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New Clinical Study for Recurrent Glioblastoma Being Conducted at City of
Neural Stem Cell Platform: First-In-Human Trials
A landmark pilot study using neural stem cells (NSCs) to treat gliomas will start testing in patients. This first-in-oncology trial utilizes NSCs that have the natural ability to rapidly home to malignancies, migrating to and distributing themselves within tumors as well as tracking to other tumor sites within the brain.
This Pilot Feasibility Study of Oral 5-Fluorocytosine and Genetically-modified Neural Stem Cells Expressing E.Coli Cytosine Deaminase for Treatment of Recurrent High-grade Gliomas is currently enrolling patients over the age of 18 with recurrent high-grade glioma. During resection of tumor, research participants will receive local injections of genetically-modified NSCs. These stem cells 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 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 selectively kill rapidly dividing tumor cells, but leaving non-dividing healthy cells alone. This strategy is likely to have a large “bystander effect,” meaning that one NSC can kill off many surrounding tumor cells while minimizing toxicity to healthy tissues.
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 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 contact Greta Manila, N.P., at 626-471-9393 or via e-mail at neurosurgery@coh.org. For a summary of this study including the full eligibility criteria, visit City of