ASH News Daily 2013 - Day 4 - (Page A-1)
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7:00 a.m. - 1:30 p.m.
7:15 - 9:15 a.m.
Special Symposium on the Basic Science
of Hemostasis and Thrombosis*
7:30 - 9:00 a.m.
Simultaneous Oral Sessions
Various locations in convention center
7:30 - 9:00 a.m.
Late-Breaking Abstracts Session
9:30 - 9:50 a.m.
Announcement of Awards
Dameshek Prize, Stratton Medal,
and Mentor Award
9:50 - 11:20 a.m.
11:20 - 11:30 a.m.
12:00 noon - 1:00 p.m.
Best of ASH
*Dr. Zaverio Ruggeri, co-chair, is unable to
attend the symposium due to weather.
IN THIS SECTION
T Cells in CLL
The Gene Therapy Voyage: Finally Land in Sight
By Shaji Kumar, md
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
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
Table of Contents for the Digital Edition of ASH News Daily 2013 - Day 4
ASH News Daily 2013 - Day 4