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Positive Effects of Stem Cell Therapy in Progressive MS Revealed


Core Concepts
Stem cell therapy shows promising results in treating progressive MS.
Abstract

The content discusses the positive effects of intrathecal injection of autologous bone marrow-derived mesenchymal stem cells (MSCs) in patients with progressive multiple sclerosis (MS). Here is a breakdown of the key points:

  • Study presented at ACTRIMS Forum 2024
  • Favorable effects on cognitive function, neurological tests, quality of life, and biomarkers
  • Initial clinical study published in Brain in 2020
  • No disease activity in 58.6% of patients receiving intrathecal injections
  • Intrathecal injections showed greater efficacy and benefits
  • Interim results on 23 patients in the extension study
  • Improvements in cognitive function, walk test, and quality of life
  • Reduction in serum neurofilament light chain (sNfL) levels and glial fibrillary acid protein (GFAP)
  • Safety and tolerability of therapy remained encouraging
  • Urgent need for more therapies for progressive MS
  • MSCs associated with immunomodulatory and neuroprotective effects
  • Results suggest possible neuroprotective and neurotrophic effects
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Stats
"there has been favorable effects on cognitive function, neurological functional tests, quality of life, and both of two major biomarkers linked to neurodegeneration," reported study investigator Dimitrios Karussis, MD, PhD "When evaluated at the end of 1 year, there was no evidence of disease activity in 58.6% of those receiving the two intrathecal injections of MSCs, 40.6% of those receiving two IV injections of MSCs, and 9.7% of those initially randomized to the sham group." "For sNfL, the reduction was 33.2% (P = .001), and there was further decline observed after repeated MSC injections." "The 22% (P < .0004) reduction in GFAP, which Karussis said has not been shown before, was observed in all 23 patients."
Quotes
"the results from this group are substantially better than those reported by several other groups." - Jeffrey A. Cohen, MD

Deeper Inquiries

What are the potential implications of these positive results for the future treatment of progressive MS?

The positive results of the study on intrathecal injection of autologous bone marrow–derived mesenchymal stem cells (MSCs) in patients with progressive MS have significant implications for future treatments. If further studies confirm the efficacy and safety of this therapy, it could potentially revolutionize the management of progressive MS. The observed improvements in cognitive function, neurological tests, quality of life, and biomarkers associated with neurodegeneration suggest that MSC therapy may offer a promising avenue for addressing the unmet needs of patients with progressive MS. The reduction in disease activity, lesion load, relapse rates, and improvements in cognitive function are particularly encouraging outcomes that could lead to the development of more effective treatments for progressive MS.

How can the discrepancies in results between different groups studying MSC transplantation be reconciled?

The discrepancies in results between different groups studying MSC transplantation may be reconciled through further research and standardization of protocols. Variations in study design, patient selection criteria, MSC preparation methods, dosages, and administration routes could contribute to the differences in outcomes observed across studies. By establishing standardized protocols for MSC transplantation in progressive MS, researchers can ensure consistency in treatment approaches and outcomes. Additionally, conducting multicenter trials with larger sample sizes and longer follow-up periods can help validate the findings and address discrepancies between studies. Collaborative efforts among researchers to share data, methodologies, and results can also facilitate a better understanding of the potential benefits and limitations of MSC therapy in progressive MS.

How might the neuroprotective and neurotrophic effects of MSC therapy impact other neurodegenerative conditions?

The neuroprotective and neurotrophic effects of MSC therapy observed in the study on progressive MS have implications for other neurodegenerative conditions. MSCs have been shown to produce soluble factors with immunomodulatory, neuroprotective, and repair-promoting properties, which could have broad applications beyond MS. In conditions such as Alzheimer's disease, Parkinson's disease, amyotrophic lateral sclerosis (ALS), and spinal cord injury, where neurodegeneration plays a central role, MSC therapy may offer potential benefits. By modulating the immune response, promoting neuronal survival, and enhancing tissue repair, MSCs have the potential to mitigate neurodegenerative processes and improve clinical outcomes in various neurological disorders. Further research is needed to explore the therapeutic potential of MSCs in different neurodegenerative conditions and elucidate the mechanisms underlying their neuroprotective and neurotrophic effects.
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