WoS İndeksli Yayınlar Koleksiyonu

Permanent URI for this collectionhttps://hdl.handle.net/20.500.14627/6

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  • Article
    Efficacy and Safety of Deferiprone for Parkinson’s Disease: A Systematic Review and Meta-Analysis of Motor Function and Overall Disease Severity
    (Springer-Verlag Italia SRL, 2026) Ehab, Menna; Ibrahim, Ismail A.; Hekal, Rawan Gameel; Solah, Israa A.; Ezz-Alarab, Moaz; Elewa, Mandy; Zidat, Ayham R. A.
    Background The accumulation of iron in brain regions is one of the characteristics of Parkinson's disease (PD). Deferiprone (DFP) is an iron chelator that reduces iron overaccumulation in certain diseases. The efficacy and safety of DFP in the management of PD have been assessed; however, the results remain controversial. Method A systematic review and meta-analysis were conducted in accordance with PRISMA guidelines. A comprehensive search was conducted across five databases to identify all randomized clinical trials and observational studies. The primary outcomes included changes in motor function (MDS-UPDRS III) and overall disease severity (MDS-UPDRS total score). Additionally, the safety of DFP was assessed by analyzing adverse events. A network meta-analysis using a random-effects model was conducted. Results Four randomized clinical trials were identified. The studies included 567 patients with early-stage PD. The DFP doses across the included studies ranged from 3.75 to 15 mg/kg twice daily. None of the doses showed a significant Improvement in motor function (I2 = 0%), or in overall disease severity (I2 = 82.5%), compared with placebo in the network analysis. However, a non-significant improvement in motor function was observed at 7.5 mg/kg twice daily, whereas higher doses were associated with worsening clinical scores. Adverse events were generally mild, although there were some safety concerns at higher doses. Conclusion DFP can reduce accumulated iron across brain regions. However, current evidence does not support the use of DFP for the clinical and symptomatic management of PD within the assessed range of doses and treatment periods.
  • Article
    Unraveling the Potential of Stem Cell Therapy in Motor Neuron Disease: A Narrative Review
    (Bentham Science Publ, 2025) Essa, Syed Muhammad; Khosa, Noor Ahmed; Kakar, Amanullah; Ozturk, Basar; Ibrahim, Ismail A.; Haq, Noman
    Motor neuron disorders (MNDs), including ALS, are deadly neurodegenerative conditions that cause progressive motor neuron degeneration. With neuroprotection and the potential for neuron regeneration employing MSCs, ESCs, iPSCs, and NSCs, stem cell treatment presents a viable alternative to current medicines, which only control a limited number of symptoms. Following PRISMA criteria, this narrative review methodically screened 1248 records from the Cochrane, Web of Science, PubMed, and Scopus databases. Following a thorough screening process, 22 studies, including preclinical models and 19 clinical trials, were analysed to assess the therapeutic mechanisms, safety, and efficacy of stem cell therapies for MNDs. Mesenchymal stem cell (MSC) therapy has shown a promising safety profile and possible therapeutic efficacy in ALS, with no substantial transplant-related toxicity noted. ALS functional rating scale-revised (ALSFRS-R) scores and forced vital capacity (FVC) assessments from clinical trials, such as those evaluating autologous bone marrow-derived MSCs, demonstrated stabilisation in ALS development. Studies have also emphasised as to how immunomodulation and neurotrophic factors play a part in MSC-based therapies. Recent data indicate that repeated intrathecal MSC injection could extend the duration of therapeutic advantages. Clinical trials have shown safety and early efficacy signals for motor neurons produced from embryonic stem cells (ESCs), especially using AstroRx (R). This suggests that ESCs could be a viable option for regenerative medicine. Nonetheless, issues, like host integration and differentiation optimisation, still exist. Although clinical translation is still in its early stages, induced pluripotent stem cells (iPSCs) and their derivatives provide disease modelling and patient-specific therapeutic applications. Stem cell therapy holds promise for treating MND, with MSCs leading the way in current trials. It is necessary to enhance ESC- and iPSC-based techniques to tackle integration issues. To ensure long-term safety and efficacy, therapies must be developed using standardised protocols, patient stratification, optimised delivery, and large-scale studies.