Scientist Earns Gene Therapy Research Award
CINCINNATI -- Christof von Kalle, MD, PhD, a physician / researcher at Cincinnati Children's Hospital Medical Center is one of only three scientists in the United States to earn a Young Investigator Award this year from the American Society of Gene Therapy (ASGT). Dr. Kalle earned the award for his groundbreaking research in human gene therapy trials.
Last fall, gene therapy trials in France were halted when a three-year-old boy who had been cured of a fatal immune deficiency developed a leukemia-like disease. A few months later, a second boy who had been successfully treated for his severe combined immune deficiency also developed a similar disease.
Studies by Dr. Kalle revealed that the virus vector carrying the "therapeutic" gene had inserted itself next to a gene called LMO-2, which is known to play a role in leukemia. This apparently activated the LMO-2 gene, which resulted in abnormal cell growth. Dr. Kalle's studies focus on identifying these events at the gene level and developing an understanding of the underlying causes of the leukemia that developed.
"Dr. Kalle's contributions this year have rapidly converted a mysterious and very frightening event into a clear and focused pathway towards preventing such complications in the future," says Cynthia Dunbar, MD, a member of the Board of Trustees of the ASGT. "His incredible dedication to the tasks at hand, and his openness (along with that of the French investigators leading the clinical trial) have served as a model for how adverse events in this field should be approached."
Dr. Kalle is an internationally recognized expert in the study of human hematopoietic stem cells using "stem cell tracking" methods in human gene therapy trials. He developed over a period of years the methodology to identify and track specific insertion sites of vectors in the human genome.
He joined the Division of Experimental Hematology at Cincinnati Children's in 2002 as associate professor and program leader of the Molecular and Gene Therapy Program. The program's goal is to determine methods for making gene therapy more effective while assuring patient safety. Dr. Kalle came to Cincinnati Children's from Freiburg University in Germany, where he was group leader of the Section of Hematopoiesis and Gene Transfer.
"Dr. Kalle's novel observations in the French gene therapy trial have significantly increased our understanding of the molecular basis of the important side effects seen in this trial," says David Williams, MD, director of Experimental Hematology at Cincinnati Children's. "He has been asked by regulatory agencies in both the United States and in Europe for expert testimony regarding these events. Dr. Kalle's recognition is timely and thoroughly warranted. We are all very proud of his work and thrilled that he has joined us at Cincinnati Children's."
Contact Information
Jim Feuer, 513-636-4656,
jim.feuer@cchmc.org
