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Smart design to benefit rare cancer patients

Paris, France, 10 September 2022 – Significant improvements in progression-free survival and response rates with fewer symptoms and better quality of life have been reported as the results of a new treatment approach for patients with desmoid tumors, which are benign, But there are locally aggressive and aggressive. soft tissue tumours. By targeting the Notch pathway with the novel gamma secretase inhibitor nirogacetate, researchers in the DeFi trial have achieved positive results for the first time with this approach. The results are reported at ESMO Congress 2022.

Desmoid tumors are rare, with an incidence of 3–5 cases per million people worldwide each year (2,3). Patients have an unpredictable disease course and, although usually not malignant, soft tissue tumors can cause symptoms that greatly impair quality of life. “Because of localized and aggressive growth, desmoid tumors can cause pain, deformity and functional problems that can be a real burden for patients,” said lead author Bernd Kasper, from the Mannheim Cancer Center, Germany.

The Defy study included 142 patients with progressive desmoid tumors who were recruited from 37 centers around the world. “This is the largest and most rigorous randomized controlled study to date in this tumor type,” Kasper explained. “The results showed a statistically significant improvement in progression-free survival in patients randomized to nirogastat compared to the placebo group, which had a 71% lower risk of disease progression on average.” Response rates were also very high – 41% in the nirogastat arm and only 8% in the placebo arm; About one in ten patients (7%) showed a complete response with the agent.


The patient-measured study reported results because of the major impact of desmoid tumors on quality of life. “We saw a statistically significant benefit in reducing the burden of pain and symptoms and in improving physical and role functioning and health-related quality of life, which was really impressive,” said Kasper. “In providing treatment we try to optimize local tumor control and reduce the burden of symptoms. But we have not previously had an approved therapy for desmoid tumors. Drugs to treat patients with this disease in this study has the potential to lead to earlier registration of

“This is a unique study, very important in many aspects,” said Jean-Yves Bleu, the Cancer Center in Lyon, France, who was not involved in the study. “The results show for the first time benefit with a novel treatment with a new mode of action in patients where treatment options are currently limited.” The Notch signaling pathway is implicated in the development and progression of many types of tumors.

“The findings are changing practice,” Blay said. He predicted: “We are going to use nirogacetate as part of the treatment arsenal for patients with desmoid tumors. But we have to figure out how to best use it.” Remaining questions include which patients should be offered this treatment, where it fits with respect to current approaches, how to identify responders and the optimal duration of treatment. DeFi included patients with tumor progression, but both Blay and Kasper said nirogacetate may also be considered in patients with pain and impaired functioning.

“It was a very smart study: it demonstrated the feasibility of carrying out a large, placebo-controlled trial – the highest quality clinical study to examine the activity of an agent – ​​in a multinational group of patients. in a rare cancer by recruiting reference centers and this demonstrated the importance of targeting the right patients with the right drug when designing clinical trials,” Blay said.

“The trial largely included patients with progressive disease, which provided a measurable way to select patients who needed treatment.” He added: “The success of this study places even greater emphasis on the concept of referring patients with rare cancers to reference centers, where clinical studies with the potential to deliver new treatments to patients with orphan diseases can be conducted in record time. can be accomplished.” The number of cancer patients being referred to reference centers is increasing, but it may still be better in some areas, improving the outlook for patients with rare cancers. (4)

Source: Eurekalert

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