A Pilot Trial to Test the Benefit of Real Time Drug Screening to Determine Individualized Treatment Plans for Children and Young Adults with Relapsed or Refractory Medulloblastoma

Pediatric Cancers

John Crawford (Rady)

Robert Wechsler-Reya (Sanford Burnham)

Medulloblastoma is the most common malignant brain tumor in children. Although significant advances have been made in treating medulloblastoma, many patients still die from the disease, and those who survive suffer severe long-term side effects from therapy. Patients who relapse have extremely poor outcomes, and new approaches to treating them are particularly important. We propose to carry out a unique clinical trial in which we take tissue from a patient’s tumors at the time of surgery, sequence it to identify mutations that may be causing the tumor, and also test the sensitivity of tumor cells to drugs that have been used against other cancers.Using this approach, we hope to identify more effective therapies for each patient. Our previous studies have demonstrated the feasibility of performing sequencing and drug testing on patient samples, and have shown that these analyses can provide insight into possible therapies. Our proposed trial will test whether this type of analyses can be done in a clinical setting, whether results can be used by a panel of experts to make recommendations for therapy, and whether these recommendations are beneficial for patients. If our approach is successful it will improve outcomes for relapsed MB patients and provide a new paradigm for personalized therapy of other pediatric cancers.