Winthrop Family Scholar in ALS Sciences; director, MGH Neurological Clinical Research Institute (NCRI); and chief, Division of ALS and Motor Neuron Diseases, Massachusetts General Hosptial

James Berry, MD, MPH

"We don’t want to create this altar of homogenous population that we sort of bow to because there’s some statistical benefit to it, while ignoring the critical importance of getting potential new therapies to people now, and allowing them to help the community, help themselves, help other people, by participating in trials. That balance is critical.”

There are a lot of unknowns surrounding the role that genetic variants play for patients with amyotrophic lateral sclerosis (ALS), but many researchers feel as though this is an area that needs further examination. To date, there has not been conclusive evidence that indicates which genetic mutations of the disease may progress earlier rather than later, or the degrees of severity to which they progress.

Similar to other basic sciences, the statistical power can be boosted by having a more homogenous group coming into the trial, according to James Berry, MD, MPH. Berry, director of the Massachusetts General Hospital ALS Care Center, feels as though there needs to be the right balance of homogenous populations while not forgetting some of the other more sporadic and unpredictive genetic backgrounds.

Berry detailed some of the complexities within ALS clinical trials, adjusting for genetic backgrounds, and the pros and cons to focusing on a more homogenous gene population.

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The director of the Massachusetts General Hospital ALS Care Center detailed the role genetic backgrounds can have on outcomes within ALS clinical trials.

Genetic Variants Impact on ALS Clinical Trials: James Berry, MD, MPH

"There needs to be a focus on patient-reported outcome measures on digital outcome measures that can quantify behavior and show that a drug can not only affect some scale, but how it impacts patients and their lives. That is a new developing important part of what we’re doing in clinical trials."

Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disorder that involves gradual onset, with a number of symptoms that can affect quality of life functions. In some cases, the problem presents first in the muscles controlling speech, producing alterations in the vocal quality, or swallowing, which can lead to coughing and choking. Other symptoms such as constipation, impairment of the stomach, bloating, and urinary urgency.

At the moment, there are a limited number of treatments for not only the motor disabilities associated with ALS, but other quality of life issues that patients face daily. For James Berry, MD, MPH, understanding the impact of the disease on people’s lives is among the top concerns, so that outcome measures in clinical trials correctly measure the impact of the disease and what’s most important about the progression.

Berry, director of the Massachusetts General Hospital ALS Care Center, believes that there are a number of issues that need addressing along with trying to slow or halt disease progression. In an interview with 

he details some of the most pressing unmet needs within the ALS community, as well as advocating for gaining a better understanding of the overall biology of the disease.

The director of the Massachusetts General Hospital ALS Care Center discussed some of the biggest challenges for patients with ALS and what should be prioritized in research. 

Tackling Quality of Life Issues in ALS: James Berry, MD, MPH

"If you have the data, it draws new interest, and allows people to move faster. We’ve seen a similar thing with some of the bio repositories that we have.”

As telemedicine use has grown, so has the use of shared data-based registries. These banks of data can contain research from a wide range of global studies and can act as a central foundation for future studies by identifying trial participants and building upon previous research.

One such effort tabbed by Answer ALS is its most recent campaign, “The Data is Here,” which includes the release of the world’s largest open-sourced data portal for amyotrophic lateral sclerosis (ALS). James Berry, MD, PHD, director, ALS Care Center, Massachusetts General Hospital (MGH), notes that rarer disease states that do a great job of sharing resources are the ones who have made breakthroughs.

Berry, who also serves as the program director of Answer ALS and director of the MGH Neurological Clinical Research Institute (NCRI), used cystic fibrosis as a prime example of the benefits of data collaboration. In an interview with 

, Berry shared his thoughts on how these registries can help further research within the ALS community.

The director of the Massachusetts General Hospital ALS Care Center spoke on the importance of data-based registries and how they can grow ALS research.

Benefits to Data-Based Registries in ALS: James Berry, MD, MPH

Answer ALS recently just announced the release of the world’s largest open-sourced amyotrophic lateral sclerosis (ALS) data portal, which collected data from over 1000 ALS patient participants and controls. Launched as “The Data is Here,” the event becomes the first to publicly make data from nearly all biological assessments or “multi-omics” combined with clinical data. The datasets, which include genomic, transcriptomic, metabolomic, and epigenomic sequence data, were all derived from individual Answer ALS study participants.

James Berry, MD, MPH, director, ALS Care Center, Massachusetts General Hospital (MGH), feels as though there is a growing understanding of ALS from the range of clinical trials currently being conducted. The portal will be able to continue to empower researchers while serving as a foundation for new clinical trials and locating ideal trial participants.

Berry, who also serves as the program director of Answer ALS and director of the MGH Neurological Clinical Research Institute (NRCI), sat down with 

 to discuss how the campaign was formed, what benefits it may bring, and why it adds to the growing momentum within the ALS community.

Answer ALS launches ‘The Data is Here’ campaign to announce the release of the world’s largest open-sourced ALS data portal. News release. January 8, 2021. Accessed January 26, 2021. 

https://www.prnewswire.com/news-releases/answer-als-launches-the-data-is-here-campaign-to-announce-the-release-of-the-worlds-largest-open-sourced-als-data-portal-301203439.html

The director of the Massachusetts General Hospital ALS Care Center discussed the origins of ‘The Data is Here’ campaign and the reason to launch it now. 

James Berry, MD, MPH

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Genetic Variants Impact on ALS Clinical Trials: James Berry, MD, MPH

Tackling Quality of Life Issues in ALS: James Berry, MD, MPH

Benefits to Data-Based Registries in ALS: James Berry, MD, MPH

Answer ALS’s Campaign Opens Door to Increased Data Access: James Berry, MD, MPH

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