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 trials — Testing checkpoint inhibitors, CAR T-cells, and other immune-based treatments to see if they can shrink tumors or prevent recurrence.
- Targeted therapy trials — Testing drugs aimed at specific mutations or proteins in osteosarcoma cells, often for tumors that have returned after standard treatment.
- Chemotherapy trials — Testing new drug combinations or schedules, sometimes with higher doses followed by stem cell rescue.
- Surgery trials — Studying different surgical approaches and timing, particularly how surgery coordinates with chemotherapy.
- Prevention trials — Testing treatments aimed at stopping osteosarcoma from spreading or returning after initial treatment.
Recently added Osteosarcoma trials
Domatinostat With Sirolimus for Relapsed, Refractory Sarcoma and Osteosarcoma
This is a multicenter, open-label, Phase 1/2 study evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, and antitumor activity of domatinostat in combination with sirolimus in adolescents and adults with relapsed or refractory sarcoma and osteosarcoma.
Prospective Evaluation of Dynamic Serum ProGRP for Treatment Response Monitoring in Ewing Sarcoma
This single-center prospective observational study aims to evaluate whether longitudinal changes in serum pro-gastrin-releasing peptide (ProGRP) reflect treatment response in patients with newly diagnosed Ewing sarcoma. Serum ProGRP levels will be measured before systemic treatment, during neoadjuvant chemotherapy, before local treatment, and after local treatment. Changes in ProGRP will be compared with radiographic tumor response assessed according to RECIST version 1.1. The study will also explore the ability of early ProGRP changes to predict objective radiographic response and the association between ProGRP patterns and event-free survival. ProGRP results obtained for research purposes will not be used to guide clinical treatment decisions.
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