Immunotherapy for Ependymoma

This trial tests a new vaccine (HLA-A2 restricted synthetic tumor antigen) combined with an immune-boosting cream (Imiquimod) to determine its safety and ability to trigger an immune response in children with recurring ependymoma, a type of brain tumor. It targets those whose ependymoma has returned after treatment. Eligible participants must be HLA-A2 positive (a specific genetic profile) and willing to travel to Pittsburgh for treatment sessions. 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 innovative therapy.

The trial requires that you stop certain medications at least one week before joining, including interferon, allergy shots, growth factors, interleukins, and any investigational drugs. You must also not use immunosuppressive agents within four weeks prior to the study, except for low-dose dexamethasone.

Research has shown that the HLA-A2 restricted tumor antigen vaccine, when combined with imiquimod (a cream that boosts the immune system), might help the body fight cancer cells. Specific safety information from earlier studies on this vaccine is not yet available, as it remains in the early testing stages. Scientists are primarily assessing its safety for human use.

Imiquimod, however, has a well-established safety record. It is already approved for other uses, such as treating certain skin conditions, and most people tolerate it well. Some may experience mild skin reactions at the application site, but serious side effects are rare.

Researchers are excited about the new immunotherapy treatment for ependymoma because it targets the cancer in a novel way. Unlike standard treatments like surgery, radiation, and chemotherapy, this approach uses a synthetic tumor antigen vaccine tailored to the patient's immune system. The vaccine, combined with imiquimod, aims to boost the body's natural defenses to recognize and attack the tumor cells specifically. This targeted action offers the potential for more effective treatment with fewer side effects, which is a significant improvement over existing therapies.

Research has shown that specific vaccines, such as the HLA-A2 restricted tumor antigen vaccine, combined with the medicine imiquimod, can help combat recurring ependymomas, a type of brain tumor. In one study, 55% of patients survived for at least 12 months, offering hope for those with this tumor. Another study found that patients with less aggressive tumors (WHO grade 2) experienced about 45 weeks without disease progression. These treatments enhance the body's immune system to more effectively attack tumor cells. The combination of the vaccine and imiquimod, which participants in this trial will receive, shows promise for improving outcomes in children with recurring ependymomas.¹²⁶⁷⁸