Immunotherapy for Pediatric Atypical Teratoid/Rhabdoid Tumor
A diagnosis of atypical rhabdoid/teratoid tumor (AT/RT) in the nervous system of an infant or young child is devastating. AT/RT is a very aggressive tumor that exhibits a five-year survival of less than 20%, and the types of intensive chemo- and radiotherapeutic treatment regimens most likely to extend life do so only at wholly unacceptable cost to cognitive function. This is true not just of AT/RT but for many other kinds of brain tumors as well: curative radiotherapy can lead to neurocognitive incapacitation and a life of permanent institutionalization.
Hence there exists a clear medical need for the development of more efficacious and less toxic treatment regimens for AT/RT and other pediatric brain tumors. Treatments should be able to both effectively fight cancer while sparing neurocognitive function.
To address this issue, our group has filed an IND for intent to treat pediatric AT/RT using a novel immunotherapeutic treatment approach we have previously shown to be safe and potentially efficacious in an outbred canine model of spontaneous brain cancer. The procedure uses the patient's own blood cells and own tumor cells to generate a powerful vaccine that can attack the tumor while sparing normal tissue. Successful funding of this application will allow the study investigators to generate manufacturing data required by the FDA. Generation of these data will allow a clinical trial to proceed.