In May, clinical research teams activated a phase 1/2 study led locally by Sarah Rumler, DO, Assistant Professor of Pediatric Hematology and Oncology. The multi-center trial evaluates the optimal dose, side effects, and benefits of CBL0137—a drug that blocks signals cancer cells use to grow and survive—in pediatric patients with relapsed or treatment-resistant brain tumors.

Brain tumors are the number one cause of cancer death in kids, said Dr. Rumler, which emphasizes the need for more comprehensive and creative solutions to help a population that does not have the same variety of treatments as adults.

CBL0137 is an exciting treatment option for recurrent or refractory solid tumors, including central nervous system tumors and lymphoma, because it shows strong anti-cancer activity without harming nearby cells. CBL0137 is in a class of drugs called curaxins, which inhibit cancer cell division and tumor growth. Curaxins work by epigenetically modifying cancer cells, disrupting the interaction between DNA and the cell’s histone complex. These drugs have also been shown to activate the anti-cancer gene, p53, Dr. Rumler said.

“Historically we have used drugs that cause direct DNA damage to kill cancer cells, which injures many healthy cells in the process and causes significant toxicity,” Dr. Rumler said. “By adding a different way of targeting the unique changes in the cancer cell, this is another tool in our multi-pronged approach to treating some of the most aggressive, resistant types of tumors.”

The trial demonstrates MCW’s commitment to bringing innovative oncology care to the “historically underfunded” pediatric population, Dr. Rumler said. The research team is currently screening for enrollment and hope to enroll three to five pediatric patients at MCW.