The Phase III STELLAR trial found that combining Eflornithine with Lomustine significantly improves outcomes for patients with recurrent IDH-mutant Grade 3 Astrocytoma, an aggressive type of brain tumor. While the combination did not benefit the entire study population, patients in this specific molecular subgroup saw median overall survival increase from 23.5 months with lomustine alone to 34.9 months. Progression-free survival more than doubled, from 7.2 to 15.8 months.
Eflornithine works by targeting an enzyme that fuels tumor growth, slowing cell division rather than directly killing cancer cells, making it particularly effective in Grade 3 tumors. Modern molecular testing was critical, as the drug’s benefit was confined to true Grade 3 tumors under the latest World Health Organization classification.
The combination therapy was generally manageable, though it increased certain side effects, including low blood counts, hearing impairment, and gastrointestinal issues. Researchers say eflornithine plus lomustine could become a new standard of care for this patient population, offering a meaningful survival advantage in a cancer type with few treatment options.