TOWARDS CHIMERIC ANTIGEN RECEPTOR TRANSGENIC T CELL THERAPY FOR TUBEROUS SCLEROSIS COMPLEX

We have performed some initial experiment that support the idea, that immunotherapy can be used to treat tuberous sclerosis complex. The ultimate objective is to take a patient’s own immune cells obtained by a blood draw, and genetically modify them so that they recognize the affected cells in TSC. What makes this different from existing therapeutics is that pharmaceuticals marketed thus far, such as rapamycin, focus on keeping the disease stay in check and preventing it from getting worse. Immunotherapy, however, makes use of the immune system to attack affected cells and eliminate them, thereby radically reducing disease burden. We want to test this concept in further pre-clinical studies to firmly establish whether treatment can be effective even in advanced stages of disease, and we need to understand whether modified T cells can be effective in the presence of commonly used rapamycin treatment. We also want to make the treatment safer by including a gene that can eliminate host immune cells when exposed to the drug ganciclovir. That can help prevent T cells from overreacting. Finally, we will focus on bringing Federal approvals in place so we can plan for a clinical trial in follow-up studies. The team brings together a clinician who treats patients with TSC, a clinician scientist who has been actively involved in designing genetically modified immune cells for cancer immunotherapy, and a scientist (PI) with a great interest in developing therapeutic for TSC. If the investigations are successful, recruitment for an early stage clinical trial can begin shortly after the current project is completed.