By delivering functional bowl reconstruction to patients with SBS through an autologous tissue engineering strategy, INTENS will generate the scientific basis to initiate a clinical trial in patients with intestinal failure (IF) due to SBS.
The potential benefit of this innovative treatment is extremely relevant and targets infants with SBS following bowel atresia, gastroschisis or necrotizing enterocolitis (NEC), as well as children affected by extensive intestinal resection from disorders such as midgut volvulus and mesenteric ischaemia. This application has the long-term potential to increase the survival rate of children with SBS, improve quality of life for patients and their families, decrease long-term disability and reduce treatment costs for families and society.
INTENS will have an impact on:
- Advance the development of new therapeutic options for patients living with the rare disease Short Bowel Syndrome:
We aim, within the 5-year time frame, to prepare a tissue engineered intestine and be ready to start a first in-man clinical trial in patients with SBS and IF with the ultimately aim of delivering a definitive therapy for the rare disease SBS.
The potential benefit of this innovative treatment is large as it can be applicable not only to infants with SBS as a consequence of bowel atresia, gastroschisis or necrotizing enterocolitis, but also to children and adults, who have developed SBS as a result of extensive intestinal resection from various intestinal disorders.
- Contribute towards International Rare Diseases Research Consortium (IRDiRC) objectives:
Furthermore, we aim to contribute to the IRDiRC objectives by coordinating and networking patient registries, changing practice, working towards improving the regulatory framework to facilitate the development of a novel therapy and fostering industry-academia collaborations.