Dr. Nader Sanai
Designated Research Grant
– Ependymoma

Barrow Neurological Foundation and the Ivy Brain Tumor Center

Give to
Pediatric Cancer Research Foundation

Audrey Coon is a beautiful little girl who has faced more than her share of challenges in her life. But, thanks to the availability of extraordinary medical care and cutting-edge research, Audrey’s story has a happy ending.

In honor of the care team that made her outcome possible, the Coon Family has designated a special fund to forward-thinking researchers in Audrey’s name.

The Project:

Ependymomas are tumors that form in the brain or the spinal cord and can occur at any age, but most often in children. The current standard-of-care for ependymoma includes surgical resection combined with radiation therapy. With this approach, the disease remains incurable for most patients. There are no FDA-approved chemotherapies that prolong the life of pediatric or adult ependymoma patients.

Recent genetic characterization of ependymomas, however, reveal a subset that harbor YAP1 gene fusions. This genetic anomaly has been shown to drive ependymoma tumor growth. Hence, this unique subpopulation of ependymoma patients may be responsive to drugs that target the YAP signaling pathway. Laboratory studies at the Ivy Brain Tumor Center have identified a new drug, a targeted inhibitor from Vivace Therapeutics, that may be effective in blocking YAP signaling.

We propose a clinical trial studying this new drug in adult YAP1 fusion ependymomas. Should the drug be effective, this will open the door to pediatric ependymoma studies using the same treatment strategy. We will conduct a Phase 0 clinical trial in eligible patients who need surgical resection of their tumor. These patients will receive several days of the experimental therapy prior to surgery, at which time we will remove their tumor and test whether this new drug is adequately penetrating the brain tumor (a common challenge in brain tumor drug development) and adequately hitting its molecular target. Patients whose tumors respond to the drug will receive the drug long term in hopes of preventing tumor recurrence.

Successful identification of YAP1-targeting drug in ependymoma patients will potentially open the door to the first-ever FDA approval of a chemotherapy for pediatric and adult ependymoma patients.

Change a Life Today

Some people say that children make up 10% of our cancer population, but they are 100% of our future! ~ Dr. Alex Huang, Case Western