The harvested umbilical cords were transported to the laboratory under monitored conditions and processed within 48 h of delivery. WJ-MSCs were donated from healthy newborns following maternal qualification based on a medical questionnaire conducted after the parents signed an informed consent form. The following laboratory tests were mandatory: C-reactive protein (CRP), sodium, potassium, glucose, blood cell count, coagulation, urea and creatinine. Enrolled patients met the following profile: clinical diagnosis of definite ALS based on the El Escorial World Federation of Neurology criteria, ventilatory independence, age 20–78 years and the ability to attend clinic visits alone or with support. Patients enrolled into the study were recruited between 20. Prior to starting the experiment, all patients signed an informed consent form. The treatment was reviewed by the Ethics Committee of the School of Medicine, University of Warmia and Mazury in Olsztyn, Poland and performed in accordance with the Declaration of Helsinki. in Olsztyn, Poland (Cell Therapies Institute, FamiCord Group) in cooperation with the Departments of Neurology and Neurosurgery, School of Medicine, Collegium Medicum of the University of Warmia and Mazury in Olsztyn, Poland. The study was conducted at Instytut Terapii Komórkowych S.A. This was done in the form of a medical therapeutic experiment (described elsewhere ) with retrospective case-control analyses. The aim of our study was to evaluate the effect of compassionate use of Wharton’s jelly-derived mesenchymal stem cells (WJ-MSC) in terms of progression and survival in ALS patients. Evidence from preclinical studies conducted with the animal ALS (SOD1) model provided promising results and led to the initiation of some clinical studies -, Previously, the safety and efficacy of mesenchymal stem cells (MSC) administration was described in small studies which did not evaluate overall survival. There is therefore still a huge unmet need for a treatment that could effectively alter the progression rate of this debilitating disease.Īlthough it is well known that MSC differentiate into neural cells and neuroglia, over the course of the last several years stem/stromal cell therapy was proposed for treating ALS based on small observational studies. Recently, another drug, edaravone, has been registered in Japan and the USA. No new drugs have been registered for this indication since 1994, partly due to our incomplete understanding of the complex pathogenesis of motor neuron degeneration. There is still only one approved drug in Europe – riluzole, which extends survival by 3 months on average. 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, hyperexcitability, and channel deficits. There is no causal therapy for amyotrophic lateral sclerosis (ALS) – a rare, fatal, progressive disease that has been rapidly increasing and occurs de novo in 90%. The female sex and a good therapeutic response to the first administration are significant predictors of efficacy following further administrations. 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. No serious adverse drug reactions were observed. Risk-benefit ratios were favorable 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%). Median survival time increased two-fold in all groups. Survival times were followed-up until March 2020. Patients were assessed using the ALSFRS-R scale. All patients received three intrathecal injections of Wharton’s jelly-derived mesenchymal stem cells every two months at a dose of 30 × 10 6 cells. Progression rates prior to treatment varied within a range of ± 0.5 points. 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. 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). This case-control study involved 67 patients treated with Wharton’s jelly mesenchymal stem cells (WJ-MSC). 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. Although different therapies can affect the health and survival of patients. Amyotrophic lateral sclerosis (ALS) is still incurable.
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