The FARA Team returned from the four-day International Ataxia Research Conference (IARC) in Pisa, Italy a bit jet-lagged but buzzing with energy that results from the sharing of good research results. IARC2017 was co-organized by goFAR, Ataxia UK and FARA and simply by the numbers included:
- over 400 attendees- researchers, clinicians, patients, and government, regulatory, and industry representatives.
- Representatives from 22 countries
- >15 pharma/ biotech companies
- >30 platform presentations on FA
- Over 95 poster presentations on FA!!!
In addition to the impressive engagement numbers, the IARC had many meaningful research and collaboration take-aways such as:
Young Investigators
The IARC2017 welcomed an increasing number of young investigators, demonstrating growing interest in
research for the ataxias. The host organizations believe in developing and mentoring the next generation of
researchers and were honored to give nine scholarships to graduate students and post-doctoral fellows who
submitted competitive abstracts. In order to help these young investigators make contacts in the field and
develop their careers in ataxia research, FARA provided a lunch at the start of the meeting to help young
investigators meet more senior investigators and learn about options for career development within the
field. In addition, among more than 50 competitive Young Investigator Posters, Nesli Ece Sen and Saba
Saqlain earned honors for best Young Investigator Posters for their work in ataxia and Friedreich’s ataxia
respectively.
International Collaboration
Ataxia is not just a US or a European disease. It is a global disease, requiring international collaboration on all
fronts. These collaborations are the power behind research advancement and treatments for FA. The
research advocacy organizations held a meeting at IARC2017 and discussed the importance of the FA Global
Patient Registry as an essential resource for both clinical trial readiness and advancing research. The partner
organizations committed to further collaboration in expanding and growing the patient registry. https://curefa.net/registry/
Exercise is Good
Several researchers presented data on the benefits of exercise for people living with FA. Dr. Zen Yan from
University of Virginia presented data in mice showing that long term aerobic exercise is seen to slow FA onset
(Click here for more information) Dr. Sarah Milne from Monash University in Australia also presented a poster demonstrating that physical rehabilitation improved physical and emotional health. There were also several
other poster and platform presentations drawing attention to the need to treat symptoms of visual dysfunction,
hearing loss, speech and swallowing and depression.
Patient & Clinical Trial Design Roundtables
Individuals with ataxia and parents and caregivers joined the meeting for a special patient roundtable
discussion to share their personal stories and need for treatment. This roundtable made clear statements about
the need to not only focus on stopping and curing the disease, but also asserted that treatments which target
symptoms are meaningful. If therapies could improve symptoms such as vision and hearing loss or speech
impairment, this would be significant for people living with ataxia. (Click here for more information) There was also informative roundtable on clinical trial design for ataxias that featured representation from the European
Medical Association, ataxia patient community, clinical neurologists and pharmaceutical companies. This
allowed for important discussion among all stakeholders on good clinical trial design. Some themes that arose
from this discussion included the need for better communication between study sponsors and study
participants and the need for objective outcome measures.
Advancing Therapies
The conference included more than 20 platform and poster presentations focused on drug discovery, preclinical
and clinical outcomes in FA. These presentations demonstrated progress in many aspects of the treatment
pipeline. Encouraging results of recent clinical trials were reported including Reata's Phase 2 MOXIe study, as
well as a number of talks on new therapeutic options that may reach the clinic soon. There has been
significant growth and advancement in drug discovery and development for therapies that target frataxin (ie the
root cause of FA). This was most notable in the number of presentations on genetic based therapies including
oligonucleotide and gene replacement strategies. To read more about some of the research presented at the
conference, click one of the links below:
- Omaveloxolone Can Aid Neurological Function in FA Patients, MOXIe Trial Shows
- Synthetic Molecules Seen to Restore Frataxin Protein Levels in Cells from FA Patients
- Gene Therapy Seen to Reverse Sensory Ataxia in FA Mouse Model
- Protein-producing Frataxin Gene Delivered to Brain, Heart of Primates in Gene Therapy Study
- Small Molecules Mimicking Brain Factors Raise Frataxin Protein Levels in FA Mice in Study
