Study of the Safety and Efficacy of Dichloroacetate in Glioblastoma and Other Recurrent Brain Tumours

Study of the Safety and Efficacy of Dichloroacetate in Glioblastoma and Other Recurrent Brain Tumours

Phase I, Open-Label, Single-Arm, Clinical and Metabolomics Study of Dichloroacetate (DCA) in Adults With Recurrent Malignant Brain Tumours.

Cancer types
  • Brain cancer
Trial phase


ACF donation
Estimated trial cost

Why this trial?

Recurrent malignant brain tumours (RMBTs) are defined as either: 1) malignant tumours originating in the brain (World Health Organization grade III-IV glioma) that have recurred at least once or 2) malignant tumours originating elsewhere in the body that have spread to the brain at least once.

RMBTs share an increasing incidence, clinical and radiographic characteristics, pathobiology, scarcity of effective therapies and fatal outcome. Thus, the international oncology community considers phase I/II trials involving RMBTs scientifically valid and efficient for investigating promising agents.

Amongst the general public, DCA is widely promoted and used as cancer treatment, although there is insufficient proof of its safety and efficacy.  By supporting this trial, we want to add to the scientific information available, so that an informed decision can be made regarding the use of DCA for its claimed anticancer effect.

Why this intervention?

RMBTs characteristics may be put to use by an emerging class of pharmacological agents called “metabolic modulators” of which dichloroacetate (DCA) is most thoroughly investigated clinically. Metabolic modulators like DCA exert their anti-tumour effects by causing fundamental changes in the manner in which tumours convert substrate fuels (such as glucose) into the energy that allows cancer cells to proliferate and metastasize. DCA has shown anti-tumour activity against brain tumours in animal and human studies.

Trial design

This phase I dose-escalation trial is the first comprehensive clinical study of DCA in adults with RMBTs.

15 patients were assigned an oral DCA dose per kilogram body weight, as used in prior DCA clinical trials. A “3+3” study design was employed, which means testing 3 subjects at a given dose and escalating to the next dose if no patients experience dose limiting toxicity (DLT).

The primary outcome was to determine the safety and tolerability of DCA in this patient population. Secondary outcomes included probative studies on the metabolism of DCA in these patients and investigations of the metabolic profile of RMBTs, and the effects of DCA thereon.


Results, published here, showed that DCA can be used safely in patients with RMBT but individualisation of the dose of DCA is required to avoid side effects.



  • Erin Dunbar, MD, University of Florida, Florida, USA (Principal Investigator)
  • Peter Stacpoole, MD, University of Florida, Florida, USA (Co-investigator)


  • University of Florida, Florida, USA

Our role

Financial support + Consortium building
Why we support this trial
Benefits a population with high unmet needs
To verify the treatments’ anticancer claim
Matching focus area


Estimated trial cost
ACF donation
Questions about this trial?
The Anticancer Fund
studies [at]


More info: NCT01111097

Author: Kristine Beckers (Trial Manager)

Last Updated: May 2019.