Chordoma Foundation

Accelerating drug therapies from the bench to the clinic

Thanks to the robust scientific infrastructure built by the Chordoma Foundation over the last decade, chordoma research is thriving. With hundreds of researchers worldwide studying the disease, and a growing number of drugs entering clinical trials, we are coming into a new phase in our mission to lead the search for better treatments. The knowledge and tools we have gained in this process – from disease models to drug targets – have enabled us to begin translating basic scientific discoveries about the biology of chordoma into what we believe will become the first drug therapies for the disease.

To accelerate the translation of these discoveries from bench to bedside, the Chordoma Foundation has invested in building and growing two landmark research initiatives:

  • Our Drug Screening Program, which tests potential therapies in preclinical mouse models of chordoma, and
  • Our Clinical Trials Program, which facilitates testing the most promising treatments in clinical trials for chordoma patients.

Testing new treatments in preclinical models of chordoma

Identifying drug therapies for chordoma patients requires the quick and cost-effective assessment of potential treatments in preclinical models of the disease. It is for this reason that the Foundation launched the Drug Screening Program in 2015, operated through a partnership with South Texas Accelerated Research Therapeutics (START), a contract research laboratory that specializes in preclinical cancer drug development.

Since the program’s inception, 26 drugs have been tested in different mouse models of chordoma. The models have either been acquired from collaborating investigators or created at START with tissue collected through the Foundation’s Tumor Donation Program. Another 10 drugs are expected to be tested this year alone, with more to follow.

To date, data generated from the Drug Screening Program has resulted in two peer-reviewed publications, and is also now publicly available on our website, as well as on the digital data repository, Figshare. These two resources provide researchers worldwide with free and easy access to valuable information generated from preclinical models of chordoma in order to accelerate findings to the clinic.

Testing new treatments in patients

Data from the Drug Screening Program has also provided scientific rationale for two new chordoma-specific clinical trials:

  • Afatinib (EGFR inhibitor) – This Phase 2 trial studying the effectiveness of the drug afatinib is currently in development and expected to begin enrolling patients with locally advanced or metastatic tumors this year.
  • Palbociclib (CDK 4/6 inhibitor) – This Phase 2 trial studying the effectiveness of the drug palbociclib began enrolling patients with locally advanced and metastatic tumors in December at the National Center for Tumor Diseases in Heidelberg, Germany.

These two trials are supported by the Foundation’s Clinical Trials Program, which provides financial support, clinical trial design support, as well as patient and provider outreach and education. Currently, we are supporting a total of seven trials, three of which are already enrolling, and four are currently in various stages of planning. The Foundation’s goal is to initiate a total of 10 clinical trials by 2020.

Chordoma Foundation Clinical Trials Pipeline

Advancing new therapies to the clinic

At the end of 2017, the Foundation released two new Request for Proposals (RFPs) for new therapeutic concepts to test through the Drug Screening and Clinical Trials Programs.

Proposals are currently being sought from investigators at academic institutions, non-profit organizations and for-profit companies and are due on February 9, 2018. All proposals will be reviewed by the Foundation’s Scientific and Medical Advisory Boards, and awardees will be notified by early April.

Request for proposals

Due February 9, 2018

Clinical Trials Program RFP

Through this RFP, the Foundation aims to identify one additional trial to support in 2018, in service of our goal of opening 10 chordoma-specific trials by 2020. Funding to of up to $300,000 is available through this RFP. Please review our RFP and submit an application by February 9, 2018.

Drug Screening Program RFP

Through this RFP, the Foundation aims to identify up to 10 well-justified therapeutic concepts for evaluation in any of the six currently available and validated chordoma mouse models. Please review our RFP and submit an application by February 9, 2018.


If you have any questions, please contact our Director of Research, Joan Levy, at joan@chordoma.org