Chordoma Foundation

New clinical trial opens testing targeted cancer therapy, palbociclib

A new clinical trial has opened for chordoma patients with locally advanced or metastatic tumors testing the effectiveness of a targeted cancer drug called palbociclib. Also known by its trade name, Ibrance®, palbociclib works by blocking two key proteins – cyclin-dependent kinase 4 (CDK4) and cyclin-dependent kinase 6 (CDK6) – which are believed to be involved in driving the growth of chordoma tumors. It is currently approved for the treatment of certain types of breast cancer. The goal of this research study is to evaluate how effective palbociclib is at slowing or stopping the growth of chordoma tumors.

Rationale for the trial

The majority of chordoma tumors have a loss of the CDK2NA gene. This gene encodes a protein called p16, which plays a critical role in controlling cell growth. When p16 is lost, proteins called CDK4 and CDK6 are able to promote the growth of tumor cells in an uncontrolled manner. Therefore, researchers believe that blocking CDK4/6 may stop the growth of chordoma cells. Indeed, drugs that inhibit the activity of the CDK4 and CDK6 proteins have been shown to be effective in chordoma cell lines and mouse models. Building on of this preclinical evidence, the palbociclib study aims to determine whether this particular CDK4/6 inhibitor can have the same effect in humans, and either shrink or stop the growth of chordoma tumors in patients with recurrent or metastatic disease that have loss of the p16 protein.

About the trial

This study is now open at the National Center for Tumor Diseases (NCT) in Heidelberg, Germany, under the leadership of Principal Investigator Prof. Dr. Stefan Fröhling and the Scientific Coordinator Prof. Dr. Richard F. Schlenk. NCT is the first of three study sites to begin enrolling patients. In the coming months, two additional study sites will open at the University of Ulm and the University of Duisburg-Essen.

It is designed specifically for chordoma patients whose tumors have come back in the same area (locally recurrent), have spread to other parts of the body (metastatic), and have not responded to or are no longer responding to treatment with a tyrosine kinase inhibitor such as imatinib or erlotinib.

Treatment plan

  • Treatment cycles last for 28 days.
  • Participants take a 125 mg oral dose of palbociclib daily for 21 days, followed by a break for 7 days.
  • These cycles continue for a minimum of 6 months unless there is tumor growth, intolerable side effects are experienced, or the participant leaves the study independently.
  • At the end of the initial 6-month period, participants who have received benefit from the treatment will be offered continuation of treatment with agreement from their treating physician and the principal investigator and the scientific coordinator.
  • Check-up exams will be performed every 4 weeks and imaging done every 12 weeks.
  • After completion of the treatment, a follow-up period of 6 months is planned with visits to the trial center every 3 months.

All exams, treatments, and evaluations are provided at the trial site where the patient enrolled in the study. Participants not local to the trial sites MUST be willing and able to travel to their trial site for all visits required by the trial.

Additional information for patients

For more information about the study, eligibility requirements, and answers to frequently asked questions, visit the trial information page on our website. Full eligibility requirements can be found at NCT03110744.

To enroll or to find out if you qualify for this trial, contact the scientific coordinator, Prof. Dr. Richard F. Schlenk at

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


Medical information provided by the Chordoma Foundation in any medium is for informational and educational purposes only. After reading content from the Chordoma Foundation, review the information carefully with your physician. Information from the Chordoma Foundation is not a substitute for consultations with qualified health care professionals who are familiar with your medical conditions and needs. The Chordoma Foundation strongly recommends that care and treatment decisions be made in consultation with your physician or other qualified health care professionals who are familiar with your specific health situation.

Pin It on Pinterest

Share This
Comodo SSL