Prof. H Lee Sweeney

International Awards

Hamdan Award for Medical Research Excellence - Molecular Therapy in Drug Targeting (Pharmacogenomics)
Dr H Lee Sweeney PhD is a William Maul Measey Professor and Chair, Department of Physiology of the University Of Pennsylvania School Of Medicine in Philadelphia of the United States of America. His other School of Medicine affiliations are Pennsylvania Muscle Institute, Department of Medicine (Division of Cardiology), Cell and Molecular Biology Graduate Program. He has obtained the following degrees: S.B., Massachusetts Institute of Technology, 1975, A.M., Harvard University, 1980, Ph.D., Harvard University, 1984.
His Honors have been as William Maul Measey Chair in Physiology, Fellow of the American Heart Association. His Professional Affiliations are with Biophysical Society, American Heart Association (Basic Research Council), American Society for Biochemistry & Molecular Biology, Society of General Physiologists, American Society for Cell Biology. His Research Interests are in molecular motors; muscle injury and disease; gene transfer into striated muscle; myofibrillogenesis.
Dr Sweeney has spent a lifetime of research in the study of genetics of X-linked degenerative disorders of muscles that result from lack of the protein dystrophin. A mouse model of Duchenne's muscular dystrophy responds to therapy with aminoglycoside antibiotics such as gentamicin that results in the expression of dystrophin in the muscles of the mouse model.
Sweeney and his colleagues were able to demonstrate the presence of dystrophin in the muscle of the mouse model. Subsequently, in collaboration with a biotechnology company (PTC Therapeutics), Sweeney selected a candidate compound PTC124 that bore no structural similarity to aminoglycoside antibiotics but nevertheless was capable of recovering striated muscle function in the Duchenne I s muscular dystrophy mouse model. Moreover, the candidate compound PTC 124 was not associated with the ototoxicity or nephrotoxicity associated with aminoglycoside antibiotics. In the words of one reviewer, "this work represents an exciting and innovative example of the translation of basic science observations for clinical therapeutic application."
In the course of his work, Sweeney has established an important paradigm for scientific development of molecular therapeutics. First, the underlying genetic abnormality has been identified. Second, the protein deficiency or abnormality associated with the genetic abnormality has been identified. Third, an intervention has been shown to modify the genetic abnormality and to correct the protein deficiency/disorder. Finally. a candidate or family of candidates has been developed that addresses the need to optimise the therapeutic intervention while minimising adverse effects. Presumably candidates for commercial development will be explored from all the usual perspectives, e.g. suitability for oral dosing, pharmacokinetics enabling simple dosing regimens, metabolism, distribution, excretion, potential for drug-interactions and toxicology. 
The work of Dr Sweeney is noteworthy in that he has been involved in each of the major steps. Although his involvement as the major player in each component of the process can be debated, it is clear that his detailed knowledge of the muscular dystrophies and their genetics, his recognition of the non-specific aminoglycoside therapeutic intervention with its limitations and his partnership with the biotechnology company that had the ability to generate other candidate therapies placed him at the forefront of this field. In other word, his "prepared mind" enabled the discovery and progress that he has demonstrated.
While Dr. Sweeney's candidacy would be further strengthened by the development of PTC124 to a drug that has achieved formal approval of the US Food and Drug Administration and other international regulatory bodies, his pathway of discovery of PTC124 as a candidate for treating muscular dystrophy is certainly worthy of recognition at this time.
Therefore, Professor H. Lee Sweeney is truly worthy being awarded for his outstanding contributions innovator in Pharmacogenomics, the Hamdan Award for Medical Research Excellence for the term 2007-2008. 
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