Brain Tumor Fact Sheet

CIRM funds many projects seeking to better understand brain tumors and to translate those discoveries into new therapies.


A type of brain tumor called a high-grade glioma, or malignant brain tumor, is among the hardest tumors to treat. An estimated 17,000 Americans will die from brain tumors in 20171.

Gliomas are particularly difficult to treat because they are made up of tumor cells that spread throughout the brain, not remaining in a single area where they could be removed through surgery or targeted by radiation. Chemotherapy has not been effective at eradicating all of the glioma cells.

Stem cell approaches look promising for treating gliomas. Certain types of stem cell tend to migrate toward the tumor cells wherever they are in the brain. CIRM-funded researchers are trying to genetically engineer those stem cells to produce cancer-killing molecules. Transplanted into the brain, these cells would seek out the cancer cells and deliver their therapy directly where it is needed. This approach could significantly decrease toxic side-effects to normal tissues, preserving or improving the patient’s quality of life.

Clinical Stage Programs

City of Hope

A team at the City of Hope led by Dr. Christine Brown is pursuing a Phase 1 trial targeting an aggressive brain cancer called malignant glioma. City of Hope will re-engineer a patient’s immune system central memory T cells (TCM cells) to express chimeric antigen receptors (CAR). These CAR-T cells will recognize a molecular marker on the surface of glioma cancer stem cells and kill the tumors. Dr. Brown’s award to pursue CAR-T therapy for solid cancers comes at an exciting and opportune time with the recent U.S. Food and Drug Administration (FDA) approval of the first CAR-T therapy, called Kymriah, for patients with acute lymphoblastic leukemia, a deadly form of blood cancer.

City of Hope

This clinical trial is testing a therapy to treat brain metastases that came from breast cancers expressing high levels of a protein called HER2.   The therapy consists of a genetically-modified version of the patient’s own T cells, which are an immune system cell that can destroy foreign or abnormal cells.  The T cells are modified with a protein called a chimeric antigen receptor (CAR) that recognizes the tumor protein HER2.  These modified T cells (CAR-T cells) are then infused into the patient’s brain where they are expected to detect and destroy the HER2-expressing tumors in the brain.

Immunocellular Therapeutics

This trial is targeting six proteins that are found on the surface of cancer stem cells in glioblastoma, a brain cancer. Immune cells from the patient’s own immune system are exposed to fragments of these cancer cell proteins in the lab. When returned to the patient’s body, these immune system cells can now help the patient’s immune system identify, and then hopefully kill, the cancer stem cells responsible for the tumor’s recurrence and growth. This Phase 3 trial was suspended in June 2017 due to lack of sufficient financial resources.

CIRM Grants Targeting Brain Tumors

Researcher NameInstitutionGrant TitleGrant TypeAward Amount
Frank PajonkUniversity of California, Los AngelesDevelopment of novel small molecules against cancer stem cells in solid cancersQuest – Discovery Stage Research Projects$2,340,000
Munjal M AcharyaUniversity of California, IrvineStem cell-derived extracellular vesicles to reverse radiation-induced brain injuryQuest – Discovery Stage Research Projects$1,064,724
Dr. Hideho OkadaUniversity of California, San FranciscoNon-viral reprogramming of the endogenous TCRα locus to direct stem memory T cells against shared neoantigens in malignant gliomasQuest – Discovery Stage Research Projects$900,000
Dr. Nicole K PaulkSiren Biotechnology, Inc.Development of an AAV gene therapy immunotherapy for the treatment of glioblastomaTherapeutic Translational Research Projects$3,997,919
Dr. Hideho OkadaUniversity of California, San FranciscoDevelopment of novel synNotch CART cell therapy in patients with recurrent EGFRvIII+ glioblastomaTherapeutic Translational Research Projects$4,556,536
Dr. Hideho OkadaUniversity of California, San FranciscoDevelopment of novel synNotch CART cell therapy in patients with recurrent EGFRvIII+ glioblastomaTherapeutic Translational Research Projects$525,000
Dr. Albert J WongStanford University2nd Generation Vaccine for the Treatment of GlioblastomaTherapeutic Translational Research Projects$2,929,889
Dr. Hideho OkadaUniversity of California, San FranciscoPhase 1 Study of Autologous E-SYNC T Cells in Adult Participants with EGFRvIII+ GlioblastomaClinical Trial Stage Projects$10,927,618
Dr. Reena Parada ThomasStanford UniversityPhase I Trial of Locoregionally Delivered Autologous B7-H3 CAR T Cells (B7-H3CART) in Adults with Recurrent Glioblastoma MultiformeClinical Trial Stage Projects$11,999,991
Dr Jana PortnowCity of Hope, Beckman Research InstituteA Phase I Study of Multiple doses of NSC-Based Oncolytic Virotherapy Administered Intracerebrally to Patients with Recurrent High-Grade GliomasClinical Trial Stage Projects$11,999,984
Crystal MackallStanford UniversityPhase 1 Clinical Trial of Autologous GD2 Chimeric Antigen Receptor T Cells for Diffuse Intrinsic Pontine Gliomas and Spinal Diffuse Midline GliomaClinical Trial Stage Projects$11,998,310
Dr. Leo D WangCity of Hope, Beckman Research InstitutePhase I Study of IL13Rα2-Targeting CAR T Cells After Lymphodepletion for Children with Refractory or Recurrent Malignant Brain TumorsClinical Trial Stage Projects$8,401,309
Dr Saul J PricemanCity of Hope, Beckman Research InstituteA Phase I Study of Chimeric Antigen Receptor Engineered Stem/Memory T Cells for the Treatment of HER2-Positive Brain MetastasesClinical Trial Stage Projects$9,015,149
Dr. Christine E. BrownCity of Hope, Beckman Research InstitutePhase I Study of Chimeric Antigen Receptor Engineered Central Memory T cells for the Treatment of Malignant GliomaClinical Trial Stage Projects$12,753,854
Anthony J. GringeriImmunoCellular TherapeuticsA Phase III randomized double-blind, controlled study of ICT 107 with maintenance temozolomide (TMZ) in newly diagnosed glioblastoma following resection and concomitant TMZ chemoradiotherapyClinical Trial Stage Projects$5,391,016
Dr. Angelo Manuel Almeida Cardoso Ph.D., MDCity of Hope, Beckman Research InstituteEx Vivo Gene Engineering of Blood Stem Cells for Enhanced Chemotherapy Efficacy in Glioblastoma PatientsLate Stage Preclinical Projects$3,684,259
Dr. Michael E. Barish Ph, D.City of Hope, Beckman Research InstituteGenetically-modified neural stem cells for treatment of high-grade gliomaDisease Team Planning$55,000
Michelle MonjeStanford UniversityWhite matter neuroregeneration after chemotherapy: stem cell therapy for “chemobrain”New Faculty Physician Scientist$2,800,526
Dr. Stephen J FormanCity of Hope, Beckman Research InstituteTargeting glioma cancer stem cells with receptor-engineered self-renewing memory T cellsEarly Translational III$5,215,447
Dr. Albert J WongStanford UniversityRecombinant Bispecific Antibody Targeting Cancer Stem Cells for the Therapy of GlioblastomaDisease Team Therapy Planning I$109,750
Dr. Noriyuki KasaharaUniversity of California, Los AngelesStem cell-based carriers for RCR vector delivery to glioblastomaEarly Translational II$3,340,625
Dr. Robert J Wechsler-ReyaSanford Burnham Prebys Medical Discovery InstituteThe role of neural stem cells in cerebellar development, regeneration and tumorigenesisResearch Leadership$5,226,049
Dr. Mitchel S BergerUniversity of California, San FranciscoStem Cell-Mediated Oncocidal Gene Therapy of Glioblastoma (GBM)Disease Team Research I$6,214,914
Prof. Karen S Aboody M.D.City of Hope, Beckman Research InstituteStem Cell-mediated Therapy for High-grade Glioma: Toward Phase I-II Clinical TrialsDisease Team Research I$17,890,623

CIRM Brain Tumor Stem Cell Videos

Brain Tumors: Advancing Stem Cell Therapies – 2011 CIRM Grantee Meeting

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