Chordoma Foundation

New clinical trial testing targeted therapy, afatinib, opens in Europe


Afatinib clinical trial for chordoma
This is a derivative of “Leiden University Medical Center” by Dmflan
and is licensed under CC BY 3.0

A new Phase 2 clinical trial testing the effectiveness of a targeted drug therapy called afatinib is now open to chordoma patients 18 years or older with recurrent, locally advanced, or metastatic tumors.

Afatinib works by blocking a protein known as the Epidermal Growth Factor Receptor (EGFR), which drives the growth of chordoma tumor cells. Afatinib belongs to a class of drugs called EGFR inhibitors, which have been used to treat non-small-cell lung cancer, pancreatic cancer, breast cancer, and colon cancer.

This research study is currently enrolling patients at Leiden University Medical Center (LUMC) in The Netherlands, under the direction of principal investigator (PI) Dr. Hans Gelderblom. In the coming months, it will also open at Istituto dei Nazionale Tumori (INT) in Milan, Italy, under the direction of PI Dr. Silvia Stacchiotti, and at University College London in England, under the direction of PI Dr. Sandra Strauss.

The Chordoma Foundation is proud to support this clinical trial with a $300,000 research grant, made possible by your contributions.

Rationale for the trial

Epidermal Growth Factor Receptor (EGFR), a protein found on the surface of certain cells throughout the body, ordinarily helps to regulate cell growth and plays a role in wound healing. However, in certain cancers, including most chordomas, EGFR becomes overactive, leading the cancer cells to multiply out of control.

This drug is relevant to chordoma patients because the majority of chordoma tumors express the EGFR protein, and afatinib has proven to be more effective than other EGFR inhibitors at slowing or stopping the growth of chordoma cells and tumors in mice. In mouse model tests conducted through our Drug Screening Pipeline last year, afatinib slowed the growth of some tumors and caused others to shrink considerably.

Based on this evidence, the Chordoma Foundation’s Medical and Scientific Advisory Boards endorsed the concept of a clinical trial testing whether afatinib can have the same effect in humans, slowing or stopping the growth of chordoma tumors in patients with recurrent or metastatic disease.

About this trial

This trial is designed specifically for adult chordoma patients (18 years and older) whose tumors have come back in the same area, are locally advanced, or have spread to other parts of the body.

All study participants will take a 40 mg oral dose of afatinib daily. This will continue in 4-week cycles until there is tumor growth or the participant chooses to leave the trial. Check-up exams will also be performed every 4 weeks, and imaging will be done every 12 weeks.

All doses of afatinib will be provided at no cost to participants, and a portion of the cost of the check-ups and tests required by the study will be covered as well. Study participants and their insurance carriers will be responsible for imaging costs, as this is considered standard of care.

All exams, treatments, and evaluations are provided at the site where the patient enrolled in the study. Participants not local to any of the three European trial sites MUST be willing and able to travel to their trial site for all visits required by the trial. Participants from out of town are responsible for any travel or lodging expenses necessary to make the required clinic visits every 4 weeks.

Who can participate

Patients must meet the following criteria to be eligible:

  • Confirmed diagnosis of a chordoma tumor that expresses the EGFR protein
    (Note: Expression of EGFR protein is different than mutations in the EGFR gene. The EGFR protein is often highly expressed in chordoma tumors, whereas EGFR gene mutations are almost never found in chordomas. This means that patients can be eligible for this trial even if EGFR mutations are not found in molecular profiling tests such as FoundationOne. Expression of the EGFR protein will be determined by a pathologist at one of the trial sites.)
  • Recurrent, locally advanced, or metastatic disease confirmed by imaging
  • Progression of disease documented in last 6 months
  • No major surgery less than 4 weeks before beginning treatment
  • Age 18 years or older
  • No major laboratory abnormalities
  • Willing and able to travel to the enrolling study site for all treatment and follow-up visits

The first step to determining eligibility is to schedule a clinic visit with a member of the trial team to discuss the screening and enrollment process. Currently Leiden University Medical Center is the only site enrolling patients. Interested patients should contact Dr. Astrid Lipplaa at or Prof. H. Gelderblom at to learn how to register as a patient at LUMC and schedule an appointment.



For more information about the study, eligibility requirements and answers to frequently asked questions, visit the trial information page on our website. Full eligibility criteria can be found at

A Chordoma Foundation Patient Navigator is also available to help answer any other questions you may have.

The Chordoma Foundation maintains a Clinical Trials Catalogue on our website which lists all chordoma-specific trials, as well as all other trials deemed relevant to chordoma patients by our MAB (it currently includes 17 trials worldwide).

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