Treatment of amyotrophic lateral sclerosis: the first stage
Researchers at the University of Washington Medical School and the Massachusetts General Hospital have announced the successful completion of a phase I clinical trial of an experimental method for the treatment of one of the hereditary forms of amyotrophic lateral sclerosis, or Charcot's disease.
Amyotrophic lateral sclerosis is a genetically determined neurodegenerative disease in which there is a gradual death of motor neurons. Ultimately, this leads to muscle paralysis and death of patients. The only drug approved by the US Food and Drug Administration for the treatment of this disease, riluzole, has only a mild effect.
Most cases of amyotrophic lateral sclerosis are sporadic and only about 10% are caused by inherited mutations. Experts have already identified 10 genes whose mutations can lead to the development of this disease, but the search continues.
The study involved patients with a form of the disease caused by mutations of the SOD1 gene, underlying approximately 2% of all cases of amyotrophic lateral sclerosis. To date, more than 100 mutations of this gene are known to cause the disease. At the molecular level, these mutations affect the properties of the SOD1 protein in different ways, but they all invariably lead to the development of amyotrophic lateral sclerosis.
Instead of understanding the mechanisms by which each of the mutations disrupts the work of the SOD1 gene, the authors, working under the guidance of Dr. Timothy Miller, developed a technique for blocking the production of its protein product using the antisense mRNA method.
After successfully testing this method on a mouse model of amyotrophic lateral sclerosis, the researchers conducted a clinical study involving 21 patients who were injected with an experimental drug or placebo in the form of 11-hour spinal injections.
Examination of patients revealed no serious side effects. The most common complaints of patients were headache and back pain, often accompanying spinal injections. The results of the analysis of the cerebrospinal fluid, carried out immediately after the procedure, confirmed the presence of the drug in it.
Currently, scientists are planning to conduct phase II clinical trials, in which increased doses of the experimental drug will be administered and the effectiveness of treatment will be evaluated.
Article by Timothy M Miller et al. An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomized, first-in-man study published in The Lancet Neurology.
Evgeniya Ryabtseva
Portal "Eternal youth" http://vechnayamolodost.ru based on the materials of Washington University School of Medicine:
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