Abstract
ObjectivesAmyotrophic lateral sclerosis (ALS) is a progressive, fatal disease with no curative treatment up to now. This study aims to analyze ALS progression of patients treated with mouse nerve growth factor (mNGF), as well as the effects, side effects, and adverse events of the therapy.Materials and MethodsA retrospective, observational study was performed including 94 patients with ALS from July 2020 to July 2021. Thirty-two of them were treated with at least one course of mNGF on a regular riluzole use, and the rest 62 were treated with riluzole only. The declining rates of body mass index (BMI) and ALS Functional Rating Scale-Revised (ALSFRS-R) scores were compared between the two groups to indicate ALS progression.ResultsNo significant differences in ALS progression indicated by the declining rates of BMI and ALSFRS-R score were observed between the two cohorts. ALS progression before and after the first treatment course of mNGF also showed no discernible difference. However, we noticed a moderate 62.7 and 25.1% reduction in the declining rate of BMI and ALSFRS-R motor subscore when comparing mNGF + riluzole treatment to riluzole only. The mNGF treatment was overall safe and well-tolerated, and a rare case of diarrhea was reported after mNGF injection.ConclusionsOur study revealed that mNGF treatment was overall safe and well-tolerated in patients of ALS. Application of mNGF combined with regular riluzole treatment had no significant clinical effects on delaying ALS progression. Prospective cohort studies and randomized clinical trials based on larger cohorts and longer follow-up times are needed to make a more convincing conclusion.
Highlights
Amyotrophic lateral sclerosis (ALS) is a motor neuron neurodegenerative disease
Some prognostic indicators of ALS, such as body mass index (BMI), onset domain, ALSFRS-R score, and forced vital capacity (FVC) % were compared at baseline and no significant difference was observed
Considering the missing data of our patients with ALS cohorts, not all patients in the initial Riluzole + Mouse NGF (mNGF) Group were included in further studies
Summary
Amyotrophic lateral sclerosis (ALS) is a motor neuron neurodegenerative disease. It is characterized by progressive weakness, atrophy, and spasticity of muscles, which is a consequence of progressive degeneration of both upper and lower motor neurons [1]. ALS exhibits a fatal, progressive disease course, with most patients dying 3–5 years after disease onset or within 15–20 months after diagnosis, mainly due to respiratory failure [1, 2]. Many clinical trials have been devoted to finding effective therapies for ALS, such as drugs such as pramipexole [6], memantine [7], flecainide [8], and cellular therapies such as stem cell transplantation [9]. No significant progress in treating ALS has been achieved until now
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