Umbilical Cord Mesenchymal Stem Cells in Amyotrophic Lateral Sclerosis: an Original Study
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Umbilical Cord Mesenchymal Stem Cells in Amyotrophic Lateral Sclerosis: an Original Study Monika Barczewska 1,2,3 & Stanisław Maksymowicz 2,4 Mariusz Grudniak 5
&
Izabela Zdolińska-Malinowska 5 & Tomasz Siwek 2,3,6 &
# The Author(s) 2020
Abstract Objective Amyotrophic 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. Methods This 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. Results Median 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. Interpretation Wharton’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.
Keywords Amyotrophic lateral sclerosis . WJ-MSC . ALS . Umbilical cord . Survival . Progression rate . Medical experiment
Introduction 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]. The studies using iPSC-derived motor neurons revealed the complicated molecular nature of this disease related to mitochondrial dysfunction and ER stress, NF aggregate formation,
Electronic supplementary material The online version of this article (https://doi.org/10.1007/s12015-020-10016-7) contains supplementary material, which is available to authorized users. * Stanisław Maksymowicz [email protected] 1
Department of Neurosurgery, University of Warmia and Mazury, Olsztyn, Poland
2
Instytut Terapii Komórkowych S.A., FamiCord Group, Olsztyn, Poland
3
University Clinical Hospital, Olsztyn, Poland
4
Departm
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