The annual meeting of the American Academy of Neurology was held from May 4 to 10 in Philadelphia and attracted approximately 12,000 participants from around the world, including clinicians, academicians, allied health professionals, and others interested in neurology. The conference highlighted recent advances in neurological disorders, with presentations focusing on the diagnosis, management, and treatment of disorders impacting the brain and nervous system.
In a dose-finding study, Timothy M. Miller, M.D., Ph.D., of the Washington University School of Medicine in St. Louis, and colleagues found that for patients with amyotrophic lateral sclerosis (ALS), treatment with tofersen 100 mg during approximately a three-month period resulted in a statistically significant lowering of superoxide dismutase 1 (SOD1) protein levels in the cerebrospinal fluid compared with placebo. The researchers also noted a numerical trend toward slowing of clinical decline as measured by the ALS Functional Rating Scale-Revised (ALS-FRS-R), as well as better scores on tests measuring breathing capacity and muscle strength.
On a 48-point scale, the investigators found that the mean change in ALS-FRS-R score from baseline to day 85 was −1.1 in the tofersen 100 mg group and −5.3 in the placebo group. The observed differences between tofersen and placebo were more apparent in patients with rapidly progressing disease.
"The interim analysis gives us new understanding for ALS, which is a historically difficult-to-treat disease. We're encouraged by the positive results and are excited to further investigate the therapeutic potential of this treatment in the phase 3 study," Miller said. "In March 2019, the first patient was dosed in the phase 3 VALOR study of tofersen in adults with ALS with a confirmed SOD1 mutation; the study aims to assess the efficacy and safety of tofersen versus placebo."
The study was supported by Biogen. Biogen licensed the global rights to develop, manufacture, and commercialize tofersen (BIIB067) from Ionis Pharmaceuticals.
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In another study, Carmela Tartaglia, M.D., of the University of Toronto and Toronto Western Hospital, and colleagues found that multiple concussions put some people at risk for neurodegeneration as measured with cerebrospinal fluid total tau.
"About half the group of former professional athletes who had suffered multiple concussions showed elevated total tau in their cerebrospinal fluid, while the other half of the players had levels similar to controls," Tartaglia said.
Specifically, the group of former players with elevated total tau had lower white matter integrity in their brain and performed worse on an executive task compared with the group of former players with normal tau. The researchers noted no difference in the number of concussions between the two groups.
"We need to find a marker of who has evidence of neurodegeneration, possibly chronic traumatic encephalopathy (CTE), in the brain so we can accurately diagnose this disease while people are alive. Not all people who have had multiple concussions will get CTE, but we need to identify who has it. That's the first step toward treating this disease," Tartaglia said. "We need more education so as to prevent concussions, including changing some sports, and more research to find vulnerability factors -- why do some people get neurodegeneration and others don't?"
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Tanya Simuni, M.D., of the Northwestern University Feinberg School of Medicine in Chicago, and colleagues compared the effect of isradipine versus placebo on the progression of Parkinson disease (PD).
