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Brain Tumor Program

Tackling one of the toughest disease sites to unlock the promise of cell and gene therapy in solid tumors

Why This Research

Brain tumors are one of the toughest diseases to treat across all cancer types. It’s difficult for therapies to get past the brain’s natural protections to find and penetrate tumors. The brain’s microenvironment makes it tough for the immune system to do its job. And brain tumors have a wide variety of mutations, so one targeted therapy can’t do it alone. Because of this, brain tumor patients have not yet benefited from immunotherapy the way patients with other types of cancers have.

PICI exists to tackle the toughest problems in cancer research. Our unique collaboration of top researchers and partners are unlocking the benefits of immunotherapy and cell and gene therapy for a group of patients who desperately need better options. The challenges of brain tumors also apply broadly to solid tumors, so success in this effort will also have an impact across the entire field of cancer immunotherapy.

What We’re Doing

PICI’s brain tumor program will launch with two projects. The first is designed to identify new targets for the treatment of pediatric and adult aggressive brain tumors with immunotherapy. It is a collaboration among PICI and researchers at City of Hope, Children’s Hospital of Philadelphia, Stanford, UCLA and UCSF, and it will be conducted in partnership with the Cancer Research Institute and the Alliance for Cancer Gene Therapy.

The second project is a clinical trial focused on Diffuse Midline Gliomas (DMG), primarily pediatric tumors that currently have no effective treatment options. The trial is a proof of concept, first-in-human clinical study for a novel cell therapy approach. It is based on research and discovery led by UCSF’s Hideho Okada, MD, PhD, who will continue to be a key leader in this effort.

Taken together, these two projects address the major challenges presented by all solid tumors, and the findings could forge a path to a clinical trial that not only brings new options for brain tumor patients, but also unlocks new immunotherapies for other cancer types as well.


Meet the scientists and clinicians leading the charge:


  • Mike Angelo, MD, PhD | Stanford University School of Medicine
  • Sean Bendall, PhD | Stanford University School of Medicine
  • Christine Brown, PhD | City of Hope National Medical Center
  • Kristina Cole, MD, PhD | Children’s Hospital of Philadelphia
  • Jonathan Esensten, MD, PhD | University of California, San Francisco
  • Crystal Mackall, MD, PhD | Stanford University School of Medicine
  • Sabine Mueller, MD | University of California, San Francisco
  • Hideho Okada, MD, PhD | University of California, San Francisco
  • Robert Prins, PhD | University of California, Los Angeles

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