Trametinib Safe, Effective for Juvenile Leukemia

On Tuesday, June 18, 2024, a study published in Cancer Discovery highlighted promising findings for children suffering from relapsed or refractory juvenile myelomonocytic leukemia (JMML). According to the research, trametinib, an oral MEK1/2 inhibitor, demonstrates both safety and efficacy in treating advanced JMML.

Relapsed or refractory JMML has typically shown poor prognoses, prompting a team led by Dr. Elliot Stieglitz at the University of California San Francisco to investigate trametinib's potential. Conducted as a phase 2 trial, the study involved 10 patients with severe cases of JMML.

The outcomes were noteworthy. The researchers observed an objective response rate of 50 percent among the participants. Of particular interest, four patients with refractory disease managed to proceed to hematopoietic stem cell transplantation (HSCT) after receiving trametinib. Another three patients completed the 12 cycles of trametinib allowed in the study and continued the medication off-protocol, without undergoing HSCT. Unfortunately, the remaining three patients experienced disease progression, with two showing molecular evolution by the end of the second cycle.

Dr. Stieglitz noted the trial's importance as an alternative for parents who preferred to avoid subjecting their children to a repeat HSCT. "Our trial offered an option for parents who did not want to subject their children to a repeat HSCT and, in some cases, helped patients avoid HSCT entirely," he stated. The findings suggest that trametinib could serve as a less toxic option compared to HSCT for selected patients.

It is worth mentioning that two of the study's authors disclosed connections to the biopharmaceutical industry, with Novartis providing trametinib for the trial.