LINKÖPING, Sweden; March 10, 2021—AMRA and Fulcrum’s recent work on developing an optimized whole-body magnetic resonance imaging (MRI) protocol and analysis that correlates with clinical outcome assessments in Facioscapulohumeral Muscular Dystrophy (FSHD) will be presented at the 2021 MDA Virtual Clinical & Scientific Conference. The conference will highlight preclinical research, clinical trials, and clinical management in Neuromuscular Diseases (NMD) as well as topics on COVID-19 and the impact on the NMD community.
The use of MRI and AMRA’s automated measurements can detect pathological changes in muscle — changes explicitly in lean muscle volume, muscle fat fraction, and muscle fat infiltration — potentially before changes in FSHD-relevant clinical endpoints, providing critical information about disease severity and progression. This tool is particularly relevant for companies like Fulcrum, which is developing a small molecule, losmapimod, for the possible treatment of FSHD via reduction of DUX-4 driven gene expression, the root cause of the disease.
To optimize an MRI-based muscle assessment protocol and analysis, AMRA and Fulcrum conducted a prospective, longitudinal, observational, multicenter study including 17 adults with FSHD1. The patients were imaged at baseline and 4 to 12 weeks later. The researchers volumetrically analyzed 36 muscles per patient within the upper extremities, torso, and lower extremities using AMRA’s whole-body MRI protocol and identified each muscle using AMRA’s automated atlas-based segmentation and manual verification.
Following imaging, the researchers generated composite scores—total muscle fat fraction or total muscle fat infiltration—for muscles associated with clinical outcome assessments: Lower extremities for timed up and go (TUG); upper and lower extremities and torso for FSHD-TUG; and upper extremities and trunk muscles for reachable workspace (RWS).
Total, muscle fat infiltration, and muscle fat fraction correlated between imaging timepoints and whole-body MRI-based composite scores were highly associated with clinical outcome assessments—reflecting the clinical potential of MRI-based muscle assessment using AMRA’s technology.This research supports that MRI muscle assessment measurements correlate to clinical endpoints in NMD, helping Fulcrum to address the limited sensitivity associated with functional assessments.
Fulcrum’s Senior Medical Director, Dr. Michelle Mellion, will present this research in more detail as a poster and oral presentation at the conference scheduled for March 15-18, 2021.
Poster Presentation Details
Posters will be viewable throughout the entire conference and showcased during the following poster session times:
- Monday, March 15, 6 PM – 9 PM EDT
- Tuesday, March 16, 6 PM – 9 PM EDT
- Wednesday, March 17, 6:30 AM – 9 AM EDT
- Thursday, March 18, 12 PM – 1:30 PM EDT
15-Minute Oral Presentation and Q&A Details
Session Name: Quantitative Muscle Analysis in FSHD using Whole-Body MRI: Composite Muscle Measurements for Cross-Sectional Analysis
Session Date: Thursday, March 18, 2021 at 11:30 – 11:45 AM EDT
Connect with our team by visiting AMRA’s virtual booth and participate in the virtual presentations via the 2021 MDA Virtual Conference Platform.