We are excited to share a promising milestone in the Foundation-supported Phase 2 BN-Brachyury trial. Yesterday, we received confirmation from trial sponsor Bavarian Nordic that one of the first chordoma patients treated on the trial has had a partial response (tumor shrinkage) that met the pre-defined threshold of activity for the first stage of the trial.
As a result, recruitment will be expanded to include an additional 19 patients with locally advanced or metastatic chordoma. New participants will join the first 10 patients in the trial, who will continue to be treated and evaluated, bringing the total enrollment up to 29.
Trial sites currently enrolling new patients include:
- Massachusetts General Hospital – Boston (Principal Investigator Greg Cote)
- Mayo Clinic – Jacksonville, Florida (Principal Investigator Steven Attia)
- Mayo Clinic – Rochester, Minnesota (Principal Investigator Scott Okuno)
- Mayo Clinic – Scottsdale, Arizona (Principal Investigator Mahesh Seetharam)
Though only a single response so far, this initial sign of activity provides further evidence that generating an immune response against brachyury, a protein which is highly expressed in chordoma cells, can enable the immune system to shrink tumors. It also adds to the growing body of evidence that immunotherapies may have a role to play in the treatment of chordoma and highlights the importance of research in this area.
Moreover, the fact that this trial has moved forward so quickly demonstrates the feasibility of conducting well-designed clinical trials for chordoma and sets a precedent that could encourage investment in future trials.
“We are encouraged by the rapid progression of this trial, which may offer final results sooner than anticipated and hope this initial finding is confirmed in a larger number of patients and that BN-Brachyury may improve treatment options for patients with chordoma.”
– Paul Chaplin, President and Chief Executive Officer of Bavarian Nordic.
Much credit for this rapid progress goes to principal investigator and CF Medical Advisory Board member Dr. Greg Cote at Massachusetts General Hospital along with his colleagues from the Mayo Clinic who worked diligently to open the trial and enroll patients.
As the first chordoma therapy to be granted an orphan drug designation by the US Food and Drug Administration (FDA), BN-Brachyury has the potential to pave the way for the development of future chordoma treatments.
Nineteen additional patients will now have the opportunity to participate in the trial. For those interested in considering this option, Chordoma Foundation Patient Navigators are available to help answer questions and provide more information.
The primary goal of the trial is to increase the objective response rate from less than 5% with radiation alone (based on historical data) to at least 14% with radiation plus the BN-Brachyury vaccine. Additional measures of success will include stopping tumor growth for an extended period of time (progression-free survival) and symptomatic improvement.
We are optimistic that as time goes on additional patients will benefit from this trial. Meanwhile, we are grateful to all of the scientists at the National Cancer Institute who created this vaccine, Bavarian Nordic for investing in this trial for chordoma, all of the study investigators and staff for their dedication to making this trial a success, and, most importantly, the patients whose participation is helping advance the science of chordoma treatment.
We will continue to provide additional updates here and on social media as more information becomes available.