To be continued
The biopharmaceutical company NeuroGenesis, specializing in the development of methods for the cellular treatment of neurodegenerative demyelinating diseases, and the Hadassah Medical Center announced positive results of a phase 2 randomized placebo-controlled clinical trial evaluating the effect of an autologous subpopulation of bone marrow cells NG-01 on the expression levels of the biomarker of neurogenerative diseases NF-L in patients with progressive multiple sclerosis (MS).
Neurofilament light chains (NF-L) is a protein secreted into the cerebrospinal fluid by damaged neurons, it is a reliable biomarker of neurodegeneration in patients with multiple sclerosis and other diseases. NF-L can be used as a biomarker of multiple sclerosis activity, as well as a response to various types of treatment.
In phase 2 of a randomized, double-blind, placebo-controlled clinical trial, the safety, tolerability and efficacy of NG-01 transplantation in people with progressive multiple sclerosis were evaluated. Treatment involves taking bone marrow from the patient. Then, using a patented process, a unique subpopulation of bone marrow stem cells is identified, which is cultured and enhanced in the direction of remyelinating biofactor cells (NG-01), also possessing neurotrophic and neuroprotective properties. A population of NG-01 cells is injected directly into the central nervous system (into the cerebrospinal fluid), where cells accumulate in the damaged area and produce a significant amount of neurotrophic factors.
The study involved 48 patients with progressive multiple sclerosis who were randomized into three groups receiving endolumbal (into the spinal canal), intravenous injection of NG-01, or placebo. Samples of cerebrospinal fluid were taken from the study participants before and 6 months after treatment with NG-01 or placebo, the level of the biomarker NF-L was assessed in each sample. The material of four patients technically defied testing for NF-L.
The results showed that the initial concentration of NF-L in the cerebrospinal fluid was exceptionally high in almost all patients, which indicates the activity and aggressiveness of the disease before treatment. This is also evidenced by the Expanded Disability Assessment Scale (EDSS) and MRI results. In the group of endolumbal administration of NG-01, 6 months after treatment, the level of NF-L significantly decreased compared to the baseline values before treatment (by an average of 63.5%). A less pronounced effect was observed in the group of intravenous administration of NG-01, whereas in the placebo group the concentration of NF-L actually increased (by an average of 47.5%), indicating the progression of the disease. Nine out of 15 patients in the endolumbal injection group showed a decrease in NF-L levels by more than 50% compared to only one out of 15 in the placebo group.
A decrease in the level of NF-L correlated with a significant improvement in the condition of patients. Eight out of nine patients in the NG-01 endolumbal injection group who had a decrease in NF-L levels by more than 50% showed stable or elevated EDSS scores after 6 and 12 months of follow-up. In the same study, scientists reported a clear decrease in proinflammatory chemokine in the cerebrospinal fluid after NG-01 endolumbal therapy, which indicates local immunomodulation in the central nervous system induced by stem cell therapy.
The NF-L level provides the first objective evidence of neuroprotection induced by endolumbally injected stem cells. The results obtained echo the clinical success of NG-01 in two studies of progressive multiple sclerosis and amyotrophic lateral sclerosis, published in the journals Frontiers of Neurology and Frontiers in Bioscience, respectively.
The authors are currently working on expanding production capacity in preparation for a new multicenter phase 2b study, which is scheduled to begin at the end of 2022.
Article by P.Petrou et al. The Effects of Mesenchymal Stem Cell Transplantation on Cerebrospinal Fluid Biomarkers in Progressive Multiple Sclerosis is published in the journal Stem Cells Translational Medicine.
Aminat Adzhieva, portal "Eternal Youth" http://vechnayamolodost.ru