Stem cell therapy may do most good when given early

People with multiple sclerosis (MS) who have never received treatment with a disease-modifying therapy tend to have better long-term outcomes after stem cell transplant, according to a recent report from a center in Mexico.

The findings suggest that stem cell therapy may be most beneficial when given early in the disease course, according to researchers.

The study, “Long-term results of autografting persons with multiple sclerosis are better in those not exposed to prior disease-modifying therapies,” was published in Multiple Sclerosis and Related Disorders.

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Autologous hematopoietic stem cell transplantation (aHSCT), also known as stem cell transplant or stem cell therapy, is a procedure that basically aims to reset a person’s immune system to reduce the excess inflammation that drives MS.

The procedure involves collecting stem cells living in the bone marrow, which give rise to all immune cells. Patients then are treated with chemotherapy and/or radiation therapy to completely wipe out their immune system and the stem cells are infused back to repopulate the immune system with healthy cells.

This procedure is not specifically approved to treat MS in the U.S., though it is widely used to manage select MS patients.

In this study, a team of scientists in Mexico reported on outcomes for patients with all types of MS who underwent aHSCT at their center. Out of more than 1,100 patients who underwent aHSCT since 2015, 74 were followed for at least three years.

Among these 74 patients, 55 had been treated previously with disease-modifying therapies (DMTs) — anti-inflammatory medications that can slow the progression of MS — while the other 19 had not. Patients who had been treated previously had generally been living with MS longer and had more advanced disease at the time of aHSCT.

EDSS scores

To compare long-term outcomes, the researchers looked at scores on the Expanded Disability Status Scale (EDSS), a standardized measure of disability in MS. This scale ranges from 0, indicating no disability, to 10, for death. Prior to receiving aHSCT, median EDSS scores tended to be increasing over time, which is consistent with MS progressing to cause more severe disability.

Among patients who had been on prior DMTs, the median EDSS score was 5.5 at the time of aHSCT. This score suggests that patients’ disability caused substantial impairments in day-to-day life, but patients still could walk moderate distances without an aid. At three years after aHSCT, the median EDSS had not changed, suggesting disease had stabilized.

Among the patients who had not been on DMTs before undergoing aHSCT, the median EDSS score was 4.5, meaning patients had significant disability but still were able to do most daily tasks independently. Three years after stem cell therapy, the median EDSS score had decreased to 4, suggesting a slight improvement in functional abilities for most patients.

The researchers stressed that this analysis is limited by the small number of patients. Also, because there was a lot of variability in this group, the researchers were unable to perform analyses to compare outcomes for patients who had been on different DMTs prior to aHSCT.

“Additional studies are needed to further analyze the impact of the use of DMT therapies prior to the aHSCT in MS, as well as the timing of the procedure,” they wrote.