Date of Award
Spring 3-24-2023
Document Type
Open Access Thesis
Degree Name
Master of Medical Science (MMSc)
First Advisor
Zachary Corbin, MD, MHS
Abstract
Glioblastoma is the most common aggressive primary brain tumor in adults, and there is no current standard of care for progression. The Warburg effect is a known part of cancer’s metabolic reprogramming and has been studied as a possible target for limiting tumor growth. Dichloroacetate, a pyruvate dehydrogenase kinase inhibitor, has been proposed as a treatment for glioblastoma, and the drug has had success in phase 1 trials. The proposed study is an open-label, randomized phase 2 clinical trial. Participants will be randomized to oral dichloroacetate, with dosing based on genetic testing, or to a control group treated with lomustine. We propose that treating patients with recurrent glioblastoma with dichloroacetate will result in a statistically significant increase in survival when compared to patients treated with lomustine. In addition, this study will constitute an advance toward the metabolic targeting of the Warburg effect as a treatment for glioblastoma.
Recommended Citation
Macia, Megan, "Dichloroacetate as a Metabolic Treatment for Glioblastoma" (2023). Yale School of Medicine Physician Associate Program Theses. 153.
https://elischolar.library.yale.edu/ysmpa_theses/153
