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Condition Guide

New Treatments & Clinical Trials for Osteosarcoma

Last updated June 2026Data from ClinicalTrials.gov217 active trials
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Osteosarcoma is the most common bone cancer in children and young adults, typically appearing in the long bones near the knee or shoulder. Standard treatment combines surgery to remove the tumor with chemotherapy before and after the operation. Despite these treatments, about 30% of patients develop metastatic disease, and survival rates haven't improved substantially in decades.

What's actually going on in research

Trials are testing immunotherapy drugs that train the immune system to attack osteosarcoma, targeted drugs against specific mutations found in some tumors, and new chemotherapy combinations. Researchers are also studying ways to predict which tumors will respond to treatment and testing drugs that prevent the cancer from spreading to the lungs, where metastases most often appear.

Immunotherapy approaches

Drugs like pembrolizumab and nivolumab are being tested alone and with other treatments for osteosarcoma that has spread or returned. CAR T-cell therapy targeting HER2, a protein on some osteosarcoma cells, is also in trials.

Targeted therapies

Trials are testing drugs against specific mutations, including mTOR inhibitors and drugs targeting receptor tyrosine kinases. Some osteosarcomas have actionable mutations that may respond to these targeted approaches.

Metastasis prevention

Researchers are testing drugs like mifamurtide, an immune stimulant approved in Europe, to see if they can prevent osteosarcoma from spreading to the lungs after surgery. Aerosol chemotherapy delivered directly to the lungs is also being studied.

What to know before you search

Eligibility typically depends on whether the cancer is newly diagnosed or has returned, whether it has spread, and what prior treatments have been tried.

What types of trials are currently open

  • Immunotherapy trialsTesting checkpoint inhibitors, CAR T-cells, and other immune-based treatments to see if they can shrink tumors or prevent recurrence.
  • Targeted therapy trialsTesting drugs aimed at specific mutations or proteins in osteosarcoma cells, often for tumors that have returned after standard treatment.
  • Chemotherapy trialsTesting new drug combinations or schedules, sometimes with higher doses followed by stem cell rescue.
  • Surgery trialsStudying different surgical approaches and timing, particularly how surgery coordinates with chemotherapy.
  • Prevention trialsTesting treatments aimed at stopping osteosarcoma from spreading or returning after initial treatment.

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