Chordoma Foundation

New clinical trial options available for chordoma patients

Introducing a new treatment pathway for patients with advanced or recurrent chordoma

Historically, treatment options for patients with residual, recurrent, or advanced chordoma have been limited and inadequate. Several approved drugs such as imatinib (Gleevec) have shown some modest benefit for chordoma patients, but they are not curative and only slow progression of the disease for some patients and for a limited period of time. Recently, as research has revealed more about the biology of chordoma – including its molecular drivers and key vulnerabilities – new and potentially more effective treatment options have begun to emerge.

Researchers are now testing the safety and efficacy of these new treatment options in a series of ongoing and planned clinical trials. These trials not only offer individuals battling chordoma today the opportunity to benefit from the latest science about our disease, they also contribute to the knowledge that can guide therapy (and, hopefully, cures) for years to come.

In light of the availability of clinical trials testing new therapies which researchers have reason to believe could be better than standard therapies, our Medical Advisory Board (MAB) has recommended a new pathway for patients with advanced or recurrent chordoma to pursue when seeking treatment:

The Foundation is working to initiate a pipeline of new trials specifically for chordoma.

As a result of intensive research and widespread collaboration, the Foundation has been able to put mechanisms in place to initiate and support clinical trials for highly promising treatments identified by our research partners. We have worked hard to eliminate barriers that typically hinder clinical trials for patients with rare cancers, ensuring that scientifically-sound treatment approaches are tested in chordoma patients as quickly as possible.

Of the seven trials currently included in our Clinical Trials Program, one (GI-6301) is already enrolling patients; the other six are in varying stages of implementation. These trials have all undergone a rigorous vetting and approval process by our MAB and Scientific Advisory Board (SAB).

We remain focused on cutting-edge research, and continue to invite trial concept proposals from companies and academic investigators, with the goal of initiating 10 clinical trials by 2020.


The MAB has also identified a number of other open trials that are relevant to chordoma patients right now.

These trials have been added to our Chordoma-Relevant Clinical Trials Catalogue, featuring straightforward descriptions of the treatment being tested, eligibility criteria, relevance to chordoma patients, trial location(s), and contact information.


We will be updating both of these lists – the Chordoma Foundation Clinical Trials Pipeline and the Chordoma-Relevant Clinical Trials Catalogue – in real-time as new information becomes available, so be sure to check back for updates.

If you cannot find a clinical trial that is right for you, off-label therapy may still be an option.

Contact our Patient Navigators for information on medical oncologists who have experience treating chordoma patients with off-label drugs.


Questions about open trials? Contact a Chordoma Foundation Patient Navigator.

Want to propose a new trial concept? Contact