ASH News Daily - Tuesday, December 13, 2011 - (Page A-4)

Page A–4 ® 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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ASH News Daily - Tuesday, December 13, 2011

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