Health

Smart design for the benefit of patients with rare cancers


Paris, France, September 10, 2022 – Significant improvement in survival and progression-free response combined with reduced symptoms and better quality of life reported as a result of a single therapeutic approach. New treatment for patients with malignant, benign, but locally invasive soft-tissue tumors. By targeting the Notch pathway with the novel gamma secretase inhibitor nirogacestat, researchers from the DeFi trial obtained promising results for the first time with this approach. The results will be reported at the ESMO 2022 congress.

Samoid tumors are rare, with an incidence of 3-5 cases per million people worldwide each year (2.3). The patient has an unpredictable course and, although generally not fatal, soft tissue tumors can cause symptoms that greatly reduce quality of life. “Due to their local and aggressive growth, desmoid tumors can cause pain, deformity, and functional problems that can be a real burden,” said lead author Bernd Kasper, Cancer Center Mannheim, Germany. patient care.

The DeFi study included 142 patients with advanced samoid tumors recruited from 37 centers around the world. “This is the largest and most rigorous randomized controlled study ever conducted on this tumor type,” Kasper said. “The results showed a statistically significant improvement in progression-free survival in patients randomized to nirogacestat compared with placebo, with a 71% lower mean risk of disease progression.” Response rates were also much higher – 41% in the nirogacestat group and only 8% in the placebo group; Nearly one in ten patients (7%) showed a complete response to the agent.

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The study measured patient outcomes reported for the primary impact of desmoid tumors on quality of life. “We saw statistically significant benefits in reducing pain and symptom burden as well as in improving physical function and health-related roles and quality of life, which is not the case,” notes Kasper. really impressive. “When delivering treatments, we strive to optimize local tumor control and reduce symptom burden. But we have not previously had an approved therapy for desmoid tumors.”

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

Blay added: “The findings are changing. “We’ll be using nirogacestat as part of a treatment program for patients with desmoid tumors. But we’ll have to figure out the best way to use it,” he predicts. The remaining questions include which patients should receive this treatment, where it fits with current approaches, how responders are determined, and the optimal duration of treatment. DeFi includes patients with advanced tumors, but both Blay and Kasper say nirogacestat may also be considered in patients with pain and functional impairment.

“This is a very clever study: it demonstrated the feasibility of conducting a large, placebo-controlled trial – the highest quality clinical study to investigate the activity of a agent – in a rare cancer by recruiting patients from a multinational group including Blay added.

“The trial included patients with volume-progressing disease, providing a measurable way to select patients who needed treatment.” He added: “The success of this study further underscores the concept of bringing patients with rare cancers to reference centers where clinical studies can be performed in record time. with the potential to provide new treatments for orphans.” An increasing number of cancer patients are referred to reference centers but may still be better in some regions, improving the outlook for patients with rare cancers. (4)

Source: Eurekalert



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