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First proven drug for aggressive desmoid tumors thanks to Leuven researchers

13 March 2023

Leuven-based researchers collaborated on brand new gamma-secretase inhibitor for rare connective tissue tumors

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.

Prof. Dr. Patrick Schöffski, head of general medical oncology at UZ Leuven and involved in the study: "Desmoid tumors are very different from traditional cancerous tumors. You don't die from them and they cannot spread to other places. The disease can remain stable for a long time and sometimes go away spontaneously. But patients who deal with an aggressive form suffer a lot of pain and especially bothersome growths that limit their movement. Their daily or social functioning at work or school often becomes very difficult. For them, this new targeted oral treatment is excellent news. It is the first time we have found a drug against aggressive desmoid tumors with convincing scientific evidence: based on this study, nirogacestat will certainly become the new standard approach for people with desmoid tumors in need of drug treatment."

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

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