Brain Tumor Clinical Trials

City of Hope currently has many clinical trials in progress, a number of which address malignant brain tumors. Clinical trials offer patients new and promising experimental treatments not available elsewhere. In fact, nearly one in two patients at City of Hope is part of a clinical trial. These trials evaluate the safety and efficacy of prospective therapies. Participants in clinical trials receive excellent care and are closely monitored. We encourage all brain tumor patients to participate in clinical trials since that will enable us to find better treatments for brain tumors.
For more information about the studies listed below including eligibility criteria, please call: 626-471-9393. For a summary of these studies including eligibility criteria, visit the City of Hope clinical trials website.
Newly Diagnosed
A Phase III Clinical Trial Evaluating DCVax®-Brain, Autologous Dendritic Cells Pulsed with Tumor Lysate Antigen for the Treatment of Glioblastoma
Recurrent Disease
A Phase I Study of Cytosine Deaminase-Expressing Neural Stem Cells with Oral 5-Fluorocytosine and Leucovorin for Treatment of Recurrent High-Grade Gliomas is currently enrolling patients over the age of 18 with recurrent grade III or IV gliomas.
Neural Stem Cells have a natural ability to home to tumor cells throughout the brain. They can be genetically-modified to produce chemotherapy at sites of tumor. Neural stem cells are being investigated as a possible treatment for brain tumors.
During removal or biopsy of tumor, research participants will receive local injections of genetically-modified neural stem cells (NSCs).  These NSCs express the activating enzyme cytosine deaminase (CD), which converts the prodrug 5-fluorocytosine (5-FC) into the chemotherapy agent 5-fluorouracil (5-FU). Research participants will then take 5-FC orally for seven days. As the 5-FC crosses into the brain, the CD-expressing NSCs (which have migrated to residual cancer sites) are expected to convert the 5-FC into 5-FU.  The 5-FU and its toxic metabolites will diffuse out of the NSC to preferentially kill rapidly dividing tumor cells. It is hoped that this strategy will have a large “bystander effect,” meaning that one NSC can kill off many surrounding tumor cells while minimizing toxicity to healthy tissues. Some study patients will also take leucovorin with 5-FC. Leucovorin is an oral medication that can help 5-FU work better against cancer cells.  A Rickham catheter, placed in the brain at the time of surgery, will be used to administer additional doses of NSCs every 2 weeks, followed each time by 7 day courses of oral 5-FC (and possibly leucovorin).
Partial Eligibility Requirements:
  • Patient has had a prior, histologically-confirmed diagnosis of a grade III or grade IV glioma (including glioblastoma, anaplastic astrocytoma, gliosarcoma, anaplastic oligodendroglioma or anaplastic oligoastrocytoma.
  • Patient is eligible for a debulking craniotomy or biopsy independent of intended treatment with genetically-modified NSCs and 5-FC.
  • Patient's high-grade glioma has recurred or progressed after chemoradiation.
If you are interested in learning more about this clinical trial or in referring a patient for enrollment, please call 626-471-9393 or email  For a summary of this study including the full eligibility criteria, visit City of Hope’s clinical trials website at and enter “13401” in the keyword search.
A Phase I Gene Therapy Trial of the Safety and Tolerability of Toca 511 in patients Recurrent High Grade Glioma
TOCA 511 & TOCA FC mechanism
This is a very exciting new experimental gene therapy treatment for high grade brain tumors. The basic concept is that a virus (Toca 511) is injected into the tumor. This virus was designed to infect only the brain tumor cells and leave the normal cells alone. When it infects a cell, it adds a gene to the cell which encodes for an enzyme that can convert an antibiotic drug (Toca FC) into a toxic chemotherapy (5-FU), selectively in the tumor. This drug (Toca FC) is given orally every few weeks, and it kills the tumor cells that have enough copies of this enzyme to convert Toca FC to 5-FU. The tumor cells that are infected but don't have enough of the enzyme act as a reservoir - they start the process over again - spreading the infection for a few more weeks, and these cycles are repeated over and over again until the entire tumor is potentially gone.
If you were diagnosed with Recurrent High Grade Glioma (HGG) (glioblastoma multiforme, anaplastic astrocytoma, anaplastic oligodendroglioma and anaplastic oligoastrocytoma) that have increased in size following treatment with surgery, radiation therapy and temozolomide. The Toca 511 and Toca FC studies might be the studies for you.
Who can participate in the Toca 511 & Toca FC studies? You may qualify for a Toca 511 & Toca FC study if you:
  • Are at least 18 years old (upper limit of 80 years in one of the studies)
  • Have recurrent HGG
Your doctor will be able to review with you these and other eligibility criteria. For more information about the Toca 511 & Toca FC studies, please contact Jana Portnow M.D. or Behnam Badie M.D. at  626-471-9393 or visit
Feasibility of Intraoperative Optical Imaging and Spectroscopy in Brain Tumors
Phase 2, Open-Label, Imaging Trial of I-124-CLR1404 in Patients with Newly Diagnosed or Recurrent Glioblastoma Multiforme. (CELLECTAR)