Autologous Stem Cell Transplant in Individuals with Progressive Multiple Sclerosis Reduced Levels of Biomarker Neurofilament Light  

03/02/2022

As published in Stem Cells Translational Medicine, autologous stem cell transplant (NG-01; Neurogenesis, Rochester, NY) significantly reduced levels of neurofilament light (NfL) in individuals with progressive multiple sclerosis (PMS) in a clinical trial (NCT04823000).

Participants were individuals with primary progress MS (PPMS) or secondary progressive MS without relapses (nSPMS) who had high levels of NfL. Participants also had increasing disability on the Expanded Disability Status Scale (EDSS) and evidence of active aggressive disease on MRI evaluation before treatment.

In those treated intrathecally, 8 of 9 who had >50% reduction in NfL levels also had stable or improved in EDSS scores at 6 months. All 8 of those individuals had improved EDSS scores at 12 months.

"Progressive MS is a debilitating disease without effective treatment options that can substantially suppress the progression of disability," said professor Dimitrios Karussis, lead principal investigator and director of MS Center at Hadassah Medical Center, Jerusalem. "This novel trial evaluated not only the clinical effects induced by the stem cell therapy, but also the effects on the most reliable and accepted biomarker of neuro-degeneration, NfL. We therefore believe that our findings are extremely encouraging and taken together with the long-term clinical results, that showed stabilization and even functional improvement in 22 out of the 24 participants up to 4 years after the initiation of treatment, provide substantial optimism for the future of management of progressive MS and neurodegenerative diseases in general."
 

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