Clinical Trials

ALS Finding a Cure has contributed to clinical testing of multiple potential ALS treatments. In addition to directly funding clinical trials, ALS Finding a Cure has also funded the development of the tools necessary for the clinical trials of the future, including imaging biomarkers for both the brain and spinal cord. Here is a list of clinical trials that ALS Finding a Cure is involved in.


Principal Investigator(s): 
Sabrina Paganoni, MD, Massachusetts General Hospital

Collaborators involved: ALS Finding a Cure Foundation, Amylyx Pharmaceuticals Inc., ALS Association, Northeast ALS Consortium, Massachusetts General Hospital Neurology Clinical Research Institute, Leandro P. Rizzuto Foundation

Overview and description:
Josh Cohen and Justin Klee, co-founded Amylyx Pharmaceuticals

Amylyx is a for-profit company that has developed a novel therapeutic, AMX0035, for the treatment of ALS. AMX0035 is a combination of two compounds, Sodium Phenylbutyrate (PB) and Tauroursodeoxycholic Acid (TUDCA). Each compound has exhibited strong efficacy in several cellular and animal models of ALS. Furthermore, PB and TUDCA have been individually tested in clinical trials of ALS and both showed safety and tolerability and preliminary signs of efficacy. Amylyx discovered a synergy between these two compounds when administered together in a particular range of ratios across multiple preclinical models. In combination, the compounds powerfully block the types of cellular death and inflammation that characterize the ALS disease process. This trial will test AMX0035 in order to determine:

  1. The safety and tolerability of AMX0035;
  2. The ability of AMX0035 to block ALS cell death and neurotoxic inflammation as measured by two novel biomarkers;
  3. The correlation between these biomarkers and functional outcomes.

If successful, this trial will show that AMX0035 is safe for use in ALS patients, validate AMX0035’s mechanism of action, correlate promising biomarkers with functional measurements, and set AMX0035 on a path towards a larger trial to confirm efficacy.

Funding:  ALSFAC, For-profit Investors, and ALS Association

Web site:, Amylyx

Results: Trial of Sodium Phenylbutyrate–Taurursodiol for Amyotrophic Lateral Sclerosis

Principal Investigator(s): 
Merit Cudkowicz, M.D., MSc, Massachusetts General Hospital in Boston
Robert J. Brown, Jr. M.D., D.Phil., University of Massachusetts in Worcester, Mass
Stanley H. Appel, M.D., Houston Methodist Hospital System in Houston
Nazem Atassi, Ph.D., University of Massachusetts in Worcester, Mass
Clive Svendsen, Ph.D., Cedars-Sinai in Los Angeles
Nadeem Ishaque, Ph.D., and Thomas Gentile, M.B.A., GE Healthcare

Collaborators involved: ALS Finding a Cure

Overview and description:
ALS ACT is a novel academic-foundation-industry partnership with ALS Finding a Cure®, initiated with researchers from General Electric (GE) Healthcare and four academic Northeast ALS Consortium (NEALS) sites to accelerate treatments for people living with ALS. Through a multi-pronged approach, each partner will contribute to expediting clinical trials in ALS.

Funding:  ALS Association, ALS Finding a Cure

Web site:  ALS Accelerated Therapeutics (ALS ACT) | The ALS Association

Principal Investigator(s): 
Michael Weiss, MD University of Washington
Phone: 206-598-7688

Collaborators involved: Massachusetts General Hospital

Overview and description:
The purpose of this research study is to find out whether the drug mexiletine will be effective in lowering motor neuron electrical activity in the brains and nerves in the arms of people with ALS. The investigators will also determine if there are any signs that the drug may slow down the progression of ALS and reduce muscle cramps and muscle twitching. This will be determined through transcranial magnetic stimulation (TMS) and threshold tracking nerve conduction studies (TTNCS). In this trial, the participants will be taking either 300mg/day of mexiletine, 600mg/day of mexiletine, or placebo (non-active study drug).

Funding:  University of Washington


Web site: Mexiletine in Sporadic Amyotrophic Lateral Sclerosis – Full Text View –

Principal Investigator(s): 
Katherine Nicholson, MD
James Berry, MD
Alberto Ascherio

Collaborators involved: Massachusetts General Hospital, Harvard School of Public Health, The Broad Institute

Overview and description:
There is growing interest in how the interplay between the intestinal microbiota (i.e., bacteria in the gut) and its host influence the onset and course of neurodegenerative diseases, potentially mediated by immune mechanisms that modulate the microglial environment. No studies have previously examined whether the overall composition of the intestinal microbiota, or specific bacterial species, are associated with ALS. In this pilot study, the intestinal microbiota of 100 people with ALS and 100 healthy controls was compared. The study used novel technology and was a collaboration project among the Harvard School of Public Health, Broad Institute, and the Neurological Clinical Research Institute at Massachusetts General Hospital, involving leading epidemiologists, microbiome analysts, and ALS clinical researchers.


Principal Investigator(s):
Merit Cudkowicz MD, Massachusetts General Hospital
Robert Brown D.Phil., MD, University of Massachusetts Medical School
James Berry MD, Massachusetts General Hospital

Collaborators involved: University of Massachusetts and Massachusetts General Hospital

Overview and description:
About 10% of ALS cases are familial. Most of the known genetic defects in familial ALS act by triggering one or more toxic processes that impair the viability of motor neurons, leading to motor neuron death. This project uses viruses to introduce reagents into the brain and spinal cord that block the mutant genes from triggering toxicity. This is accomplished by turning off the activity of those genes using new technologies for so-called “gene silencing.” The viruses are used to penetrate the blood-brain barrier to deliver the gene silencing reagents, which then inactivate the genes in question. This technology is in process of being tested in cases of ALS arising from mutations in the well-defined ALS gene SOD1. This project is intended to take the gene silencing all the way to a pilot human trial. We are hopeful that in the long term these studies will lead to a general platform or method for treating other types of familial ALS and potentially also some cases of non-familial ALS.

Web site: Silencing strategies for therapy of SOD1-mediated ALS – PubMed (

Results: SOD1 Suppression with Adeno-Associated Virus and MicroRNA in Familial ALS

Principal Investigator(s): 
Merit Cudkowicz, MD, MSC
Director, Sean M. Healey & AMG Center for ALS

Collaborators involved:
Tackle ALS
Massachusetts General Hospital
Barrow Neurological Institute
Northeast ALS Consortium
University of Rochester
Berry Consultants
Biohaven Pharmaceuticals
Clene Nanomedicine
Implicit Bioscience
Seelos Therapeutics
Overview and description:
Borrowing from successes in cancer drug development, the Sean M. Healey & AMG Center for ALS at Mass General, in partnership with Tackle ALS, are leading the first Platform Trial initiative for ALS. Traditionally, each trial evaluates only one drug at a time, and requires lengthy start-up and execution times. Platform trials, instead, are trials where multiple drugs are tested at the same time, using specialized statistical tools. New regimens (drugs) can be added as they become available thereby decreasing or eliminating the gap in time from identification of a rationale therapy to testing. Thus, the focus is on the disease, rather than any individual experimental agent, and the platform remains open long-term until successful cures are found. The HEALEY ALS Platform Trial will test multiple promising experimental therapeutics using an efficient and informative early-phase design with increased access for people with ALS. Overall, this approach can reduce the cost of research by 30%, decrease the trial time by 50% and increase patient participation by 67%.

Funding:  Sean M. Healey, the AMG Charitable Foundation, The ALS Association, Northeast ALS Consortium, Tackle ALS, ALS Finding a Cure, Muscular Dystrophy Association (MDA), ALS ONE, sALSa For a Cure, participants in the Ice Bucket Challenge 2, IAMALS, Run2Revive, and countless other donors and individuals who have motivated their communities to make an impact.


Web site:
HEALEY ALS Platform Trial (
HEALEY ALS Platform Trail – Master Protocol (Clinical
Trial Sites for the HEALEY ALS Platform Trial

Principal Investigator(s): 
Study Director: Stanley H. Appel, MD The Methodist Hospital Research Institute
Jason R. Thonhoff, MD, PhD The Methodist Hospital Research Institute
James D. Berry, MD, MPH Massachusetts General Hospital”

Collaborators involved:
Massachusetts General Hospital
The Center for Clinical and Translational Sciences (CCTS)
Clinical Research Unit at The University of Texas
Health Science Center at Houston
North East Amyotrophic Lateral Sclerosis Consortium

Overview and description:
This study is a randomized, placebo-controlled, phase 2a trial to study the biological activity, safety, and tolerability of regulatory T Lymphocytes (Tregs) taken and expanded outside of the body and returned back to the same person whose Treg were removed, given back by IV (intravenously) and in combination with low-dose IL-2 in people with Amyotrophic Lateral Sclerosis (ALS).

Funding:  Coya Therapeutics, The Methodist Hospital Research Institute

Phase 1: Houston Neurologist Discovers Treatment That Could Halt the Progression of ALS
Phase 2: Underway

Dr. Stanley Appel Finds Immune Cells Hold Promise in Slowing Down ALS
Expanded autologous regulatory T-lymphocyte infusions in ALS

Web site: