Trastuzumab Deruxtecan for Bone Cancer

This trial tests the effects of trastuzumab deruxtecan (also known as Enhertu or DS-8201a), a targeted cancer treatment, on patients with specific tumors: osteosarcoma, Wilms tumor, or desmoplastic small round cell tumor. This treatment combines a monoclonal antibody, which seeks out cancer cells, with a chemotherapy drug that attacks them directly. The study seeks participants with these tumors who have not responded to previous treatments or have experienced a recurrence. Those with Wilms tumor or desmoplastic small round cell tumor after initial treatments or with inoperable tumors might be eligible. Participants will receive the treatment through an IV every three weeks, with their condition monitored through imaging tests and blood samples. 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 potentially groundbreaking therapy.

The trial protocol does not specify if you need to stop taking your current medications. However, certain medications like corticosteroids, other investigational drugs, and anti-cancer agents are not allowed. It's best to discuss your specific medications with the trial team.

Research shows that trastuzumab deruxtecan is usually well-tolerated, but some side effects require attention. Studies have identified common side effects such as nausea and blood-related issues, though most were mild. Importantly, serious side effects occurred in more than 1% of patients, including vomiting, lung inflammation, pneumonia, fever, and urinary infections.

Trastuzumab Deruxtecan is unique because it combines a targeted therapy approach with chemotherapy. Unlike traditional chemotherapy, which attacks all rapidly dividing cells, Trastuzumab Deruxtecan specifically targets cancer cells that express the HER2 protein, delivering the chemotherapy directly to those cells. This targeted delivery can potentially minimize damage to healthy cells and reduce side effects. Researchers are excited about this treatment because it offers a new way to address cancers that are hard to treat, such as osteosarcoma, Wilms tumor, and desmoplastic small round cell tumor, potentially improving outcomes for patients with fewer side effects than conventional options.

Research has shown that trastuzumab deruxtecan, also known as Enhertu, holds promise for treating certain cancers. It targets HER2-positive cells and delivers chemotherapy directly to them. In earlier studies, Enhertu reduced the risk of cancer recurrence or death by 53% compared to another treatment in patients with high-risk breast cancer. It also achieved a 35.9% overall response rate, meaning about a third of patients experienced tumor shrinkage. In real-world use, more than half of the patients saw their tumors shrink, with high survival rates after one year. These findings suggest potential benefits for treating osteosarcoma, Wilms tumor, and desmoplastic small round cell tumor, due to similar mechanisms involved. Participants in this trial will receive trastuzumab deruxtecan as the investigational treatment.⁵⁶⁷⁸⁹