Preclinical evaluation of BRD inhibitors to treat Posterior Fossa A ependymoma

Dr. Oren Becher - Icahn School of Medicine at Mount Sinai, New York, NY

There are different types of brain cancers in children. One of them is called ependymoma. There are also different subtypes of ependymoma. Here we are proposing to study an ependymoma subtype called PFA ependymoma which has a long-term survival of approximately 50%. This type arises in young children and needs new treatments. In fact, we, the medical field, have not identified a new effective therapy for ependymoma in the past 30 years. Here we are proposing to study whether new medications that target specific types of proteins called BRD proteins can be effective to treat this subtype of ependymoma. Results from this study will help us better understand if the new medications are effective against this type of ependymoma and if we should plan to test these new medications in children with this type of brain cancer in the future. We will be using a novel genetic model of PFA ependymoma which is a useful model because this is a model with a normal immune system.