Background Several pieces of evidence have shown the neurotrophic effect of erythropoietin (EPO) and its introduction in the therapeutic practice of neurological diseases. However, its usefulness in the treatment of spinocerebellar ataxia type 2 (SCA2) has not been proven despite the fact that it is endogenously reduced in these patients. Objective The study aims to investigate the safety, tolerability, and clinical effects of a nasally administered recombinant EPO in SCA2 patients. Methods Thirty‐four patients were enrolled in this double‐blind, randomized, placebo‐controlled, phase I–II clinical trial of the nasally administered human‐recombinant EPO (NeuroEPO) for 6 months. The primary outcome was the change in the spinocerebellar ataxia functional index (SCAFI), while other motor, neuropsychological, and oculomotor measures were assessed. Results The 6‐month changes in SCAFI score were slightly higher in the patients allocated to NeuroEPO treatment than placebo in spite of the important placebo effect observed for this parameter. However, saccade latency was significantly decreased in the NeuroEPO group but not in placebo. The frequency and severity of adverse events were similar between both groups, without evidences of hematopoietic activity of the drug. Conclusions This study demonstrated the safety and tolerability of NeuroEPO in SCA2 patients after 6 months of treatments and suggested a small clinical effect of this drug on motor and cognitive abnormalities, but confirmatory studies are warranted. © 2022 International Parkinson and Movement Disorder Society.
Background: The Center of Molecular Immunology (CIM) is a center in Cuba devoted to the research, development and manufacturing of biotechnological products. CIMAvax ® EGF is a vaccine for the treatment of non-small cell lung cancer patients (NSCL). Purpose: The aim of this work is to evaluate the effects of some potential prognostic factors on the overall survival of patients treated with CIMAvax ® EGF vaccine, based on data collected in a phase II and a phase III clinical trials. Methods: The stratified Cox regression model is used to evaluate the effects of these prognostic factors, based on separate analysis for each trial, and on the combined data from both trials. Results: Patients with Performance status 0 or 1, with IV stage of tumor and male under 60 years obtain more benefit in terms of overall survival if they receive CIMAvax ® EGF. Conclusions: Vaccinated group has a better performance if patients have a performance status 0 or 1, stage IV and age under 60 years. These prognostic factors influence overall survival in a positive way for those patients that received CIMAvax ® EGF.
RESUMENEl Centro de Inmunología Molecular ha venido desarrollando biomoléculas para el tratamiento del cáncer. En la medida que se avanza en el desarrollo de estos productos, se avanza en las fases I, II, III y IV de los ensayos clínicos, lo que trae aparejado un incremento en el número de pacientes a tratar y en el número de hospitales involucrados. Se cuenta con diez productos diferentes, implicados en más de 50 ensayos clínicos, nacionales y multinacionales, con un pronóstico de inclusión de 2 500 pacientes nuevos por año. En este proceso están incluidos alrededor de 30 hospitales en 13 provincias del país. Para mejorar el acceso a la información de ensayos clínicos por parte de los investigadores comprometidos, se creó un sitio Web formado por cuatro secciones fundamentales, relacionadas con los productos y sus indicaciones, buenas prácticas clínicas, la entrada remota de
Background: ALK rearrangements have been described in approximately 4-5% of patients with non-squamous non-small cell lung cancer (NSCLC). Crizotinib is initially effective in the treatment of ALK-rearranged NSCLC, but the disease eventually progresses. PLB1003, a highefficiency second generation ALK inhibitor, was developed due to the increased resistance of EML4-ALK fusion genes. Preclinical data show that PLB1003 is safe and effective in cell-based assays and Crizotinibresistant animal models. This is the ongoing phase Ia study of PLB1003.Method: An open-label, multicenter phase I clinical trial was conducted in patients with locally advanced or metastatic NSCLC who had previously failed or were intolerable to Crizotinib or chemotherapy. It consisted of dose-escalation cohorts and dose-expansion cohorts. In the dose-escalation cohorts, patients were orally given 50-500mg/d of PLB1003 at 6 dose levels. In each cohort, patients' plasma were collected for pharmacokinetic evaluation. The safety, tolerability, pharmacokinetics, maximum tolerated dose (MTD), dose limiting toxicities (DLT) and recommended phase 2 dose (RP2D) of PLB1003 were determined. Result: A total of 21 patients were enrolled in doseescalation cohorts as of 31 August 2018. The dose-escalation cohort is ongoing at the dose of 200 and 250 mg BID. A lipase elevation of DLT event was observed at 250 mg BID. MTD has not been reached in this study. Additionally, the most common treatment-emergent adverse events (TEAEs) (>10%) were grade1/2, including: (1) gastrointestinal toxicities: diarrhea (24%), vomiting (14%); (2) hepatotoxicity: increased GGT (g-glutamyltransferase) (48%), increased ALP (33%), elevated ALT (43%) and AST (33%); (3) others: increased blood glucose level (43%), hyperuricemia (24%), increased creatinine (19%), anemia (19%), hypercholesterolemia (14%). All the treatment-related adverse events (TRAEs) were reversible. TRAEs of grade 3, increasing of GGT (-glutamyl transferase) (33%), alkaline phosphatase (10%) and lipase (10%), mostly appeared during 7-13 weeks of initial study. Patients all recovered from TRAEs of grade 3 with symptomatic treatments. Among the 14 evaluable patients in 200mg/d cohorts, 10 patients had PR (71%), 2 patients had SD (14%), and the disease control rate (DCR) was 86%. Among the 7 patients who progressed with previous treatment of Crizotinib, 5 patients had PR (71%), 1 patient had SD (14%), and the DCR was 86%. Conclusion: PLB1003 is safe, tolerable and has potential clinical benefit to locally advanced or metastatic NSCLC patients with ALK rearrangement mutation and had disease progression or were intolerable to previously treatment of Crizotinib or chemtherapy. (ClinicalTrials.gov number, NCT03130881
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