Clinical Research Directory

Ultra-Rapid Profiling and Drug Screening for Treatment Selection in Patients With Relapsed or Recurrent Rare Brain Tumors

The current study will use a new treatment approach based on the molecular characteristics of each participant's tumor. The study will test the feasibility in of performing real-time drug screening on tissue taken during surgery in patients with relapsed ultra rare brain tumors (BCOR -altered, MN1-altered, CNS Sarcomas, other rare brain tumors) and of having a specialized tumor board assign a treatment plan based on the results of the drug screening and genomic sequencing. The aim of this trial is to allow every child and young adult with relapsed ultra rare brain tumors to receive the most effective and least toxic therapies currently available and will pave the way for improved understanding and treatment of these tumors in the future. Moreover, if successful, it could serve as a paradigm for personalized medicine programs for other types of cancer.

Observational Study of Responses to Treatments in Advanced Central Nervous System (CNS) Tumors

Background: Primary central nervous system (CNS) tumors grow in the brain and spinal cord. These tumors are rare, but they are difficult to treat and often fatal. SmartMatch is a new technology that tries to help find the best medicines for the particular tumor by testing how small pieces of surgically removed tumor tissue react to different drugs. The study team wants to see if SmartMatch can complete the analysis and generate a report within 21 days from the time of the surgery. Objective: To better understand CNS tumors so doctors can find better ways to treat them in the future. Eligibility: People aged 15 years and older with recurrent CNS tumors. Patient must already be scheduled for a surgery for the tumor at the NIH. Design: The study team will collect a small amount of tumor samples during the planned surgery. They may also use tumor samples from previous procedures. No new or additional procedures will be done for the purpose of this study. The tumor samples will be sent to a lab for SmartMatch analysis. Once completed, the results will be shared with the patient and his/her local doctor. Together they can decide whether to incorporate the results into the treatment plan. It is important to know that the results may or may not be helpful. There will only be one blood test for research. Blood and tissue samples collected may be used for additional analysis. Tumor tissue may be used to grow additional samples for further study. Participants will receive a pathology diagnosis and mutation profile generated by pathologists who specialize in CNS tumors. The study team will seek updates on participant's health approximately every 6 months for 3 years. Tumor samples may also be collected from any additional surgery done at NIH during this time....