A Novel Hedgehog Signalling Inhibitor for Targeted Cancer Treatment

Josep Roma.

Fundació Hospital Universitari Vall d’Hebron - Institut de Recerca (VHIR), Spain.

Cancer remains a major global health challenge, particularly in children. A biological pathway known as Hedgehog signalling is known to be overactive in many cancers, but existing targeted treatments are limited and often cause severe side effects. This project aims to develop a new type of drug that specifically targets this pathway to create a safer and more effective treatment option, particularly for children with aggressive cancers such as sarcomas.

The team has identified a compound that effectively blocks the pathway effectively in animal models. However, the compound is processed too quickly in the body, limiting its effectiveness. Researchers will use computer models to understand how it is metabolised and redesign its structure to improve stability while preserving its anti-cancer activity. The new compound will then be assessed for efficacy and safety.

The results of this project could fill a critical gap in cancer treatment, offering hope for improved survival and quality of life for children with aggressive sarcomas. The same approach might also benefit patients with other aggressive cancers linked to Hedgehog pathway overactivity.

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