4: HEALEY ALS Platform Trial with Dr. Merit Cudkowicz and Dr. Melanie Quintana

In this episode of the podcast, we sit down with Dr. Merit Cudkowicz and Dr. Melanie Quintana to discuss the inception and execution of the Healy ALS Platform Trial, a revolutionary approach designed for efficiency and impactful data collection. With insights from both medical and statistical perspectives, this episode offers a comprehensive understanding of the trial's structure and outcomes, shedding light on its potential to reshape neuro-therapeutics research.

Key Highlights

• Dr. Merit Cudkowicz discusses the motivation behind adopting master platform trials for ALS and the collaboration that brought it to life.

• Dr. Melanie Quintana explains the statistical design of the trial, emphasizing the sharing of control groups and Bayesian methods for efficiency.

• Insights into the FDA's enthusiastic support and the iterative process to align on innovative statistical approaches.

• The dual roles of the trial: significant patient engagement and industry collaboration as facilitating factors for successful trial implementation.

• Discussion on the future adaptation of trial designs based on collected data and emerging biomarkers.

Quotes

• "There was such an energy about your group… everybody had really done their homework." – Dr. Melanie Quintana

• "ALS is a very complex disorder, and we actually did learn a lot." – Dr. Merit Cudkowicz

• "We're adapting with learnings." – Dr. Merit Cudkowicz