Chordoma Foundation

New results from our Drug Screening Program presented at leading international cancer conference

Our Drug Screening Program enables researchers and companies to test promising drugs in chordoma cell and mouse models at a fraction of the time and cost of doing it themselves, and helps generate the preclinical data packages necessary to initiate new, well-justified clinical trials as quickly as possible. And this week, we were pleased to be co-authors on two posters sharing new drug screening data presented at the annual meeting of the American Association for Cancer Research (AACR):

  • In collaboration with Sphaera Pharma, we tested a drug that targets the PI3K/mTOR pathway, one of the most commonly upregulated targets in chordoma. Experiments in mouse models suggested that the drug could significantly inhibit chordoma tumor growth on its own and potentially enhance the efficacy of palbociclib, a CDK4/6 inhibitor in clinical trials for chordoma, when tested in combination.
  • With Kronos Bio, we studied a drug that aims to inhibit CDK9, which is thought to play a crucial role in stopping chordoma cell proliferation by suppressing brachyury, the Achilles’ heel of chordoma. The data demonstrated dose-dependent antitumor activity in chordoma mouse models, as well as increased efficacy when the drug was combined with afatinib, an EGFR inhibitor in clinical trials for chordoma.

Both sets of results could provide rationale for future chordoma clinical trials. And they exemplify the efficient progress made possible by our Drug Screening Program, which is a powerful tool enabling companies and investigators to bring their expertise and resources to bear to pursue better therapies for chordoma patients.

In the year to come, we’ll continue our collaborative efforts to evaluate promising chordoma treatment concepts, with a focus on drugs and combinations of drugs capable of shrinking tumors, rather than just stopping their growth. Moreover, with the recent opening of CF Labs — the first laboratory 100% dedicated to chordoma research — we’ll further accelerate the pace at which we and our academic and industry partners can get better treatments to chordoma patients.

Additionally, our grantees at the Institute of Cancer Research and the Broad Institute presented a poster on their impressive body of work defining the effects of direct and indirect inhibition of brachyury in chordoma.

The AACR meeting also provided an opportunity to highlight the Foundation’s progress and lessons learned in two other arenas. In one session, we shared how our collaboration with the National Cancer Institute on a natural history study of chordoma is advancing clinical research. And, in a panel focused on successful therapeutic development for rare cancers, we presented recent milestones toward drugs that target brachyury.

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