MS news notes: Stem cell therapy, early Aubagio use, DMTs

Welcome to “MS News Notes,” a Monday morning column where I comment on multiple sclerosis (MS) news stories that caught my eye last week. Today I’ll focus on stories from the American Academy of Neurology (AAN) 2023 Annual Meeting, held April 22-27 in Boston. Here’s a look at what’s been happening:

Stem cell therapy receives a good report card

The MS News Today article “AAN 2023: Low rates of disease activity after stem cell transplant” reports on a follow-up study of 22 autologous hematopoietic stem cell transplant (aHSCT) patients who received the treatment at a center in Mexico.

After two years, all 22 met NEDA3 (no evidence of disease activity 3) criteria, which includes no new relapses, no sustained worsening of disability, and no new disease activity on MRI scans. After seven years, 16 of them were still at NEDA3. Seven of the 22 patients had significant safety issues related to the aHSCT procedure itself, including severe infections and fever, though none died due to complications related to it.

aHSCT is a one-time procedure to reset a person’s immune system by collecting blood stem cells (which give rise to immune cells) from a patient’s bone marrow, then administering intensive chemotherapy and/or radiation to destroy existing immune cells. The stem cells are then transplanted back into the patient, with the aim of repopulating healthy immune cells that are less prone to the type of inflammatory attack that drives MS.

Unfortunately, no stem cell therapy has been approved by the U.S. Food and Drug Administration specifically to treat people with MS, but some stem cell-based therapies are in clinical testing.

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A stem cell therapy appears to improve walking

A slightly different kind of stem cell therapy is the focus of a study reported on in “AAN 2023: MSC-NP aids walking in advanced progressive MS patients.” In this case, the therapy involves collecting mesenchymal stem cells, or MSCs, from a patient. These cells then are engineered in a laboratory to grow into neural progenitor cells, or NPs — a more specialized type of stem cell that’s able to grow into neurons (nerve cells) and other types of cells critical for nervous system health. The engineered cells are injected into the patient’s nervous system through the spinal canal.

A Phase 2 clinical trial using MSC-NP reported the treatment improved walking ability for people with more advanced disease — those who need an aid to walk short distances. Patients treated with MSC-NP also often reported fewer bladder problems following treatment. However, MSC-NP failed to show a significant effect on overall disability scores among people with progressive forms of MS.

Good results from early Aubagio use

My interest in reading “AAN 2023: Aubagio reduced risk of developing MS symptoms in RIS trial” comes from the fact that according to the MS Society, Aubagio (teriflunomide) is considered a moderately effective disease-modifying therapy (DMT) and that it was administered in the study before an actual MS diagnosis was made.

Radiologically isolated syndrome, or RIS, is a condition in which people have MS-like brain and spinal cord lesions without showing disease activity. Several years ago, MS News Today reported that researchers disagreed at the time about whether to start any treatment for people with RIS.

New data from a Phase 3 clinical trial show that 28 of the 89 participants in the study developed new MS symptoms: 20 of them had been given a placebo and eight had been treated with Aubagio. Statistical analyses showed the risk of progression to MS was significantly lowered, by approximately 63%, in the patients who were treated with Aubagio.

But the question for me that wasn’t addressed by this study is whether any of the other available DMTs, particularly those considered high-efficacy, might have reduced the risk of progression even more.

Two follow-up DMT studies

A number of stories from the AAN conference are about how well some DMTs are doing their job a number of years after the first treatment. Here’s a brief look at two of them:

“AAN 2023: MS disability not worse for most on Kesimpta over 5 years” reports that more than 80% of patients with relapsing forms of MS who were treated with Kesimpta (ofatumumab) continuously for up to five years in a clinical trial had no documented worsening of their disability.

“AAN 2023: Tolebrutinib safely lowers relapse rates over 2.5 years” says that an ongoing, open-label extension of a Phase 2b trial for that treatment was associated with low relapse rates and stable disability levels.

Note: Multiple Sclerosis News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Multiple Sclerosis News Today or its parent company, BioNews, and are intended to spark discussion about issues pertaining to multiple sclerosis.