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. Now available on the Foundation’s website, the information is designed as a simple, printable table that 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 reduce the complexity of researching treatment options and help facilitate discussions with healthcare providers.
Treatments in the table are organized by mechanism of action. Each mechanism listed in the table is hyperlinked to a summary of the published supporting evidence. The “mechanism of action” of a systemic therapy refers to the way it attacks tumor cells. In the case of targeted therapies, 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.
The different types of therapies listed on the table include targeted drug therapies, immune therapies including checkpoint inhibitors, therapeutic vaccines, and an oncolytic bacteria. There are several targeted therapies and immune checkpoint inhibitors that are currently available through both clinical trials and off-label prescription, meaning that they have been approved for other types of cancer and are available to patients outside a clinical trial if prescribed by an oncologist. The therapeutic vaccines GI-6301 and MVX-ONCO-1 and the oncolytic bacteria C. Novyi NT* are only available through clinical trials at this time.
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 a list of clinical trials open to chordoma patients. If clinical trials are not possible, patients can consult with a medical oncologist about which drugs might be accessible off-label.
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.
*The C. Novyi NT trial is currently ongoing but not currently recruiting new participants.