New and Noteworthy Information—August 2016
The FDA has accepted the Biologics License Application for Ocrevus (ocrelizumab) for the treatment of relapsing multiple sclerosis and primary progressive multiple sclerosis. The agency granted the application Priority Review Designation with a targeted action date of December 28, 2016. The Ocrevus Marketing Authorization Application has also been validated by the European Medicines Agency. The Ocrevus marketing applications are based on results from three phase III studies that met primary and key secondary end points. Data from OPERA I and OPERA II in people with relapsing multiple sclerosis showed superior efficacy of Ocrevus in reducing annualized relapse rates and disability progression sustained for at least three and for at least six months, respectively, compared with interferon beta-1a. Genentech, which will manufacture the drug, is headquartered in South San Francisco, California.
Thirty-eight independent genomic regions are associated with migraine, according to a study published online ahead of print June 20 in Nature Genetics. The identified loci showed enrichment for genes expressed in vascular and smooth muscle tissues, which is consistent with a predominant theory of migraine that highlights vascular etiologies. To identify new genomic loci associated with susceptibility to migraine, researchers carried out a genetic study of migraine on 59,674 subjects with migraine and 316,078 controls who participated in 22 genome-wide association studies. Investigators identified 44 independent single-nucleotide polymorphisms significantly associated with migraine risk that mapped to 38 distinct genomic loci, including 28 loci not previously reported and a locus that is the first to be identified on chromosome X. The findings may promote the development of personalized treatments for migraine.
Long-term risks of recurrent stroke and poststroke dementia remain high after stroke and are substantially influenced by prestroke risk factors, emphasizing the need for optimizing primary prevention, according to a study published online ahead of print July 14 in Stroke. Researchers monitored 1,237 patients with first-ever stroke and 4,928 stroke-free participants, matched by age, sex, examination round, and stroke date, for the occurrence of stroke or dementia. Beyond one year after stroke, patients retained a threefold increased risk of recurrent stroke and an almost twofold increased risk of dementia, compared with people without stroke. In all, 39% of recurrent strokes and 10% of poststroke dementia cases were attributable to prestroke cardiovascular risk factors. These percentages were similar for first-ever stroke and dementia in the matched stroke-free population.
Molecular evidence indicates that nilotinib significantly increases brain dopamine and reduces toxic proteins linked to disease progression in patients with Parkinson's disease or dementia with Lewy bodies, according to a phase I study published July 11 in the Journal of Parkinson's Disease. Twelve participants were randomized to 150 mg/day or 300 mg/day of oral nilotinib for 24 weeks. The CSF levels of homovanillic acid were significantly increased between baseline and 24 weeks of treatment. The researchers found that nilotinib is safe and well tolerated for people with advanced Parkinson's disease. In addition, nilotinib is detectable in the CSF and engages the target ABL1. Improvements in motor and cognitive function suggest that nilotinib may have clinical benefits. Nilotinib should be evaluated in larger randomized trials, said the researchers.
The FDA has approved ExAblate Neuro, the first focused ultrasound device to treat essential tremor in patients who have not responded to medication. ExAblate Neuro uses MRI to deliver focused ultrasound to destroy brain tissue in a small area thought to be responsible for causing tremors. Of 76 patients with essential tremor, 56 randomly received the ExAblate Neuro treatment in one study. Patients in the control group were able to cross over into the treatment group three months later. Patients treated with ExAblate Neuro showed a nearly 50% improvement in tremor and motor function three months after treatment, compared with their baseline scores. To determine whether ExAblate Neuro treatment is appropriate, patients should first have MRI and CT scans. InSightec, the device's manufacturer, is headquartered in Tirat Carmel, Israel.
The driving ability of patients with mild cognitive impairment (MCI) and Alzheimer's disease is related to their degree of cognitive impairment, according to a systematic review published May 11 in the Journal of Alzheimer's Disease. Researchers investigated the predictive utility of cognitive tests and domains, and the areas and degree of driving impairment in patients with MCI and Alzheimer's disease. Effect sizes were derived and analyzed in a random effects model. Executive function, attention, visuospatial function, and global cognition significantly predicted driving performance. Trail Making Test Part B and Maze test were the best single predictors of driving performance. Patients with very mild Alzheimer's disease and mild Alzheimer's disease were more likely to fail an on-road test than healthy control drivers, with failure rates of 13.6%, 33.3%, and 1.6%, respectively.
