Abstract #2521

Monitoring of therapeutic response using lesion segmentation and DTI in adult cerebral adrenoleukodystrophy treated with Leriglitazone

Marianne Golse¹, Isabelle Weinhofer², Bernardo Blanco ³, Magali Barbier³, Camille Huiban³, Silvia Pascual⁴, Marc Martinell⁴, Johannes Berger², Vincent Perlbarg⁵, Damien Galanaud⁶, and Fanny Mochel⁷

¹Sorbonne Université, INSERM, CNRS, Laboratoire d'Imagerie Biomédicale, Paris, France, ²3Department of Pathobiology of the Nervous System, Center for Brain Research, Medical University of Vienna, Vienna, Austria, ³Department of Medical Genetics, Reference Centers for Adult Neurometabolic diseases and Adult Leukodystrophies, AP-HP, Pitié-Salpêtrière University Hospita, Paris, France, ⁴Minoryx Therapeutics, Barcelona, Spain, ⁵Braintale (Software As a Service), Paris, France, ⁶Department of Neuroradiology, AP-HP, Pitié-Salpêtrière University Hospital, Paris, France, ⁷Department of Medical Genetics, Reference Centers for Adult Neurometabolic diseases and Adult Leukodystrophies, AP-HP, Pitié-Salpêtrière University Hospital, Paris, France

Synopsis

Keywords: White Matter, Brain, Adrenoleukodystrophy, Leriglitazone, Volumetric analysis, Diffusion Tensor Imaging

Motivation: Cerebral adrenoleukodystrophy (CALD) is a rare and devastating demyelinating disease. The standard of care is hematopoeitic stem cell transplantation (HCST), which efficacy is limited to early-stage patients.

Goal(s): We studied the efficacy of Leriglitazone in CALD patients, either not eligible or awaiting HSCT.

Approach: Leriglitazone was administered to thirteen adult male patients. They underwent brain MRI every 3 months, with a quantitative analysis of lesion volumes on FLAIR, diffusion tensor parameters and gadolinium enhancements.

Results: The disease stabilized in all patients awaiting HSCT. Corticospinal tract lesions downsized in most patients and turned gadolinium-negative, showing a selective effect of this molecule on motor pathways.

Impact: Cerebral adrenoleukodystrophy is a devastating demyelinating disease, fatal for all patients without early stem cell transplantation. We showed that Leriglitazone represents a therapeutic alternative, allowing clinical and radiological stabilization in ten men. Remarkably, motor pathways lesions downsized and turned gadolinium-negative.

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