Volume 9.48 | Dec 8

Cell Therapy News 9.48, December 8, 2008
     In this issue: Science | Policy | Business | NIH | CBER | Regulatory
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Top Stories

Gene Therapy Corrects Sickle Cell Disease in Laboratory Study: Using a harmless virus to insert a corrective gene into mouse blood cells, scientists at St. Jude Children’s Research Hospital have alleviated sickle cell disease pathology.


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A Novel Human Stem Cell-Based Model of ALS Opens Doors for Rapid Drug Screening
Researchers from the Salk Institute for Biological Studies for the very first time established a human embryonic stem cell-based system for modeling ALS.

Next Generation RNAi Technology Discovered by AiRNA Pharmaceuticals and Boston Biomedical 
Research from this study highlights that the smaller 15 bp aiRNA can target a variety of genes with superior efficiency than the standard 19-21 bp siRNA and can target genes more selectively with significantly less off-target effects than the standard 19-21 bp siRNA.

New ‘Control Knobs’ for Stem Cells: Changes in Membrane Voltage Control Timing of Differentiation
Natural changes in voltage that occur across the membrane of adult human stem cells are a powerful controlling factor in the process by which these stem cells differentiate, according to research published by Tufts University scientists.

Dormant Stem Cells for Emergencies
A small group of stem cells in the bone marrow remains dormant almost throughout life and only in case of injury or blood loss do they awaken and become active.

Bone Marrow-Derived Stem Cells May Offer Novel Therapeutic Option for Skin Disorder
Stem cells derived from bone marrow may serve as a novel therapeutic option to treat a disease called epidermolysis bullosa, a disorder characterized by extraordinarily fragile skin.

Regulus Therapeutics, Alnylam Pharmaceuticals, and Collaborators Publish First Ever In Vivo Efficacy Data for a microRNA Therapeutic in a Disease Model
Regulus Therapeutics LLC, a joint venture between Alnylam Pharmaceuticals, Inc. and Isis Pharmaceuticals, Inc. announced recently the publication of new research in the journal Nature on the role of a microRNA, known as miR-21, in heart failure.

First Ever Treatment of Stroke Patient with Stem Cell Therapy Product
The International Neuroscience Institute is pleased to announce that the first patient has been treated in the first ever clinical trial for the treatment of stroke with a stem cell product.


Human Embryonic Stem Cell-Derived Motor Neurons Are Sensitive to the Toxic Effect of Glial Cells Carrying an ALS-Causing Mutation
The researchers ask whether specific neuronal subtypes produced from human embryonic stem cells can be used to investigate the mechanisms leading to neural degeneration in amyotrophic lateral sclerosis.

Gene Therapy Using TRAIL-Secreting Human Umbilical Cord Blood–Derived Mesenchymal Stem Cells against Intracranial Glioma
In the present study, the researchers evaluated the tumor targeting properties and anti-tumor effects of UCB-MSCs as gene delivery vehicles for glioma therapy.

The Relationship Between Circulating Natural Killer Cells after Reduced Intensity Conditioning Hematopoietic Stem Cell Transplantation and Relapse-Free Survival and Graft-Versus-Host Disease
In this study serial peripheral blood absolute natural killer cell counts were prospectively measured by flow cytometry of lymphocytes expressing CD56 and CD16 in 167 patients.

Biologic Properties and Enucleation of Red Blood Cells from Human Embryonic Stem Cells
This paper shows that it is feasible to differentiate and mature human embryonic stem cells into functional oxygen-carrying erythrocytes on a large scale (1010-1011 cells/6-well plate hESCs).


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Putting State Money to Good Use
The Maryland Stem Cell Research Commission recently announced that it had received 187 letters of intent from researchers vying for $18 million in state funds available from the Maryland Stem Cell Research Fund, which substantially survived the state’s recent budget difficulty with a modest $1 million cut for fiscal 2009.

Lawmakers Divided Over Next FDA Head, Fixing Safety Issues
A top House Democrat is asking President-elect Barack Obama to avoid naming any current officials of Food and Drug Administration to lead the agency, even temporarily, reflecting a divergence of views on Capitol Hill on how to fix the FDA’s problems.


Pluristem Retains KKS as Its Contract Research Organization for Upcoming Limb Ischemia Clinical Trial 
Pluristem Therapeutics Inc. recently announced that the Company signed an agreement with KKS, retaining them as the Contract Research Organization for its upcoming Critical Limb Ischemia multi center clinical trial utilizing Pluristem’s placental-derived adherent stromal cell product, PLX-PAD.

Neuralstem Collaborates with Hospital in Taiwan to Develop Additional ALS Stem Cell Clinical Trials
Neuralstem Inc. announced recently that it has entered into a collaboration with the China Medical University & Hospital of Taiwan, to advance development of Neuralstem’s human spinal cord neural stem cell therapies.

Burnham and HeadNorth Foundation Join Forces to Advance Spinal Cord Research
The funding, part of HeadNorth’s Chronic Spinal Cord Injury Project, will support efforts to use stem cells to treat chronic spinal cord injuries.

British Team Lead Stem Cell Heart Surgery that could End Need for Transplants
A top cardiovascular researcher who is trying to harness the body’s own mechanisms to help the heart repair itself has won a prestigious award to help turn his research into a treatment for heart disease patients.

Introgen Files Voluntary Chapter 11 Petition to Facilitate Restructuring
As part of its ongoing restructuring, Introgen Therapeutics, Inc. recently announced that the company has elected to file Chapter 11 petitions in the U.S. Bankruptcy Court, Western District of Texas.

Roche May Have Trouble with Genentech Financing
The prospects of Swiss drugmaker Roche raising a $45 billion syndicated loan to finance the buyout of the rest of U.S. biotech group Genentech are becoming increasingly remote.

$2.5 Million Towards Gene Therapy for Human Degenerative Retinal Diseases
A Canadian and American research group including the team of Dr. Robert Koenekoop from the Research Institute at the Montreal Children’s Hospital of the MUHC has just been awarded $2.5 million to fund an ambitious research project to develop innovative gene therapies for a number of human degenerative retinal diseases.

Products for Cold Storage of Embryos Approved in USA
Vitrolife is now the first company to have received approval from the American Food and Drug Administration for products within the fertility area used for the rapid freezing and thawing of cleavage embryos.

Bioheart Files for Reimbursement Approval in Europe for MyoCell(R) Myogenic Stem Cell Therapy for Heart Failure
Bioheart, Inc. announced recently it has filed with appropriate agencies in Italy, Germany and the Netherlands for reimbursement approval for its MyoCell(R) myogenic cell therapy for heart failure.

Intradigm Announces Issuance of Key Patent Related to Enhancing Efficacy and Potency of RNAi Therapeutics
Intradigm Corporation recently announced the issuance of United States patent 7,459,547 generally claiming methods of enhancing the RNA silencing activity of an RNAi agent through certain structural modifications in various cell types, including mammalian.


NIH to Migrate Approximately 100 FOAs in Early Dec; Applicants Will Receive Automated E-mail from Grants.gov (NOT-OD-09-022)

Enhancing Peer Review: The NIH Announces New Scoring Procedures for Evaluation of Research Applications Received for Potential FY2010 Funding (NOT-OD-09-024)

Enhancing Peer Review: The NIH Announces Enhanced Review Criteria for Evaluation of Research Applications Received for Potential FY2010 Funding (NOT-OD-09-025)

Notice of Correction to Application Instructions for: PAR-09-012 “2009 NIH Directors Pioneer Award Program (X02) (NOT-RM-09-006)

Notice of Correction to Application Instructions for: PAR-09-013 “2009 NIH Directors New Innovator Award Program (X02)” (NOT-RM-09-007)

NLM Grants for Scholarly Works in Biomedicine and Health (G13) (PAR-09-030)


Guidance for Industry Cooperative Manufacturing Arrangements for Licensed Biologics



Guidance for Industry: Potency Tests for Cellular and Gene Therapy Products

Guidance for Sponsors, Clinical Investigators, and IRBs; Data Retention When Subjects Withdraw From FDA-Regulated Clinical Trials; Availability

Guidance for Industry: Cooperative Manufacturing Arrangements for Licensed Biologics; Availability 73940–73941 [E8–28693]


An Updated List of Members of the GLP Compliance Programme

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