Nick has been involved in The AKU Society for over 10 years; becoming trustee in 2003, chairman in 2005 and CEO in 2010. He is now leaving his CEO position to take on a role in AstraZeneca, but will remain chairman and continue to be involved in the society! In this weeks blog, Nick talks about what the society has achieved over the past 5 years and his plans for the future.

After five years working as CEO of the AKU Society, I’m now moving on. It’s been a big adventure and we’ve come a long way during that time. Thanks to all the hard work of the AKU teams in Cambridge, Liverpool and around the world, we’re now well underway for developing successfully the first ever treatment for AKU.

It hasn’t been easy. When we first started putting together the DevelopAKUre consortium five years ago, the AKU community was disheartened. We’d just had the results of the unsuccessful clinical trial of Nitisinone at the National Institutes of Health in the USA. AKU patients and researchers were devastated: such a promising treatment, yet it was proving so difficult to show that it worked.

Fortunately, Prof Ranganath of the Royal Liverpool University Hospital was undeterred. I can distinctly remember the email he sent through in mid 2010 to Prof Jim Gallagher (of the University of Liverpool) and myself, where he said,‘We are ready to do a new clinical trial.’

Indeed, the AKU Society had funded a natural history study at the Royal Liverpool University Hospital, as well as a PhD programme and an animal model at the University of Liverpool. The information gathered had led to the development of a new way of measuring AKU more accurately as well as further evidence that nitisinone was a promising drug.

We teamed up with Dr Tony Hall, one of the co-founders of PSR Group, a company that specialises in clinical trials for rare diseases, and with Sobi, the company that owned the license to nitisinone. Everyone got to work to plan a European clinical trial – one that would learn from the challenges of the past in order to prove beyond doubt that nitisinone was effective at treating AKU.

Thanks to the indefatigable help of Oliver Timmis, who had just finished at Cambridge University and joined the AKU Society, we toiled for more than a year to prepare a 150-page document that we submitted to the European Commission for funding in May 2012.

We were overjoyed to hear that we’d scored 15/15 and were to receive €6m in funding to support the new clinical development programme.

At the same time, we also learnt that we’d been successful in securing funding to set up the world’s first treatment centre for AKU, at the Royal Liverpool University Hospital. With support from the AKU Society’s Lesley Harrison, this meant that patients from England and Scotland would be able to access nitisinone and have a full check-up once per year in a ground-breaking move that has transformed how AKU is treated in the UK.

Three years later, both the European clinical trial and the National AKU Centre are thriving. Initial results from the National AKU Centre already indicate that nitisinone is having a positive effect on AKU patients, which is excellent news.

None of this would have been possible without the hard work of many people – too numerous to thank all of them here. My thanks go out especially to all the AKU patients who are dedicating time and effort to participating in the European trial and the National AKU Centre. Without you, none of this would be possible.

As for me, I’ll be moving on to a position in patient engagement with AstraZeneca, the pharmaceutical company. I’ll continue as chairman of the AKU Society, with Oliver Timmis taking over the role of CEO, so I’ll continue to be part of the journey for some time to come!

The AKU team wish Nick the best of luck in his new role at AstraZeneca and thank him for his hard work and determination over the past 5 years. We are very pleased to say that Nick will remain Chairman of the society and continue to be involved in the AKU journey!