NRF2 activators for the prevention and/or treatment of axonal degeneration
Problema a solucionar
X-linked adrenoleukodystrophy (X-ALD) is a rare and progressive pathology caused by an inborn error of β- oxidation. It is the most common rare genetic disorder of the brain white matter. Patients may be asymptomatic or present with various clinical phenotypes ranging from severe and lethal childhood cerebral adrenoleukodystrophy (ccALD) to mild progressive chronic adrenomyeloneuropathy (AMN). Current therapeutic options are unsatisfactory, restricted to bone narrow transplant and gene therapy for cerebral inflammatory patients; no treatment is available for AMN, which affects 60% of the patients.
Tecnologia
Oral administration of an FDA-approved NRF2 activator, in the mouse models of X-ALD indicate that therapies based on NRF2 activation will be a valuable strategy to treat X-ALD/AMN and other axonopathies in which the AKT/GSK-3β /NRF2 axis is impaired. This therapeutic solution for axonal degeneration including NRF2 activators will be useful for prevention and/or treatment of axonal degeneration. Preclinical tests with an NRF2 activator and current treatment for multiple sclerosis, dimethyl fumarate (DMF), were encouraging as all main molecular and cellular disease pathogenetic mechanisms were restored.
Autors
Aurora Pujol
Technology Readiness Level
TRL6
Què busquem?
Licensing opportunities to invest in the expansion to phase II/III internationally of the clinical trials.