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Dr. Pier Paolo Pandolfi de Rinaldis

International Awards

Hamdan Award for Medical Research Excellence - Therapy in Leukemia
1999-2000
Dr. Pier Paolo P. de Rinaldis is a member of the Human Genetics Department of the world-renowned Memorial Sloan-Kettering Cancer Center in New York. Dr Pandolfi de Rinaldis received his Medical School education in Roma and Perugia, and he started research work at a very early stage in the laboratory of Dr Pier Giuseppe Pelicci. In that laboratory he played a key role in the work that led to the cloning of the t(15.17) breakpoint characteristically associated with acute promyelocytic leukemia (APL, or AML-M3).
This work was followed by extensive characterization of the modalities of transcription of the PML gene, and by the spearheading of important clinical applications, including the development of an assay for minimal residual disease after treatment of APL.
 
Since that time Dr Pandolfi has devoted himself in a single-minded fashion to understanding the molecular basis of leukemia in order to improve its treatment. Among his achievements are: 1) A systematic in-depth analysis of the pathogenesis of acute promyelocytic leukemia by a powerful genetic approach. He has now succeeded in inactivating by homologous recombination each one of the genes concerned (PML, PLZF, NPMj; and he has produced transgenic mice with the respective fusion genes (PML/RAR?, PLZF/RAR? etc.) under the control of appropriate promoters. 2) Genetic inactivation of APL-related genes has revealed a variety of phenotypic features, some of them completely unexpected. 3) By systematic analysis of several RAR ? partners in the chromosomal translocation characteristic of PML, Dr. Pandolfi has been able to rationalize the molecular basis for several features of PML, including the response to retinoic acid. 
 
Dr Pandolfi has been able to use this information to construct an entirely original model on the pathogenesis of APL based on the notion that a multi-protein complex is involved in the transcriptional regulation of specific genes. This in turn has led him to put forward the novel concept that one might attempt to treat leukemia by specifically interfering with an abnormal transcription complex (transcription therapy). Dr Pandolfi's contribution is substantial, original, and innovative. Animal models that faithfully mimic specific types of human leukemia will make it possible for the first time to optimize clinical protocols effectively, safely and economically to the best advantage of patients with leukemia.
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