Cure Parkinson’s is excited to announce that we are now accepting expressions of interest for our new combination therapy funding call. In August, Cure

Introduction Only symptomatic treatments are available for patients with Parkinson’s disease (PD), the second most common chronic neurodegenerative disease worldwide, and it is therefore imperative to...

A phase 2 study shows risvodetinib improves safety and symptoms in early Parkinson disease, suggesting potential as a disease-modifying therapy.

npj Parkinson's Disease - Severe GBA1 variants drive the GBA1-PD clinical phenotype: implications for counselling and clinical trials

Differential Peripheral NLRP3 Inflammasome Expression in Parkinson

The relationship between plasma or cerebrospinal fluid (CSF) amino acid levels and the pathophysiology of Parkinson’s disease (PD) remains unclear. We aimed to examine the association of plasma and CSF amino acid levels with the nigrostriatal dopaminergic neurons and the clinical symptoms of newly diagnosed drug-naïve patients with PD. This was a cross-sectional study using the Jikei University PD registry. A total of 45 amino acids were measured in both plasma and CSF, and their associations with key clinical parameters were investigated. Multiple linear regression analyses were conducted to evaluate whether amino acids served as major explanatory variables for clinical parameters in PD. Forty-five drug-naïve patients were enrolled. The mean age was 68.6 ± 9.6, and disease duration was 2.5 years (± 2.1). MDS-UPDRS Part III score averaged 28.1 (± 13.3). Among the measured plasma and CSF amino acids, only CSF taurine levels were identified as independent factors for SBR, MDS-UPDRS Part II, and Part III scores in multivariate analysis (β = 0.39 [95% CI 0.13 to 0.65, p = 0.005], –0.46 [95% CI –0.76 to –0.15, p = 0.004], –0.39 [95% CI –0.73 to –0.05, p = 0.02], respectively). Higher CSF taurine levels were associated with more preserved SBR on 123I-FP-CIT SPECT and better activities of daily living and motor symptoms, suggesting that CSF taurine may be involved in the mechanism of nigrostriatal dopaminergic degeneration and exert a neuroprotective effect on dopaminergic neurons.

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Background Multiple system atrophy (MSA) is a rare, rapidly progressive alpha-synucleinopathy characterized by autonomic dysfunction, parkinsonism and marked cerebellar ataxia, with very limited treatment options. N-acetyl-DL-leucine (ADLL) (Tanganil™), a racemic derivative of leucine is usually well tolerated and has shown promise in improving cerebellar symptoms and reducing REM sleep behavior disorder (RBD), a common non-motor dream-sleep disorder feature of alpha-synucleinopathy. Given this dual potential, we treated three patients with advanced-stage MSA under compassionate use rules. Case presentations Three patients with advanced-stage MSA and severe cerebellar symptoms—two of whom also had moderate RBD—were treated with ADLL (TanganilTM), titrated to 5g/day over 10 days. While both patients with RBD reported self-assessed decrease of RBD symptoms within 2–3 weeks on the full dosage (approximately four weeks after treatment initiation), all three patients had major worsening of gait and balance during the same period, leading to sudden falls and overall health decline. These adverse events prompted early discontinuation of therapy. Gait improved within two weeks after discontinuation of therapy in two patients. In parallel, the RBD phenotype reoccurred. In the third, who had very advanced MSA and concurrent infections, gait only slowly improved over time and it remains unclear whether his worsening of truncal ataxia was attributable to ADLL (TanganilTM), or whether it was related to his concurrent infections with slow recovery. Conclusions While ADLL improved RBD, it was poorly tolerated in these three patients with advanced-stage MSA of predominantly cerebellar type. Further studies are needed to evaluate its safety and efficacy in different, preferably early, stages of MSA.

Key Milestone in ACTIVATE Phase 2 Clinical Trial of BIA 28-6156