Research

City of Hope is a hub of innovation and progress when it comes to treating brain tumors. Our research and clinical teams work together to advance therapies that more effectively — and directly — target brain tumors. A groundbreaking case study conducted at City of Hope, and published in the New England Journal of Medicine, found that using CAR T cell therapy, a type of immunotherapy, can be effective in the treatment of glioblastoma. And we are researching immunotherapy as a way of disabling cancer cells and finding unique ways to allow cancer drugs to cross the blood-brain barrier.

We are world leaders in the field of neural stem cell therapy, a groundbreaking method of delivering chemotherapy drugs directly to tumor sites within the brain — and were the first in the world to apply this exciting breakthrough therapy to brain cancer.

Brain tumor clinical trials and research

The key to finding better treatments for aggressive disease is the constant collaboration between researchers, clinicians and oncologists — who move therapies swiftly from our on-campus research labs to patients. The quality and frequency of this interaction allows us to get potentially life-extending therapies to our patients quickly.

For more information about the brain tumor studies listed below, including eligibility criteria, please call: 844-333-HOPE (4673). For a summary of these studies visit the City of Hope clinical trials website.

Clinical trials for recurrent disease

IRB #13384: Phase I Study of Cellular Immunotherapy Using Central Memory Enriched T Cells Lentivirally Transduced to Express an IL13Rα2-Specific, Hinge-Optimized, 41BB-Costimulatory Chimeric Antigen Receptor and a Truncated CD19 for Patients with Recurrent/Refractory Malignant Glioma

In this ‘first in human’ clinical study, we are evaluating the safety and feasibility of reprogramming a patient’s own immune system to target malignant glioma. For this study:

  • White blood cells known as T cells are isolated and engineered to express a protein of novel design called a chimeric antigen receptor (CAR).
  • This CAR instructs the T cells to recognize and kill target cells that express IL13Rα2, a receptor readily detected on the majority of malignant gliomas, but not on normal brain tissue.
  • At the time of surgery, a catheter system will be placed for local delivery of the reprogrammed T cells at the tumor site. 
  • Following recovery from surgery and cell manufacture, research participants will receive weekly injections of the CAR T cells for three weeks, with an option for an additional three week treatment course.

If you are interested in learning more about this clinical trial, or in referring a patient for enrollment, please call 844-333-HOPE (4673) or email [email protected]. For a summary of this study including the full eligibility criteria, visit City of Hope’s clinical trials website at http://clinicaltrials.coh.org and enter “13384” in the keyword search.


IRB# 13401: A Phase I Study of Cytosine Deaminase-Expressing Neural Stem Cells with Oral 5-Fluorocytosine and Leucovorin for Treatment of Recurrent High-Grade Gliomas

Neural Stem Cells (NSCs) 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.

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  • During removal or biopsy of tumor, research participants will receive local injections of genetically-modified 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 two weeks, followed each time by seven day courses of oral 5-FC (and possibly leucovorin).

If you are interested in learning more about this clinical trial or in referring a patient for enrollment, please call 844-333-HOPE (4673) or email at [email protected]. For a summary of this study including the full eligibility criteria, visit City of Hope’s clinical trials website at http://clinicaltrials.coh.org and enter “13401” in the keyword search.

Clinical trials for newly diagnosed patients

IRB #16062: Phase III Randomized, Double-blind, Controlled Study of ICT-107 in Glioblastoma

ICT-107 consists of dendritic cells, prepared from autologous mononuclear cells that are pulsed with six synthetic peptides that were derived from tumor associated antigens (TAA) present on glioblastoma tumor cells. This is a phase III study to evaluate ICT-107 in patients with newly diagnosed glioblastoma. Subjects will be randomized to receive standard of care chemoradiation (temozolomide (TMZ) with either ICT-107 or a blinded control. Reinfusion with the pulsed dendritic cells should stimulate cytotoxic T cells to specifically target glioblastoma tumor cells.

  • Patients in arm 1 will receive ICT-107 in combination with the standard of care, temozolomide (TMZ).
  • ICT-107 will be given once a week for four weeks in the induction phase.
  • During the maintenance phase, ICT-107 will be given monthly for the 11 months after induction and once every six months thereafter until depletion of supply or confirmation of progressive disease.
  • Patients in arm 2 will receive TMZ with a blinded control.
  • Control will be given once a week for four weeks in the induction phase.
  • During the maintenance phase, Control will be given monthly for the 11 months after induction and once every six months thereafter until depletion of supply or confirmation of progressive disease.


Clinical trials for patients with metastatic disease

IRB #14312: An Intracerebral Microdialysis Study to Determine the Neuropharmacokinetics of Eribulin in Patients with Brain Tumors

Despite the use of surgery, radiation and chemotherapy, malignant brain tumors are very difficult to treat successfully. One reason that chemotherapy drugs might not be effective is that the drugs may not be able to get into the brain tumor and kill the cancer cells. Eribulin is approved for use by the Food and Drug Administration to treat advanced breast cancer. However, it is experimental in the treatment of brain metastases. This study will see if doses in the human brain can also reach levels high enough to be effective. 

  • In this study, the neurosurgeon will place up to two microdialysis catheters in the brain tumor (if only a biopsy is being done) or in the nearby brain tissue. The catheter (which is about the size of a piece of spaghetti) is smaller than the standard needle used to do brain biopsies. 
  • Beginning at least 24 hours later, patients receive eribulin mesylate intravenously (IV) over two to five minutes on day 1.
  • Brain fluid samples are collected for approximately 72 hours and the microdialysis catheter is then removed.
  • Beginning at least two weeks after tumor resection or biopsy, patients may continue to receive eribulin mesylate IV over two to five minutes on days 1 and 8. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity.

Another objective of this study is to evaluate the safety and effectiveness of eribulin in participants who choose to continue to receive the drug after the microdialysis portion of the study is complete. Your treatment on this part of the study will last for as long as your tumor is not growing and you are not having any unmanageable side effects. However, if you were previously treated with eribulin, you are eligible to participate in the microdialysis portion of this study only.

After your participation in the study ends, your condition will be followed for an additional 30 days or until all side effects have resolved, whichever is longer. About six to eight people will take part in this study.


IRB #15331: A Phase 2 Study of Abemaciclib in Patients with Brain Metastases Secondary to Hormone Receptor Positive Breast Cancer, Non-small Cell Lung Cancer, or Melanoma

This study is being done to see how safe an investigational drug is and how well it will work to help people with cancer that has spread to the brain due to certain types of breast cancer, lung cancer and melanoma. The main reason for you to take part in this study is to help in answering the following research question: Whether abemaciclib (study drug) can help patients with breast cancer, nonsmall cell lung cancer, or melanoma whose disease has spread to the brain.