Treating a rare cancer using a precision medicine approach

Genomic analysis visualisation credit Tetiana Lezunova
Cambridge clinical researchers have, for the first time, shown that the drug nirogacestat can be effective in treating a rare type of cancer arising from the windpipe (trachea).

There are no established treatment options for tracheal adenoid cystic carcinoma once it has spread beyond the windpipe, and standard chemotherapy is ineffective in this disease.

Using DNA sequencing of a patient’s cancer, the researchers identified what changes were driving the cancer and used a personalised, precision medicine approach to successfully target this using the experimental drug nirogacestat.

Dr Gary Doherty (Thoracic Cancer Programme Member, Consultant Medical Oncologist and Clinical Lead for Cancer at the East Genomic Laboratory Hub) was the senior author of the research which is published in the journal JCO Precision Oncology.

Dr Doherty said: “I hope that this important proof-of-principle will now lead to clinical trials to improve outcomes for patients with this cancer.

“In future, offering broad tumour sequencing within the NHS Genomic Medicine Service will enable more patients with any cancer, including other rare cancers, to benefit from such a personalised medicine approach.”

Kieran, R et al Response of NOTCH1-activated tracheal adenoid cystic carcinoma to the gamma-secretase inhibitor nirogacestat JCO Precision Oncology no. 5 (2021) 1579-1583. Published online October 6, 2021 DOI: 10.1200/PO.21.00228 

6 Oct 2021