Pimozide (Orap)

Drug Name: Pimozide (Orap)
Company: N/A
Location: Canada
Drug Type: Small Molecule
Conditions: ALS, Neuropsychiatric Disorders
Mechanism Type: NMJ stabilization
Mechanism: Pimozide may help stabilize NMJs by blocking calcium channels, which may stimulate neurotransmitter release (Patten et al., 2017). Researchers at the University of Montreal in Canada discovered that drug in a high-throughput screen that aimed to identify potential therapies for ALS by looking for drugs that improved mobility of a zebrafish model of the disease.
U.S. Status for ALS: Phase II

[1] Getting to Know Pimozide in ALS. ALS Canada Virtual Research Forum. 9 Aug 2017.
[2] A Clinical Trial of Pimozide in Patients With Amyotrophic Lateral Sclerosis (ALS) (Pimozide2) Clinicaltrials.gov, 27 Nov 2017. Accessed 15 Feb 2017 from https://www.clinicaltrials.gov/ct2/show/NCT03272503.
[3] Neuroleptics as therapeutic compounds stabilizing neuromuscular transmission in amyotrophic lateral sclerosis. Patten, SA et al. JCI Insight. 2017 Nov 16;2(22). pii: 97152.

Last updated February 15th, 2018

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