ASH News Daily - Tuesday, December 13, 2011 - (Page A-4)
Page A–4
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NEUTROPENIA
Christoph Klein, 2011 Dameshek Prize Winner
“
for my whole team of physicians
and scientists who dedicated their
lives with great enthusiasm hoping
that their work will make a difference
for children with rare blood
disorders,” said Christoph Klein
MD, PhD, of Ludwig Maximilian
University of Munich in Munich,
Germany. This morning, the 2011
William Dameshek Prize will be
awarded to Dr. Klein for his innovative
contributions to hematology,
namely his landmark discovery
of mutations on HAX1 genes
in patients with severe congenital
neutropenia (Kostmann disease)
and his discovery of certain genetic
defects that can cause this disease.
The Dameshek Award, named for
the late William Dameshek, MD,
a noted hematologist, past president
of ASH, and first editor of
Blood, honors an individual who
has made a contemporary and exceptional
contribution to the field
of hematology. As a result of Dr.
Klein’s research, new hope is available
to patients whose diseases
T
ALL
Champion of ALL Wins Henry M. Stratton Medal
search Hospital with the 2011 Henry
M. Stratton Medal for 30 years
of extraordinary achievements in
childhood leukemia research and
treatment. The Stratton Medal,
named after the late Henry Maurice
Stratton, co-founder of the Grune
and Stratton publishing house, celebrates
an individual whose contributions
in hematology and to
the Society have been noteworthy
throughout an illustrious career.
Dr. Pui has been at the forefront of
T
leukemia research for the past three
decades. Dr. Pui was instrumental
in increasing St. Jude’s cure rate for
childhood ALL from approximately
70 percent in the early 1980s to an
unprecedented 90 percent today.
His work has also showed that the
once standard cranial irradiation
treatment for ALL could be omitted
altogether, sparing patients from
devastating side effects and enhancing
their quality of life. A champion
of patients with ALL, Dr. Pui advises
the next generation of hematologists
to “Be focused and persistent in the
pursuit of your professional goals.
Work hard on your research interests,
challenge conventional wis-
Ching-Hon Pui, MD
dom, and collaborate at every opportunity
with scientists and clinicians
engaged in translational research.”
Dr. Pui credits hematology as the
means by which he could pursue his
dream of discovering a permanent
cure for ALL. Many of his research
findings are widely accepted in the
field of pediatric oncology. While he
credits the field for drawing him in,
it is often the patients who have kept
him, and while there are many happy
memories of time spent with his
patients, some are rather emotional.
Dr. Pui recalls one patient who had
his morning the Society will
present Ching-Hon Pui, MD,
of St. Jude Children’s Re-
endured multiple relapses and, at
the end, tearfully apologized to his
mother that he had to leave her. “The
poignancy of the moment stayed
with me,” Dr. Pui said, “strengthening
my resolve to combat childhood
cancers through research.”
Had he not entered the field of
hematology, Dr. Pui disclosed that
he might have pursued a career in
teaching, noting that it was two of
his early teachers who encouraged
him to consider medical education.
Surprisingly, he might have chosen a
very different route altogether. “Music
has always provided me with a
relaxing diversion from work, and
I could easily see myself as a choral
director,” he said. But, as fate would
have it, perhaps he did not stray too
far from his musical inclinations. In
an article published this past summer
in the Memphis Daily News
(chronicling yet another award Dr.
Pui received), St. Jude Director and
CEO Dr. William E. Evans compared
the doctor to a “great conductor
drawing together the best musicians
to create something that is extraordinary
and far greater than any one
person playing alone.”
Harmonization is a major chord
in Dr. Pui’s life. Not only does Dr.
Pui draw from his own well of re-
sources as a skilled researcher, pediatrician,
humanitarian, and educator,
but he also recognizes the need
to pool together resources across
international boundaries and work
toward a common goal. “Extending
curative treatment to children with
leukemia in developing countries
has perhaps been the greatest challenge.
This task is much too difficult
for any individual to attempt,”
Dr. Pui said. Instead, he works as a
medical director for the International
Outreach at St. Jude Children’s
Research Hospital program sharing
organizational skills, contemporary
knowledge, and the latest technology
with developing countries.
He also founded the International
Childhood ALL Working Group,
which is responsible for standardizing
cost-effective treatment for
those countries.
A member of ASH since 1983,
Dr. Pui has served as chair of a Scientific
Program session at the ASH
annual meeting and as a member
of the Blood editorial board. He has
also given numerous presentations
at ASH’s annual meetings and published
approximately 100 articles in
Blood. Dr. Pui will receive the Henry
M. Stratton Medal today at 9:30
a.m. in Hall AB.
he William Dameshek
Award is a truly exceptional
honor, not just for me, but
were not fully understood not too
long ago.
Dr. Klein’s work has included the
discovery that the severe early onset
of inflammatory bowel disease
(IBD) is linked to certain genetic
mutations; he then used stem cell
transplantation to put IBD into remission
for one child. Additionally,
he successfully ran the first-ever
gene therapy trial to correct symptoms
of the rare, inherited, and
life-threatening immunodeficiency
Wiskott-Aldrich syndrome (WAS).
In nine out of 10 children enrolled
in this clinical trial, treatment was
successful.
Looking back, Dr. Klein’s deci-
sion to practice pediatric hematology
came after a scholarship allowed
him to conduct research and perform
clinical rotations at the Children’s
Hospital and Dana-Farber Cancer
Institute in Boston. Early in his career
he encountered many inventive
scientists who encouraged him to
pursue academic medicine.
Still, Dr. Klein’s pursuit was not
without a unique set of challenges.
Gene therapy trials have had few
successes and many failures since
ASH NEWS DAILY
Tuesday, December 13, 2011
principle, ‘never give up.’ In most
instances, it has worked. However,
there were experiences that have
taught me that even a strong conviction
and enduring perseverance
cannot overcome all barriers. Failing
is part of being human, and we
should accept this humbly.”
While at work, Dr. Klein finds
Christoph Klein, MD, PhD
the first in 1990, and there has yet to
be an FDA-approved gene therapy
treatment in the United States. But
in 2003, Dr. Klein and colleagues
at Children’s Hospital in Boston
successfully inserted a retrovirus
with a healthy duplicate of a gene
into the hematopoietic stem cells of
WAS-deficient mice.
“I have seen many challenges,”
Dr. Klein said, “and I adhered to the
pleasure in seeing patients cured
based on the innovations of his
colleagues. While away from the
office, he cherishes spending time
with family, traveling extensively,
studying philosophy, learning
about foreign cultures, and relishing
nature’s treasures. He also enjoys
music and admits that had he
not chosen hematology he might
have been an organist. But hematology
it was, and he has continued
to enjoy a successful career in the
field. To those who would consider
this field, Dr. Klein advises, “Don’t
give up your enthusiasm and curiosity,
be persistent in your endeavors,
be honest and authentic, and
find your own way.”
Dr. Klein will receive the William
Dameshek Prize today at 9:30 a.m.
in Hall AB.
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