Targeted Therapy for Chordoma, a Rare Cancer

According to a story from CheckOrphan, there are currently very few effective treatment options for chordoma, a rare, slow growing tumor that originates from bone. However, a recent research discovery may reveal an effective therapeutic approach that is based on the genetic characteristics of chordoma tumors that have reached an advanced stage. These findings could result in significant improvements to outcomes for patients with these rare tumors.

About Chordoma

Chordoma is a slow growing rare cancer that is thought to originate from the cells of the notochord. The tumor often appears at the base of the skull or along the spinal column. The precise of cause of this cancer is unknown. There are no known external risk factors for it. However, duplication of the brachyury protein appears to be a risk factor, and the presence of the cancer within certain families suggest that it could have a genetic component as well. Symptoms of chordoma include double vision, problems with bowel or bladder function, headaches, back pain, and numbness or weakness in the back, legs, or arms. While there are no treatments specifically approved for chordoma, surgery followed by radiation therapy is the most common approach. This cancer is somewhat resistant to radiation and requires heavy doses. To learn more about chordoma, click here.

About the Study

The team looked at data from 11 patients that had all received extensive treatment but had not seen much response; as a result, their chordoma had reached an advanced stage. The researchers found that these tumors displayed genetic abnormalities that resulted in a flawed homologous recombination (HR) process, which is normally used to repair DNA. PARP inhibitors, a targeted therapy used to treat some forms of ovarian cancer and breast cancer, has proven effective in cancers that have the same genetic abnormality.

A single patient received PARP inhibitor therapy on an experimental basis after genetic analysis determined that the patient’s genetic profile was favorable. The treatment saw the patient experience long lasting improvements and the chordoma tumor shrank in size. Once the patient began to see disease progression again, the team was able to identify the resistance mutation as affecting the PARP1 enzyme. 

The original study was posted in the academic journal Nature Communications.


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