Effect of Riluzole as a Symptomatic Approach in Patients With Chronic Cerebellar Ataxia
Status:
Completed
Trial end date:
2008-08-01
Target enrollment:
Participant gender:
Summary
Cerebellar disorders are often disabling and symptomatic therapies are limited to few options
that are partially effective. It seems therefore appropriate to search for additional
approaches.
Purkinje cells are the sole output of the cerebellar cortex: they project inhibitory signals
to the deep cerebellar nuclei (DCN), which have a critical role in cerebellar function and
motor performance. DCN neurons fire spontaneously in the absence of synaptic input from
Purkinje neurons and modulation of the DCN response by Purkinje input is believed to be
responsible for coordination of movement. Recent evidences support the notion that an
increase in DCN excitability may be an important step in the development of cerebellar ataxia
and point to the underlying molecular mechanisms: the inhibition of small-conductance
calcium-activated potassium (SK) channels, that causes an increase of the firing frequency in
DCN, correlates with cerebellar ataxia.
The rationale of the present project is that SK channel openers, such as riluzole, may have a
beneficial effect on cerebellar ataxia.
The researchers propose to perform a pilot study investigating safety and efficacy of
riluzole, an approved treatment for amyotrophic lateral sclerosis, as a symptomatic approach
in patients with chronic cerebellar ataxia.