Development of First Immunotherapy to Treat Chordoma

Cancer vaccines harness the immune system to identify and destroy cancer cells, and are a promising new approach to fighting cancer. In contrast to preventative vaccines, cancer vaccines identify antigens from cancer cells and immunize cancer patients against those antigens to stimulate the body’s immune cells to attack and kill the cancer cells. The National Cancer Institute (NCI) has developed investigational cancer vaccines that induce a specific, targeted immune response against cancer cells expressing the brachyury protein. The discovery may be the first medical treatment for chordoma, a rare cancer with no alternative medical therapy. 

Brachyury is a type of genetic on-switch, also known as a transcription factor. It is a driver of a process associated with cancer progression and resistance to therapy. Brachyury is an attractive vaccine target because it is not generally found in normal tissues, but is abnormally found in many cancers and chordoma, a difficult-to-treat bone cancer. When brachyury is expressed in tumor cells, it enhances their invasiveness and induces resistance to chemotherapy and radiation.

Before NCI’s discovery, brachyury was deemed “undruggable” because of challenges associated with developing therapies targeting transcription factors. The first NIH patent application covering brachyury as a cancer vaccine was filed in 2007. Since then, the invention has attracted significant commercial interest. 

NCI is currently developing brachyury vaccines through Cooperative Research and Development Agreements (CRADAs) and license partnerships with GlobeImmune, Inc.; Bavarian Nordic; and Etubics Corporation, respectively. These collaborations led to the rapid translation of investigational therapeutic vaccines with the potential to revolutionize how researchers and physicians treat many cancers.

NCI’s collaborations led to the creation of new intellectual property and licensing activities. Currently, there are several issued patents and pending patent applications. NCI’s commitment to collaborate with multiple partners is helping to exploit the discovery’s full potential. The rapid translation and clinical development of brachyury vaccines has been well-served by careful management of a complex technology transfer process. 

Awardees

NIH - National Cancer Institute:

• Dr. James Gulley
• Dr. Christopher Heery
• Dr. Claudia Palina
• Dr. Jeffrey Schlom
• Kevin Brand (formerly of NIH)
• Dr. Michael Pollack

NIH - Office of Technology Transfer:

• Dr. Kevin Chang
• Dr. Sabarni Chatterjee
• Mojdeh Bahar (formerly of NIH)