# Merit Cudkowicz
Merit E. Cudkowicz is an American neurologist and clinical trialist who leads amyotrophic lateral sclerosis (ALS) research at [Massachusetts General Hospital](https://www.edgepedia.org/massachusetts-general-hospital) (MGH) in Boston. She became Chief of Neurology at MGH and directs the MGH Neurological Clinical Research Institute[1](https://researchers.mgh.harvard.edu/profile/1520993/Merit-Cudkowicz), and she is Director of the Sean M. Healey & AMG Center for ALS, Executive Director of the Mass General Brigham Neuroscience Institute, and the Julieanne Dorn Professor of Neurology at Harvard Medical School[2](https://www.massgeneral.org/doctors/16904/merit-cudkowicz). Her field is ALS drug development, where she has led or co-led numerous clinical trials, and she launched the HEALEY ALS Platform Trial, described as the first platform trial initiative in ALS[2](https://www.massgeneral.org/doctors/16904/merit-cudkowicz) • [3](https://doi.org/10.1097/01.wnt.0001162356.70502.01).
| Fact | Detail |
|---|---|
| Chief of Neurology, MGH | Also Director of the MGH Neurological Clinical Research Institute[1](https://researchers.mgh.harvard.edu/profile/1520993/Merit-Cudkowicz) |
| Harvard roles | Julieanne Dorn Professor of Neurology; Director, Sean M. Healey & AMG Center for ALS; Executive Director, MGB Neuroscience Institute[2](https://www.massgeneral.org/doctors/16904/merit-cudkowicz) |
| Training | BS in chemical engineering, MIT; MD, Harvard Medical School, 1990; MSc in clinical epidemiology, Harvard T.H. Chan School of Public Health, 1996[2](https://www.massgeneral.org/doctors/16904/merit-cudkowicz) • [1](https://researchers.mgh.harvard.edu/profile/1520993/Merit-Cudkowicz) |
| Signature work | CENTAUR trial of sodium phenylbutyrate–taurursodiol, New England Journal of Medicine, 2020[4](https://www.nejm.org/doi/full/10.1056/NEJMoa1916945) |
| Consortium building | Co-founder of the Northeast ALS Consortium (NEALS), 1995; grew to more than 150 research centers worldwide[5](https://advances.massgeneral.org/neuro/article.aspx?id=1598) |
| Platform trial | HEALEY ALS Platform Trial: over 500 participants at 52 US sites; 1,500 participants and 8 treatment groups as of July 2026[6](https://docs.house.gov/meetings/IF/IF14/20210729/113983/HHRG-117-IF14-Wstate-CudkowiczM-20210729.pdf) • [7](https://www.centerwatch.com/clinical-trials/listings/NCT04297683/healey-als-platform-trial-master-protocol) |
| Recognition | American Academy of Neurology Lifetime Achievement Award, April 2025[8](https://www.healio.com/news/neurology/20250407/merit-e-cudkowicz-md-msc-receives-lifetime-award-for-als-research-im-not-done-yet) |
## Training and career
Cudkowicz completed an [undergraduate degree](https://www.edgepedia.org/undergraduate-degree) in chemical engineering at the [Massachusetts Institute of Technology](https://www.edgepedia.org/massachusetts-institute-of-technology) and obtained her medical degree in the Health Science and Technology program of Harvard Medical School, earning the MD in 1990[2](https://www.massgeneral.org/doctors/16904/merit-cudkowicz) • [1](https://researchers.mgh.harvard.edu/profile/1520993/Merit-Cudkowicz). According to her Mass General profile, her clinical training included an internship at Beth Israel Hospital, a residency and fellowship at MGH, and a master's degree in clinical epidemiology from the Harvard School of Public Health, completed in 1996[1](https://researchers.mgh.harvard.edu/profile/1520993/Merit-Cudkowicz). Her listed research interests center on ALS biomarkers and clinical epidemiology[1](https://researchers.mgh.harvard.edu/profile/1520993/Merit-Cudkowicz).
In conjunction with NEALS she planned and completed over 15 multi-center clinical research studies in ALS[1](https://researchers.mgh.harvard.edu/profile/1520993/Merit-Cudkowicz). She also serves as Principal Investigator of the Clinical Coordination Center for NeuroNEXT, the National Institute of Neurological Disorders and Stroke's Neurology Network of Excellence in Clinical Trials[2](https://www.massgeneral.org/doctors/16904/merit-cudkowicz).
## Representative work
The CENTAUR trial of sodium phenylbutyrate–taurursodiol (AMX0035), published in the New England Journal of Medicine in 2020, was a randomized, double-blind, placebo-controlled study run at 25 NEALS centers in the United States from June 2017 through September 2019; 137 of 177 screened participants were assigned to the drug combination (89) or placebo (48)[4](https://www.nejm.org/doi/full/10.1056/NEJMoa1916945). On the primary endpoint, the ALS Functional Rating Scale-Revised score declined 1.24 points per month with the drug versus 1.66 points per month with placebo, a difference of 0.42 points per month (95% confidence interval, 0.03 to 0.81; P=0.03)[4](https://www.nejm.org/doi/full/10.1056/NEJMoa1916945). Secondary outcomes did not differ significantly between groups, and the drug's adverse events were mainly gastrointestinal[4](https://www.nejm.org/doi/full/10.1056/NEJMoa1916945). She described the study as designed and run through NEALS and supported by a partnership between The ALS Association and ALS Finding a Cure[9](https://www.amylyx.com/news/amylyx-pharmaceuticals-announces-new-england-journal-of-medicine-publication-of-pivotal-amx0035-data-demonstrating-statistically-significant-benefit-in-people-with-als).
She also had a senior role in the antisense oligonucleotide program for SOD1 familial ALS, the first antisense treatment developed for a neurological disorder[2](https://www.massgeneral.org/doctors/16904/merit-cudkowicz); the VALOR trial of tofersen enrolled 108 participants carrying 42 unique SOD1 mutations, assigning 72 to the drug and 36 to placebo[10](https://www.nejm.org/doi/full/10.1056/NEJMoa2204705). That program led to FDA approval of Qalsody (tofersen, Biogen) for SOD1 ALS[8](https://www.healio.com/news/neurology/20250407/merit-e-cudkowicz-md-msc-receives-lifetime-award-for-als-research-im-not-done-yet).
## The HEALEY ALS Platform Trial
The HEALEY ALS Platform Trial tests several investigational treatments simultaneously under one master protocol[6](https://docs.house.gov/meetings/IF/IF14/20210729/113983/HHRG-117-IF14-Wstate-CudkowiczM-20210729.pdf). Testifying before a House Energy and Commerce subcommittee in July 2021, she reported that despite COVID-19 the trial had enrolled over 500 people across 52 US clinical sites, with enrollment 4 times faster than any prior ALS trial, and that a fourth regimen had been added within 30 days of launch[6](https://docs.house.gov/meetings/IF/IF14/20210729/113983/HHRG-117-IF14-Wstate-CudkowiczM-20210729.pdf). Operationally, the first three regimens were approved by a single institutional review board in 120 days, enrollment for those regimens completed in 15 months, and a fourth regimen (pridopidine, sponsored by Cudkowicz with Prilenia as collaborator) enrolled 163 participants from December 2020 to July 2022[11](https://doi.org/10.1002/mus.28442) • [12](https://clinicaltrials.gov/study/NCT04615923).
## How it compares with standard ALS drug development
Shared controls and Bayesian methods are the trial's statistical core. Conventional parallel-group trials in ALS, she testified, are too large, long, and expensive, and too often exclude people who want to take part[6](https://docs.house.gov/meetings/IF/IF14/20210729/113983/HHRG-117-IF14-Wstate-CudkowiczM-20210729.pdf). The platform design cuts the time to evaluate a treatment roughly in half and greatly reduces drug-development costs while minimizing placebo allocation[6](https://docs.house.gov/meetings/IF/IF14/20210729/113983/HHRG-117-IF14-Wstate-CudkowiczM-20210729.pdf). Its control cohort shares placebo-treated participants across regimens and across time, which increases statistical efficiency and reduces overall allocation to placebo without loss of power relative to 1:1 randomization, with [Bayesian hierarchical modeling](https://www.edgepedia.org/bayesian-hierarchical-modeling) used to account for differences among the shared controls[13](https://doi.org/10.1002/ana.26714). A 2024 commentary by the trial leadership described the framework as enabling concurrent enrollment into four distinct regimens under a single adaptable infrastructure[14](https://www.nature.com/articles/s43587-024-00740-x). She has said the idea came after reading an article on master platform trials by a former FDA Principal Deputy Commissioner[15](https://www.biospace.com/drug-development/the-healey-platform-trial-quick-failures-and-new-hope-for-als).
## Recognition and recent developments
In April 2025 the [American Academy of Neurology](https://www.edgepedia.org/american-academy-of-neurology) honored Cudkowicz with its Lifetime Achievement Award for her work in ALS research[8](https://www.healio.com/news/neurology/20250407/merit-e-cudkowicz-md-msc-receives-lifetime-award-for-als-research-im-not-done-yet). Earlier honors include the 2009 Sheila Essay ALS award, the 2017 Forbes Norris Award from the International MND Alliance, and the 2019 Ray Adams Award from the American Neurological Association[2](https://www.massgeneral.org/doctors/16904/merit-cudkowicz).
In February 2025, results from the first four drugs evaluated through the HEALEY Platform Trial were published; none met its primary endpoint[5](https://advances.massgeneral.org/neuro/article.aspx?id=1598) • [16](https://www.medscape.com/viewarticle/als-trials-fail-meet-key-endpoints-its-still-win-and-heres-2025a10004q5). She argued that the negative results showed the platform model working as intended, weeding out ineffective agents: "These are not failed trials"[16](https://www.medscape.com/viewarticle/als-trials-fail-meet-key-endpoints-its-still-win-and-heres-2025a10004q5). The revised master protocol lengthens the testing period for each regimen, enrolls participants earlier in the disease, and adds biomarkers; seven drugs had been tested to date, with new regimens slated for 2025 and 2026[5](https://advances.massgeneral.org/neuro/article.aspx?id=1598). Her other current initiative is the ALS MyMatch Program, which matches patients to drugs in phase 2a development[8](https://www.healio.com/news/neurology/20250407/merit-e-cudkowicz-md-msc-receives-lifetime-award-for-als-research-im-not-done-yet). As of the master protocol's July 17, 2026 update, the trial is active but not recruiting, with 1,500 total participants and 8 treatment groups[7](https://www.centerwatch.com/clinical-trials/listings/NCT04297683/healey-als-platform-trial-master-protocol).
## References
1. [Merit Cudkowicz, MD, MSc - MGH researchers profile](https://researchers.mgh.harvard.edu/profile/1520993/Merit-Cudkowicz)
2. [Merit Cudkowicz, MD, MSc - Department of Neurology, Mass General](https://www.massgeneral.org/doctors/16904/merit-cudkowicz)
3. [Building Platforms for Progress: Merit Cudkowicz on the Challenges, and Hope, of a Life in ALS Research (Neurology Today)](https://doi.org/10.1097/01.wnt.0001162356.70502.01)
4. [Trial of Sodium Phenylbutyrate–Taurursodiol for Amyotrophic Lateral Sclerosis (CENTAUR), NEJM 2020](https://www.nejm.org/doi/full/10.1056/NEJMoa1916945)
5. [Innovative Platform Trials Reshape the Future of ALS Drug Discovery (Mass General Advances in Motion)](https://advances.massgeneral.org/neuro/article.aspx?id=1598)
6. [Testimony of Merit Cudkowicz, House Energy & Commerce Subcommittee on Health, July 29, 2021](https://docs.house.gov/meetings/IF/IF14/20210729/113983/HHRG-117-IF14-Wstate-CudkowiczM-20210729.pdf)
7. [HEALEY ALS Platform Trial, Master Protocol (NCT04297683), CenterWatch](https://www.centerwatch.com/clinical-trials/listings/NCT04297683/healey-als-platform-trial-master-protocol)
8. [Merit E. Cudkowicz, MD, MSc, receives lifetime award for ALS research (Healio)](https://www.healio.com/news/neurology/20250407/merit-e-cudkowicz-md-msc-receives-lifetime-award-for-als-research-im-not-done-yet)
9. [Amylyx announcement of NEJM publication of pivotal AMX0035 data](https://www.amylyx.com/news/amylyx-pharmaceuticals-announces-new-england-journal-of-medicine-publication-of-pivotal-amx0035-data-demonstrating-statistically-significant-benefit-in-people-with-als)
10. [Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS (VALOR), NEJM 2022](https://www.nejm.org/doi/full/10.1056/NEJMoa2204705)
11. [Operational Development and Launch of an Adaptive Platform Trial in ALS (Muscle & Nerve)](https://doi.org/10.1002/mus.28442)
12. [HEALEY ALS Platform Trial, Regimen D Pridopidine (NCT04615923), ClinicalTrials.gov](https://clinicaltrials.gov/study/NCT04615923)
13. [Design and Statistical Innovations in a Platform Trial for Amyotrophic Lateral Sclerosis (Annals of Neurology)](https://doi.org/10.1002/ana.26714)
14. [Lessons from the HEALEY adaptive platform trial in amyotrophic lateral sclerosis (Nature Aging, 2024)](https://www.nature.com/articles/s43587-024-00740-x)
15. [The HEALEY Platform Trial: Quick Failures and New Hope for ALS (BioSpace)](https://www.biospace.com/drug-development/the-healey-platform-trial-quick-failures-and-new-hope-for-als)
16. [ALS Trials Fail to Meet Key Endpoints…But It's Still a Win (Medscape, 2025)](https://www.medscape.com/viewarticle/als-trials-fail-meet-key-endpoints-its-still-win-and-heres-2025a10004q5)
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*Topic: Encyclopedia › Life and health › Life and health scientists › Medical and health researchers › Researchers in clinical neuroscience, neurology, and psychiatry research › Amyotrophic lateral sclerosis and frontotemporal dementia*
*Initially written Sep 21, 2026 · Reviewed: Sep 25, 2026 · Edited: Sep 25, 2026; Oct 11, 2026; Oct 15, 2026; Oct 21, 2026 · Last review: Sep 25, 2026*
*Copyright 2026 EdgeChat AI, a subsidiary of Biostate AI.*
License: Edgepedia Community License 1.0, https://www.edgepedia.org/edgepedia/license