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    ISSN : 2328-9791
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Dichloroacetate - Phase 1 Trial in Adults with Malignant Brain Tumors
     
 
Ryan J. Lorenzo and Stephan C. Jahn
University of Florida

Department of Medicinal Chemistryand UF Health Cancer Center, UFL, Gainesville, FL  32610, USA

scjahn@ufl.edu

Dichloroacetate (DCA) has been used for many years as an investigational drug to treat children born with mitochondrial disorders. Through its inhibition of Pyruvate Dehydrogenase Kinase (PDK), a key component of the pyruvate dehydrogenase complex, DCA has shown promise in facilitating oxidative phosphorylation in cell mitochondria.  Recently, DCA has received more attention as a possible treatment for numerous forms of cancer.  In this research highlight, we will discuss a paper from Dunbar et al. in which a Phase 1 trial for DCA was run in adults with recurrent malignant brain tumors (RMBTs). These tumors often carry a poor prognosis. As DCA has the ability to pass through the blood-brain barrier, it is being investigated as a possible treatment for RMBTs. The goal of the trial was to establish dose ranges that are safe to administer to patients with recurrent malignant gliomas.

 
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