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.
"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 NeurologyLive, 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.