Researchers Halt Trial of Promising Sickle Cell Treatment

Two patients in a gene therapy study developed cancer years after treatment. It is not clear whether the therapy was responsible.
Source: NYT Health - Category: Consumer Health News Authors: Tags: Sickle Cell Anemia Leukemia Bone Marrow Genetic Engineering Clinical Trials Bluebird Bio Inc Williams, David II DNA (Deoxyribonucleic Acid) National Heart, Lung and Blood Institute Busulfan Drugs (Pharmaceuticals) Source Type: news

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Publication date: 15 February 2021Source: Journal of Hazardous Materials, Volume 404, Part BAuthor(s): Yanhua Liu, Yang Li, Shanshan Dong, Lu Han, Ruixin Guo, Yourong Fu, Shenghu Zhang, Jianqiu Chen
Source: Journal of Hazardous Materials - Category: Environmental Health Source Type: research
We describe the most highly recommended generic and disease-specific PRO tools in SCD and discuss the challenges of incorporating them in clinical practice. EXPERT OPINION: PRO measures are essential to incorporate into SCD clinical trials either as primary or secondary outcomes. The use of PRO measures in SCD facilitates a patient-centered approach, which is likely to lead to improved outcomes. Significant challenges remain in adapting PRO tools to routine clinical use and in developing countries. PMID: 33034214 [PubMed - as supplied by publisher]
Source: Expert Review of Hematology - Category: Hematology Tags: Expert Rev Hematol Source Type: research
Authors: Musio F Abstract INTRODUCTION: Anemia has and will continue to be a central theme in medicine particularly as clinicians are treating a burgeoning population of complex multi-organ system processes. As a result of multiple randomized controlled trials (RCTs), meta-analyses, and societal recommendations overly restrictive paradigms and under-administration of erythropoiesis stimulating agents (ESAs) have likely been followed by clinicians among all specialties. AREAS COVERED: A review of anemia in the context of chronic kidney disease, hematologic malignancies and cancer is presented with focus on the e...
Source: Expert Review of Hematology - Category: Hematology Tags: Expert Rev Hematol Source Type: research
Publication date: Available online 9 October 2020Source: Mutation Research/Reviews in Mutation ResearchAuthor(s): Klaudia Kulczynska-Figurny, James J. Bieker, Miroslawa Siatecka
Source: Mutation Research Reviews in Mutation Research - Category: Genetics & Stem Cells Source Type: research
Publication date: Available online 25 June 2019Source: Life SciencesAuthor(s): Mehdi Derakhshani, Hossein Abbaszadeh, Ali Akbar Movassaghpour, Amir Mehdizadeh, Majid Ebrahimi-Warkiani, Mehdi YousefiAbstractHematopoietic stem cells (HSCs) are a rare cell population in adult bone marrow, mobilized peripheral blood, and umbilical cord blood possessing self-renewal and differentiation capability into a full spectrum of blood cells. Bone marrow HSC transplantation has been considered as an ideal option for certain disorders treatment including hematologic diseases, leukemia, immunodeficiency, bone marrow failure syndrome, genet...
Source: Life Sciences - Category: Biology Source Type: research
Discussion This case demonstrates successful cure of pre-B-ALL complicating XLA by alloSCT with restoration of B-cell development and functional antibody response. We are aware of only one previous case of pre-B-ALL in an XLA patient (21), which suggests that human BTK deficiency in itself does not predispose to pre-B-ALL. However, there are data to suggest that BTK may act as a tumor suppressor, and BTK deficiency may predispose to tumor development following a “second hit.” Mice with a genetic deficiency in Slp65, a gene encoding an adaptor protein that functions together with BTK, have a block in progenito...
Source: Frontiers in Immunology - Category: Allergy & Immunology Source Type: research
Conclusions:Our study demonstrates frequent variants in histone modification, splicing, and transcription-associated genes in JMML specimens in addition to known pathogenic RAS pathway mutations. We further report histopathologic CD123+ PDC clustering in JMML specimens analogous to that observed in CMML, which may aid in the workup of this often difficult-to-diagnose disease. Our findings of genetic and immunophenotypic overlap between JMML and CMML suggest similarities in pathogenesis despite typical presentation at extremes of age.DisclosuresTasian: Aleta Biopharmaceuticals: Membership on an entity's Board of Directors o...
Source: Blood - Category: Hematology Authors: Tags: 636. Myelodysplastic Syndromes-Basic and Translational Studies: Poster I Source Type: research
With the proposed changes to the Affordable Care Act (ACA), 20 million Americans are at risk of losing their health care coverage. A survey, conducted by Brunswick Partners, found that “75 percent of Americans agree that the proposed changes to Medicaid in the AHCA are a bad idea. And that we should not allow 14 million Americans to become uninsured even if there is a potential to reduce Medicaid spending. These results are significant because they find majorities of Americans identifying as conservatives (55 percent), moderates (82 percent) and liberals (90 percent) are opposed to the AHCA’s Medicaid pro...
Source: Healthy Living - The Huffington Post - Category: Consumer Health News Source Type: news
NCI’s Center for Cancer Research (CCR) Grand Rounds Dr. Lucio Luzzatto received his M.D. from the University of Genova and then went on to do a fellowship in hematology at the University of Pavia and receive his Ph.D. in biochemistry from the Ministry of Education in Rome. The leitmotiv of his research has been to understand, in depth, blood diseases with the ultimate purpose to improve their management. In the area of G6PD he did extensive studies in population and biochemical genetics, and in the early eighties cloned the G6PD gene. He took part in solving the 3D structure of human G6PD, and thus eventually he an...
Source: Videocast - All Events - Category: Journals (General) Tags: Upcoming Events Source Type: video
CHICAGO (AP) -- Barely 2 years old, Talia Pisano is getting tough treatment for kidney cancer that spread to her brain. She's also getting a chance at having babies of her own someday.  To battle infertility sometimes caused by cancer treatment, some children's hospitals are trying a futuristic approach: removing and freezing immature ovary and testes tissue, with hopes of being able to put it back when patients reach adulthood and want to start families. No one knows yet if it will work. It has in adults - more than 30 babies have been born to women who had ovarian tissue removed in adulthood, frozen, and put back af...
Source: Science - The Huffington Post - Category: Science Source Type: news
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