James Berry, MD, MPH, is working to build tools and identify biomarkers to improve clinical trial methodology. His goal is to speed up the process of identifying the next drug that will slow, reverse or even prevent ALS in the future.

When a patient has strep throat, their care provider knows which antibiotics to prescribe to cure the ailment.

But when a disease has no known cure, as is the case with amyotrophic lateral sclerosis (ALS), research often becomes part of clinical care.

“There are still a lot of limitations in terms of what we can do to modify the disease for people with ALS,” says James Berry, MD, MPH, a researcher in the Neurological Clinical Research Institute (NCRI) at Massachusetts General Hospital. “We see research as a way forward, and for many patients, participating in clinical trials is a way of giving back to the ALS community at large.”

Berry and his colleagues at the NCRI are working to build tools that will improve clinical trial methodology, making for faster trials with fewer participants. In quickening the trials process, they can get their answers in less time and either continue down the pipeline if their results seem promising or move on to another drug.

The long-term goal is to speed up the process of identifying the next drug that will slow, reverse, or even prevent ALS in the future.

Role of Biomarkers and Technology

Berry says improving trials involves developing better outcome measures. “We are identifying factors, known as biomarkers, that we can measure at the very beginning of a trial and look for changes even one or two months later.”

Berry and his collaborators across the country are investigating markers of inflammation and nerve degeneration in the blood and the spinal fluid to find clues to ALS disease progression.

He is also seeking new biomarkers by integrating mobile technology with his research. Along with fellow NCRI researcher Sabrina Paganoni, MD, PhD, Berry is using cellphone apps to monitor patients’ behavior, function, and strength.

Berry and his team are testing an app they developed to track a patient’s daily movements, providing insight into behavioral changes which could indicate disease progression. “In our pilot studies, we’ve found this data to correlate quite nicely with the functional ratings scale we currently use to assess a patient’s function. However, this gives us more granular data that we can possibly use as a biomarker.”

In collaboration with Jordan Green, PhD, CCC-SLP, a speech language pathologist at the MGH Institute for Health Professions, Berry is also using technology to measure how patients’ speech changes throughout their disease progression.

“Slurred speech and eventually the inability to speak are two major effects of the disease,” says Berry. “For the first time, we’re developing an outcome measure around when, why, and how ALS patients experience vocal changes. Through our research, we can see that over time, there clearly is a change in speech even before patients or speech pathologists can detect it.”

Impact on Care

Berry says his work could have applications in both ALS research and clinical care.

“A lot of our focus is on the research side, but we’re also increasingly focused on care innovation. How do we use the tools available to do a better job of caring for people? My hope is to give us a way to intervene with the right tool at the right time.”

Berry imagines a scenario in which a patient is asked to make brief voice recordings every week, allowing their care team to monitor the recordings and bring in a speech pathologist when they begin to notice vocal changes. Or, integrating physical therapy when they see a patient isn’t moving around as much based on data from a cellphone app.

An Optimistic Outlook

Overall Berry feels optimistic about where the field is moving.

“If you go back five or ten years, there was one trial at a time. Now we have ten trials at a time and a pipeline of drugs. It’s incredibly exciting. It increases our chances that we’re going to find a new drug.”

“ALS is a difficult disease. I think when we as care providers are involved in research and we see the field moving forward, we can walk into the room with a patient with a pocket full of hope.”