Simultaneous Spinal, Blood Infusions of MSCs May Delay ALS Progression

Patricia Inácio, PhD avatar

by Patricia Inácio, PhD |

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Simultaneous injections of mesenchymal stem cells (MSCs) into the blood and spinal canal were found to delay disease progression in people with amyotrophic lateral sclerosis (ALS) in a small clinical trial.

“Our results demonstrated that the IT [intrathecal, or into-the-spine] and IV transplantation of [MSCs] in ALS patients is a safe procedure and could slow down the progression of the disease,” the researchers wrote.

Further research must be done to determine the effects of repeated MSCs injections, “with more patients and longer follow-up periods,” the team said.

The study, “Safety and efficacy of bone marrow derived-mesenchymal stem cells transplantation in patients with amyotrophic lateral sclerosis,” was published in the journal Regenerative Therapy.

MSCs have emerged as a potential cell-based therapy for people with ALS, due to their ability to enhance the survival of nerve cells and halt the inflammatory responses that contribute to disease progression.

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A number of studies have investigated these cells in ALS, but their results have been largely dependent on an optimal cell preparation, the dose used, and the site of administration.

Now, researchers in Iran and Germany conducted a single-center trial (IRCT20160809029275N2) to assess the safety and efficacy of MSCs, infused simultaneously into the bloodstream and spinal canal, in people with ALS. The stem cells used in this trial were autologous, meaning they were isolated from the patients’ own tissue — their bone marrow.

The trial included 15 adults — four women and 11 men, with a median age of 35. The majority (13 patients) had spinal-onset ALS.

The participants received concomitant, or simultaneous MSC infusions into the blood (intravenous) and spinal canal (intrathecal), and were then followed for six months.

The study’s main goal was to determine the safety of the dual transplants, which was measured in the three days following the infusions. Secondary measures included changes in the rate of disease worsening — measured as the rate of decline in the ALS Functional Rating Scale (ALSFRS-R) score — and forced vital capacity (FVC), a measure of lung function.

No serious adverse reactions were seen in any of the 15 patients. Following three days of infusion, all patients experienced the mild-to-moderate headaches that are often seen after lumbar punctures. One patient had an allergic reaction, respiratory distress, nausea, and vomiting that resolved within 24 hours. Two patients had urinary incontinence that lasted for three days.

Regarding efficacy, while no differences were observed in the first three months following the infusions, the patients experienced a significant reduction in their ALSFRS-R scores in the six months after treatment. Those results suggest that the MSCs were slowing disease progression, the researchers said.

A significant reduction in FVC also was observed after six months, the team said.

Overall, these findings suggest that infusion of bone marrow-derived MSCs simultaneously to the blood and spinal canal is safe and could slow disease progression, according to the researchers.

“Our data indicate a temporary reduction of ALS progression after a single application” of the dual injections, the researchers wrote.

“Further clinical investigations with more patients and longer follow-up periods,” as well as with repeat infusions of MSCs, are now required to confirm the safety and long-term efficacy of this approach, the team said.