An initial French study, supported by the AFM and using zebrafish models of giant axon neuropathy (GAN), has identified new drug candidates:
- Using pharmacological screening to identify small molecules that can restore both physiological and cellular deficits in the animal model.
- High-resolution behavioural, computational and imaging analyses enabled the researchers to select five drug candidates.
- These molecules restore locomotion and axonal growth and stabilise neuromuscular junctions in the zebrafish model.
- They could be integrated into a repositioning approach to accelerate the development of a treatment for this neuropathy.
A Canadian team has developed a new mouse model of NAG :
- These mouse models were developed by inactivating gigaxonin and overexpressing peripherin, two cytoskeletal proteins.
- At 12 months, they develop cognitive disorders as well as major sensory and motor deficits.
- In vitro administration of Tubastatin A (an HDAC6 inhibitor) to the neurons of these mice restored the level of axonal transport of microtubules to its normal level.
- Treatment of the mice with this molecule slightly improved motor function.
