Smart Cholesterol-Mopping Polymer Nanoparticles in Niemann-Pick Disease Type C

Start Date and End Date

01 April 2018
31 March 2019

Turkish Partner(s)

Istanbul Technical University

Coordinator

Dr. Antonino Puglisi

Budget

145,845 Euro

Programme

Horizon 2020 Marie-Curie

Project Web Page

Scientific Outputs

Synthesis, characterisation and activity of smart polymer nanoparticles (‘NanoMops’) as a cholesterol-mopping, cyclodextrin-based, therapeutic agents in Niemann-Pick Disease Type C (NPC)

Tackling ‘grand’ or societal challenges

Niemann-Pick Disease Type C (NPC), also known as “childhood Alzheimer’s”, is a rare genetic disorder caused by the accumulation of cholesterol mainly in the brain. In the European Union (EU) NPC has an incidence of 1 per 100,000 people. Cyclodextrins (CDs) have recently gained considerable attention as a potential therapeutic intervention in the treatment of the disease. The project addresses an important question that needs to be answered urgently and will contribute to the treatment of the neglected disease in line with the Horizon2020 health goal of providing better and more effective therapies. Moreover, this project’s expected results may also contribute towards the treatment of other neurodegenerative disorders exhibiting impaired cholesterol metabolism such as Alzheimer’s, Parkinson’s and Huntington’s diseases.

Industrial Innovation (including innovation in services as well as products and processes)

The project will reach out to industry (secondment at Cyclolab a world-leading R&D company researching CD derivatives – Hungary) in order to have a multiple knowledge-transfer effect and where I will also gain an inside viewpoint into marketable CD-based products.

Research-influenced changes in policy, agenda-setting

During the project the researcher will collaborate with the International Niemann-Pick Disease Alliance (INPDA), networking internationally with 14 non-profit NPC group members and supporting hundreds of families though which he would like to make his contribution to influence policy-making and raise awareness about NPC internationally and in Turkey.

The provision of Improved Public Goods

Currently, there is no effective treatment for NPC which present itself with challenging symptoms imposing a major burden upon patients, health services and society. Further understanding of the condition and how it may be treated is urgently required to address the unmet needs of this patient community.

The improved exercise of professional skill

This interdisciplinary proposal is designed to build on my specific skillset in organic synthesis of CD derivatives by providing the researcher with advanced training in polymer chemistry. At the same time, this project will take him on a personalised professional development journey into the field of biosciences where he will learn about a range of bio-assays.

Human capital development

Together with the supervisor. the researcher have formulated a customised Personal Career Development Plan which will strengthen his professional independence while reinforcing his professional maturity during the fellowship helping him to head decisively towards the establishment of his own research group.