Evidence Explorer / MS-N218 / Investigational metabolic therapies / Appraised November 2, 2026
Dichloroacetate (DCA) has a plausible metabolic laboratory rationale, but human cancer evidence is early. In a phase I study of advanced solid tumors, no objective responses were observed and toxicity—including neuropathy—limited dosing.
Evidence at a glance
- Population: 24 adults with advanced solid tumors enrolled; 23 were evaluable.
- Design: open-label, single-arm 3+3 phase I dose escalation.
- Regimen studied: oral DCA 6.25 or 12.5 mg/kg twice daily in 28-day cycles.
- Results: no objective responses; eight participants had stable disease. Three of seven at 12.5 mg/kg twice daily had dose-limiting fatigue, vomiting or diarrhea.
- Evidence label: investigational phase I safety evidence; no established cancer-control benefit.
What was studied
The trial’s purpose was safety, pharmacokinetics and selection of a recommended phase II dose—not proof of efficacy. The recommended dose for future study was 6.25 mg/kg twice daily.
Common toxicities were fatigue, peripheral neuropathy and nausea. Drug levels and neuropathy varied substantially between participants. A trend in FDG-PET uptake was exploratory and cannot substitute for objective tumor response.
What this evidence cannot establish
Stable disease in a small uncontrolled phase I cohort cannot show that DCA delayed progression; advanced cancers can remain stable for a time because of natural history or prior treatment.
The study does not establish survival benefit, an effective dose, long-term neurologic safety or a role outside research. Preclinical metabolic effects and imaging biomarkers do not prove cancer control.
Practical safety context
Peripheral neuropathy may be painful, disabling or difficult to distinguish from chemotherapy toxicity. Higher-dose participants also experienced dose-limiting gastrointestinal symptoms and fatigue.
Online or industrial DCA may not meet pharmaceutical standards. Self-treatment can obscure toxicity, interact with care and delay a clinical evaluation of progression.
Questions for the care team
- Is access through a registered clinical trial with pharmaceutical-grade product?
- What baseline neuropathy, liver function and medication interactions need review?
- What objective endpoint would show benefit, and when would treatment stop?
Sources
- Phase I advanced-solid-tumor study — PMID 25762000
- Journal article — DOI 10.1007/s10637-015-0221-y
Source note: The PubMed abstract and bibliographic record were checked; full-text appraisal was not available. The abstract reports no responses and the stated toxicities, but detailed participant-level outcomes, funding and conflicts remain not verified. No correction, retraction or expression of concern was identified on November 2, 2026.
This record is educational. DCA remains investigational for cancer and should not be self-prescribed or substituted for evidence-based treatment.
