Miguel Sena-Esteves, PhD

The prognosis for brain tumor patients has not improved significantly despite many innovations in surgery and radiation therapy, and the introduction of many new drugs that work well for other types of cancer. There is an urgent need for new approaches to treat brain tumors. Here, we propose investigating a new approach to brain tumor therapy based on the genetic modification of normal brain cells to create an environment that prevents tumor growth.  

Adeno-associated virus (AAV) vectors will be used as they are very efficient in introducing genes into normal brain cells. These AAV vectors will be used to introduce in normal brain cells or blood vessels a gene that makes a protein which can be released from modified normal cells and that is selectively active against tumor cells.  

In these experiments we will investigate the efficiency of this therapy principle by modifying the brain surrounding the tumor by direct injection of AAV vectors into the brain or by modifying the blood vessels in the brain by injection of these vectors into the bloodstream. These approaches may lead to the creation of widespread anti-tumor networks capable of preventing brain tumors from growing or appearing again after surgery.  

“I have no doubt that gene therapy is the path to the future. Recent clinical trials show this unequivocally.”