Expanded Access and Related Treatment Options for Brain Tumors

The following information summarizes selected treatment programs and related clinical trials for brain tumors.

Last updated: 5/13/26

Important: This page is for informational purposes only and is not medical advice. Availability may change. Patients should discuss these options with their treating physician, who can contact the program sponsor or listed contact for details.

AVM0703

ClinicalTrials.gov ID:
NCT05974410

Indication:
Recurrent glioblastoma.

Mechanism of Action:
Corticosteroid derivative that modulates the immune system, potentially enhancing anti-tumor immune responses.

Notes: No recent data available for glioblastoma.

Chemo-immunotherapy Using Ibrutinib Plus Indoximod

ClinicalTrials.gov ID:
NCT05106296

Indication:
Pediatric brain cancer, including glioblastoma, DIPG, medulloblastoma, and ependymoma.

Mechanism of Action:
Combining ibrutinib and indoximod with chemotherapy to block the BTK and IDO pathways that tumors use to alter immune cell metabolism and suppress T-cell activity, with the goal of restoring a stronger anti-tumor immune response.

Notes: Program is only open to patients previously treated with indoximod-based therapy, including prior treatment via the phase 2 indoximod study GCC1949 / NCT04049669, the now closed phase 1 study NLG2105 / NCT02502708, or any expanded access protocols.

DOC1021

ClinicalTrials.gov ID:
Not available for expanded access program.

Indication:
Newly diagnosed and recurrent glioblastoma.

Mechanism of Action:
Dendritic cell vaccine loaded with autologous tumor mRNA and lysate, administered near tumor-draining lymph nodes.

Related Trial:
NCT06805305 Phase 2 for newly diagnosed glioblastoma.

Notes: Diakonos Oncology, the company developing DOC1021, is not currently offering an expanded access program in the United States. A different company, Immunocine, offers a similar vaccine in Mexico. Patients must pay privately.

Gallium Maltolate (GaM)

ClinicalTrials.gov ID:
NCT06404034

Indication:
Recurrent glioblastoma.

Mechanism of Action:
Orally bioavailable gallium compound that disrupts iron metabolism in tumor cells, leading to inhibition of DNA synthesis and tumor growth.

Related Trial:
NCT04319276 Phase 1 in relapsed glioblastoma.

Notes: For patients with relapsed or refractory histologic or molecular glioblastoma who have exhausted available treatments. There is a cost to the patient for this program, and the Musella Foundation may be able to offer financial assistance to help with the cost.
The program remains active for currently enrolled patients. Further limited capacity may be available at select sites.

NanO2 (Dodecafluoropentane Emulsion)

ClinicalTrials.gov ID:
Not available.

Indication:
Newly diagnosed glioblastoma.

Mechanism of Action:
IV-infused therapy that helps oxygen delivery to hypoxic tumor regions, with the goal of enhancing the effectiveness of radiation and chemotherapy.

Notes: Single-patient expanded access may be available on a case-by-case basis. For more information, a patient’s treating physician may contact Nuvox Therapeutics.

RZ-001

ClinicalTrials.gov ID:
NCT06676891

Indication:
Recurrent glioblastoma.

Mechanism of Action:
Recombinant adenovirus carrying a modified ribozyme construct with the HSV-tk gene, targeting hTERT-positive glioblastoma cells.

Notes: Small patient population. Contact Rznomics for availability.

Ulixertinib (BVD-523)

ClinicalTrials.gov ID:
NCT04566393

Indication:
Advanced solid tumors with MAPK pathway alterations, including glioblastoma.

Mechanism of Action:
ERK1/2 inhibitor targeting the MAPK signaling pathway.

Notes: Open to patients 12 years and older.


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