Nature Journal: The First Clinical Trial Of Embryonic Stem Cells For The Treatment Of Multiple Sclerosis

Jan 12, 2023

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Multiple sclerosis (Multiple Sclerosis, MS) is a kind of neurodegenerative disease, is due to the immune system error attack and destroy the myelin, involving the optic nerve, spinal cord, brainstem, white matter around the ventricle, lead to abnormal vision, limb movement disorder and ataxia clinical symptoms, patients will gradually lose the limbs and parts of the other parts and function of the body.

Although multiple treatments have been developed, they are incurable, and these therapies mainly target relapsing-remitting multiple sclerosis (RRMS), and effective treatments for progressive multiple sclerosis (PMS) remain lacking,

On January 9,2023, researchers from the IRCCS San Raffaele Institute of Science and other institutions in Italy published a research paper titled: Neural stem cell transplantation in patients with progressive multiple sclerosis: an open-label, phase 1 study in the top international medical journal Nature Medicine.

The study tried a new treatment for multiple sclerosis. The team collected donated embryonic stem cells from 10-12 weeks and injected them into the spine of patients with multiple sclerosis, and successfully reduced disease markers. This is also the first phase I clinical trial to evaluate the safety of neural stem cell / precursor cell (hfNPC) transplantation in the human fetal central nervous system in patients with progressive MS.

Neural stem cells / progenitor cells (NPC) are mitotically active, self-renewing multipotent cells, which can differentiate into mature brain cells, reduce myelin demination, astrogliosis and axonal loss, and play nutritional support and anti-inflammatory functions. NPC, derived from the human fetal central nervous system (hfNPC), due to its replication capacity in standardized and quality control conditions, can generate cell lines available for nerve transplantation therapy, and its therapeutic properties have been demonstrated in non-human primates.

This clinical trial included four injections of embryonic stem cells into 12 patients with multiple sclerosis. Prior to the injection, all patients had severe symptoms and were already bedridden. In addition, each patient underwent a lumbar puncture to extract spinal fluid from the spinal cord to measure the MS marker —— neuroprotective and anti-inflammatory molecular levels. Each patient also underwent an MRI scan to measure brain gray matter volume, as patients with MS gradually lose brain mass.

Three months later, each patient underwent a second lumbar puncture, and the research team found an increase in both the neuroprotective and anti-inflammatory molecules in their spinal fluid. Each patient also underwent an MRI scan two years after the ES cell treatment, and those with the most injected ES cells had the least gray matter loss in the brain.

Based on the results, the team says it is too early to determine if their symptoms have decreased or the disease has slowed. The research team will also continue to monitor these patients over the coming years to further see if ES cell therapy relieves symptoms and disease progression in patients with multiple sclerosis.

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