Abstract
ObjectiveAmyotrophic lateral sclerosis (ALS) is still incurable. Although different therapies can affect the health and survival of patients. Our aim is to evaluate the effect of umbilical mesenchymal stem cells administrated intrathecally to patients with amyotrophic lateral sclerosis on disability development and survival.MethodsThis case-control study involved 67 patients treated with Wharton’s jelly mesenchymal stem cells (WJ-MSC). The treated patients were paired with 67 reference patients from the PRO-ACT database which contains patient records from 23 ALS clinical studies (phase 2/3). Patients in the treatment and reference groups were fully matched in terms of race, sex, onset of symptoms (bulbar/spinal), FT9 disease stage at the beginning of therapy and concomitant amyotrophic lateral sclerosis medications. Progression rates prior to treatment varied within a range of ± 0.5 points. All patients received three intrathecal injections of Wharton’s jelly-derived mesenchymal stem cells every two months at a dose of 30 × 106 cells. Patients were assessed using the ALSFRS-R scale. Survival times were followed-up until March 2020.ResultsMedian survival time increased two-fold in all groups. In terms of progression, three response types measured in ALSFRS-R were observed: decreased progression rate (n = 21, 31.3%), no change in progression rate (n = 33, 49.3%) and increased progression rate (n = 13, 19.4%). Risk-benefit ratios were favorable in all groups. No serious adverse drug reactions were observed.InterpretationWharton’s jelly-derived mesenchymal stem cells therapy is safe and effective in some ALS patients, regardless of the clinical features and demographic factors excluding sex. The female sex and a good therapeutic response to the first administration are significant predictors of efficacy following further administrations.Graphical Medical therapeutic experiment with retrospective case-control analyses
Highlights
There is no causal therapy for amyotrophic lateral sclerosis (ALS) – a rare, fatal, progressive disease that has been rapidly increasing [1] and occurs de novo in 90% [2]
Signs of the PLP syndrome were short-lived and resolved without any. In her March 2020 JAMA Neurology publication, Vijayaraghavan highlighted the problem of limited access to clinical studies for patients with ALS
Without calling into the obvious objections related to unproven therapies based on stromal vascular fraction (SVF) [20, 21] or ill-defined “stem cells” [22] administered by clinicians without the proper background or even by persons without a medical license [23], this case clearly shows that there are risks attached to action, and to omission
Summary
There is no causal therapy for amyotrophic lateral sclerosis (ALS) – a rare, fatal, progressive disease that has been rapidly increasing [1] and occurs de novo in 90% [2]. No new drugs have been registered for this indication since 1994 [4], partly due to our incomplete understanding of the complex pathogenesis of motor neuron degeneration [5]. Another drug, edaravone, has been registered in Japan and the USA. There is still a huge unmet need for a treatment that could effectively alter the progression rate of this debilitating disease
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