Skip to content

Innovations in ALS Clinical Trials

In This Video

  • Merit Cudkowicz, MD, MSc, is the chief of Neurology, director of the Amyotrophic Lateral Sclerosis Clinic and the Neurological Clinical Research Institute at Massachusetts General Hospital and Partners HealthCare
  • In this video, she discusses innovations in ALS clinical trials since she started in the field in 1994

In this video, Merit Cudkowicz, MD, MSc, chief of Neurology Service, director of the Sean M. Healey & AMG Center for ALS, director of the Amyotrophic Lateral Sclerosis (ALS) Clinic and the Neurological Clinical Research Institute (NCRI) at Massachusetts General Hospital and Partners HealthCare, discusses innovations in ALS clinical trials since she started in the field in 1994.

This is Part 1 of a three-part series on ALS treatment.

  • Part 2: Innovations in Care for Patients with ALS
  • Part 3: Biomarkers to Accelerate ALS Therapy Development


I started in the field of ALS in 1994 and at time there was one drug in the market, Riluzole, and there was just a handful of people studying this illness and really not many people doing clinical trials. That has completely changed now where there’s thousands of investigators all over the world studying ALS. There’s a lot of knowledge about how to do trials and there’s a lot of therapeutic ideas.

My passion has been always to find treatments for people with ALS and to speed up the process to get rid of inefficiencies because our families don’t have time for any inefficiency. So, one of the things we’ve done is to set up something called a central IRB where if we’re going to do a clinical trial, there’s one review of the protocol and all the centers can start. That alone has cut down development time by a year. I like to sit back and say we have accomplished something. Now I’m tackling the next inefficiency, and that is we have a lot of therapeutic ideas, but if we do one trial at a time, we’re going to still be here 20 years from now and that’s way too slow.

I want to adopt some technologies and approaches that are used in oncology and that’s the idea of a platform trial, testing multiple things in the same trial and having quick readouts of something if something works or not so that we can take the best ideas forward faster. If we can set this up in ALS, we are going to speed up the process ten or twenty-fold. There’s interest in doing this. We’re getting the right people together. We’re learning from our colleagues in oncology. This is really a must for ALS. We really want to raise some philanthropy to support setting up this process, so we can get the cures for our patients much, much faster.

Learn about the ALS Multidisciplinary Clinic at Mass General

Refer a patient for ALS care


In this video, Ghazaleh Sadri-Vakili, PhD, director of the NeuroEpigenetics Laboratory and the MassGeneral Institute of Neurodegenerative Disease, discusses her genetic research, specifically concerning neurodegenerative diseases like ALS and X-linked dystonia parkinsonism. 


In this video, Dr. Merit Cudkowicz, MD, MSc, chief of Neurology, discusses biomarkers that can help to diagnose patients quicker and target individualized treatments to patients.