Charles Mullighan, MD

Funded by the Dick Vitale Pediatric Cancer Research Fund

Most children who die from cancer have had either leukemia (blood cancer) or medulloblastoma (brain cancer). These two types of cancer cause children to be really sick. Dr. Mullighan studies how genes cause the most common form of leukemia. Dr. Roussel studies how the brain cancer medulloblastoma grows in the brains of children. Both Dr. Mullighan and Dr. Roussel want to develop new drugs that are better at destroying cancer cells.  Their idea is to develop new medicines that will have fewer side effects than the medicines that are being used today. They have discovered a new method called molecular glues to create new cancer medicines. The doctors have found that one molecular glue called SJ42872 kills both blood and brain cancer cells. They have already shown that SJ42872 kills these cancer cells in mice.  They now want to find out how SJ42872 works in so many different types of blood and brain cancers. Dr. Mullighan and Dr. Roussel already know that SJ42678 works inside the cancer cells by destroying a protein call PPIL4. By doing this research, the doctors want to understand why SJ42872 is so good at destroying this protein.  Then they could learn how to use SJ42872 along with other cancer medicines to kill larger numbers of cancer cells.  The research will also help them understand which children will benefit from using SJ42872 as part of their treatment.