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Medicine Made to Measure

Lead Research Organisation: University of Cambridge
Department Name: Clinical Neurosciences

Abstract

Modern medicine seeks to personalize diagnosis and treatment to the needs of individual patients. This need is heightened in particular for those individual patients where no treatments are available - and where current standard treatment development paradigms exclude to generate such. The ultimate goal of Medicine made to Measure (MMM) is to go even beyond the latest genetic medicine approaches, implementing a novel paradigm of treatment development: the field of single patient tailored antisense oligonucleotide (ASO) treatments for patients with nano-rare disease mutations. Specifically, MMM will develop core modules of a
novel "out-of-the-box", but still EMA-advised treatment development path for these patients - from omics-based theranostics, via preclinical target validation to innovative trial methodology and a pioneering ethical framework for individualised genetic therapies.
MMM provides a unique opportunity for doctoral candidates to be involved in building - and to receive training - in all core modules needed for tailored ASO treatment in Europe. This new scientific field requires a new generation of open-minded, technology-fluent and applications-oriented experts.
MMM will help educate future drug development and translational experts in adopting and disseminating such a cross-specialty approach. Our idea for this innovative training network is built on our personal experience and understanding of the importance of single patient tailored genetic treatments in the years to come. We propose a consortium of world-leading experts and partners that present with extended experience and relevant know-how in molecular biology, neurology, translational medicine, biochemistry, bioinformatics, philosophy, mathematics, regulatory science and engineering. Thus, MMM provides an exceptional platform to young, ambitious and talented researchers who like to engage in supporting the concept of single patient tailored treatments.
 
Title Extraction of extracellular vesicles from cell culture media of cellular models and from serum of patients wityh mitochondrial disease 
Description In collaboration with Susan Ozanne`s group at the Institute of Metabolic Science, University of Cambridge, we have established the method of extraction of vesicles and confirmed their mitochondrial content. 
Type Of Material Technology assay or reagent 
Year Produced 2024 
Provided To Others? No  
Impact We wrote a new grant application based on the pilot data. 
 
Description Collaboration on mitochondrial extracellular vesicles 
Organisation University of Cambridge
Department Institute of Metabolic Science (IMS)
Country United Kingdom 
Sector Academic/University 
PI Contribution In collaboration with Susan Ozanne we started to isolate extracellular vesicles and study the mtDNA in these in serum of patients with primary mitochondrial disease. We wrote a joint grant application to fund further joint research.
Collaborator Contribution Prof. Ozanne`s lab is expert in studying extracellular vesicles and helped us with the methods.
Impact We wrote a joint grant application to fund further joint research.
Start Year 2024
 
Title Deferiprone in neuroferritinopathy 
Description The clinical trial with deferiprone, an iron chelator in neurogferritiniopathy has been started in 2024. I am the PI of the trial. 
Type Therapeutic Intervention - Drug
Current Stage Of Development Refinement. Clinical
Year Development Stage Completed 2024
Development Status Under active development/distribution
Clinical Trial? Yes
Impact the trial is ogoing