NeuroVoices: James Berry, MD, MPH, on Answer ALS’s Expanded Data Campaign


The director of the Massachusetts General Hospital ALS Care Center sat down to discuss ‘The Data is Here’ campaign from Answer ALS, the world’s largest open-sourced ALS data portal.

James Berry, MD, MPA

Answer ALS, in collaboration with the Stephen Hawking Foundation, recently launched ‘The Data is Here’ campaign on January 8, 2021, to celebrate what would have been Hawking’s 79th birthday. The campaign gives global, and free, access to the most comprehensive ALS research in history through the recently developed and expanded Data Portal.

The portal empowers researchers with the critical tools necessary to explore the trove of data collected from over 1000 patients with ALS participants and controls. Through the use of artificial intelligence (AI) and machine learning, Answer ALS will serve as a foundation for new clinical trials, identifying trial participants, and to find drug-responsive biomarkers or diagnostics.

James Berry, MD, MPH, director, ALS Care Center, Massachusetts General Hospital (MGH), believes this campaign only adds to the increasing momentum within the ALS community. Berry, who also serves as the program director of Answer ALS and director of the MGH Neurological Clinical Research Institute (NCRI), sat down with NeurologyLive as part of the latest installment of NeuroVoices to provide insight on the data portal, the importance of data-based registries, and how it will continue to further ALS research.

NeurologyLive: Can you first talk a little bit about the data is here campaign, how it came about and what the sudden urge was for pushing this out?

James Berry, MD, MPH: Like most things, it starts slowly and gains speed in the end. The reality is that Answer ALS is a project that started 6 years ago, as an idea to create a shared resource for the ALS and neurodegeneration communities. We had learned a lot in different venues, including induced pluripotent stem cells (iPSC), the motor neurons from them, and then biology that they can contribute to leading us to clinical trials. We learned a lot about the omics fields and the complexities of those, as well as gained a lot of knowledge from doing biomarker studies in patients with ALS.

We understood the incredible importance of having a bio repository and large data set that was available to us from previously projects that those at Answer ALS had previously worked on. We brought that understanding to the leadership of the project and say, “Let’s do this bigger than we’ve done in the past but learn from the lessons before.” One of the cornerstones of the project was always that there would be a data repository, and that we needed to have a clear focus of that.

It’s not brand new, so I wouldn’t particularly say there’s been a push to get this done. What happened now is that its crystallized; we have the omics data, we have the underlying data structure, and now the community was fully ready for this because of some of the public relations work that other parts of our Answer ALS team had done.

What is the importance of data-based registries? What can they do for the ALS population specifically?

ALS is an uncommon disease. It’s not vanishingly rare, therefore we do have the opportunity to aggregate data and make it available to the community. But of note, because of its commonality, we don’t routinely have studies with 10,000 people in them. You can look at 1 study and reanalyze that data easily. We’re finding ourselves in this uncommon disease area, where we’ve seen again and again that in medicine, the disease areas that are uncommon but do a great job of sharing resources, are the ones who have made breakthroughs. Cystic fibrosis is a prime example of this.

In ALS, there was a move supported by a number of organizations in the past to aggregate clinical trial data into a data portal called Proact, which is widely available to the community and led to better trial planning, more insights into the disease, more clinically wide sharing of data, and more interest in the disease. That was a very powerful example. 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.

What’s unique about Answer ALS is that, from the inception, it was a collaboration between bench researchers and clinical researchers to put together what had been done in the clinical world of aggregating data, with the complexities of biological data, which has been harder to aggregate, normalize, collate, and share again. Prior to this, biofluids would be shared, the analysis would be done, and kept in a small data set. This gave us an opportunity to crowdsource, which fed data back into the project, enabled a broad biological data set, and target a whole new audience. That to me, is incredibly exciting.

ALS experts would say there is growing momentum within the community, do you agree?

I absolutely would agree there’s a growing momentum within the ALS community. We’re not seeing the combination of a lot of community building, shared resources, and shared knowledge that has been built up over the years. The same momentum that brought us to Answer ALS from a lot of learning projects, resources, and the thought that we could do even better on both the biological side and clinical side, has brought us to more and more effective treatments and positive clinical trials.

If you look at the history of clinical trials, there was always success in the early 90s. To modify the disease, new data was a success in treating a symptom of ALS pseudobulbar affect. We subsequently did studies looking at motor speech effects using Nuedexta and found that it actually can help speech in at least a subset of patients with ALS. Following that, within a couple of years we saw that positive trials of edaravone (Radicava; MT Pharma). And only a couple more years after that, we have the promising trial of AMX0035. The milestones are passing faster and faster. That’s because we can do trials fast, operate them better, in part because we can design them better because of shared resources and the better understanding of the biology.

This is also in part because we have a well-organized group of clinical researchers connected to basic researchers across the country. That’s what makes Answer ALS possible, but it also is what makes it so powerful. That community is ready to take advantage of the resources that are being made available. Why is there a pressure to release this data now? It’s because we see this momentum picking up, and we want to push it even faster.

Transcript edited for clarity.

For more information on the data portal, click here.

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.
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