New Therapy for Deadly Childhood Disease
(Ivanhoe Newswire) -- A new study shows the drug riluzole could be a treatment option for children with a deadly disease.
Severe spinal muscular atrophy (SMA) is a disease of the motor neurons that is generally diagnosed before a child is 6 months old. Often a child is unable to sit without support and has a life expectancy of two years or less. There is no known treatment for SMA and until recently, no therapeutic trials have been attempted.
Riluzole is a drug that has been shown to slow the rate of decline in patients with amyotrophic lateral sclerosis, another form of motor neuron disease. Researchers from Oregon Health Sciences University and Shriners Hospital for Children in Portland conducted a study to determine if riluzole is tolerated and could have a positive effect on life expectancy.
For the study, babies with SMA were randomly selected to receive the drug or placebo in a two to one ratio. This meant, for every two patients on the drug, one patient was on a placebo. Complete blood workups were done at the beginning of the study, two weeks, one month, two months, three months, six months and nine months after the drug or placebo was started. Treatment was stopped after nine months and blood work was repeated at 12 months. Treatment was continued at that point if requested by the parent. Originally, researchers hoped to enroll 30 patients but only 10 patients were enrolled in the study due to loss of funding.
Seven patients received riluzole and three patients received placebo. All three of the patients in the placebo group died in the first year. Three of the seven who received the drug are still living at ages 5 1/3 years, 4 years and 30 months. None of the children experienced any adverse side effects from the medication and none showed any change in motor abilities. The surviving children are receiving an increase in dosage of the drug as they grow, but none of them have achieved the ability to sit by themselves.
Study authors write, "The present study suggests that riluzole is safe in infants and toddlers with SMA and may have a mitigating effect on the natural course of the disease." While this study was limited, they suggest further research on this drug for pediatric patients with SMA.
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SOURCE: Archives of Neurology, 2003;60:1601-1603
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