ASH News Daily 2013 - Day 4 - (Page A-1)

Read this issue online at www.hematology.org/ashnewsdaily2013_tuesday Follow us on Twitter using #ASH13 Schedule 7:00 a.m. - 1:30 p.m. Registration Great Hall 7:15 - 9:15 a.m. Special Symposium on the Basic Science of Hemostasis and Thrombosis* (invited presentations) Room 265-268 7:30 - 9:00 a.m. Simultaneous Oral Sessions Various locations in convention center 7:30 - 9:00 a.m. Late-Breaking Abstracts Session Hall F 9:30 - 9:50 a.m. Announcement of Awards Dameshek Prize, Stratton Medal, and Mentor Award Hall F 9:50 - 11:20 a.m. Presidential Symposium Hall F 11:20 - 11:30 a.m. Business Meeting Hall F 12:00 noon - 1:00 p.m. Best of ASH Hall F *Dr. Zaverio Ruggeri, co-chair, is unable to attend the symposium due to weather. IN THIS SECTION Choosing Wisely A-3 Late-Breaking A-3 update T Cells in CLL A-4 Awards A-6 Microangiopathies A-7 The Gene Therapy Voyage: Finally Land in Sight By Shaji Kumar, md D evelopment of allogeneic stem cell transplantation led to a paradigm shift in the management of numerous hematologic conditions - malignant and benign, acquired and inherited. We owe a great deal to Professor E. Donnall Thomas for pioneering the concept, a discovery that led to the well-deserved Nobel Prize in Medicine in 1990. It is only apt that the E. Donnall Thomas Lecture and Prize at this year's annual meeting is dedicated to another therapeutic modality that can potentially alter the lives of hundreds of thousands. This year's E. Donnall Thomas Lecture was delivered by Dr. Katherine A. High, Children's Hospital of Philadelphia. The William H. Bennett Professor of Pediatrics at the Perelman School of Medicine, University of Pennsylvania took the audience through the enthralling journey of gene therapy over the last several decades. Dr. High has led the way in bringing the gene- therapy approaches to the clinic, particularly in the setting of hemophilia. The concept of altering the fundamental building block of the human body, literally its identity, has often been depicted as "playing God." However, the societal bur- den of inherited diseases has only increased during the past decade as advances in medical and supportive care have allowed patients to live progressively longer with disorders once considered incompatible »» E. DONNALL THOMAS Page A-2 Incoming President Dr. Linda Burns talks about her priorities and goals for the coming year. Genomics in Hematology: Ready for Prime Time or OMG TMI!? By marK m. udden, md T here are more than 8 billion species of plant and animal life thought to exist on earth, and we now accept evolution as the explanation for the origin of this biodiversity. Darwin's concept of evolution as an explanation for this incredible diversity of life was made possible by the work of biologists like Karl Linnaeus who spent a lifetime collecting, naming, describing, and obsessively classifying plants and animals based on shared characteristics. This morning's Presidential Symposium, Using Genomics for Clinical Decision Making: Are We There Yet?, will address the molecular taxonomy of a different level of biodiversity: the diversity of expression of genetic disorders of hemostasis and the di- verse cellular evolution leading to leukemia and myelodysplasia. Our ability to identify, describe, and characterize this genetic diversity has been greatly expanded based on the incredible mass of genetic information derived from genomewide association studies (GWAS) and whole-genome sequencing (WGS) of an individual's germline DNA and the DNA of cell populations arising from neoplastic transformation. Today's session will be chaired by outgoing ASH President Dr. Janis Abkowitz, University of Washington, and will start with a presentation by Dr. James Downing, St. Jude Children's Research Hospital, Memphis, TN, who will provide an introduction to genomics and will describe how next-generation DNA sequencing has retrospective- ly identified new genetic subtypes of both ALL and AML. He stated, "For a subset, there are targeted agents against the specific altered proteins." Though promising, Dr. Downing noted, "Next-generation DNA/RNA sequencing remains expensive. The costs can be managed by focusing on selected genes, the so-called 'actionable mutations.'" Next, Dr. Matthew Walter, Washington University School of Medicine, St. Louis, MO, will discuss recent results of WGS studies of myelodysplasia at diagnosis and during evolution to leukemia. The analysis of these patients indicates that there are typically founder clones; then one or more subclones, which are present at diagnosis, can evolve. Dr. Walter noted, "The »» PRESIDENTIAL Page A-7 http://www.hematology.org/ashnewsdaily2013_tuesday

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ASH News Daily 2013 - Day 4

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