On Thursday 16th October our Eighth International Workshop on AKU was held in Stockholm, Sweden. Just the day before, on Wednesday 15th October, we received the news that Robert Gregory, co-founder of the AKU Society, and himself an AKU patient, had sadly died. In this week’s blog we take a look at the workshop, which was dedicated to Robert Gregory.

Robert Gregory

At the start of the conference we heard a dedication to Robert Gregory from Prof Ranganath. Together Prof Ranganath and Robert co-founded the AKU Society. Prof Ranganath is now the Lead Clinician at the National Alkaptonuria Centre (NAC), and Principle Investigator for our #DevelopAKUre trials.

However, he recalled how he had little interest in AKU before meeting Bob. It was Bob who suggested setting up the AKU Society, and before Prof Ranganath had time to think about it, Bob had filled in all of the forms and paperwork to set up the charity. Since then Prof Ranganath has dedicated his life and his career to the study and treatment of the disease, none of which would have happened without Bob. Prof Ranganath spoke fondly of Bob as not only a co-founder, but as a friend and inspiration.

Without Bob Gregory our workshop showcasing the thriving research and knowledge around AKU would never have happened. The AKU Society as we know it would not exist. It is only fitting that we dedicated the Eighth International Workshop on AKU to Bob, as the whole day really was a credit to his hard work. We hope to keep working towards his vision to support patients and cure AKU.

The Workshop

The workshop opened with an introduction from our hosts, Swedish Orphan Biovitrum (Sobi). Sobi are the pharmaceutical company who produce nitisinone, and we are working with them closely in our clinical trial consortium. They introduced us to Sweden, and their work with rare diseases.

After an introduction from Prof Ted Lock, Prof Elisabeth Holme from the University of Gothenburg spoke about the use of nitisinone in the rare disease it was originally licensed for- hereditary tyrosinaemia 1. It was incredibly interesting to hear about the origins of nitisinone, and how it came to be identified as a potential treatment for AKU.

Prof Ranganath spoke next on the results of our SONIA 1 clinical trial. This trial finished earlier this year, and its goal was to determine the best dose of nitisinone to be used in AKU patients. The dose recommendation of the trial is higher than the current dose taken by UK patients at the NAC, who take the drug off license. This highlights the importance of studying the effectiveness of nitisinone in AKU to get the best treatment for our patients.

Dr Jean-Baptiste Arnoux was the next speaker, and gave a fantastic presentation on the importance of dietary management in patients with AKU. Dr Arnoux is from Hospital Necker in France, which is one of the sites for our clinical trial. He explored the research around diet in AKU patients, and the importance of a low protein diet in patients taking nitisinone. He also emphasised the benefits of a healthy diet in those who don’t take the drug.

Gait analysis is a relatively new technology being used to study AKU patients. Dr Gabor Barton leads the gait analysis project at Liverpool John Moores University. He gave a fascinating presentation on why gait analysis is so useful for studying AKU. Through a series of videos, graphs and animations, he showed the depth of data gathered by simply getting a patient to walk across a room. With the help of sensors and cameras this data can be analysed, and can even be used to help a patient walk differently and reduce their pain.

Prof Virginia Kraus was next to speak on her wide ranging research of different types of osteoarthritis. She shared a very interesting message about the study of arthritis, pointing out that we don’t just treat heart disease after an attack. Instead we work towards prevention to try to prevent the worst outcome. Prof Kraus said osteoarthritis should also be approached in this way.

Patient Session

As talks continued after lunch, the patients visiting the workshop were brought in to another room and given the chance to have their own discussion session. Along with staff from the NAC, and our Patient Support Manager, Lesley Harrison, we discussed all sorts of topics. These ranged from dietary needs when taking AKU, to the production of a new patient handbook, and even the possibility of an International Patient Workshop, where patients from around the world could meet each other.

This session was valuable for both us and the patients, allowing them to contribute their ideas to our future projects, and also giving them a chance to voice any concerns they have.

Pain Management and Patient Groups

The patients re-joined the talks just in time to hear Dr Andrew Jones speak to us about pain management in AKU. Dr Jones provides pain management sessions for patients who attend the NAC in the UK. His session is often a favourite among the patients, as the results of his injections are often immediate, and his judgement and advice are well respected. He spoke about the different types of pain management he uses, and how effectiveness varies from person to person.

The final talk of the day was from our CEO, Nick Sireau, who gave a patient group perspective. His talk was on the development of the AKU Society since Bob helped found it around 12 years ago. He focused on the impact Robert Gregory has made for AKU patients, and how he has continued to influence the work of the AKU Society. He will be greatly missed, but his legacy will live on in the work of the AKU Society, and all of our partners, both in our clinical trial and at the NAC.