Chordoma Foundation

Systemic Therapy Table

Learn more about systemic therapyTo assist chordoma patients who are considering systemic therapies, we have created a resource designed to clarify the options that are currently available through clinical trials or off-label prescription. This resource is meant to make it easier for chordoma patients and caregivers to research treatment options.

The table below includes therapies corresponding to all mechanisms of action our Medical Advisory Board (MAB) has identified as relevant to chordoma treatment. A mechanism of action is the way a systemic therapy works to attack tumor cells. In the case of targeted therapies, for instance, the mechanism of action is to block or attack a molecular target that the tumor depends on for survival. For example, the mechanism of action of an EGFR inhibitor is to block the EGFR protein, which would be referred to as the drug’s “target”.

How to use the Systemic Therapy Table:

  • We encourage patients who are considering systemic therapies to show this table to their doctors to help guide discussions about treatment options. Click here to view the table as a spreadsheet that you can download or print out to take with you to your appointments.
  • Follow the links under “Mechanism of action” to see a summary of the published supporting evidence.
  • Follow the available links under “Open clinical trial” for more information about those trials.
  • Visit the Systemic Therapy for Chordoma page to learn more about when these treatments are recommended.
Systemic therapy class Mechanism of action Drug Brand name Open clinical trial Available off-label
Targeted Therapy EGFR inhibitor afatinib Gilotrif Yes Yes
BCA101 Yes
cetuximab Erbitux  Yes Yes
gefitinib Iressa Yes (outside the U.S.)
erlotinib Tarceva Yes
lapatinib Tykerb Yes
CDK4/6 inhibitor palbociclib Ibrance Yes Yes
CDK9 inhibitor KB-0742 Yes
mTOR inhibitor sirolimus Rapamune Yes
everolimus Afinitor Yes
nab-rapamycin Yes
EZH2 inhibitor tazemetostat Tazverik Yes
PDGFR inhibitor imatinib Gleevec Yes
sorafenib Nexavar Yes
sunitinib Sutent Yes Yes
SINE/XPO1 inhibitor selinexor Xpovio Yes
VEGF inhibitor regorafenib Stivarga Yes
pazopanib Votrient Yes
bevacizumab Avastin Yes
Immune Checkpoint Inhibitor PD-1/PD-L1 inhibitor atezolizumab Tecentriq Yes
avelumab Bavencio Yes
durvalumab Imfinzi Yes Yes
nivolumab Opdivo Yes Yes
pembrolizumab Keytruda Yes Yes
Therapeutic Vaccine Anti-brachyury vaccine TAEK-VAC-HerBy Yes
Autologous tumor vaccine MVX-ONCO-1 Yes
Chemotherapy INT230-6 Yes
Antifolate pemetrexed Alimta Yes
Alkylating agent cisplatin Platinol Yes


All medical publications that contain case reports or clinical trial data on chordoma patients’ responses to systemic therapy can be accessed in this publications table.


Chordoma Foundation Medical Advisory Board recommendations

Although each patient’s situation is unique, in general, our Medical Advisory Board (MAB) recommends that patients considering systemic therapy treatment options do so in the following order of priority, in consultation with a chordoma-experienced medical oncologist:

1. Chordoma-specific clinical trials
Chordoma-specific clinical trials are trials designed specifically for chordoma patients or those that are enrolling a group of chordoma patients to a specific arm of the trial.

2. Other relevant clinical trials recommended by an experienced physician
If you are not eligible for any chordoma-specific trials, ask your oncologist about other clinical trials that may be open to you.

3. Off-label therapy with evidence of clinical benefit for chordoma patients 
While there are currently no systemic therapies approved for the treatment of chordoma, some therapies that are approved for the treatment of other cancers have shown activity in chordoma. The Systemic Therapy Table above includes information on these treatments. In some countries, doctors can choose to prescribe these to chordoma patients if they believe they will be of benefit to the patient.

The Chordoma Foundation is a resource for clinical trials. Our Clinical Trials Catalogue lists all chordoma-specific clinical trials, as well as trials that our MAB has determined are relevant to chordoma patients based on what we know about the biology of chordoma in relation to the type of drug being studied in the trial. Listings in the Catalogue include information on the treatment being studied, the locations of trial sites, and links to learn more about the trial.

Read more about clinical trials for chordoma »    Search our Clinical Trials Catalogue »


Patient Navigation ServiceRequest Help

If you or someone you love might have chordoma, Chordoma Foundation Patient Navigators are here to help you get the best care possible. Our Patient Navigators are available by email and phone, Monday through Friday from 8 AM to 5 PM EST.

Contact a Patient Navigator »


To be included in this table, the mechanism of action (MOA) must be:

1. An MOA that the Chordoma Foundation Medical Advisory Board has recommended be explored in a clinical trial for chordoma
2. Any MOA that has been studied in a Phase 2 trial for chordoma
3. The drugs/therapies listed for each mechanism are either currently being studied in a clinical trial that is enrolling chordoma patients or are available off-label

The information provided herein is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your or your loved one’s physician about any questions you have regarding your or your loved one’s medical care. Never disregard professional medical advice or delay in seeking it because of something you have read on this Website. 

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