The first conference of the SCA Global initiative was held in Las Vegas from 27 to 29 March. Researchers from around the world, industry and patient organizations have formed a global collaboration in clinical research efforts for Spinocerebellar Ataxias (SCA). SCA Global was created to address the obstacles that clinical investigators are having with a lack of natural history study data and validated biomarkers for Ataxia.
In view of the emerging treatment for SCAs, there is the urgent need to increase trial readiness. To this end, SCA Global will strive to achieve the following objectives:
- to provide added value through a global SCA research platform that is based on exchange and sharing
- to establish large, well characterized, standardised trial ready cohorts,
- to precisely define the evolution of clinical outcomes across all SCAs and different world regions,
- to better characterise the pre-ataxia stage of all SCAs,
- to develop and validate imaging and biochemical biomarkers,
- to attract young talent and to improve education and training of clinical investigators.
As an outcome of the SCA Global conference four working groups (WG) were formed:
- Clinical outcomes
- MR biomarkers
- Standards of biosampling
- SCA Global policies
ERN-RND will be partnering with SCA Global. Two of the four working groups will be led by ERN-RND members: Thomas Klockgether leads the Clinical Outcome WG and Holm Graessner leads the SCA Global Policies WG.
The next SCA Global conference will be organised in April 2020 in Bonn, Germany. More information about SCA Global can be found here.