Formulating 3D printed tablets containing antioxidants for the management of patients with rare metabolic diseases

Lead Research Organisation: University College London
Department Name: School of Pharmacy

Abstract

Patients with rare metabolic diseases collectively form a large group, but are stratified into heterogeneous subsets, each being small and with a specific genetic defect. Currently, the majority (~95%) of rare diseases still have no MHRA or FDA approved treatment, making their formulation development crucial in the pharmaceutical field (1). As a result, there is a large patient group whose clinical needs are not being adequately addressed by the current approach to pharmaceutical product development. Rare metabolic diseases in adults require tightly controlled diets with amino acid supplementation, which require supplementation with antioxidants such as coenzyme A (CoA), riboflavin, or biotin (2). This project will utilise the flexibility offered by 3D printing to formulate patient-specific and tailored dose medicines to deliver antioxidant to rare metabolic disease patients.

The advent of 3D printing offers a new manufacturing route to development and production of dosage forms for these patients; small production batches of medicines with doses and dose combinations personalised to the specific needs of individual patients can be produced. In this project, we will design, engineer and manufacture 3D printed oral dosage forms for treating rare metabolic diseases. Once diagnosed, treatment of a particular rare metabolic disease requires careful supplementation of the diet with a specific number and concentration of antioxidant; immediately following diagnosis titration may be needed to determine the optimal concentration of antioxidants required. The 3DP approach is therefore ideal for creating dosage forms for individual patients; the required antioxidants can be formulated into chewable tablets and manufactured on a patient-by patient basis. Thus, the main objective of this work is to develop a printable dosage form that can be configured to deliver numerous antioxidant blends and concentrations for patients with rare metabolic diseases.

References:
(1) Bienstock, R.J., Data Sharing Advances Rare and Neglected Disease Clinical Research and Treatments. ACS pharmacology & translational science, 2019. 2(6): p. 491-496.
(2) Boyer, S.W., L.J. Barclay, and L.C. Burrage, Inherited Metabolic Disorders: Aspects of Chronic Nutrition Management. Nutrition in clinical practice : official publication of the American Society for Parenteral and Enteral Nutrition, 2015. 30(4): p. 502-510.

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Studentship Projects

Project Reference Relationship Related To Start End Student Name
EP/S023054/1 01/10/2019 31/03/2028
2425913 Studentship EP/S023054/1 28/09/2020 27/09/2024 Patricija Januskaite