Cookies on this website

We use cookies to ensure that we give you the best experience on our website. If you click 'Accept all cookies' we'll assume that you are happy to receive all cookies and you won't see this message again. If you click 'Reject all non-essential cookies' only necessary cookies providing core functionality such as security, network management, and accessibility will be enabled. Click 'Find out more' for information on how to change your cookie settings.

A successful Phase 3 clinical trial of N-acetyl-leucine (NALL) in the lysosomal storage disorder Niemann-Pick disease type C has been published this month in the New England Journal of Medicine.

Following the successful trial, a new drug application (NDA) has been submitted to the Food and Drug Administration (FDA).

The study, sponsored by IntraBio, evaluated the safety and efficacy of N-acetyl-l-leucine (NALL), an agent that potentially ameliorates lysosomal and metabolic dysfunction, for the treatment of Niemann–Pick disease type C. The trial found that, among patients with Niemann–Pick disease type C, treatment with NALL for 12 weeks led to better neurologic status than placebo. A longer period is needed to determine the long-term effects of this agent in patients with Niemann–Pick disease type C.

IntraBio is a spin out from Pharmacology co-founded by Antony Galione, Grant Churchill and Fran Platt. For the full paper, see https://www.nejm.org/doi/full/10.1056/NEJMoa2310151

Similar stories

Amazing Brain Festival at Cheney Secondary School

Pharmacology staff and students contributed to a special afternoon of events last week to engage 200-300 students and families on the theme of the brain organised by their school ‘Rumble Museum’. A team of ten members of our department led by Dr Lizzie Burns developed five zones of activities to engage students with different aspects of neuroscience: memory, focus, build-a-brain, illusion and microscopy.