Abstract
Stem cells (SCs) may constitute a perspective alternative to pharmacological treatment in neurodegenerative diseases. Although the safety of SC transplantation has been widely shown, their clinical efficiency in amyotrophic lateral sclerosis (ALS) is still to be proved. It is not only due to a limited number of studies, small treatment groups, and fast but nonlinear disease progression but also due to lack of objective methods able to show subtle clinical changes. Preliminary guidelines for cell therapy have recently been proposed by a group of ALS experts. They combine clinical, neurophysiological, and functional assessment together with monitoring of the cytokine level. Here, we describe a pilot study on transplantation of autologous adipose-derived regenerative cells (ADRC) into the spinal cord of the patients with ALS and monitoring of the results in accordance with the current recommendations. To show early and/or subtle changes within the muscles of interest, a wide range of clinical and functional tests were used and compared in order to choose the most sensitive and optimal set. Additionally, an analysis of transplanted ADRC was provided to develop standards ensuring the derivation and verification of adequate quality of transplanted cells and to correlate ADRC properties with clinical outcome.
Highlights
Amyotrophic lateral sclerosis is a fatal neurodegenerative disease characterized by a progressive loss of motor neurons in the central and peripheral nervous system
Nine patients presented clinically possible amyotrophic lateral sclerosis (ALS) according to the El Escorial criteria [34], 31 had a disease duration longer than 24 months, and 10 patients were at the advanced disease stage (ALSFRS R < 25)
Qualification of ALS patients to controlled pharmacological clinical trials encounters a number of obstacles resulting from the disease character
Summary
Amyotrophic lateral sclerosis is a fatal neurodegenerative disease characterized by a progressive loss of motor neurons in the central and peripheral nervous system. Stem Cells International edaravone, recently registered in Japan and US, relents disease progression in a subgroup of patients without respiratory involvement [2, 3]. Stem cells (SCs) may constitute a perspective alternative to pharmacological treatment in ALS. The safety of SC transplantation has been widely shown [6,7,8], their efficiency in ALS is still unproven. It is due to a limited number of studies, high variability of transplanted cell types, small treatment groups, and fast but nonlinear disease progression and the lack of objective methods able to prove subtle clinical changes
Sign-in/Register to view highlights & summary Talk to us
Join us for a 30 min session where you can share your feedback and ask us any queries you have
Schedule a callDisclaimer: All third-party content on this website/platform is and will remain the property of their respective owners and is provided on "as is" basis without any warranties, express or implied. Use of third-party content does not indicate any affiliation, sponsorship with or endorsement by them. Any references to third-party content is to identify the corresponding services and shall be considered fair use under The CopyrightLaw.
