HER2-specific Chimeric Antigen Receptor (CAR) T Cells for Children with Ependymoma (PBTC-059)


This study enrolls patients who have ependymoma that has come back or has not gone away after standard treatment. To qualify for the study, tumor screening must indicate that the cancer-associated protein HER2 is present.


This is a Phase I study to evaluate the safety profile of a type of immune therapy called HER2 CAR T cells (short for HER2 chimeric antigen receptor T cells). In addition to looking for side effects, we will study how well this treatment works against a brain tumor called ependymoma that has come back after treatment (recurrent) or has not responded well to treatment (progressive) in children.

The HER2 CAR T cells used in this trial are made from the patient's own blood. A new gene, called the HER2 CAR, will be inserted into patient's T cells to allow them to recognize a protein on the tumor called HER2. These HER2-specific CAR T cells may be able to target and kill ependymoma tumors that express HER2. This research is also studying how doable it is to provide this type of CAR T cell treatment to children being treated at different hospitals.

PBTC-059 is a multi-center, Phase I and surgical study of the treatment with HER2-specific CAR T cells for patients with refractory or recurrent ependymoma. This national study is led by Dr. Meenakshi Hegde from Texas Children’s through the Pediatric Brain Tumor Consortium (PBTC).

Eligibility Criteria

  • 1 to 22 years old
  • Relapsed or refractory ependymoma
  • Acceptable organ function
  • Tumor HER2 expression confirmed by histology

Detailed inclusion and exclusion criteria are listed at clinicaltrials.gov


For patients interested in treatment at Texas Children’s Hospital:

Shoba Navai, MD
Texas Children’s Cancer and Hematology Center


Kathy McCarthy (research nurse)