3 full days in the sharing of important ALS research discoveries and conversation.

View the full 2022 Symposium Speaker and Sponsor Guide with bio’s for each speaker and clickable links to all sponsors, by clicking HERE.

DAY 1 (Thursday, Oct 6th, 2022): 

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, Neurology Department - Director, Sean M. Healey & AMG Center for ALS Mass General Hospital and Julianne Dorn Professor of Neurology Harvard Medical School.

• RECORDING: https://youtu.be/sa9dT9ppEPs

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

• Speaker: Sabrina Paganoni, MD, PhD, Co-Director of the Neurological Research Institute (NCRI) at the Massachusetts General Hospital, physician scientist at the Healey & AMG Center for ALS, and Associate Professor at Harvard Medical School.

• RECORDING: https://youtu.be/WIcwKyZVN3A

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

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

  • RECORDING: https://youtu.be/Y1m-HfzmPvk

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.

• RECORDING: https://youtu.be/eGNN96xMB1k

• 12:20 - 12:45pm Gene therapies: Update on Tofersen

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

• RECORDING: https://youtu.be/2Ax6kwlnsRU

• 12:45 - 1:05pm Update on AMX0035

• Speaker: Jamie Timmons, MD, Head of Scientific Communications at Amylyx Pharmaceuticals; along with Josh Cohen and Justin Klee, Co-Founders & Co-CEO's, Amylyx Pharmaceuticals.

• RECORDING: https://youtu.be/W3b39JSbm44

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

• Speaker: Sabrina Paganoni, MD, PhD, Co-Director of the Neurological Research Institute (NCRI) at the Massachusetts General Hospital, physician scientist at the Healey & AMG Center for ALS, and Associate Professor at Harvard Medical School.

• RECORDING: https://youtu.be/W6b_8da3XWE

1:30 – 2:30pm: Lunch/Break  

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

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

• 2:30 - 3:00pm Mitochondria, Inflammation and ALS.

• Speaker: Erika L.F. Holzbaur, Ph.D., William Maul Measey Professor of Physiology at the University of Pennsylvania Perelman School of Medicine.

• RECORDING: https://youtu.be/2CgoSceb9gA

• 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.

• RECORDING: https://youtu.be/3dSjGk5zjcY

3:30 – 3:45pm: Break  

3:45 - 4:15pm: NEW BIOMARKERS, BCI, NEUROTECH & BEYOND: A prelude to Neurotechnology Day.

• Speakers:

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

• Leigh Hochberg, MD, PhD, Director, Center for Neurotechnology and Neurorecovery, Neurocritical Care and Stroke Services, Department of Neurology, Massachusetts General Hospital; Senior Lecturer on Neurology, Harvard Medical School; L. Herbert Ballou University Professor of Engineering and Brain Science, Brown University; Director, VA RRR&D Center for Neurorestoration and Neurotechnology, Providence VA Medical Center.

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

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

• RECORDING: https://youtu.be/a2kMfWXW5Lg
  

DAY 2 (Friday, Oct. 7th, 2022):

9:00 - 9:15am: Welcome & Introductions   

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

Continuing lessons from TDP43 and related ALS genes (continuation from Thursday’s segment).

9:15 - 9:45am New ALS splicing targets.

• Speaker: Aaron Gitler, PhD, Stanford Medicine Basic Science Professor in the Department of Genetics at Stanford University.

• RECORDING: https://youtu.be/5kSOoRVdRfk

9:45 - 10:45am: Industry Presentations

9:45-10:05am: AC Immune. Speaker: Tariq Afroz, PhD. 

• Abstract Title: “Immunotherapy targeting the C-terminal domain of TDP-43 decreases neuropathology and confers neuroprotection through microglial engagement in mouse models of ALS/FTD”. 

• RECORDING: https://youtu.be/3Dle40U81_Y

10:05am – 10:25am: Axoltis Pharma. Speaker: Sighild Lemarchant, PhD, Head of Preclinical R&D, Axoltis Pharma.

• Abstract Title: “A multi-properties peptide (NX210c) for the treatment of ALS”. 

• RECORDING: https://youtu.be/Tr9eqYuTOlA

10:25am – 10:45am: Biogen. Speaker: Michael Benatar, MBChB, MS, DPhil, Walter Bradley Chair in ALS Research; Professor of Neurology; Chief, Neuromuscular Division; Executive Director of The ALS Center; Vice Chair, Clinical & Translational Research Department of Neurology, Miller School of Medicine at the University of Miami.

• Abstract Title: “Rationale and design of a phase 3, randomized, placebo-controlled trial of tofersen initiated in clinically presymptomatic SOD1 mutation carriers: The ATLAS Study.”

• RECORDING: https://youtu.be/LthdaarjLqc

10:45 - 11:00: Break    

11:00 - 12:00pm: Industry Presentations continued

11:00 – 11:20am: BrainEver. Speaker: Julien Spatazza, PhD., Head of Pharmacology at BrainEver.

• Abstract Title: "ENGRAILED-1 homeoprotein transcription factor as a novel therapeutic strategy for ALS." 

• RECORDING: https://youtu.be/rZFeBVqCWxY

11:20 – 11:40am: Brainstorm-Cell Therapeutics. Speaker: Stacy Lindborg, PhD, Executive Vice President and Chief Development Officer at Brainstorm-Cell Therapeutics.

• Abstract Title: “The Relationship Between CSF Biomarkers and Efficacy of Treatment with NurOwn (MSC-NTF).”

• RECORDING: https://youtu.be/qwttt4VZbe8

11:40 – 12:00pm: Eledon. Speaker: Jeffrey Bornstein, MD, Chief Medical Officer, Eledon Pharmaceuticals.

• Abstract title: "Tegoprubart (AT-1501) is safe and well tolerated and reduces inflammation in patients with ALS."

• RECORDING: https://youtu.be/bXA6nkhsJ9M

12:00 - 12:30: Lunch/Break   

12:30 - 1:30pm: Industry Presentations continued.

MODERATOR: Fernando Vieira, MD

12:30 – 12:50pm: Ionis Pharmaceuticals. Speaker: Amy Shea, Director, Diagnostic & Strategic Initiatives at Ionis.  

• Abstract Title: “Ionis’ ALS Genetic Testing Program”.

• RECORDING: https://youtu.be/4_iyn9szMOo

12:50 – 1:10pm. NeuroSense. Speaker: Ferenc Tracik, MD, Chief Medical Officer at NeuroSense.

• Abstract Title: “Shifting the Paradigm: PrimeC as a novel therapeutic strategy for ALS.”

• RECORDING: https://youtu.be/dHnN1ltdrek

1:10 – 1:30pm: QurAlis. Speaker: Angela Genge, MD, Chief Medical Officer at QurAlis.

• Abstract Title: "QRL-201-01 - A multi-center, randomized, double-blind, placebo-controlled multiple‑ascending dose study to evaluate the safety and tolerability of QRL‑201 in amyotrophic lateral sclerosis"

• RECORDING: https://youtu.be/HvdVcRbu1y8

1:30 - 1:45pm: Break   

1:45 - 2:45pm: Industry Presentations continued.

1:45 – 2:05pm: SOLA Biosciences. Speaker: Akinori Hishiy, PhD, Chief Science Officer at SOLA.

• Abstract Title: "Preclinical proof of concept of SOL-257, a gene therapy targeting misfolded TDP-43 in ALS.” 

• RECORDING: https://youtu.be/aN2RMLIvpUo

2:05 – 2:25pm: VectorY. Speaker: Pavlina Konstantinova, MSc, PhD, MBA, Co-Founder and Chief Scientific Officer of VectorY.

• Abstract Title: “Reduction of oxidized phospholipids or misfolded protein aggregates by AAV-VecTabs in an Amyotrophic Lateral Sclerosis pre-clinical models.”

• RECORDING: https://youtu.be/s7yatzU1Dkc

2:25 – 2:45pm: Verge Genomics. Speaker: Irene Y. Choi, PhD, Senior Director/ Head of Drug Discovery at Verge Genomics. 

• Abstract Title: “Accelerating discovery and development of ALS therapeutics with an AI-powered All-in-Human Platform.”

• RECORDING: https://youtu.be/_p0zz1Kt-mw

2:45 - 3:00pm: Break    

3:00 - 4:30pm: Roundtable Discussion - a review of the exciting new developments reported during the first two days of the Symposium, and promising future directions in ALS investigations and trials. 

Panelists:

• James Berry, MD, MPH

• Merit Cudkowicz, MD, MSc

• Angela Genge, MD

DAY 3 (Tuesday, Oct. 11h, 2022)

9:00am - 4:00pm Eastern Time.

We proudly present “NEUROTECH NOW”, a collaboration with Indu Navar, MSCS, Founder of Everything ALS, Dr. Shelena Lalji, MD, F.A.C.O.G.,, Founder of ALS Heroes, and Dr. Leigh Hochberg, MD, PhD, of the Center for Neurotechnology and Neurorecovery at MGH.

Morning Session (9:00am - 12:00pm ET): Neurodiagnostics: Digital Biomarkers

9am – 12:00pm ET: Neurodiagnostics: Digital Biomarkers

• 9:00am: Intro: James Berry, MD, MPH

• 9:10am: Patient Perspective: Tina Cascio

• 9:15am: Intro: Indu Navar, MSCS, CEO and Founder of EverythingALS.

Moderator: James Berry, MD, MPH

Speakers:

• 9:20am: Biogen Digital Health Science, NMD: Kelley Erb, PhD, Principal Investigator, Biogen Digital Health Science, NMD.

• 9:30am: Moderator: James Berry, MD, MPH

• 9:35am: MT-Pharma: Satoshi Iwasaki, PhD, Senior Director, Digital Healthcare Innovation, Corporate & Product Strategy at MT-Pharma.

• 9:45am: Moderator: James Berry, MD, MPH

• 9:50am: Novartis: Ram Miller, MD, MBA, FRCPC, Executive Director at Novartis.

• 10:00am: Moderator: James Berry, MD, MPH

• 10:05am: NeuroSense Therapeutics: Alon Ben-Noon, Co-Founder and CEO of NeuroSense Therapeutics.

• 10:15am: NeuraLight Micha Breakstone, PhD, Co-Founder and CEO of NeuraLight.AI

• 10:25am: Moderator: James Berry, MD, MPH 

10:30am: Break 

• 10:45am: EverythingALS/Vision: Indu Navar, MSCS & Meera Gandhi

• 10:50am: EverythingALS Data Science Team: Alan Taitz & Julian Peller, PhD

• 11:00am: Modality.AI: David Suendermann-Oeft, Founder and CEO of Modality.AI

• 11:10am: Moderator: James Berry, MD, MPH

• 11:15am: Amazon AWS: Rishi Madhok, MD, Venture Capital & HCLS Startups at Amazon Web Services (AWS)

• 11:35pm: Panel Discussion of Industry Speakers moderated by: James Berry, MD, MPH, & Indu Navar, MSCS

12:00 – 1:00pm: Break

Afternoon Session (1:00 - 4:15pm ET): Brain-Computer Interfaces (BCI's)

Revolutionary implanted brain-computer interfaces (BCIs) could restore communication and mobility for people with ALS. Recent industry investment in this field underscores its potential.

During this session, CEOs from three BCI companies (Blackrock Neurotech, Paradromics, and Synchron) will describe their plans for the future of their products and the field, as well as the challenges ahead for bringing technologies to market. Leading BCI researchers from academia will reflect on the industry presentations, asking questions that highlight opportunities for even more exciting BCIs for people with ALS, followed by a roundtable with the industry leaders.

The session will close with a Q&A where Symposium attendees can ask questions of the industry and academic panelists. The session will be moderated by Leigh Hochberg, MD, PhD, director of the internationally respected BrainGate consortium and clinical trials.

Moderators

• Merit Cudkowicz, MD, MSc, Chief, Neurology Department - Director, Sean M. Healey & AMG Center for ALS Mass General Hospital and Julianne Dorn Professor of Neurology Harvard Medical School.

• Leigh Hochberg, MD, PhD, Director, Center for Neurotechnology and Neurorecovery, Neurocritical Care and Stroke Services, Department of Neurology, Massachusetts General Hospital; Senior Lecturer on Neurology, Harvard Medical School; L. Herbert Ballou University Professor of Engineering and Brain Science, Brown University; Director, VA RRR&D Center for Neurorestoration and Neurotechnology, Providence VA Medical Center.

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

Schedule of Speakers:

• 1:00pm: Intro: Shelena C. Lalji, MD, F.A.C.O.G., Founder of ALS Heroes and The Dr.Shel Wellness & Aesthetic Center. 

• 1:08pm: BrainGate: Leigh Hochberg, MD, PhD, Director, Center for Neurotechnology and Neurorecovery, Neurocritical Care and Stroke Services, Department of Neurology, Massachusetts General Hospital; Senior Lecturer on Neurology, Harvard Medical School; L. Herbert Ballou University Professor of Engineering and Brain Science, Brown University; Director, VA RRR&D Center for Neurorestoration and Neurotechnology, Providence VA Medical Center.

• 1:23pm: Blackrock: Florian Solzbacher, PhD, Co-Founder, Executive Chairman & President, Blackrock Neurotech.

• 1:38pm: Paradromics: Matt Angle, PhD, Founder & CEO at Paradromics

• 1:53pm: Synchron: Tom Oxley, MD, PhD, Founder & CEO at Synchron

• 2:08pm: Intro to Panel: Questions from academics/nonprofit experts: Leigh Hochberg, MD, PhD,

• 2:09pm: Mariska Vansteensel, PhD, Assistant Professor at the UMC Utrecht Brain Center in Utrecht.

• 2:14pm: Brian Dekleva, PhD, Research Scientist, Rehab Neural Engineering Labs, University of Pittsburgh.

• 2:19pm: Nathan Crone, MD, Professor of Neurology at The Johns Hopkins University School of Medicine.

• 2:24pm: Sydney Cash, MD, PhD, Associate Professor of Neurology at Harvard Medical School and attending epileptologist at Massachusetts General Hospital.

• 2:29pm: Tracy Laabs, PhD, Chief Development Officer at the Wyss Center in Geneva. 

• 2:34pm: David Moses, PhD, Postdoctoral Scholar in the Chang Lab at UCSF.

2:39 – 2:50pm: Break

2:50pm: Roundtable 1: Responses to presentations and inquiries - moderated by Leigh Hochberg, MD, PhD, Merit Cudkowicz, MD, MSc, Shelena Lalji, MD.

3:20pm: Roundtable 2: Q&A from ALS community, moderated by Leigh Hochberg, MD, PhD, Merit Cudkowicz, MD, MSc, and Shelena Lalji, MD, FACOG.

3:50pm: Wrap-up/Conclude: Wrap-Up: Merit Cudkowicz, MD, MSc and Shelena Lalji, MD.

RECORDING OF NEUROTECHNOLOGY DAY: https://youtu.be/aARkkUOZiHc

This concludes our 3-day 2022 Symposium. With immense gratitude to all speakers, sponsors, and attendees.

Updated on 10/4/2022.