Defying the Odds Through a Clinical Trial

As part of the inaugural American Association for Cancer Research (AACR) Pediatric Cancer Report for 2025, Dr. Carl Koschmann is featured in a new video highlighting the journey of a young patient diagnosed with diffuse midline glioma (DMG). 

In 2020, eight-year-old Kaley Ihlenfeldt began experiencing persistent headaches and nausea that ultimately led to a devastating diagnosis: DMG, an aggressive pediatric brain tumor with historically no effective treatments and a survival rate of less than 5% beyond two years.

Determined to find options, Kaley’s family connected with Dr. Koschmann’s clinical trial of ONC201—now known as dordaviprone, the first FDA-approved therapy for recurrent DMG. Kaley began the trial in late 2020 and has continued treatment with minimal side effects, allowing her to enjoy a full, active teenage life while the medication helps slow the tumor’s progression.

The video profiles both Kaley’s remarkable resilience and Dr. Koschmann’s pioneering research, which helped uncover how dordaviprone works by reprogramming cancer cell metabolism and partially restoring molecular features of healthy brain cells. His work, supported by patient families and organizations such as the ChadTough Defeat DIPG Foundation, contributed to a milestone moment: the 2025 FDA approval of dordaviprone—the first-ever approved therapy for this devastating disease.