40 Participants Needed

DCA for Glioblastoma

Recruiting at 4 trial locations
BU
PW
TF
Overseen ByTom Franklin, BA
Age: 18+
Sex: Any
Trial Phase: Phase 2
Sponsor: University of Florida
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Prior Safety DataThis treatment has passed at least one previous human trial
Approved in 1 JurisdictionThis treatment is already approved in other countries

Trial Summary

Will I have to stop taking my current medications?

You can keep taking your current medications, except if you need insulin or sulfonylurea for diabetes. If you use these, you cannot join the trial.

What data supports the effectiveness of the drug DCA for treating glioblastoma?

Research shows that DCA can penetrate the blood-brain barrier and has potential antitumor effects on brain tumors, including gliomas, by inhibiting cell growth and inducing cell death. Additionally, DCA has shown effectiveness in stabilizing other types of cancer, such as melanoma, suggesting it may help manage tumor growth.12345

Is DCA generally safe for humans?

DCA has been studied for safety in humans, showing some dose-related, reversible side effects like nerve damage (neurotoxicity) when taken orally. However, high-dose intravenous DCA has been confirmed as safe in both healthy and critically ill patients, with no systemic toxic effects observed in animal studies.12467

What makes the drug DCA unique for treating glioblastoma?

DCA is unique because it can cross the blood-brain barrier and targets mitochondria in cancer cells, helping to stop tumor growth and induce cancer cell death. This is different from many other treatments that may not effectively reach brain tumors or work through this specific mechanism.12489

What is the purpose of this trial?

This trial tests if DCA, a medication taken by mouth, can help treat patients with returning brain tumors who are scheduled for surgery. DCA may change how tumor cells use energy, potentially slowing their growth. DCA has shown potential activity against several human cancers, including brain tumors.

Research Team

PS

Peter Stacpoole, PhD, MD

Principal Investigator

University of Florida

Eligibility Criteria

Adults aged 18-80 with recurrent Glioblastoma Multiforme (GBM) who have already undergone surgery, radiation, and chemotherapy with temozolomide are eligible. They must not be pre-terminal or pregnant, nor can they have severe liver insufficiency, end-stage renal failure, or be on insulin/sulfonylurea therapy for diabetes.

Inclusion Criteria

I've had surgery, radiation, and TMZ for my cancer but it didn't work. If my GBM is unmethylated, I haven't had TMZ.
I am being treated at Johns Hopkins or Wake Forest University and can take my current medications, except insulin or sulfonylureas.
I am not at risk for drug interactions with DCA based on its unique metabolism and past trial data.
See 1 more

Exclusion Criteria

Patients considered pre-terminal (life expectancy ≤ 2 months)
Patients with Hgb A1c level less than 6.0 at screening
Those who are pregnant will be excluded
See 3 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Pre-Surgical Treatment

Participants receive DCA or no DCA for one week prior to surgery

1 week

Surgery

Participants undergo clinically indicated debulking surgery

1 day

Post-Surgical Treatment

Participants start DCA 12-24 hours postoperatively, depending on their ability to safely receive medication

Variable

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

  • Dichloroacetate (DCA)
Trial Overview The trial is testing oral Dichloroacetate (DCA) in patients with GBM to establish safe dosing based on genotyping. Participants will either receive DCA or no treatment for one week before their clinically indicated tumor removal surgery.
Participant Groups
2Treatment groups
Active Control
Group I: No Pre-Surgical Dichloroacetate (DCA)Active Control2 Interventions
Subject randomized to start DCA after surgery will do so 12-24 hours postoperatively, depending on their ability to safely receive medication.
Group II: Pre-Surgical Dichloroacetate (DCA)Active Control2 Interventions
Study medication begins in subjects randomized to preoperative DCA. All subjects will be given the 12.5 mg/kg/12 hour DCA for pre-surgical dosing. Post-surgery the GSTZ1 haplotype will be utilized to dose all patients.

Dichloroacetate (DCA) is already approved in Canada for the following indications:

🇨🇦
Approved in Canada as Dichloroacetic acid for:
  • Topical treatment of warts
  • Cauterization and removal of skin and tissue lesions

Find a Clinic Near You

Who Is Running the Clinical Trial?

University of Florida

Lead Sponsor

Trials
1,428
Recruited
987,000+

Food and Drug Administration (FDA)

Collaborator

Trials
184
Recruited
1,553,000+

Findings from Research

Dichloroacetate (DCA) effectively inhibits the growth of C6 glioma cells by inducing apoptosis and arresting the cells in the S phase, demonstrating its potential as a treatment for brain tumors.
In animal models, DCA significantly reduced tumor growth in C6 glioma-bearing rats and mice, while also increasing reactive oxygen species (ROS) production and exhibiting antiangiogenic effects, suggesting a multifaceted mechanism of action against gliomas.
Antitumor activity of dichloroacetate on C6 glioma cell: in vitro and in vivo evaluation.Duan, Y., Zhao, X., Ren, W., et al.[2021]
Sodium dichloroacetate (DCA) effectively reduces the viability of various leukemia cell lines, with sensitivity varying by cell type; CEM/C1 cells were most sensitive (IC50 of 30 mM) while HL-60/MX2 cells were most resistant (IC50 of 75 mM).
DCA treatment alters gene expression profiles related to apoptosis in leukemia cells, but does not activate a specific apoptosis pathway, indicating that further research is needed to understand the molecular mechanisms behind DCA's anticancer effects.
Effect of sodium dichloroacetate on apoptotic gene expression in human leukemia cell lines.Abramek, J., Bogucki, J., Ziaja-Sołtys, M., et al.[2020]
Dichloroacetate (DCA) selectively induced apoptosis in pediatric tumor cells but only moderately inhibited growth across 18 different tumor cell lines, suggesting limited efficacy as a standalone treatment.
DCA was found to reduce the effectiveness of cisplatin and doxorubicin in several cell lines, indicating that its use in combination with standard chemotherapy drugs may require careful evaluation to avoid compromising treatment outcomes.
Dichloroacetate metabolically targeted therapy defeats cytotoxicity of standard anticancer drugs.Heshe, D., Hoogestraat, S., Brauckmann, C., et al.[2022]

References

Antitumor activity of dichloroacetate on C6 glioma cell: in vitro and in vivo evaluation. [2021]
Effect of sodium dichloroacetate on apoptotic gene expression in human leukemia cell lines. [2020]
Dichloroacetate metabolically targeted therapy defeats cytotoxicity of standard anticancer drugs. [2022]
Dichloroacetate induces autophagy in colorectal cancer cells and tumours. [2022]
Long-term stabilization of metastatic melanoma with sodium dichloroacetate. [2020]
A novel form of dichloroacetate therapy for patients with advanced cancer: a report of 3 cases. [2019]
Sodium dichloroacetate attenuates the growth of B16-F10 melanoma in vitro and in vivo: an opportunity for drug repurposing. [2021]
Use of oral dichloroacetate for palliation of leg pain arising from metastatic poorly differentiated carcinoma: a case report. [2021]
Dichloroacetate modulates cytokines toward T helper 1 function via induction of the interleukin-12-interferon-γ pathway. [2021]
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