Expanded Access Programs for Brain Tumors

Last updated 5/8/25

Expanded access, also known as compassionate use, allows patients with serious or life-threatening conditions to access investigational treatments outside of clinical trials. These programs are typically for patients who have exhausted all available standard therapies and are not eligible for ongoing clinical trials. Expanded access programs provide an opportunity for patients to receive potentially life-saving treatments while also contributing to the collection of valuable data for future research.

Below is a list of expanded access programs available for patients with brain tumors. These programs provide access to investigational treatments for those who may not qualify for clinical trials or have exhausted available standard treatments.

Expanded Access Programs for Glioblastoma (GBM)

• Gallium Maltolate (GaM)
  ClinicalTrials.gov ID: NCT06404034
  Indication: Recurrent GBM
  Mechanism of Action: Orally bioavailable gallium compound that disrupts iron metabolism in tumor cells, leading to inhibition of DNA synthesis and tumor growth.
  Notes: For patients with relapsed/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!
  Related trial: NCT04319276 (Phase 1 in relapsed GBM)
• RZ-001
  ClinicalTrials.gov ID: NCT06676891
  Indication: Recurrent GBM
  Mechanism of Action: Recombinant adenovirus carrying a modified ribozyme construct with the HSV-tk gene, targeting hTERT-positive GBM 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 GBM
  Mechanism of Action: ERK1/2 inhibitor targeting the MAPK signaling pathway.
  Notes: Open to patients 12 years and older.
• AVM0703
  ClinicalTrials.gov ID: NCT05974410
  Indication: Recurrent GBM
  Mechanism of Action: Corticosteroid derivative that modulates the immune system, potentially enhancing anti-tumor immune responses.
  Notes: No recent data available for GBM.
• DOC1021
  ClinicalTrials.gov ID: NCT06805305
  Indication: Recurrent GBM
  Mechanism of Action: Dendritic cell vaccine loaded with autologous tumor mRNA and lysate, administered near tumor-draining lymph nodes.
  Notes: Contact clinical@diakonosoncology.com for evaluation.

Expanded Access Programs for Diffuse Midline Glioma (DMG)

• ONC201 (Dordaviprone)
  ClinicalTrials.gov ID: NCT04617002
  Indication: Recurrent DMG with H3 K27M mutation
  Mechanism of Action: ClpP agonist and DRD2 antagonist that induces apoptosis in tumor cells.
  Notes: Effective against H3 K27M-mutant gliomas.
• OKN-007
  ClinicalTrials.gov ID: NCT05518838
  Indication: Recurrent DMG
  Mechanism of Action: Small molecule that inhibits tumor growth, angiogenesis, and reduces inflammation.
  Notes: Currently listed as "No Longer Available."