This leads to the accumulation of PUT in cells [71,72] and DFMO treatment, which has been safely used for decades in the treatment of African trypanosomiasis and hirsutism and which is now in clinical trials as a chemopreventative (e.g., prostate and colon cancer) or therapeutic (e.g., neuroblastoma) agent for several malignancies, may provide a therapeutic strategy for treatment of patients with this gain-of-function ODC1 variant. Here, ODC1 is linked to neuroblastoma.