UCL Queen Square Institute of Neurology


AIM-PD: new clinical trial testing a potential disease-modifying agent in Parkinson’s disease

17 February 2017


A new trial that investigates whether a drug called ABX can influence the levels of a crucial enzyme in patients with Parkinson’s disease (PD) has recently started at the Leonard Wolfson Experimental Neurology Centre (LWENC) Clinical Research Facility (CRF), based at the UCL Institute of Neurology, the Royal Free London hospital and the National Hospital for Neurology and Neurosurgery (UCLH).

The most statistically important risk factor for the development of PD is the presence of the GBA mutation, which is associated with low levels of glucocerebrosidase (GCase) enzyme activity.

The rationale behind the AIM-PD trial is that increasing GCase activity may have positive knock-on effects on the function of Parkinson’s patients carrying the GBA mutation. Furthermore, it has been shown that there is a two-way relationship between GCase activity and levels of the main constituent of Lewy bodies, the pathological hallmark of PD.

This provides further rationale for the hypothesis that modulation of GCase activity may represent a neuroprotective mechanism in PD. There is currently no therapy to slow down the progression of PD.

The AIM-PD trial, led by Professor Anthony Schapira (Head of Department of Clinical Neurosciences) will investigate whether ABX is able to penetrate the central nervous system (CNS), increase GCase activity, and reduce levels of the Lewy body protein, alpha-synuclein.

Researchers from the LWENC CRF, will look at blood and cerebrospinal fluid samples to determine whether ABX is able to influence GCase activity and will use a range of cognitive and motor assessments to examine whether CNS penetration influences these parameters.

AIM-PD is an open-label, intra-participant, dose escalation study meaning each participant will start on a low dose of ABX that increases over the first 4 weeks of their participation depending on how well it is tolerated. If the AIM-PD trial has encouraging results, then ABX may have positive implications for a significant proportion of PD patients.

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