5th Annual ALS Research Symposium

DAY 1 (Thursday, Oct 6th): 

10:30 – 10:45am: WELCOME & INTRODUCTIONS

10:45am – 11:45pm CURRENT STATE OF ALS TRIALS

MODERATOR: Fernando Vieira, MD

• 10:45 – 11:05am General overview of ALS Trials: What’s new, opportunities and challenges

• • Speaker: Merit Cudkowicz, MD, MSc, Chief of the Massachusetts General Hospital Neurology Service, Director, Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital and the Julieanne Dorn Professor of Neurology at Harvard Medical School in Boston.

• 11:05 – 11:25am Update on The Healey ALS Platform Trial

• • Speaker: Sabrina Paganoni, MD, PhD, Co-Director of the Neurological Research Institute at the Massachusetts General Hospital and Associate Professor at Harvard Medical School.

• 11:25 – 11:45am Functional Outcomes & Biomarkers: Regulatory Considerations

  • Speaker: James Berry, MD, MPH – Associate Chief of Neurotherapeutics and Chief of the Division of ALS and Motor Neuron Diseases at Massachusetts General Hospital and Director of the MGH Neurological Clinical Research Institute (NCRI).

11:45 – 11:55am: Break

11:55am – 1:30pm: CURRENT STATE OF ALS TRIALS continued.

MODERATOR: James Berry, MD, MPH

• 11:55am – 12:20pm Which are the best pre-clinical models?

• • Talk Title: Preclinical Modeling of ALS: Relevance and Applications

• • • Speaker: Fernando Vieira, MD, CEO and Chief Scientific Officer at the ALS Therapy Development Institute.

• 12:20 – 12:45pm Gene therapies: Update on Toferson

• • Speaker: Toby Ferguson, MD, PhD, Vice President, Head Neuromuscular Development Unit at Biogen.

• 12:45 – 1:05pm Update on AMX0035

• • Speaker: Josh Cohen and Justin Klee, Co-Founders & Co-CEO’s, Amylyx Pharmaceuticals.

• 1:05 – 1:30pm Update on EAP & ACT 4 ALS

• • Speaker: Sabrina Paganoni, MD, PhD, Co-Director of the Neurological Research Institute at the Massachusetts General Hospital and Associate Professor at Harvard Medical School.

1:30 – 2:15pm: Lunch/Break

2:15 – 3:30pm: Continuing lessons from TDP43 and related ALS genes

MODERATOR: Robert H. Brown, Jr., MD, DPhil

• 2:15 – 2:40pm Mitochondria, Inflammation and ALS.

• • Speaker: Erika L.F. Holzbaur, Ph.D., William Maul Measey Professor in Physiology, Dept. of Physiology, Perelman School of Medicine, UPenn.

• 2:40 – 3:05pm Gene Mis-splicing in ALS: from causes to therapeutic targets.

• • Speaker: Aaron Gitler, PhD, Professor, Dept. of Genetics, Stamford University.

• 3:05 – 3:30pm Stathmin2: Update on biology and approaches to therapy.

• • Speaker: Kevin Eggan, PhD, Group Vice President, Head of Research and Early Development at BioMarin.

3:30 – 3:45pm: Break

3:45 – 4:30pm: NEW BIOMARKERS, NEUROTECH & BEYOND.

MODERATOR: James Berry, MD, MPH

• Speakers:

• • Leigh Hochberg, MD, PhD, Director, Center for Neurotechnology and Neurorecovery, Neurocritical Care and Stroke Services, Department of Neurology, Massachusetts General Hospital.

  • James Berry, MD, MPH, Chief, Division of ALS and Motor Neuron Diseases at Massachusetts General Hospital, Director, MGH Neurological Clinical Research Institute (NCRI).

  • Indu Navar, MSCS, CEO and Founder of EverythingALS.

  • Shelena C. Lalji, MD, F.A.C.O.G., Founder of ALS Heroes and The Dr.Shel Wellness & Aesthetic Center.