Trastuzumab + GM-CSF for Ependymoma

This trial explores a new combination treatment for children with relapsed posterior fossa ependymoma, a type of brain tumor. The researchers aim to determine if trastuzumab, administered into the spinal fluid, can be detected in the tumor and to assess the safety of using it with GM-CSF (Granulocyte-Macrophage Colony Stimulating Factor), which boosts white blood cells. Participants should be children diagnosed with this specific relapsed brain tumor who plan to undergo surgery as part of their standard care. 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.

Yes, you will need to stop certain medications. You cannot take systemic corticosteroids, investigational drugs, or other anti-cancer agents while participating in the trial.

Yes, you will need to stop taking certain medications. Specifically, you cannot be on systemic corticosteroids, other investigational drugs, or anti-cancer agents to participate in the trial.

Research has shown that trastuzumab is usually well-tolerated when combined with chemotherapy for other conditions. One study found that patients taking trastuzumab did not experience more severe side effects than those who did not. However, the risk of heart problems necessitates careful monitoring.

GM-CSF (Granulocyte-Macrophage Colony-Stimulating Factor) can cause side effects such as fever and skin rashes. Some patients may experience capillary leak syndrome, where fluid leaks from small blood vessels into nearby tissues, causing swelling and low blood pressure.

Researchers are excited about the combination of trastuzumab and GM-CSF for ependymoma because it offers a novel approach compared to traditional treatments like surgery and radiation. Unlike standard therapies, trastuzumab targets specific cancer cell receptors, potentially offering a more precise attack on tumor cells. Additionally, GM-CSF is used to stimulate the immune system, enhancing the body's natural ability to fight the cancer. The unique delivery method, where trastuzumab is administered intrathecally (directly into the spinal fluid) after subcutaneous GM-CSF, aims to improve drug effectiveness and minimize systemic side effects, offering hope for better outcomes in this challenging condition.

In this trial, participants will receive either IT trastuzumab after a subQ GM-CSF course or IT trastuzumab combined with subQ GM-CSF. Research has shown that trastuzumab, a monoclonal antibody, can treat certain cancers with the HER2 protein. While it doesn't work for everyone, it has shown early success without causing serious side effects in children with brain tumors. GM-CSF boosts the immune system and is believed to enhance trastuzumab's effectiveness. Studies suggest that combining GM-CSF with trastuzumab can improve the immune response in patients with ependymoma, a type of brain tumor. Although direct evidence for this combination in relapsed posterior fossa ependymoma is still being gathered, the results so far are promising.²⁶⁷⁸⁹