8:25 am Chair’s Opening Remarks

  • Ralph Kern President & Chief Medical Officer, Brainstorm Cell Therapeutics

Validating Biomarkers of Target Engagement & Disease Progression to Transform Early & Sensitive Indicators of Therapeutic Efficacy

8:30 am Interrogating Neurofilament as a Marker of Disease Activity & Treatment Response


  • Assessing data from tofersen studies to explore the potential utility of neurofilament to enrich or stratify study populations
  • Evaluating the relationship between treatment-induced reductions in neurofilament and clinical outcome measures
  • Exploring neurofilament as an early marker of disease activity in presymptomatic mutation carriers

9:00 am Implementing Remote Monitoring Technology for Tracking ALS Outside the Clinic Using Wearable Sensors & Smartphone Apps

  • Amir Lahav Head of Digital R&D, Digital Healthcare Innovation, Mitsubishi Tanabe Pharma Holdings America


  • Leveraging technology for early diagnosis and remote monitoring of ALS patients
  • Aggregating digital data coming from activity monitors, speech recording apps, and smartphone-powered platforms for motor function tests at home.
  • Roadmap for integrating digital technology into ALS trials: from feasibility and usabillity testings to clinical validation of new digital endpoints

9:30 am Advancing Novel CSF Biomarkers to Evaluate ALS Target Engagement & Improve Therapeutic Outcomes

  • Ralph Kern President & Chief Medical Officer, Brainstorm Cell Therapeutics


  • CSF biomarkers interrogate multiple neuroinflammatory, neurodegenerative and neuroprotection pathways to confirm target engagement
  • Employing biomarkers at baseline and on-treatment in combination with clinical variables to predict clinical responders
  • Supporting biobanks and continuing dialogue with regulators to advance adoption of novel biomarkers

10:00 am In-Person Structured Networking


  • This session is the ideal opportunity to take advantage of face-to-face networking time, and understand who is also prioritising and overcoming challenges within ALS drug development.

10:30 am Morning Break & Networking

11:00 am Exploring Electrophysiological Biomarkers of Diagnosis & Outcome in ALS


  • Examining applications of electrophysiology as a biomarker of disease progression
  • Using electrophysiology to track clinical disease progression and efficacy
  • Enhancing repeatability in patients and investigating potential as a primary outcome measure

11:30 am Validating Biomarkers & Use in Clinical Trials to Demonstrate Target Engagement & Eligibility Criteria for Enrollment

  • Robert Bowser Chief Scientific Officer, Professor & Chair of Translational Neuroscience, Barrow Neurological Institute


  • Investigating neurodegenerative and inflammatory biomarkers to evaluate treatment effects in ALS
  • Correlating research markers with clinical outcome and interpreting conflicting results
  • Utilizing a biomarker panel as enrollment criteria for a clinical trial

Delineating Accurate Primary Outcome Measures to Correlate with Clinical Outcome

12:00 pm Innovation in ALS Trial Design & Clinical Outcome Measures

  • Jeremy Shefner Professor & Chair of Neurology, Barrow Neurological Institute


  • Inclusion criteria can be used to ensure a homogenious trial population with regard to disease progression rate and other attributes
  • Well-designed open label follow on periods can support findings from placebocontrolled treatment
  • New outcome measures can enhance trial sensitivity and reduce participant burden

12:30 pm Lunch & Networking

1:30 pm Moving Beyond ALSFRS-R & Survival Endpoints: Exploring Patient Reported & Non-Traditional Outcome Measures

  • James Berry Chief, Division of ALS & Motor Neuron Diseases, Massachusetts General Hospital


  • Evaluating patient reported outcome measures remotely
  • Measuring movement, accuracy of typing, and speech: developing and steps to validating alternative readouts
  • Correlating biomarkers and clinical outcomes to accurately reflect disease progression and ultimately demonstrate treatment response in trials
  • Integrating novel digital outcome measures into clinical trials

2:00 pm Panel Discussion: Refining Sensitive Primary Outcome Measures & Navigating Innovative Trial Design in ALS

  • Stephanie Fradette Senior Medical Director, Clinical Development, Biogen
  • Andrew Wolff Senior Vice President, Senior Fellow, Clinical Research & Development, Cytokinetics
  • Angela Genge Chief Medical Officer, QurAlis
  • Robert Bowser Chief Scientific Officer, Professor & Chair of Translational Neuroscience, Barrow Neurological Institute


  • Pursuing sensitive markers of disease activity and delineating accurate primary outcome measures beyond ALSFRS-R
  • Driving early diagnosis, patient diversity, and decentralized trial design to establish effective patient recruitment
  • Can we capture statistically meaningful changes in an acute timeframe? Balancing length of trial to demonstrate safety and clinical benefit

Trial Design, Recruitment & Patient-Centric Approaches: Innovating Clinical Trials to Advance Effective Treatments

2:30 pm Driving Experimental Trial Design in ALS to Evaluate Multiple Interventions

  • Sabrina Paganoni Co-Director, Neurological Clinical Research Institute Massachusetts General Hospital


  • Breaking ground on platform trial design: accelerating towards effective ALS treatments and enabling greater access for patients
  • Establishing a common protocol across multiple drug candidates
  • Managing patient recruitment across different sites and creatively navigating patients on placebo

3:00 pm Afternoon Break & Networking

3:30 pm Rethinking Inclusion & Exclusion Criteria: Should we be Dissecting Patient Heterogeneity?


  • Interrogating Phase II clinical trial data to inform Phase III inclusion and exclusion criteria
  • How predictive algorithms may be used as part of inclusion and exclusion criteria
  • What types of therapy could be relevant to a wider patient population?

4:00 pm Considerations for Novel ALS Clinical Trials

  • Ram Miller Director Translational Medicine, Novartis


  • Incorporation of clinical trial innovations into ALS studies
  • The role of consortia in ALS trial design and execution
  • The inclusion of the voice of the patient in ALS clinical trials

4:30 pm TRICALS & the ENCALS Prediction Model: Innovating Clinical Trials in ALS


  • TRICALS: the highway towards a cure
  • Including more patients in trials by using the ENCALS prediction model
  • Trial duration for ALS clinical trials

5:00 pm Chair’s Closing Remarks

  • Ralph Kern President & Chief Medical Officer, Brainstorm Cell Therapeutics

5:10 pm Scientific Poster Session


After the formal presentations have finished, the learning and networking carries on. The Poster Session is allows you to connect with your peers in a relaxed atmosphere and continue to forge new and existing relationships. During this session scientific posters will be presented on the very latest advancements in ALS therapeutics with a focus on better understanding and overcoming translational challenges to inform the direction of future drug development efforts.

End of Day One