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Targeted Therapy and Immunotherapy for Bone Cancer

In recent years, researchers have developed non-chemo drugs that can be used as part of treatment for some bone cancers. These work differently from standard chemotherapy (chemo) drugs, and they tend to have different side effects.

  • Targeted therapy: Some targeted therapy drugs specifically target the gene changes inside cells that can lead to bone cancer. Targeted drugs are especially important in treating types of bone cancer where chemo has not been very useful, such as chordomas.
  • Immunotherapy: Immunotherapy drugs are also used as part of treatment for some types of bone cancers. Immunotherapy is the use of medicines to help the immune system recognize and destroy cancer cells.

Note: This information focuses on bone cancers that are seen most often in adults and start in the bones (primary bone cancers). Osteosarcoma, Ewing sarcoma, and bone metastases are covered separately.

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Osteosarcoma: targeted therapy

A class of drugs called tyrosine kinase inhibitors (TKIs) are used in the treatment of some osteosarcoma tumors. Additional information can be found in Targeted Drug Therapy for Osteosarcoma.

Chondrosarcoma: targeted therapy

Targeted therapy drugs are part of treatment for some chondrosarcomas. These drugs may be used in certain situations, for example:

  • When specific gene changes are present in the tumor
  • If the cancer has spread throughout the body

TKIs work by blocking the signals cells need to grow and divide. Some examples of TKIs that have been tried in chondrosarcoma include:

  • Dasatinib (Sprycel)
  • Pazopanib (Votrient)
  • Sunitinib (Sutent)
  • Regorafenib (Stivarga)

These drugs are given as a tablet or capsule by mouth once a day.

Possible side effects

Side effects of these drugs can include fatigue, low blood counts, loss of appetite, hand-foot syndrome (redness and irritation of the hands and feet), high blood pressure, weight loss, diarrhea, and abdominal (belly) pain.

Less common but more serious side effects can include problems with blood flow to the heart, bleeding, abnormal thyroid test results, and perforations (holes) in the stomach or intestines.

Ivosidenib (Tibsovo) is an isocitrate dehydrogenase (IDH1) inhibitor used in the treatment of some chondrosarcomas.

This type of targeted drug works by blocking an enzyme (IDH1) with a gene change that supports tumor growth. By blocking it, tumors are not able to grow and cell growth and division stops. This medication can be used if IDH1 gene changes are seen in the tumor biopsy.

Ivosidenib is given as a tablet by mouth once a day.

Possible side effects

Side effects of Ivosidenib may include fatigue, joint pain, skin rashes, changes in blood test results, abdominal (belly) pain, nausea, vomiting, diarrhea, or constipation.

Chondrosarcoma: immunotherapy

Immunotherapy drugs called immune checkpoint inhibitors are sometimes used to treat chondrosarcoma.

An important part of your immune system is its ability to keep itself from attacking normal cells in your body. To do this, it uses “checkpoint” proteins on immune cells. These act like switches that need to be turned on, or off, to start an immune response. Cancer cells sometimes use these checkpoints to avoid being attacked by immune cells.

Drugs that target these checkpoints, called checkpoint inhibitors, can be used to treat some people with chondrosarcoma. They include:

  • Pembrolizumab (Keytruda)
  • Nivolumab (Opdivo)

These drugs are given as an intravenous (IV) infusion once every few weeks. Pembrolizumab is also available as an injection.

Common side effects

Common side effects of these drugs can include fatigue, cough, skin rashes, loss of appetite, constipation, joint pain, and diarrhea.

Severe side effects

In some cases, severe side effects can happen when taking these drugs. These problems can be serious and even life-threatening.

  • An infusion reaction can happen while you are getting these IV drugs. Symptoms can include fever, chills, rash, dizziness, wheezing, and trouble breathing.
  • These drugs can also cause immune cells to attack normal healthy cells throughout your body, leading to problems with your lungs, gut, hormone-making glands like the thyroid, kidneys, or other organs.

Ewing and Ewing-like sarcomas: targeted therapy

Targeted therapy drugs are part of treatment for some Ewing and Ewing-like sarcomas. These drugs may be used in certain situations, like disease that has spread throughout the body or cancer that comes back after treatment.

TKIs work by blocking the signals cells need to grow and divide. Some examples of TKIs that have been tried in Ewing sarcoma include:

  • Cabozantinib (Cabometyx, Cometriq)
  • Regorafenib (Stivarga)

These drugs are given as a tablet by mouth once a day.

Side effects

Side effects of these drugs can include fatigue, low blood counts, loss of appetite, hand-foot syndrome (redness and irritation of the hands and feet), high blood pressure, weight loss, diarrhea, and abdominal (belly) pain.

Less common but more serious side effects can include problems with blood flow to the heart, bleeding, abnormal thyroid test results, and perforations (holes) in the stomach or intestines.

Undifferentiated pleomorphic sarcoma (UPS): targeted therapy

UPS is treated similarly to osteosarcoma. A class of drugs called tyrosine kinase inhibitors (TKIs) are used in the treatment of some of these tumors. These drugs may be used when cancer has spread to other parts of the body or if the cancer comes back after treatment. Additional information can be found in Targeted Drug Therapy for Osteosarcoma.

Fibrosarcoma of the bone: targeted therapy

Targeted drugs don’t currently play a role in treating a fibrosarcoma of the bone. If there is a drug that targets a gene change in your tumor cells, your cancer care team may try those treatments. But this usually happens only after treatments like surgery and radiation, which are known to work for these types of tumors.

Giant cell tumors of the bone: targeted therapy

Denosumab (Xgeva) is a monoclonal antibody that blocks RANKL, a protein that activates osteoclasts, a type of cell that reabsorbs bone. This blocks tumor growth in giant cell tumors of the bone.

This drug is given as a shot once every few weeks.

Side effects

Side effects of this drug can include fatigue, weakness, nausea, and diarrhea.

Less common but serious side effects can include problems with the jawbone (osteonecrosis) or a broken bone in the leg. People on this drug can also experience low calcium and are at higher risk of getting an infection.

Chordomas: targeted therapy

TKIs work by blocking the signals cells need to grow and divide.

Some TKIs, like Lapatinib, work on a type of tyrosine kinase called EGFR, making the drug more effective for tumors with specific gene mutations. Other TKIs work to block signals from multiple tyrosine kinases.

Some examples of TKIs that have been tried in chordomas include:

  • Imatinib (Gleevec, Imkeldi)
  • Dasatinib (Sprycel)
  • Sunitinib (Sutent)
  • Erlotinib (Tarceva)
  • Lapatinib (Tykerb)
  • Sorafenib (NexAVAR)

Most of these drugs are given as a tablet by mouth once or twice a day. Imatinib is also available as a liquid.

Side effects

Side effects of these drugs can include fatigue, low blood counts, loss of appetite, hand-foot syndrome (redness and irritation of the hands and feet), high blood pressure, weight loss, diarrhea, and abdominal (belly) pain.

Less common but more serious side effects can include problems with blood flow to the heart, bleeding, abnormal thyroid test results, and perforations (holes) in the stomach or intestines.

mTOR inhibitors, such as sirolimus, work by blocking cells from growing and dividing.

This drug can be given as a tablet or liquid by mouth daily.

Side effects

Side effects can include high blood pressure, high cholesterol, diabetes, abdominal pain, nausea, constipation, diarrhea, low blood counts, muscle or joint pain, and mouth sores.

Sirolimus weakens the immune system, so infections are more common in people on this drug.

Chordomas: immunotherapy

An important part of your immune system is its ability to keep itself from attacking normal cells in your body. To do this, it uses “checkpoint” proteins on immune cells, which act like switches that need to be turned on, or off, to start an immune response.

Cancer cells sometimes use these checkpoints to avoid being attacked by immune cells. Drugs that target these checkpoints, called checkpoint inhibitors, can be used to treat some people with chordoma. This includes:  

  • Pembrolizumab (Keytruda).

This drug is given as an intravenous (IV) infusion once every few weeks. Pembrolizumab is also available as an injection.

Common side effects

Common side effects can include fatigue, cough, skin rashes, loss of appetite, constipation, joint pain, and diarrhea.

Severe side effects

In some cases, severe side effects can happen when taking this type of drug. These problems can be serious and even life-threatening.

  • An infusion reaction can happen while you are getting this type of IV drug. Symptoms can include fever, chills, rash, dizziness, wheezing, and trouble breathing.
  • This type of drug can also cause immune cells to attack normal healthy cells throughout your body, leading to problems with your lungs, gut, hormone-making glands like the thyroid, kidneys, or other organs.

More information

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Developed by the 黑料大湿Posts Cancer Society medical and editorial content team with medical review and contribution by the 黑料大湿Posts Society of Clinical Oncology (ASCO).

Davis DD, Taqi M, Kane SM. Fibrosarcoma. In: StatPearls. Treasure Island (FL): StatPearls Publishing; November 12, 2023.

Gelderblom AJ, Bovee J. Chondrosarcoma. UpToDate. 2025. Accessed at https://www.uptodate.com/contents/chondrosarcoma on October 8, 2025.

National Comprehensive Cancer Network. NCCN Clinical Practice Guidelines in Oncology: Bone Cancer. v.1.2026 – September 11, 2025. Accessed at https://www.nccn.org/professionals/physician_gls/pdf/bone.pdf on October 7, 2025.

National Cancer Institute. Ewing Sarcoma Treatment. Accessed at https://www.cancer.gov/types/bone/hp/ewing-treatment-pdq on October 7, 2025.

National Cancer Institute. Osteosarcoma and UPS of Bone Treatment. Accessed at https://www.cancer.gov/types/bone/hp/osteosarcoma-treatment-pdq on October 7, 2025.

Stacchiotti S, Gronchi A, Fossati P, et al. Best practices for the management of local-regional recurrent chordoma: a position paper by the Chordoma Global Consensus Group. Ann Oncol. 2017;28(6):1230-1242.

Stacchiotti S, Sommer J; Chordoma Global Consensus Group. Building a global consensus approach to chordoma: a position paper from the medical and patient community. Lancet Oncol. 2015;16(2):e71-e83. 

Last Revised: January 5, 2026

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