Chordoma Foundation

Systemic therapy

To assist patients who are considering systemic therapies, we have created a new resource designed to clarify the options that are currently available through clinical trials or off-label prescription. The table below includes therapies corresponding to all mechanisms of action our Medical Advisory Board (MAB) has identified as relevant to chordoma treatment. This resource is meant to make it easier for chordoma patients and caregivers to research treatment options and talk with their healthcare providers about those options.

Treatments in the table are organized by mechanism of action. The mechanism of action of a systemic therapy refers to the way it attacks tumor cells. In the case of targeted therapies, for example, the mechanism of action is to block 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.

Click on the hyperlinks under mechanism of action to see a summary of the published supporting evidence for that mechanism in chordoma treatment. Therapies for which there are open clinical trials are linked to more information about those trials.

Systemic therapy class Mechanism of action Drug Brand name Open clinical trial Available off label
Targeted Therapy EGFR inhibitor afatinib Gilotrif Yes 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
mTOR inhibitor sirolimus Rapamune Yes
everolimus Afinitor Yes
PI3K inhibitor taselisib Yes
EZH2 inhibitor tazemetostat Yes
PDGFR inhibitor imatinib Gleevec Yes
sorafenib Nexavar Yes
sunitinib Sutent Yes Yes
VEGF inhibitor regorafenib Stivarga Yes 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
M7824 Yes
FAZ053 Yes
PDR001 Yes
Therapeutic Vaccine Anti-brachyury vaccine GI-6301 Yes
BN-Brachyury Yes
Autologous tumor vaccine MVX-ONCO-1 Yes
Oncolytic Bacteria Bacteriolysis C. Novyi NT Yes (on hold)


You may also view this systemic therapy table as a Google Sheet here. The Google Sheet will allow you to download and/or print the table.

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

When possible, our MAB recommends that patients who have advanced or metastatic disease first consider chordoma-specific clinical trials, followed by trials relevant to chordoma patients. Visit the Clinical Trials Catalogue on our website for more information about clinical trials that are currently open to chordoma patients. If clinical trials are not possible, patients can consult with a medical oncologist about the option of off-label therapies.

We encourage patients who are considering systemic therapies to show this table to their doctors to help guide discussions. Additionally, CF Patient Navigators are available to help answer questions about systemic therapies, clinical trials, or off-label prescriptions.


Criteria for inclusion in this table:

1. Mechanism that the Chordoma Foundation Medical Advisory Board has recommended be explored in a clinical trial for chordoma.

2. Any mechanism that has been studied in a Phase 2 trial for chordoma.

3. The therapies listed for each mechanism are either currently being studied in a clinical trial that is enrolling chordoma patients or are available off-label.

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