Highlights of the International Congress for Ataxia Research 2024

The International Congress for Ataxia Research (ICAR) 2024 took place in London from 12th – 15th November. It was organised by Ataxia UK, the National Ataxia Foundation, the Friedreich’s Ataxia Research Alliance, and the Ataxia Global Initiative.

This was the biggest ICAR yet, with 600 in-person attendees and 50 attending online from 32 countries, including researchers, clinicians, representatives from the pharmaceutical industry and patient organisation representatives. The congress brought together researchers from the ataxia field to share knowledge through talks, workshops, debates and poster presentations. Around 350 scientific research posters were presented and over 80 invited talks were given. Members of the Ataxia UK research department were involved as authors on six posters and one talk. 

ICAR 2024 included sessions on disease mechanisms, models of the ataxias, preclinical and clinical research, showcasing many innovations in the field of ataxias, from gene therapies to new measures of ataxia progression. Here, we summarise some of the highlights from the Congress. 

We are grateful to the 14 pharmaceutical companies and two patient advocacy organisations whose sponsorship made ICAR 2024 possible. ICAR’s presenting sponsor Biogen hosted a breakfast symposium for clinicians in countries where Omaveloxolone is now approved for the treatment of Friedreich’s ataxia (FA). Throughout the conference, Ataxia UK hosted an information stall, sharing a range of resources including the Ataxia UK medical guidelines, which were well received. The stall facilitated discussions with numerous researchers and companies, enabling Ataxia UK to build upon and initiate new collaborations. Ataxia UK’s Head of Research, Julie Greenfield reflected,

‘This has been the largest ever meeting of ataxia researchers, with around 600 people attending. Ataxia UK has been proud to organise this conference alongside our colleagues at FARA, NAF and AGI”. 

ICAR 2024 showcased a shift from understanding the basic science of the causes of ataxias toward the many advances in clinical research into treatments for ataxias. In Friedreich’s ataxia (FA), there were a number of presentations regarding Omaveloxolone. Data was presented on the safety and tolerability of Omaveloxolone from the trial and from people taking the drug in the countries where it is available (e.g., US and France), the relationship between age of onset and FA progression, and plans for a further study assessing the long-term safety of Omaveloxolone in FA.  

A number of other new developments were discussed, including research trials testing the effect of potential new therapies such as: 

  • Nomlabofusp – a frataxin protein replacement therapy developed by Larimar Therapeutics. Nomlabofusp is currently in phase II clinical trials. 
  • Vatiquinone – a small molecule drug developed by PTC Therapeutics that targets oxidative stress currently in phase III clinical trials. PTC submitted a new drug application to the FDA in December 2024. 
  • DT216-P2 – a small molecule drug developed by Design Therapeutics that targets GAA repeats to restore frataxin gene function. Design Therapeutics plan to start phase I clinical trials of DT216-P2 in 2025.  
  • CAP-004 – a gene therapy developed by Capsida Therapeutics currently in preclinical trials. The therapy has been tested in animal models, and is able to reach the brain with just a single intravenous injection, while avoiding off-target effects in other parts of the body such as the liver. This may potentially avoid the need for invasive brain surgery.  

At the 2022 ICAR, data on the then newly discovered ataxia SCA27B were presented. At ICAR 2024, 10 talks and 6 posters featured research into SCA27B, highlighting the growing interest in understanding the condition and conducting clinical trials for new treatments, such as the compound 4-aminopyridine. In the SCAs, research was presented on antisense oligonucleotides (ASOs) such as Vico Therapeutics’ VO659 which targets the genetic change that causes SCA1, SCA3 and Huntington’s disease.  In the UK the London Ataxia Centre is a trial site for this programme.  

Research was presented by a number of different groups on the role of faulty DNA repair in different types of ataxia, including FA, SCA3 and SCA27B. Targeting genes involved in DNA repair represents a promising avenue of research in repeat expansion ataxias. Dr Stefan Pulst and his laboratory at the University of Utah presented their discovery of the gene that causes SCA4, which you can read more about on the Ataxia UK website. This discovery paves the way for new treatments for SCA4. 

On the first day of the Congress, Ataxia UK’s CEO Sue Millman hosted a discussion panel where a number of participants with ataxia, or who care for someone with ataxia, shared their experiences and perspectives on living with ataxia. The panel shared their experiences of taking part in research, from healthcare research to clinical trials of Omaveloxolone, as well as the lived experience of being a parent of someone with ataxia. Including the experience of people living with ataxia is important to highlight the impact of the condition to researchers and clinicians and is always valued. 84% of feedback survey respondents said that the patient perspective included within the conference content was either extremely valuable or very valuable.

The second day of the conference featured a clinical Grand Round – where ataxia clinicians presented unique and challenging ataxia cases to educate researchers and clinicians. For example, one case featured the temporary deterioration in ataxia symptoms following caffeine consumption on SCA27B symptoms, highlighting the importance of lifestyle modifications which can be used to manage ataxia symptoms.  

On the third day of the conference, Ataxia UK’s Senior Advocacy and Community Programme Officer, Shana De Figueiredo Scholtz, who lives with SCA3, presented a talk as part of a workshop on patient perspectives on preventive clinical trials in ataxias, sharing insights from four people with ataxia from a focus group hosted by Ataxia UK. 

The Congress ended with a late-breaking research session, designed to allow researchers to share exciting new data. In this session a representative from Biohaven presented results from their clinical trial testing Troriluzole in people with SCA. Troriluzole is currently under review by the EMA for the treatment of SCA3 and Biohaven are planning to submit a New Drug Application (NDA) to the US Food and Drug Administration (FDA) for Troriluzole in the treatment of all SCA genotypes in the last quarter of 2024. 

We will be running a series of articles spotlighting some of the research presented at ICAR 2024 on the Ataxia UK website. Until then, you can read the ICAR press release here

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