent to Healthcare Professionals in May About the Safety of Medicines
Copies of letters sent to healthcare professionals in May 2011, to inform of new safety information and advice.

Drug Safety Update: June 2011 (Volume 4, Issue 11)
Drug Safety Update is essential reading for all healthcare professionals, bringing the very latest information and advice to support the safer use of medicines.


Australian Regulatory Guidelines for Prescription Medicines (ARGPM)
Added amendment to Appendix 8

Australian Public Assessment Reports for Prescription Medicines (AusPARs)
Added Dabigatran etexilate mesilate, Rupatadine, Ferinject & Rosuvastatin

Presentations about Manufacturing Therapeutic Goods
Added presentations about quality risk management and clinical trials

Therapeutic Goods (Excluded Goods) Order No. 1 of 2011
New therapeutic goods (excluded goods) order

Scheduling News
Updates and news about scheduling medicines and poisons

Scheduling FAQs
Updated FAQs on revised scheduling arrangements from 1 July 2010 and general scheduling matters

Advisory Committees on Medicines & Chemicals Scheduling (ACMS & ACCS)
Updated ACCS and ACMS meeting dates


NEW Gordon Research Conferences: Biomaterials & Tissue Engineering
July 31-August 5, 2011
Holderness, United States

The EMBO Meeting 2011
September 10-13, 2011
Vienna, Austria

NEW American Association for Cancer Research International Conference New Horizons in Cancer Research: Biology to Prevention to Therapy
December 13-16, 2011
Gurgaon, India

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

Chemist (STEMCELL Technologies)

Research and Development Senior Technologist (STEMCELL Technologies)

Senior Research Fellowship (Masaryk University)

Junior Research Fellowship (Masaryk University)

Reintegrating/Czech Nationality/ Research Fellowship (Masaryk University)

Microscopy and Image Analysis Specialist (Masaryk University)

PhD Positions in Stem Cell Research (Masaryk University)

PhD Positions in Medical Biology (Masaryk University)

Postdoctoral Fellow – Stem Cell Biology and Immunology (NIH Center for Biomedical Research Excellence)

Graduate Student – Laboratory for Equine and Comparative Orthopedic Research (Louisiana State University)

Post Doctoral Fellow (University of California, Los Angeles)

Staff Scientist Position (Spanish National Cancer Center)

Postdoctoral Research Fellows (Stem Cells) (A*Star Bioprocessing Technology Institute) 

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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