Pediatric Cancer Immunotherapy

Our advanced cellular therapies for cancer and autoimmune disorders offer children renewed hope.
Pediatric Cancer Immunotherapy

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Why choose UCSF Benioff Children's Hospitals for immunotherapy cancer treatment?

Immunotherapy is a type of cancer treatment that uses the body's immune system to fight cancer. One of the most promising forms of immunotherapy, called cellular therapy, is transforming cancer care. It can help children with relapsed or treatment-resistant cancer have better outcomes with fewer side effects.

At UCSF Benioff Children's Hospitals, we are leading the way in advanced cellular therapies, including CAR T-cell therapy. We offer these therapies in clinical trials, giving children with challenging diseases early access to breakthrough treatments before they're widely available. We're also among the few programs in Northern California offering the only FDA-approved CAR T-cell therapy for children, adolescents and young adults with leukemia and lymphoma.

Advanced cellular therapy for childhood cancers

We often use CAR T-cell therapy to treat children with B-cell acute lymphoblastic leukemia (B-ALL) when other treatments aren't effective or cancer returns after treatment. This advanced immunotherapy trains a child's immune cells to recognize and attack cancer.

During treatment, we collect T cells (a type of white blood cell) and modify them in a laboratory. Scientists add a gene that enables the cells to target a specific protein on leukemia cells. They multiply the modified T cells and return them to the patient's body, giving them a boost of cancer-fighting cells.

Once offered only through clinical trials, CAR T-cell therapy is now available for children and young adults as an approved therapy called KYMRIAH®. In most studies, about 90% of patients achieved remission after one dose. This highly specialized gene therapy is only available at select institutions, including UCSF.

New treatment options for cancers and autoimmune disorders

Researchers at UCSF are investigating how cellular therapies can treat other childhood cancers and autoimmune disorders. We offer one of the largest selections of early-phase clinical trials in Northern California. Families across the West Coast turn to us for our pioneering research and clinical expertise.

We are actively studying cellular therapies for:

  • Leukemias

  • Brain tumors

  • Liver tumors

  • Crohn's disease

  • Lupus

  • Mucopolysaccharidosis type 1

  • Type 1 diabetes

Highlights of our cancer care

  • Leading-edge treatments

    We're among the first to offer new treatment options, as soon as they're proven safe and effective. In addition, patients can access promising experimental therapies by participating in one of our many clinical trials.
  • High-risk tumors are analyzed with the UCSF500 Cancer Gene Panel, a state-of-the-art test that identifies mutations in the tumor's DNA. The results can indicate the best treatment course and, in some cases, clarify the exact type of cancer.
  • Seamless care, near and far

    Dedicated patient navigators help families with managing referrals, insurance concerns and appointment planning. We work with patients and referring doctors well beyond the San Francisco Bay Area, thanks to our comprehensive telehealth system.
  • Our Pediatric Survivorship Program provides expert care to address any medical, psychological or social issues that arise after treatment is complete. The team also ensures smooth transitions to adult care providers as our patients leave childhood behind.

Providers

Awards & achievements

Recognized nationally for excellence, we pair world-class medical expertise with compassionate care to deliver the best possible outcomes for patients.
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  • U.S. News & World Report Best Children's Hospitals 2026-2027, cancer

    Best in Northern California and top 10 in the nation for cancer care

  • Designated an early phase clinical trials core site by the Children's Oncology Group

  • Accredited by the Foundation for Accreditation of Cellular Therapy

  • U.S. News & World Report Best Children's Hospitals 2026-2027, ranked in 11 specialties

    Ranked among the nation's best in 11 specialties

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Clinical trials

A CAR-T cell success story

Meet Siya, whose leukemia didn't respond well to chemotherapy. After one month of CAR T-cell therapy, she's cancer-free.

Resources for physicians

Whether you're referring a patient, seeking a second opinion or looking for professional education opportunities, you'll find resources on the UCSF Pediatric Cancer Program's MedConnection hub. We look forward to partnering with you!

Cancer immunotherapy for adults

UCSF Health offers a wide range of personalized immunotherapies for adults with advanced cancer. Young adults over 30 who are transitioning from our pediatric program benefit from seamlessly coordinated care.