The annual meeting of the American Academy of Neurology was held from April 2 to 7 in Seattle and attracted more than 38,000 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 randomized, placebo-controlled study, Donald L. Gilbert, M.D., of the Cincinnati Children’s Hospital Medical Center, and colleagues found that a novel, once-daily oral medication in development, referred to as ecopipam, reduces tics in children and adolescents with Tourette syndrome.

The authors sought to determine if ecopipam is safe and effective for treating Tourette syndrome in children and adolescents. One hundred, forty-nine children and adolescents were randomly assigned to receive either ecopipam (74 participants) or placebo (75 participants) for 12 weeks. The researchers found that ecopipam reduced tics, and it did not cause weight gain or marked neurological adverse effects.

“Preparations are underway to perform an additional placebo-controlled trial which, if also positive, could lead to approval by the U.S. Food and Drug Administration,” Gilbert said. “Clinicians caring for patients with Tourette syndrome may consider referring their patients to participate in this study, particularly if they are concerned about side effects or have had poor responses to currently available treatments.”

The study was supported by Emalex Biosciences, the developer of ecopipam.

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As part of the open-label, phase 2 LUMINARY study, Aaron Koenig, M.D., of Sage Therapeutics in Cambridge, Massachusetts, and colleagues found that SAGE-718, a first-in-class, oral, positive allosteric modulator of N-methyl-D-aspartate receptors, is generally well tolerated and is associated with improvement on multiple tests of executive performance and learning and memory in patients with mild cognitive impairment and mild dementia due to Alzheimer disease.

The authors evaluated SAGE-718 in patients aged 50 to 80 years with mild cognitive impairment and mild dementia (Montreal Cognitive Assessment [MoCA] scores of 15 to 24) due to Alzheimer disease. The patients underwent a comprehensive battery of tests to assess multiple domains of cognitive performance.

The researchers observed improvements from baseline on multiple tests of executive functioning (Digit Symbol Substitution, Multitasking, One Touch Stockings, Spatial Working Memory, and 2-Back tests) and learning and memory (Pattern Recognition Memory and Verbal Recognition Memory tests). Statistically significant improvement in the MoCA (+2.3 points versus baseline) was observed at day 28. The investigators did not observe any appreciable effect on measures of simple attention/psychomotor speed, in keeping with the profile of SAGE-718 based on data collected to date. SAGE-718 was generally well tolerated.

“We are encouraged by the positive results shared from the phase 2 LUMINARY study, which are consistent with signals suggesting improvement in cognitive performance seen across the SAGE-718 program, including in people with Parkinson and Huntington disease,” Koenig said. “We look forward to learning more about the potential of SAGE-718 as we continue to advance our program with multiple ongoing or planned phase 2 studies.”

The study was supported by Sage Therapeutics, the manufacturer of SAGE-718.

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Bruce Campbell, M.D., of the University of Melbourne in Australia, and colleagues found that a fully implanted endovascular brain-computer interface (BCI), referred to as the stentrode device, appears to be safe when implanted in the brains of patients with paralysis due to amyotrophic lateral sclerosis (ALS).

This was the first study evaluating the stentrode device in patients with paralysis as a result of ALS. A field clinical engineer conducted weekly sessions with each of the four study participants. The patients learned to use the device independently at home with setup by a caregiver. The researchers monitored the stentrode device for one year in all four patients, with no serious adverse events reported. The stentrode device also stayed in place for each patient, and the blood vessel into which the device was implanted remained open. This is particularly important given one of the key advantages of the stentrode is its minimally invasive endovascular delivery into the sagittal sinus compared to the craniotomy required for other BCI devices.

“The stentrode has demonstrated safety in humans, and was effective in enabling control of digital devices just by thinking in patients with advanced stages of ALS,” Campbell said. “The stentrode BCI is currently being evaluated in two additional clinical trials in the United States and Australia, and we are opening the study to a larger population of patients on both continents.”

The study was supported by Synchron, the manufacturer of the stentrode device.

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