Clinical Safety Study on the Use of 5-Aminolevulinic Acid (5-ALA) in Children and Adolescents with Brain Tumors

Tumors of the central nervous system, along with leukemias, are the second most common types of cancer in children and adolescents. These tumors develop in the brain or spinal cord. Treatment varies significantly depending on the type of tumor. Surgical removal of the tumor is often performed, as children tend to tolerate the side effects of radiation therapy or chemotherapy less well. Various techniques are used to ensure the most precise removal of tumor tissue possible and to avoid secondary damage. In adult medicine, 5-aminolevulinic acid (5-ALA)—a substance produced naturally by the body that plays an important role in metabolism—is used as standard. After ingestion, 5-ALA accumulates particularly in tumor cells. These cells convert the substance into a compound that fluoresces under UV light. This allows surgeons to better identify the tumor tissue during surgery and remove it more precisely. The procedure is approved for adults but not yet for children and adolescents.

Since the metabolism of children differs from that of adults, the safety of 5-ALA use must be specifically evaluated for children and adolescents. The current standard of care for children primarily involves imaging methods also used in adult medicine, but these are usually supplemented with 5-ALA to achieve more accurate results.

The goal of the study is to collect data on the safety and efficacy of 5-ALA fluorescence-guided removal of brain tumors in children and adolescents. All patients drink a dose of 5-ALA—a powder dissolved in water—adjusted to their body weight approximately 4 hours before surgery. Data on potential side effects and the success of the surgery will be collected for up to 6 weeks after the surgery. The treatment has not yet been approved for children and adolescents. All patients participating in the study will receive this experimental treatment.

Children and adolescents between the ages of 3 and 17 who have a brain tumor scheduled for surgical removal are eligible to participate in the study. The tumor must be located above the tentorium cerebelli (supratentorial) and in the anterior or middle cranial fossa. Brain tumors located outside the brain tissue (extra-axial), such as a meningioma or a craniopharyngioma, are excluded from participation. Patients with known hypersensitivity to 5-ALA, known porphyria (acute or chronic), or kidney or liver dysfunction are also ineligible to participate.