18 children with severe combined immunodeficiency respond to gene therapy | Allogeneic transplantation induces sustained remission of multiple myeloma | Study offers clues about hepatitis C antiviral resistance
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November 19, 2014
AABB SmartBrief
News for the transfusion medicine and cellular therapy community

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18 children with severe combined immunodeficiency respond to gene therapy
University of California, Los Angeles, researchers have announced that 18 children with adenosine deaminase-deficient severe combined immunodeficiency are thriving and have functioning immune systems after being treated with gene therapy. Blood stem cells were collected from the patients' bone marrow and modified to correct the defect that caused the illness. ABC News (11/18)
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Science & Health
Allogeneic transplantation induces sustained remission of multiple myeloma
Multiple myeloma patients who receive allogeneic cell transplants experienced sustained remission, particularly when they receive transplants as consolidation therapy, according to a study in the journal Biology of Blood and Marrow Transplantation. The five-year overall survival was 59% for all patients and 82% for those who received consolidation therapy. Healio (free registration)/HemOnc Today (11/18)
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Study offers clues about hepatitis C antiviral resistance
A study in the journal Nature Communications sheds light on how the hepatitis C virus develops drug resistance and contributes to the development of cancer. Treatments that target microRNA-122, which helps the virus infect human cells, appear to trigger drug resistance, giving rise to strains that can infect cells with low levels of miR-122. The virus may trigger cancer by reducing levels of miR-122, which acts as a tumor repressor. Genetic Engineering & Biotechnology News (11/18)
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Grafted neurons grow long axons in injured rat spinal cord
Fetal rats' neural progenitor cells that were grafted into spinal cord lesions in adult rats two weeks or six months after injury were able to grow and connect to surrounding neurons, according to a study presented at the Society for Neuroscience meeting. The neurons also grew longer axons that extend to the brain stem and regions of the spinal cord, and the astrocytes that form scar tissue around the lesion site disappeared after the neurons had grown. The Scientist online (11/18)
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Study finds protein associated with membranous nephropathy
A study in The New England Journal of Medicine has identified a second protein that causes membranous nephropathy. The discovery could lead to the development of a blood test to diagnose and monitor the condition. Business Standard (India)/Indo-Asian News Service (11/18)
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Key Elements of Cord Blood Processing
So many choices, so little time! This November 20 webinar will address key elements of cord blood processing, manual vs. automated methods of recovery, and various types of cells recovered by each. Participants will better understand the different technologies available and how they are being used so they can assess their own methods and evaluate additional options. Register Now!
Emerging TrendsSponsored By
Home dialysis more effective than in-center dialysis, study finds
A study presented at the annual Kidney Week meeting found that home hemodialysis and peritoneal dialysis resulted in better outcomes for patients. Those approaches should be considered whenever possible for end-stage kidney disease, researchers said. The study analyzed U.S. Renal Data System data involving 585,911 patients from 2005 to 2010. PhysiciansBriefing.com/HealthDay News (11/18)
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Industry News & Practice
Gates Foundation donates $5.7 million for tests of blood-based Ebola therapy
The Bill & Melinda Gates Foundation has committed $5.7 million to support studies of Ebola treatments that use convalescent plasma and other blood products from Ebola survivors. The funding also will support drug studies, and the trials will be carried out in Guinea and other nations in West Africa. BBC (11/18), Time.com (11/19)
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Wis. blood center taps N-of-One for oncology testing services
The BloodCenter of Wisconsin has entered into an oncology testing partnership with N-of-One for undisclosed terms. Under the deal, N-of-One will offer clinical interpretation services for the center's HemeOnc next-generation sequencing-based panel, which is used for the detection, risk stratification and prognosis of hematologic cancers. GenomeWeb Daily News (free registration) (11/18)
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Europe grants additional patent for OXiGENE's leukemia drug candidate
OXiGENE's acute myeloid leukemia drug candidate, OXi4503, has received a European patent covering its use as a treatment for nonsolid myeloid neoplasms. OXi4503, or combretastatin A1 di-phosphate, blocks and attacks tumor vasculature. Pharmaceutical Business Review Online (11/18)
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Now Available: Standards for a Patient Blood Management Program, 1st edition
AABB's Standards for a Patient Blood Management Program is designed for facilities seeking to improve patient outcomes by establishing or expanding a PBM program. Based on a quality framework, these standards are flexible to provide for various three activity levels that reflect the services offered by your facility. Order Now!
Government & Regulatory
Juno Therapeutics' leukemia drug receives FDA orphan drug status
JCAR015, an experimental chimeric antigen receptor-modified T-cell therapy from Juno Therapeutics, has been granted orphan status by the FDA for use in patients with acute lymphoblastic leukemia who have not received prior treatment. JCAR015 zeroes in on CD19, which is greatly expressed in B-cell malignancies and other leukemia subtypes. Healio (free registration)/HemOnc Today (11/19)
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Kite's cancer immunotherapy takes step toward orphan status in Europe
The European Medicines Agency's Committee for Orphan Medicinal Products has recommended that Kite Pharma's KTE-C19, an experimental treatment for diffuse large B cell lymphoma, be granted an orphan drug designation. The therapy modifies T cells to target CD19, a protein present in most B cell malignancies. Pharmaceutical Business Review Online (11/18)
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Association News
AABB supports creation of CPT Tier 1 code for blood group genotyping
AABB sent a letter to the American Medical Association's CPT Advisory Committee to support the creation of a Tier 1 current procedural terminology code for blood group genotyping. Genotyping for human erythrocyte antigens has become increasingly common in transfusion medicine and is especially valuable for frequently transfused patients who are at increased risk of suffering life-threatening adverse reactions due to transfusions of incompatible blood. Genotyping provides a faster and more thorough method of determining blood group antigens than current serologic typing techniques, according to AABB. Effective Jan. 1, 2015, there will be a Tier 2 CPT code for blood group genotyping. However, establishment of a Tier 1 code would facilitate reimbursement for and increase patient access to this critical service. Recognizing the importance of blood group genotyping, AABB has published Standards for Molecular Testing for Red Cell, Platelet and Neutrophil Antigens since 2008.
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Who's Hiring?
Position TitleCompany NameLocation
Laboratory quality manager/QC unitTexas Stem CellHouston, TX
Facility processing DirectorTexas Stem CellHouston, TX
Medical Blood Bank Technologists & Sr TechnologistsUniversity of Miami Miller School of MedicineMiami, FL
Clinical Laboratory ScientistEisenhower Medical CenterRancho Mirage, CA
Deputy Director, Regulatory AffairsAABBBethesda, MD
Click here to view more job listings.
It is always our own self that we find at the end of the journey. The sooner we face that self, the better."
-- Ella Maillart,
Swiss travel writer
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