Overview
Dr Pacitti graduated with a BSc in Pharmacology before obtaining his PhD in Cell biology and Genetics from St George’s, University of London. After his PhD, he continued his work as postdoctoral research assistant at St George’s University of London, where he investigated the role of thymidine phosphorylase in CNS maintenance and collaborated on progressing a European clinical trial for a novel enzyme replacement therapy for Mitochondrial Neurogastrointestinal Encephalomyopathy (MNGIE). His research background is therefore in rare mitochondrial and neurological disorders. He is deeply interested in inflammation in the context of lung and CNS diseases, with a particular interest in disease modelling using complex in vitro systems such as 3-D cultures and organoids.
He has now joined Professor Matt Whiteman as a Postdoctoral Research Fellow in Inflammatory Pharmacology, working on mitochondria-targeted H2S donors against inflammation.
Links
Qualifications
Research group links
Research
Research interests
Mitochondrial biology, inflammation, iPSCs, disease modelling, gene editing, transcriptomics, organoids, respiratory diseases.
Research projects
Current Projects:
- Currently investigating the efficacy of mitochondria-targeted H2S donors in lung disease.
Publications
Key publications | Publications by category | Publications by year
Publications by category
Journal articles
Levene M, Bain MD, Moran NF, Nirmalananthan N, Poulton J, Scarpelli M, Filosto M, Mandel H, MacKinnon AD, Fairbanks L, et al (2019). Safety and Efficacy of Erythrocyte Encapsulated Thymidine Phosphorylase in Mitochondrial Neurogastrointestinal Encephalomyopathy.
J Clin Med,
8(4).
Abstract:
Safety and Efficacy of Erythrocyte Encapsulated Thymidine Phosphorylase in Mitochondrial Neurogastrointestinal Encephalomyopathy.
Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE) is an ultra-rare autosomal recessive disorder of nucleoside metabolism that is caused by mutations in the nuclear thymidine phosphorylase gene (TYMP) gene, encoding for the enzyme thymidine phosphorylase. There are currently no approved treatments for MNGIE. The aim of this study was to investigate the safety, tolerability, and efficacy of an enzyme replacement therapy for the treatment of MNGIE. In this single centre study, three adult patients with MNGIE received intravenous escalating doses of erythrocyte encapsulated thymidine phosphorylase (EE-TP; dose range: 4 to 108 U/kg/4 weeks). EE-TP was well tolerated and reductions in the disease-associated plasma metabolites, thymidine, and deoxyuridine were observed in all three patients. Clinical improvements, including weight gain and improved disease scores, were observed in two patients, suggesting that EE-TP is able to reverse some aspects of the disease pathology. Transient, non-serious adverse events were observed in two of the three patients; these did not lead to therapy discontinuation and they were managed with pre-medication prior to infusion of EE-TP. To conclude, enzyme replacement therapy with EE-TP demonstrated biochemical and clinical therapeutic efficacy with an acceptable clinical safety profile.
Abstract.
Author URL.
Publications by year
2019
Levene M, Bain MD, Moran NF, Nirmalananthan N, Poulton J, Scarpelli M, Filosto M, Mandel H, MacKinnon AD, Fairbanks L, et al (2019). Safety and Efficacy of Erythrocyte Encapsulated Thymidine Phosphorylase in Mitochondrial Neurogastrointestinal Encephalomyopathy.
J Clin Med,
8(4).
Abstract:
Safety and Efficacy of Erythrocyte Encapsulated Thymidine Phosphorylase in Mitochondrial Neurogastrointestinal Encephalomyopathy.
Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE) is an ultra-rare autosomal recessive disorder of nucleoside metabolism that is caused by mutations in the nuclear thymidine phosphorylase gene (TYMP) gene, encoding for the enzyme thymidine phosphorylase. There are currently no approved treatments for MNGIE. The aim of this study was to investigate the safety, tolerability, and efficacy of an enzyme replacement therapy for the treatment of MNGIE. In this single centre study, three adult patients with MNGIE received intravenous escalating doses of erythrocyte encapsulated thymidine phosphorylase (EE-TP; dose range: 4 to 108 U/kg/4 weeks). EE-TP was well tolerated and reductions in the disease-associated plasma metabolites, thymidine, and deoxyuridine were observed in all three patients. Clinical improvements, including weight gain and improved disease scores, were observed in two patients, suggesting that EE-TP is able to reverse some aspects of the disease pathology. Transient, non-serious adverse events were observed in two of the three patients; these did not lead to therapy discontinuation and they were managed with pre-medication prior to infusion of EE-TP. To conclude, enzyme replacement therapy with EE-TP demonstrated biochemical and clinical therapeutic efficacy with an acceptable clinical safety profile.
Abstract.
Author URL.
Dario_Pacitti Details from cache as at 2019-08-22 21:56:31
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