Infant leukemia is an aggressive cancer with a very poor prognosis. The leukemia cells in most of these patients have a genetic change in which a gene (KMT2A) is broken and combined with other genes that typically do not interact with one another (this is called "rearranged"). A drug named SNDX-5613 has been developed that directly targets the changes that occur in a cell with a KMT2A rearrangement to specifically kill these leukemia cells, and it has shown promise in treating adult leukemia patients with a KMT2A rearrangement. An upcoming clinical trial will combine SNDX-5613 with traditional chemotherapy for children with leukemia with a KMT2A rearrangement that has come back (relapsed) or proven resistant to typical treatment (refractory). In addition to testing the safety and efficacy of SNDX-5613, studies will be done on patients’ blood and bone marrow samples to better understand how the drug functions in fighting leukemia. This trial represents the next step in evaluating this promising new targeted drug for these deserving patients, and the associated studies are key to helping us understand more about the biology of this leukemia and how to best target it.

This grant is named for the Oh Danny Boy, I Love You So: The Danny O'Brien Rhabdoid Tumor Research Fund. Danny O’Brien was just 5 months old when he was diagnosed with a malignant rhabdoid tumor on his liver. This cancer is extremely rare and aggressive. He endured chemotherapy to shrink the tumor for surgery, but the treatment was not effective. At the tender age of 9 months, Danny passed away. Fortunately, he knew nothing but love and affection all of his short life. This fund honors Danny’s courage and his unconditional love even in the midst of his battle with cancer.