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Groundbreaking Brain Stem Cell Transplant Study Shows Promising Results in Treating Secondary Progressive Multiple Sclerosis

Groundbreaking Brain Stem Cell Transplant Study Shows Promising Results in Treating Secondary Progressive Multiple Sclerosis

January 18, 2024
Dr. Lana du Plessis
January 18, 2024
Dr. Lana du Plessis

Multiple sclerosis (MS) is a neurological illness that affects over 2 million individuals worldwide and can cause difficulties with movement, vision, and thought. There are three main types of MS – relapsing, primary progressive and secondary progressive. MS affects everyone differently.

Many MS patients eventually acquire secondary progressive MS, a more severe form of the illness, even though there are medications that can help lessen the frequency and intensity of MS attacks. Myelin, the covering that protects nerve fibres, is attacked by the immune system in multiple sclerosis (MS), impairing crucial brain and spinal cord communication. Immune cells called macrophages, which usually eradicate undesired invaders, are very important in this process. Among these, microglial cells—which are distributed throughout the spinal cord and brain—are essential.

One of the pioneering stem cell therapies for MS was the clinical trial NCT04943289 registered in June 2021, which planned to treat progressive MS with the cord blood product DUOC-01. The trial with the cord blood product for MS relies on intrathecal cell delivery, which is an injection into the cerebral-spinal fluid, and it has been proven safe in children. It is very important to note that the DUOC-01 trial for MS targets patients with the primary progressive form of the disease, not the relapsing-remitting MS patient group which is eligible for autologous HSCT.

Regretfully, secondary progressive MS has few available treatments. Furthermore, no medication has been approved to treat the most severe types of the illness.

The body’s “master stem cells” may be able to lessen this harm according to recent data. A brain stem cell transplant is performed using these stem cells, and it has the potential to regenerate damaged brain cells caused by multiple sclerosis.

The brains of fifteen patients with secondary progressive multiple sclerosis received direct injections of brain stem cells as part of an early-stage clinical trial, and the results were encouraging, according to a recent study.

Prior to the surgery, each participant had a thorough evaluation of their degree of disability and disease activity over a period of three months. Most of the treated patients had substantial levels of handicap at the time of transplantation (the majority needed wheelchairs, for example).

Higher stem cell doses were associated with a smaller brain capacity, according to research using sophisticated magnetic imaging on a small sample of volunteers. Strong medications used for MS patients have shown similar results, pointing to a potential function for the cells in reducing edema and inflammation in the brain. Crucially, neither relapse-like symptoms of MS, nor significant impairment in movement or cognitive function were reported in patients during the research.

Prior studies on mice have demonstrated that skin cells that have been transformed to become brain stem cells, when inserted into the central nervous system, can potentially heal damage caused by MS and reduce inflammation. Brain stem cells can also change the metabolism, or how the body makes energy, and rewire microglia from being harmful to being beneficial.

This study investigated how treating brain stem cells influenced the brain’s mechanisms for producing energy. More specifically, patients getting higher dosages of stem cells had elevated amounts of a class of chemicals called acylcarnitine’s, which are essential for maintaining a good cellular energy metabolism.

Although these results are encouraging, caution is warranted because they came from a small sample of individuals who were already taking immunosuppressive medications. However, in patients with secondary progressive MS, this study offers the first strong evidence that a single brain stem cell transplant administered directly into the brain is safe and can have long-lasting effects.

We need more research to confirm and build upon our findings. Nevertheless, this study provides encouraging evidence that this strategy may prove to be a useful therapeutic choice for treating MS patients in its latter phases.

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