The Emergence of Novel Biomarkers and Model Systems for ALS: Rita Sattler, MD, MSc

WATCH TIME: 4 minutes

"There’s not going to be 1 single biomarker that’s going to be the golden ticket for us. We need to understand that we’re probably going to need a combination of biomarkers. And depending on what the clinical trial is being designed as, we may pick one biomarker that sort of measures the overall changes in outcome and progression of the disease as well as another biomarker that would be more of a pharmacodynamic biomarker to see target engagement."

Amyotrophic lateral sclerosis (ALS), a fatal neurodegenerative disease, is pathologically characterized by cytoplasmic aggregation and nuclear depletion of TAR DNA-binding protein 43 (TDP-43). This disease mechanism also occurs in about 50% of patients with frontotemporal dementia. In recent years, TDP-43 has been among the handful of up-and-coming promising biomarkers for patients with the disease, for which there are only a handful of approved therapies.

The 2024 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, held from March 3-6, in Orlando, Florida, will feature the latest updates in neuromuscular diseases, including ALS. The conference programming boasts 6 tracks, focusing on: clinical considerations, lab to life, clinical approaches to streamlining care, practical considerations in gene therapy, and highlights of neuromuscular diseases. One specific track session, led by Rita Sattler, MD, MSc, focuses on the translational research in ALS, including the most recent discoveries and utilization of molecular biomarkers for the disease.

Sattler, a professor in the Department of Translational Neuroscience at Barrow Neurological Institute, sat down with NeurologyLive® prior to the meeting to provide an overview of the upcoming session and what clinicians can gain from the conference. In addition, she spoke about the progress made in both serum and fluid-based ALS biomarker research, some of the emerging drug development pathways, and the need to identify a combination of biomarkers that impacts patient outcomes. Furthermore, she touched upon the advances in imaging techniques for the disease and upcoming neuropsychological measures to track progression.

Registration for the 2024 MDA Conference is now open! The meeting is set to be held at the Hilton Orlando, in Orlando, Florida, from March 3 to 6, 2024. To register and for more information, head to www.mdaconference.org