Therapeutic RNA delivery to the bone marrow (TRADBow)
This project will develop targeted nanoparticles to treat childhood leukaemia in the bone marrow
Innovative RNA Therapy for Childhood Leukemia Through Public-Private Collaboration
This project aims to develop a novel RNA-based therapy to treat childhood acute myeloid leukemia (AML) by targeting the bone marrow directly. Current RNA therapies are mainly taken up by the liver, making it difficult to treat diseases in other organs. In this public-private partnership, the Princess Máxima Center collaborates with Dutch biotech companies Mercurna and RiboPro to develop targeted lipid nanoparticles (LNPs) that deliver RNA treatments specifically to the bone marrow. This innovation could significantly improve leukemia treatment outcomes and pave the way for treating other bone marrow-related diseases.
Addressing a Critical Health Challenge
Paediatric AML remains a life-threatening disease, with 25% of affected children not surviving. Current treatments are aggressive, leading to severe side effects that impact survivors’ quality of life. New and targeted therapies are urgently needed to improve survival rates while minimizing harm. Beyond the individual impact, this innovation could reduce the long-term healthcare burden, as AML treatments often lead to chronic health issues. By improving survival and reducing treatment side effects, this approach has the potential to save millions of disability-adjusted life years (DALYs) and significantly lower healthcare costs.
A New Approach to RNA Drug Delivery
Traditional nanomedicine struggles with liver retention, limiting its use for non-liver diseases. This project aims at developing LNPs that evade the liver and efficiently reach the bone marrow. The therapy will work by (i) eliminating leukemia cells and (ii) modifying the bone marrow environment to enhance the body's immune response against cancer. The same approach could be expanded to treat other bone marrow disorders, such as myelodysplastic syndrome (MDS).
Expected Outcomes
This research will establish a new RNA therapy for AML, providing a blueprint for RNA-based treatments targeting other organs. Success in this project could revolutionize drug delivery and set new standards for treating various cancers and metabolic diseases.
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