Between November 1st – 4th 2022, Ataxia UK, the National Ataxia Foundation (NAF), and the Friedreich’s Ataxia Research Alliance (FARA) co-hosted the International Congress for Ataxia Research (ICAR) in Dallas, Texas, USA. The conference was a hugely successful event. Over 450 people attended from 19 different countries, making this one of the largest gatherings of ataxia researchers to date. This included researchers, pharmaceutical companies, and patient group representatives. The conference programme included sessions on many different aspects of ataxia, including gene discovery, disease mechanisms, biomarkers, and emerging therapeutics.

The Ataxia Global Initiative (AGI) Conference was held immediately after ICAR 2022, On November 4th – 5th. The AGI is a worldwide research platform, with the goal of facilitating development of therapies for the ataxias. It involves researchers, pharmaceutical representatives and patient groups. The AGI conference included presentations on a range of ataxias, focusing on clinical trial-readiness.

Here are some of the highlights relevant to the PROSPAX project.

Presentation on developing outcomes which are meaningful to people with ataxia
In order to become ready for clinical trials, it is important that outcomes can measure the progression of ataxia and any response to treatment. While a number of outcomes have been developed, for example clinical assessments and brain imaging markers, it is essential that these are meaningful and relevant to people with ataxia. At ICAR, PROSPAX coordinator Professor Matthis Synofzik gave a presentation on developing meaningful outcomes. He presented the results of two PROSPAX surveys which were completed by people with ataxia and HSP. These looked at the frequency, severity, and impact of symptoms. The surveys were translated into several different languages and had a high number of respondents (find out more here) The results from these were used to determine the most meaningful aspects of health in ataxia. These can be used by researchers to demonstrate that the outcome that they are using is relevant and meaningful to people with ataxia. 

Presentations on the PROSPAX natural history study
Natural history studies collect health information over time to better understand how a condition progresses and are important in preparing for future clinical treatment trials. The PROSPAX natural history study is a 2-year study looking at the progression of SPG7 and ARSACS, and includes clinician- and patient-reported, digital, and imaging outcomes. At the AGI conference, PROSPAX coordinator Professor Rebecca Schüle gave a presentation on the PROSPAX natural history study data from 97 people with SPG7 and Professor Matthis Synofzik presented a poster on the PROSPAX natural history study data from 72 people with ARSACS. These showed that several clinical measures may worsen over the course of SPG7 and ARSACS and could therefore capture progression. The data presented were from the first time point of the study, with data from Year 1 and Year 2 currently being collected and analysed. The PROSPAX natural history study has been very successful due to having a large number of participants, multiple international study sites, and many types of high-quality assessments. This study could provide a blueprint for future natural history studies in other spastic ataxias.

Conference feedback
92% of ICAR attendees thought the conference was extremely useful or very useful for advancing their work, and 96% made new contacts whilst attending. International conferences are a fantastic opportunity for ataxia researchers to present their work, connect with other researchers, and are important for inspiring and progressing further ataxia research. Click here to read scientific summaries of all talks and posters in the abstract book.