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CRÊME PHILADELPHIA, May 2, 2019 / PRNewswire / – An early clinical trial of an experimental treatment for amyotrophic lateral sclerosis (ALS) suggests that people could tolerate the experimental drug and that in the exploratory results the experimental drug was badociated with a possible slower progression of people presenting a genetic form of the disease caused by mutations of a gene called superoxide dismutase 1 (SOD1). Preliminary study released today will be presented at the 71st Annual Meeting of the American Academy of Neurology at Philadelphia cream, May 4 to 10, 2019.
ALS is a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord. People with ALS lose the ability to initiate and control muscle movements, which often leads to complete paralysis and death. The average life after diagnosis is two to five years. About 10% of all cases of ALS are genetic and about one fifth of them are caused by mutations in the SOD1 gene.
"The treatment we studied in this study, an antisense oligonucleotide called tofersen (BIIB067), works by targeting and reducing the protein created by the mutated gene," said the study's author. Timothy M. Miller, MD, PhD, of University of Washington School of Medicine at St. Louis, Mo.and member of the American Academy of Neurology. "This mutated protein is toxic and leads to ALS by damaging the nerve cells that control movement, and our research was aimed at reducing the production of this protein."
This component of the study involved 50 people with ALS who had a SOD1 genetic mutation. Participants received five doses of 20, 40, 60 or 100 milligrams (mg) of the investigational drug, or placebo, by lumbar puncture or lumbar puncture over a period of about three months. The researchers examined the safety, dosage, and exploratory efficacy of the experimental drug.
The researchers found that the 10 individuals who received 100 mg of the experimental drug had a 37% reduction in SOD1 protein in the cerebrospinal fluid compared to 12 people who received the placebo.
Miller, who received the Sheila Essey 2018 Award for ALS Research from the American Academy of Neurology, the ALS Association and the American Brain Foundation "Lower concentrations of the protein in the cerebrospinal fluid suggest that there were also lower concentrations in the brain and spinal cord.These reductions could lead to the preservation of motor neurons and the slow progression of the disease , but further studies are needed to examine this issue further. "
The researchers also found that subjects treated with the 100 mg dose scored better on tests for measuring respiratory capacity, muscle strength and activity activity compared to those receiving placebo. On the scale that measures the correct functioning of people during activities (48 points being the highest possible score), the patients who received the 100 mg dose experienced an average decrease of 1.1 points compared to people receiving a placebo who experienced an average decrease of 5.3 points. Given the short duration of treatment, the difference observed between patients treated at 100 mg and those receiving placebo was more apparent in patients with rapidly progressive ALS SOD1.
The most common adverse events were mild to moderate and included headache, procedural pain and post-lumbar puncture syndrome.
The limitations of the study include its small number of participants and its short duration. Further studies are needed to determine if the experimental drug is effective in larger groups of people and over longer periods.
The study was supported by Biogen. Biogen has licensed the worldwide rights to develop, manufacture and market tofersen (BIIB067) from Ionis Pharmaceuticals.
To learn more about ALS, visit BrainandLife.org, site of the free magazine for patients and caregivers of the American Academy of Neurology, devoted to the intersection of neurological diseases and brain health. To follow Brain and life® on Facebook, Twitter and Instagram.
The American Academy of Neurology is the world's largest badociation of neuroscientists and neuroscience professionals, with more than 36,000 members. AAN is dedicated to promoting patient-centered neurological care of the highest quality. A neurologist is a physician with specialized training in the diagnosis, treatment, and management of brain and nervous system disorders such as Alzheimer's disease, stroke, migraine, multiple sclerosis, osteoarthritis. concussion, Parkinson's disease and epilepsy.
For more information on the American Academy of Neurology, visit AAN.com or find us on Facebook, Twitter, Instagram, LinkedIn and YouTube.
SOURCE American Academy of Neurology
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