MDA and ALS ONE Work to Advance ALS Biomarker Research

MDA has awarded a human clinical trial grant totaling $750,000 to ALS ONE to explore the potential for a type of imaging called positron emission tomography (PET) to measure inflammation in the brain that could serve as a biomarker for ALS.

ALS ONE is an alliance between four institutional leaders in ALS treatment development: Massachusetts General Hospital (MGH), ALS Therapy Development Institute (ALS TDI), University of Massachusetts Medical School, and Compassionate Care ALS (CCALS). The partnership was formed with the goal of speeding the discovery of treatments for ALS and developing new approaches to improve care for individuals living with the disease.

The new project, led by Nazem Atassi, M.D., MSSc, will use PET imaging to track inflammation in healthy people who carry a known ALS gene and in symptomatic people with early-stage ALS. Atassi is a member of the ALS ONE science team, associate director for the Neurological Clinical Research Institute at Massachusetts General Hospital and associate professor of neurology at Harvard Medical School.

“This project is poised to change the paradigm of ALS drug development and have a direct impact on the design of future treatment trials for both familial and sporadic ALS,” Atassi says. “Real scientific advances in rare and orphan diseases rely heavily on the integration of the research community. Organizations such as MDA are the strongest links between all key stakeholders, including patients, academic institutions, philanthropy and industry, and we are incredibly grateful for their support.”

One of the biggest challenges hindering ALS drug development is a lack of biomarkers that can be used in clinical trials to signal that a drug is working (or not) in patients. With recent advances in the understanding of ALS and a robust and exciting drug development pipeline, biomarkers are needed to make it possible for researchers to quickly test many treatments.

In previous studies, data has shown a significant increase in inflammation in the motor regions of the brain in people with ALS. It also has been found that more inflammation tends to be associated with a more advanced stage of disease progression and greater impairment of motor function. Now, researchers at MGH are working to build on those findings and assess the ability of PET imaging to measure the degree of inflammation in the ALS-affected brain. Results from the study could contribute to the growing body of evidence suggesting PET imaging can be used as a tool to accurately measure inflammation in the brains of people living with ALS.

Implementing PET imaging technology potentially could reduce the duration of future trials from 12 months to three months, and the required patient enrollment from 400 participants to 30. This increased efficiency in the clinical trials stage of ALS drug development could translate into an ability to test more promising drugs and bring successful treatments to market faster.

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