Oxford University

Professor Frances Platt has been instrumental in progressing research and therapies for NPC. Her lab at Oxford has been involved in many of the significant projects testing potential therapies for NPC over the last 2 decades. Miglustat was the first licensed drug in Europe for NPC and the Platt Lab was influential in bringing this drug to trial. They also investigated the benefits of Curcumin in reducing the effects of NPC.

Fran was one of the driving forces behind David and Sue setting up the NPRF in 2011. Following conversations with Fran at various meetings, it became apparent that there were projects aimed at investigating potential avenues and hunches for which it was difficult to gain grant funding. It also became clear that enabling scientists to bypass an often slow and laborious process for gaining funding would help accelerate research and therefore a potential therapy. In addition, getting funding for valuable and experienced support staff and infrastructure was difficult but much needed to again help speed up progress.

The Platt Lab was the first lab to receive funding from the NPRF, with a grant of £46,767 provided to support key technician salaries and consumables. Since that first grant back in 2011, they have successfully applied for a further 4 grants with total funding of £274,922.00. Due to this funding, they have looked at metal-based therapeutic strategies, the neuropathology of iron therapy in NPC, investigated the P450 pathway and published a paper showing the benefits of NPC patients taking a bile acid supplement to aid the metabolism of drugs, food and toxins in the liver.

The expansion of the NPC mouse colony at Oxford has also ensured collaboration with many other laboratories and pharma companies, helping to bring other therapies to trial. Platt Lab collaborates with many other NPC centres including for example UCL, the University of Sussex and Orphazyme.

We are currently reviewing a further application from the Platt Lab.