First proven medicine for aggressive desmoid tumours

9 March 2023
Leuven researchers collaborated on a brand new gamma-secretase inhibitor for rare connective tissue tumours. "It is the first time that we were able to find a medicine against aggressive desmoid tumours with convincing clinical evidence."

Desmoid tumours are rare local tumours in the connective tissue. They're often abdominal, but they can also appear in the neck area and in other parts of the body. The tumours often cause pain and severe functional limitations. The disorder is extremely rare and progresses unpredictably. Most patients have slow-growing desmoid tumours but a few face a more aggressive form. Only a small percentage of patients benefit from drug treatment, so there has been little research into effective medication until now.

Improvement in 91%

A randomised phase 3 study could show that treatment with the brand new medicine nirogacestat, a gamma secretase inhibitor, resulted in a spectacular improvement in comparison to the  placebo group. 91 per cent of patients saw partial or complete improvement of their complaints and the risk of an increase of the tumours was reduced with 70%.  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.

Up to now there was very little research into efficient medication
prof. dr. Patrick Schöffski

Prof. dr. Patrick Schöffski, head of the general medical oncology department at UZ Leuven and involved in the trial: “Desmoid tumours are very different from traditional tumours. They won't kill you and they cannot metastasise to other places. The disease can remain stable for a long time and can also sometimes spontaneously disappear. Patients that are confronted with an aggressive type suffer a lot of pain symptoms and especially disturbing growths that limit their freedom of movement. Their daily or social functioning at work or at school often becomes very difficult. For them this new targeted oral treatment is excellent news. It is the first time that we have been able to find medication against aggressive desmoid tumours with convincing scientific evidence: based on this study, nirogacestat will definitely become the new standard approach for people with desmoid tumours in need of treatment with medication.”

The study was published in the New England Journal of Medicine and will be an impetus for the FDA and EMA to register the drug.

Last edit: 12 January 2024