Clinical Study using 3D Printing Revolutionises Treatment of Rare Metabolic Disorders in Children

Researchers from the UCL School of Pharmacy, Prof Abdul Basit and CDT student Patricija Januskaite, have published their recent collaboration with FABRX Ltd., a UCL School of Pharmacy spinout, and the Health Research Institute of Santiago de Compostela.     

In a remarkable advancement for paediatric healthcare, researchers have utilised the potential of 3D printing technology to transform the treatment of rare metabolic disorders in children. This innovative approach allows for real-time personalisation of medication doses, overcoming the limitations of traditional pharmaceutical compounding, which often fails to deliver the necessary precision.

Researchers prepared 3D printed chewable medicines containing amino acids such as citrulline, isoleucine, valine, and combinations thereof. The efficacy and acceptability of these formulations were compared with conventional compounded medicines in six paediatric patients. Key findings from the study include:

• Efficacy: The 3D printed medicines maintained amino acid levels within target ranges as effectively as conventional medicines.
• Patient Acceptance: The inclusion of flavours such as lemon, vanilla, and peach significantly improved the palatability of the medicines, enhancing adherence to treatment protocols. Feedback gathered via a mobile app indicated high levels of acceptance and improved quality of life among the young patients.
• Adherence and Quality of Life: The personalised chewable formulations were well-received by the children, leading to better adherence to treatment schedules and overall improvements in their quality of life.

For the first time, 3D printing enabled the combination of two active ingredients in a single formulation. This reduces the number of administrations required, simplifying the treatment process and further improving adherence. By integrating novel technology into real clinical practice, healthcare providers can offer more precise, effective, and patient-friendly treatments, marking a new era in the management of such challenging conditions.

https://www.sciencedirect.com/science/article/pii/S0378517324003740?via%3Dihub

https://basitresearchgroup.org/

https://www.fabrx.co.uk/home

Prof Abdul Basit (a.basit@ucl.ac.uk)

Dr Alvaro Goyanes (a.goyanes@fabrx.co.uk) 

https://twitter.com/basitresearch