Advancing Hope: AFTD Attends C9orf72 FTD/ALS Prevention Trial Workshop
A one-day C9orf72 FTD/ALS Prevention Trial Workshop was held in June 2024. The workshop was led by Dr. Adam Boxer (UCSF) and Dr. Michael Benatar (University of Miami) and enabled by AFTD and the ALS Association. More than 30 scientific experts attended the meeting from academia, biopharma, advocates, nonprofit organizations and the US Food and Drug Administration.
The goal of the workshop was to discuss key elements needed to design a C9orf72 prevention clinical trial. The agenda focused on crucial components needed to deploy a C9orf72 FTD/ALS prevention trial including biomarkers, statistical disease modeling, clinical trial endpoints. Many conversations highlighted the current state of research on these topics and what must be achieved before we can launch a trial to test a preventative treatment for C9 ALS/FTD. Gail Andersen, AFTD, and Jean Swidler, End the Legacy, shared remarkable stories of their lived experience of C9orf72 FTD and ALS and provided vital feedback to researchers on what is important to the community.
A mutation in the C9orf72 gene is a known genetic risk factor for developing FTD and ALS. While FTD and ALS are associated with neurodegeneration in different areas of the nervous system, the C9orf72 mutation can cause the onset of one or both diseases. To-date, researchers and clinicians are unable to predict which disease or combination of diseases will emerge in which person. As researchers continue to develop treatments to prevent or delay disease onset, simultaneous work is needed to ensure that the tools are in place to test the effectiveness and safety of a potential preventative treatment. Strengthening the collaboration between FTD and ALS researchers will be essential for these tools to be developed, particularly as there is no ability to predict which people with C9orf72 will develop which disease or combination of diseases. AFTD persists in advocating for cross-discipline collaboration through participation in initiatives like AMP-ALS.
AFTD’s support for this workshop was made possible by the Samuel I. Newhouse Foundation and their generous support of the FTD Biomarkers Initiative.
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