Building Drug-Delivery Warheads from Brain Stem Cells

Title: Generation of New Human Neural Stem Cell Lines for Therapeutic Applications
Investigator: Karen Aboody
Grantee: City of Hope National Medical Center

Even the best drugs won’t cure brain cancer if they can’t find the cancer cells.  This is especially true for glioblastoma, an extremely aggressive type of tumor that invades so deeply into the brain that it’s impossible to remove with surgery or radiation therapy.  To fight glioblastoma, we need to deliver chemotherapy drugs directly to the tumor’s doorstep – but finding that doorstep is really tricky for such an invasive tumor.

Dr. Karen Aboody at the City of Hope National Medical Center is working to solve this problem.  Dr. Aboody studies brain stem cells, chameleon-like immature brain cells that grow up to become different types of adult brain cells.  These chameleon-like cells are really good at sniffing out brain tumors, and they travel through the brain looking for them.

Dr. Aboody hopes to exploit the tumor-tracking power of these brain stem cells to deliver chemotherapy to glioblastoma’s doorstep.  In her work, she fills brain stem cells with anticancer drugs and then lets the drug-filled “warheads” home in on brain tumors.  When they find a tumor, the warheads dump their payloads – thus killing any tumor cells in the vicinity.  As an added bonus, because this treatment strategy targets chemotherapy to tumors instead of healthy brain cells, it should have fewer side-effects than traditional chemotherapy.

Dr. Aboody’s brain stem cell warheads have already been tested in a clinical trial, where they safely, successfully killed brain tumor cells.  To make this revolutionary new therapy a realistic treatment option for the wider brain cancer community, however, we need a more diverse set of drug-delivery warheads specialized to look for tumors all over the brain.

To meet this need, ABC2 funded Dr. Aboody in 2010 to develop and characterize a new, expanded set of drug-delivery warheads that travel more broadly throughout the brain.  She is using very strictly regulated methods to construct the new warheads, and she is carefully determining which ones track best to which parts of the brain – so that she can make the most useful tools available to the brain cancer community right away.

If Dr. Aboody’s project is successful, she will have generated a powerful new resource for taking chemotherapy drugs exactly where they are needed most to fight brain cancer.