PNOC ATRT Registry Update Webinar Nov 15, 2022

Atypical teratoid rhabdoid tumor (AT/RT) is a rare, malignant tumor that occurs in the brain and spinal cord of young children. The condition usually appears by 3 years old. Occasionally, it occurs in older children.

The PNOC AT/RT Working Group is a collaboration of physicians and scientists who have expertise in AT/RT. They are dedicated to furthering understanding of this disease and improving survival for children with AT/RT.

The PNOC AT/RT Registry is the first step to breakthroughs.
The best way to understand such a rare disease like AT/RT is to gather as much data as possible and have that data evaluated by a collection of expert physicians and scientists. The AT/RT Registry will gather all available patient data in one dataset. It will give the AT/RT Working Group an unprecedented tool to understanding this disease and accelerating research. It will improve data collection and data sharing across the scientific community. The registry will help identify prognostic variables and new therapies including immunotherapies. It will facilitate the collaboration between translational and clinical trials with novel agents.

How we move the science into a clinical trial
AT/RT Working Group Investigators from around the world meet regularly to share pre-clinical data, and their best ideas and strategies for research. The AT/RT Working Group is developing a clinical trial for patients with newly diagnosed AT/RT that will allow them to determine if there are clinical and/or molecular features associated with survival. The group is also 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. The pre-clinical group has defined several potential agents that they are considering for a phase I/II clinical trial in patients with recurrent AT/RT.

Donate or start a fundraising team to support the AT/RT Registry: https://fundraising.pnocfoundation.or... or reach out to [email protected].

Are you an AT/RT patient family interested in participating in the registry?
- For anyone who received, or is receiving treatment at a PNOC institution - you can request that the institution enroll your child. PNOC has sites across the United States, Canada, Europe and Australia. You can check to see if your hospital is a PNOC participating site here: https://pnoc.us/participating-sites/
- If you were treated, or are being treated at a non-PNOC Institution - you will enroll through CHLA by:
1. Emailing Jasmine Pauly [email protected]
2. Reaching out to your treating physician(s) and institution(s) and requesting your
- Tumor tissue, pathology report
- Imaging on a CD
- Medical records