Eric J. Mallack, M.D., Director of the Leukodystrophy Center and Assistant Professor of Pediatrics and Neurology at Weill Cornell Medicine is the Co-Principal Investigator of a new phase 2/3 clinical trial, sponsored by Minoryx Therapeutics. The “NEXUS” study will assess the efficacy and safety of leriglitazone in pediatric patients with early-stage cerebral adrenoleukodystrophy (cALD).
Alongside Co-PI, Patricia Musolino, MD, PhD from Massachusetts General Hospital, the team aims to confirm whether researchers can halt or stabilize early cerebral ALD, a progressive inflammatory neurodegenerative disease of childhood, with leriglitazone - a novel, orally bioavailable and selective PPAR gamma agonist. If proven effective, this will be the first non-transplant-related therapy (e.g. stem cell transplant and gene therapy) that can halt this childhood neurodegenerative disorder. And if delivered early enough, it may stop the disease in the pre-symptomatic period of disease, thus preserving full neurological function in these patients.
The imaging outcomes in the study were developed from Dr. Mallack’s quantitative MRI research in lesion volumetry (PMID: 34503945), and Dr. Musolino’s work in MR Perfusion (PMID: 22961546).