First proven drug for aggressive desmoid tumors thanks to Leuven researchers

Desmoid tumors are rare localized tumors in connective tissue. They often occur in the abdomen, but can also appear in the neck region and in other parts of the body. The growths often cause pain and severe functional limitations. The condition is extremely rare and has an unpredictable disease course. Most patients have slow-growing desmoid tumors, but a few experience a more aggressive form. Only a small percentage of patients benefit from drug treatment, so little research has been conducted until now to find an effective drug.

A randomized phase 3 trial was able to show that treatment with the brand new drug nirogacestat, a gamma-secretase inhibitor, provided dramatic improvement compared with the placebo group. 91 percent of patients saw a partial or complete improvement in symptoms, and the risk of neoplasms growing reduced by 70 percent. The researchers also noted a statistical improvement in pain symptoms, physical functioning and overall quality of life in the patients taking the drug, while side effects remained very limited.