After years of consistently negative clinical trial results, there is hope for ALS patients. The prestigious journal ‘The New England Journal of Medicine’ published the results of a study yesterday, showing that a new therapy reduces damage to motor nerve cells and that patients show a stabilization in their disease course.
Amyotrophic Lateral Sclerosis or ALS gained popularity in 2014 thanks to the ice bucket challenge. It is a deadly nerve-muscle disease in which the motor neurons die. The disease gradually paralyzes your body from the neck down. You can no longer move, speak, swallow and eventually no longer breathe, while mental and sensory faculties remain intact. Patients die on average 33 months after diagnosis, with no hope of a cure.
But today there is a glimmer of hope. For the first time, ALS patients with an SOD1 mutation could be treated successfully, according to a new international study in which UZ Leuven participated. The drug tofersen appears to counteract the disease in these patients. For example, it has been shown that ALS is in principle a treatable disease.
Several years ago, researchers developed a new treatment based on gene therapy for the group of patients with an SOD1 mutation. The drug tofersen binds to the genetic material and thus blocks the production of the harmful SOD1 protein.
The response we saw was sometimes spectacular. In some, the disease progression stopped completely. They even regained some muscle strength
Philip Van Damme (UZ Leuven), neurologist and ALS expert
The first clinical studies in a small number of patients already showed promise, but the results of a larger phase 3 study were recently announced. More than a hundred patients worldwide participated in the study. They received either tofers treatment or a placebo as a control and were followed up for a year.
“The response we saw was sometimes spectacular, especially in patients who could start the drug early in the course of the disease,” says Professor Philip Van Damme (UZ Leuven), neurologist and ALS expert. “In some, the disease progression stopped completely. They even regained some muscle strength. Patients who started treatment later showed a delay in the further course of the disease.”
Worldwide, approximately 400,000 people suffer from the nerve-muscle disease ALS. The disease affects about 6 to 7 people in 100,000 and occurs worldwide. Most patients get ALS around age 60, but there are also young adults with ALS. Men are affected slightly more frequently than women. In Belgium, 1,000 people continuously suffer from ALS. Every year more than 200 patients die and at least as many are added.
Usually the disease is not hereditary, but in about 2 percent of patients it is caused by a mutation in the SOD1 gene. This genetic abnormality leads to a toxic build-up of SOD1 proteins, which cause the motor nerve cells to die.
More info: ALS Liga Belgium
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