Atypical teratoid rhabdoid tumor (AT/RT) is a rare, malignant tumor that occurs in the brain and spinal cord of young children. AT/RT can look like many other tumors under the microscope and is diagnosed based on the presence of a mutation in SMARCB1, or rarely SMARCA4. Unfortunately, despite aggressive multi-modal therapy, children with this disease still have a poor prognosis. Fortunately, there is growing community of physicians and researchers dedicated to furthering our understanding of this disease and improving survival for children with AT/RT.
The AT/RT working group is a collection of physicians and scientists who have expertise in AT/RT and are dedicated to improving the outcomes of children with these tumors.
The specific goals of the working group are as follows:
- Better understand the factors that affect outcomes of children with AT/RT
- Identify best treatment options for these patients including development of novel therapies
- Increase awareness and funding for AT/RT research
- Provide support to families of children with AT/RT
We feel very strongly that the best way to understand a rare disease such as AT/RT is to gather as much data as possible and have those data evaluated by a collection of expert physicians and scientists. Our group understands that the only way to improve prognosis for children with AT/RT is to work together. We meet regularly to share pre-clinical data, discuss the latest AT/RT research occurring around the world and to discover new ways to collaborate. We are fortunate to work closely with our disease champions who frequently join our pre-clinical and clinical working group meetings and provide invaluable input.
We are developing a clinical trial for patients with newly diagnosed AT/RT that will allow us to determine if there are clinical and/or molecular features associated with survival. Our group is working on several clinical trial options for children with progressive/recurrent disease. These options include an expert panel review of the patients’ clinical and molecular features with recommendations for treatment inclusive of clinical trial options. Our pre-clinical group has defined several potential agents that we are considering for a phase I/II clinical trial in patients with recurrent AT/RT.