Muscular Atrophy, Spinal Clinical Trial
Official title:
A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients
The objectives of this trial are: to establish a safety profile for use of Hydroxyurea in children with Type I Spinal Muscular Atrophy; to identify reliable outcome measures for HU treatment in Type I SMA; and to detect the clinical efficacy of HU treatment in children with Type I SMA.
SMA is a neuromuscular disorder characterized by degeneration of spinal cord motor neurons
and muscular atrophy. SMA is classified into three clinical subtypes according to the
severity and age of onset (Types I, II and III). Type I SMA (also called severe, infantile or
acute SMA, or Werdnig-Hoffman disease) is the most severe phenotype. The onset of symptoms is
within the first 6 months of life, and weakness of intercostal muscles and lack of airway
protection lead to respiratory insufficiency and aspiration pneumonia, often resulting in
early infant death.
In our laboratory, our preliminary results indicate that HU treatment significantly increases
both SMN mRNA expression and intact SMN protein levels in vitro. These data confirm previous
observations that in vitro treatments of SMA lymphocytes with hydroxyurea resulted in
augmentation of the SMN2 gene expression in a dose and time related manner. Based on these
exciting pre-clinical data, coupled with the well-documented side-effect profile of HU in
children, we are conducting a pilot clinical trial using HU in children with Type I SMA. This
clinical trial study is intended to establish the safety profile in children with Type I SMA;
to identify reliable outcome measures; and to detect the possible clinical efficacy of HU
treatment in children with Type I SMA.
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