Amyotrophic lateral sclerosis (ALS), also known as motor neuron disease or Lou Gehrig’s disease, is a disease that gradually paralyzes people because the brain is no longer able to communicate with the muscles of the body that we are typically able to move at will[1]; as ALS progresses, people will lose the ability to walk, talk, swallow, and eventually breathe.

While no treatment to prevent or even slow the progression of, ALS currently exists, recent findings indicate that neurotrophic factors (NTFs) have been shown to potentially improve the survival of motor neurons in ALS. While a single administration of NTFs has not been effective in extending the life of these motor neurons, the review suggests the direct delivery of multiple NTFs by transplantation to the CNS has proven effective in animal studies.  

Specifically, the observed benefits of mesenchymal stem cells (MSC) transplanted from bone marrow or adipose suggest improved neurological stabilization in patients with ALS. As such, the authors of this review have developed a method that produces a strong synergistic effect when introducing a combined delivery of neurotrophic factors in patients with ALS. 

The authors, in this review, report on the safety and clinical effects resulting from phase 1 / 2 and 2a clinical trials in which autologous MSC-NTS cells were transplanted in patients with ALS. Both of these studies were considered open-label proof of concept studies where patients were followed up for 3 months before transplantation and 6 months after receiving MSC-NTS transplantation.

No serious adverse events were associated with MSC-NTF cells intramuscular (IM) injections, intrathecal injections (IT), or a combination of both (IT+IM) during these studies. 

Additionally, neurotrophic growth factor secretion of patients’ cells was shown to be induced in the MSC-NTF cells when compared to MSCs of the same patient prior to differentiation.  In all samples, MSC-NTF cells demonstrated increased secretion of NTFs when compared to non-differentiated MSCs from the same patient.

As a result of this study, the authors have concluded that IT and IM injections of MSC-NTF cells in patients with ALS are safe and well-tolerated.  While not the primary focus of the study, the findings also demonstrated clinically meaningful benefits specifically induced by intrathecal treatment with MSC-NTF cells, including potentially slowing the rate of ALS progression.

Considering that neurologists specializing in the treatment of ALS consider a reduction in ALS-FRS-R slope of 25% or more to be clinically significant, the change in ALS progression rate observed after MSC-NTF cell transplantation in this study may indicate a clinically meaningful effect to be confirmed in future clinical trials.   

Source:   (n.d.). Safety and Clinical Effects of Mesenchymal Stem Cells Secreting …. Retrieved from https://pubmed.ncbi.nlm.nih.gov/26751635/


[1] “Amyotrophic lateral sclerosis (ALS) – Symptoms and causes – Mayo ….” 6 Aug. 2019, https://www.mayoclinic.org/diseases-conditions/amyotrophic-lateral-sclerosis/symptoms-causes/syc-20354022. Accessed 15 Feb. 2021.

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