Targeting mitophagy in neurodegenerative diseases

Major neurodegenerative diseases show mitochondrial dysfunction. In addition, mutations in genes associated with mitophagy deficits is observed in familial forms of Parkinson disease and amyotrophic lateral sclerosis.
Targeting the mitophagy pathway therefore might protect against neurodegenerative diseases and is supported by genetic, molecular and preclinical models.
Selective small molecule mitophagy enhancement — namely, USP30 inhibitors and PINK1 activators — are entering phase I clinical trials for the first time.