stem-cells-2019-posters-abstracts

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Volume 02

Stem Cells 2019 & Pediatrics Congress 2019

November 06-07, 2019

Journal of Clinical Genetics and Genomics

November 06-07, 2019 | Tokyo, Japan

STEM CELLS AND REGENERATIVE MEDICINE

PEDIATRICS AND CHILD CARE

International Conference on

World Congress on

J Clin Gen Genomics, Volume 02

Stem cells for treatment of amyotrophic lateral sclerosis

Eva Sykova

Scimed Biotechnologies Ltd., Czech Republic

myotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disorder leading to the death of motoneurons (MN). In

our preclinical study human mesenchymal stem cells (hMSCs), were delivered intrathecally into SOD1 G93A transgenic rats.

Survival in the hMSC-treated group was prolonged and rats showed better motility and grip strength. We found greater numbers of

perineuronal nets (PNNs) in the hMSCs-treated animals and MSCs have antiapoptotic and immunomodulatory effects. The clinical

study was designed as a prospective, non-randomized, open-label study (phase I/IIa, EudraCT No. 2011-000362-35) to assess the

safety and efficacy of autologous BM-MSC in the treatment ofALS. BM-MSCwere applied via lumbar puncture into the cerebrospinal

fluid. During the 18-month follow-up period no serious adverse reactions or new cerebrospinal pathology on MR examinations were

observed. The clinical outcome was evaluated by an ALS functional rating scale (ALSFRS), norris spinal and bulbar scale (NSS

and NSB), forced vital capacity (FVC) and weakness scale (WS). In almost 80% of patients FVC values remained above 60% for a

time period of 12 months. A group of 14 patients, with remarkable pretreatment decline in functional scales (ALSFRS + NSS), had

significant reduction/stabilization in their total functional score decline at 3 months after application (p<0.02) that persisted for 6

months (p<0.05). In about 80% of the patients, FVC values remained stable or above 70% for a time period of 9 months. These results

demonstrate that the intrathecal application of BM-MSC in ALS patients is a safe procedure and that it can slow down progression

of the disease.