Expert review of neurotherapeutics
-
Spinal muscular atrophy (SMA) is an autosomal recessive degenerative neuromuscular disorder characterized by loss of spinal motor neurons leading to muscle weakness. This review article focuses on a novel antisense oligonucleotide treatment, first ever approved for SMA (nusinersen, SpinrazaTM) and describes the exciting journey from early ASO clinical trials to regulatory approval of the first ever known effective treatment for SMA. Areas covered: This article reviews the results of the published open label nusinersen studies in infants and children, and briefly covers the preliminary findings of the recently completed but as yet unpublished nusinersen-sham controlled trials, as well as the presymptomatic nusinersen trial known as Nurture. ⋯ Nusinersen was found to be safe and well tolerated across all age groups studied. Nusinersen has irrevocably altered the natural history of SMA and allowed for the first time children to transition between SMA types. Nusinersen should be considered as standard of care for the treatment of SMA of all types.
-
Expert Rev Neurother · Sep 2017
ReviewImproving understanding of trigger points and widespread pressure pain sensitivity in tension-type headache patients: clinical implications.
The underlying etiology of tension type headache (TTH) is not understood. The current paper highlights the etiologic role of muscle trigger points (TrPs) to the development and maintenance of central sensitization in TTH and its clinical repercussion for proper management of these patients. Areas covered: A literature search on Pub Med for English-language published papers between 1990 and May 2017 to provide the most updated data on the topic was conducted. ⋯ In fact, TrPs have been found to be associated with widespread pressure pain sensitivity in TTH. Temporal and spatial summation of TrP nociception suggests that inactivating TrP in the neck, head and shoulder muscles could help these patients; however, current evidence supporting the therapeutic role of TrPs in TTH is conflicting. Expert commentary: Understanding the role of TrPs in TTH in widespread pain sensitization may help to develop better management regimes and possibly prevent TTH from developing into more chronic conditions.
-
Expert Rev Neurother · Aug 2017
Intravenous thrombolysis for acute ischemic stroke: a bridge between two centuries.
Intravenous tissue-plasminogen activator (tPA) remains the only approved systemic reperfusion therapy suitable for most patients presenting timely with acute ischemic stroke. Accumulating real-word experience for over 20 years regarding tPA safety and effectiveness led to re-appraisal of original contraindications for intravenous thrombolysis (IVT). Areas covered: This narrative review focuses on fast yet appropriate selection of patients for safe administration of tPA per recently expanded indications. ⋯ Promising studies of penumbral imaging to extend IVT beyond the 4.5-hour window and in wake-up strokes are summarized. Expert commentary: This review provides an update on the role of IVT in specific conditions originally considered tPA contraindications. Novel practice challenges including NOAC's, MSU proliferation and bridging therapy (IVT&MT) for LVO patients, and the potential extension of IVT time-window using penumbral imaging are emerging as safe and potentially effective IVT applications.
-
Expert Rev Neurother · Jun 2017
ReviewInvestigational drugs in recent clinical trials for treatment-resistant depression.
The authors describe the medications for treatment-resistant depression (TRD) in phase II/III of clinical development in the EU and USA and provide an opinion on how current treatment can be improved in the near future. Areas covered: Sixty-two trials were identified in US and EU clinical trial registries that included six investigational compounds in recent phase III development and 12 others in recent phase II clinical trials. Glutamatergic agents have been the focus of many studies. ⋯ Expert commentary: Promising new agents are emerging for TRD treatment. Glutamatergic modulators likely represent a very promising alternative to monoaminergic antidepressant monotherapy. We could see the arrival of the first robust and rapid acting antidepressant drug in the near future, which would strongly facilitate the ultimate goal of recovery in persons with TRD.