LAM561 for Pediatric Brain Tumor

This trial explores a new treatment option called LAM561 (also known as 2-OHOA or Minerval) for children with serious brain tumors, such as high-grade gliomas. The goal is to determine the safest and most effective dose for future studies. Initially, the trial tests different dose levels to assess how much children can safely tolerate, then evaluates the safety of the optimal dose. It suits children under 18 with difficult-to-treat tumors that have not responded to other treatments. As a Phase 1 trial, this research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new treatment.

The trial does not specify if you need to stop taking your current medications, but you cannot participate if you need warfarin, phenytoin, or certain diabetes medications. It's best to discuss your current medications with the trial team.

Studies have shown that LAM561, a substance similar to a natural fatty acid that can reach the brain, is under testing for safety in people with various solid tumors. Previous research with adults determined the highest dose that doesn't cause serious side effects. In studies with children, researchers aim to find a safe dose based on what was safe for adults.

Researchers are excited about the treatments 2-OHOA and LAM561 for pediatric brain tumors because they offer a fresh approach compared to existing therapies like surgery, radiation, and chemotherapy. LAM561 is particularly intriguing due to its novel mechanism of action, which involves adjusting the dose based on the maximum tolerated dose in adults, potentially enhancing safety and effectiveness for children. Additionally, 2-OHOA is a new active ingredient that targets the tumor's cellular membranes, offering a different way to attack the cancer cells. These features could lead to more effective and safer treatment options for young patients.

Studies have shown that LAM561, also known as idroxioleic acid, yields promising results for treating certain brain tumors. In earlier research, patients with a recurring brain tumor called glioma had an 18.5% chance of their disease remaining stable for six months. Specifically, some patients experienced no worsening of their condition during this period. Additionally, combining LAM561 with the usual treatment for newly diagnosed glioblastoma slowed the disease's progression. This trial will explore the effectiveness of LAM561 in managing advanced brain tumors in children, focusing on dose escalation to determine the optimal dosing strategy.¹³⁶⁷⁸