Pre-Conference Workshop Day

9:00 – 12:00 pm
Workshop A

Painting the Bigger Picture: Understanding Overarching ALS Pathology to Allow Novel Sporadic Target Identification

  • Giulio Maria Pasinetti Director & Chair Of Molecular, Integrative Neuroresilience ,Cognitive & Psychological Resilience Neurology Program, Icahn School of Medicine at Mount Sinai
  • Matija Zelic Senior Scientist, Precision Neurology & Neuroinflammation Cluster, Sanofi


Understanding disease pathology remains one of the biggest challenges facing ALS drug development. With fast disease progression and complex, multi-system biology, how can we understand underlying causes for sporadic ALS to uncover novel targets to benefit the wider patient population? Join this workshop to explore the cutting-edge research understanding ALS disease biology and how this translates into identifying novel targets for sporadic ALS.

Workshop highlights include:

• Monitoring early disease progression to investigate causes and pathways to sporadic ALS disease progression

• Non-neuronal factors: piecing together global ALS pathology to uncover common threads for novel sporadic targets

• Tackling patient heterogeneity to identify ALS participant subsets for personalized target validation

1:00- 4:00 pm
Workshop C

De-risking Preclinical Strategies: Measuring Relevant Data to Optimize Successful Translation of ALS Therapies

  • Fen Huang Associate Director - Translational Science & Project Team Lead, Denali Therapeutics Inc.
  • Andrew Kaplan Senior Scientist - Neuroscience TRC, Bristol Myers Squibb


Collecting the correct preclinical data is pivotal to maximise successful translation of ALS therapies into human trials. With recent candidates displaying preclinical promise but failing in clinical trials– how can preclinical strategies be adapted to maximise success translation?

Workshop highlights include:

• Selecting the correct disease model phenotype to undergo tailored safety and efficacy testing for selective therapeutic targets

• Target engagement and disease modification: solidifying preclinical endpoints to optimize successful translation to human trials

• Developing translatable biomarkers to validate desired therapeutic effects

9:00 – 12:00 pm
Workshop B

Unlocking the Potential of Wearable Technology & Digital Biomarkers to Allow Remote ALS Participant Assessments

  • James Berry Chief, Division of ALS & Motor Neuron Diseases, Massachusetts General Hospital
  • Ram Miller Executive Director, Translational Medicine, Novartis AG
  • Marcin Straczkiewicz Research Associate, Harvard University


The use of digital biomarkers and wearable technologies creates an invaluable opportunity to conduct remote assessments of ALS patients. With technology being developed to measure vocal, motor function and respiratory biomarkers, sensitive data can be constantly collected by non-invasive means directly from patient’s homes. Attend this workshop to learn latest advances in wearable technology being developed for ALS clinical trials.

Workshop highlights include:

• Exploring the different types of wearable technology available to monitor ALS participants remotely

• Outlining the benefits of performing remote assessments: expanded data collection and participant engagement

• Advancing towards a seamless experience: understanding the necessary stages to validate and implement wearable technologies into decentralized ALS clinical trials

1:00 – 4:00 pm
Workshop D

Optimizing ALS Clinical Trials to Incorporate the Evolving Standard of Care & Standardize Trial Design

  • Sabrina Paganoni Co-Director, Neurological Clinical Research Institute, Massachusetts General Hospital
  • Olga Uspenskaya-Cadoz Vice President, Clinical Development, Prevail Therapeutics
  • Alex Sherman Director - Systems & Neurology, Massachusetts General Hospital


With an increasing number of ALS therapies entering human trials – understanding the best strategy for clinical trial design is crucial. Attend this workshop to learn the optimal clinical trial design in light of recent market approvals, differing trial duration and participant selection strategies.

Workshop highlights include:

• Understanding how to measure novel candidate efficacy magnitude against an evolving standard of care placebo

• 6-18 months: what is the ideal trial duration to bridge the gap between efficacy measurements and participant burden?

• Defining inclusion and exclusion