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Stacpoole’s federally-sponsored research is broadly focused in two areas: intermediary metabolism and new drug development, with particular emphasis on the causes and treatment of congenital and acquired mitochondrial diseases. In this regard, he collaborates with investigators at UF and across N. America with expertise in neurology, physiology, oncology, immunology, and cell and molecular biology.
Related research includes mechanistically oriented laboratory studies on the molecular and biochemical consequences of loss of function mutations in the mitochondrial pyruvate dehydrogenase complex (PDC) and therapeutic interventions for congenital and acquired causes of PDC deficiency.
With regard to new drug development, Dr. Stacpoole and his colleagues have developed a prototype for a novel class of investigational drugs for the treatment of acquired or inborn errors of mitochondrial energy metabolism and lactic acidosis. The prototype of this class, dichloroacetate (DCA), is currently undergoing two federally funded FDA-approved multicenter clinical trials: a Phase 3 trial of DCA in children with congenital defects in the PDC and a Phase 2A trial of DCA in adults with recurrent glioblastoma multiforme (GBM), the most common and deadly primary brain cancer in adults. Dr. Stacpoole and colleagues are also conducting preclinical studies in the therapeutic potential of DCA in Barth syndrome, a rare mitochondrial pediatric disease, in neonatal stress during parturition and in septic shock, a major cause of mortality in hospitalized patients.
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Peter W. Stacpoole, Kathy Dorsey
MOLECULAR GENETICS AND METABOLISMno. 3 (2023): 95-95
Biochemical pharmacology (2023): 115818-115818
FRONTIERS IN ONCOLOGY (2023): 1077461-1077461
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