Is stem cell therapy considered for multiple sclerosis?

Question

Hello doctor,

My wife is 45 years old and has been living with multiple sclerosis (MS) for the past 11 years. She has tried multiple disease-modifying therapies, including interferon, glatiramer acetate, and dimethyl fumarate, and has been on Ocrelizumab for the past two years. Despite these treatments, she continues to decline, experiencing increased fatigue, muscle weakness, and bladder control issues. Her most recent magnetic resonance imaging (MRI) showed three new lesions, and she has recently started having trouble with fine motor skills, making her work as a dental hygienist nearly impossible. The neurologist suggests staying the course with the current treatment, but we are seeing a steady decline despite doing everything right. We recently came across information about stem cell therapy, specifically hematopoietic stem cell transplantation (HSCT) for MS, where they essentially reboot the immune system. Some online MS communities have members claiming dramatic improvements or even halting of disease progression after stem cell treatments. My wife is still young, and we have teenage children, so we are desperate to try anything that might preserve her function longer.

Is there solid evidence behind stem cell treatments for MS? What are realistic expectations and major risks for someone with moderate disability (Expanded Disability Status Scale [EDSS] 4.5)? Are clinical trials available, or would we need to go overseas? Her quality of life is deteriorating, and we are willing to accept some risk if there is a real chance of improvement.

Please help.

Thank you.

Answer 1

Hello,

Welcome to icliniq.com.

I understand your concern.

Your concerns are completely valid, and I understand your urgency in exploring every possible option to slow down your wife's MS progression. HSCT (hematopoietic stem cell transplantation) is one of the most researched forms of stem cell therapy for MS, and there is solid scientific evidence that it can significantly reduce disease activity in some patients, particularly those with relapsing-remitting MS (RRMS) who still have active inflammation. However, for someone like your wife, who has been on multiple disease-modifying therapies (DMTs) and is continuing to decline, it is important to carefully weigh the potential benefits and risks.

HSCT has shown strong success in halting disease progression, particularly in younger patients (under 50) with active inflammatory MS (new lesions, relapses). A study (MIST trial) found that HSCT was significantly more effective than standard DMTs in stopping new disease activity.

Long-term data suggest up to 70 percent of patients show no progress in disease at five to ten years post-transplant, especially if done early in the disease course. However, patients with progressive MS without active inflammation tend to have less benefit because the treatment does not repair existing damage. Since your wife has new lesions on MRI, this suggests some inflammatory activity, which makes her a better candidate than someone with only neurodegeneration. Studies indicate HSCT works best when EDSS is below 6.0 (before severe disability sets in). Your wife, at EDSS 4.5, still has a window where HSCT could help preserve function.

The goal of HSCT is to stop progression, not necessarily to reverse existing damage, but some patients do see improvements in fatigue, mobility, and bladder function. So it means you can expect her to get better, but she will not get any worse. HSCT is an intensive, high-risk treatment. It involves chemotherapy to wipe out the immune system before reinfusion of stem cells. So your wife's immune system will remain suppressed for a few weeks, and during this time she will become vulnerable to any infection. Chemo can also cause permanent infertility and early menopause. You need to be mindful of that, too.

Nausea, vomiting, and weight loss are some common side effects. But the major and rare risk is mortality, which is, with modern protocols, less than one percent. But do not get scared, as a simple operation like an appendicitis operation also has a mortality rate. Every procedure has some complications, even her ongoing treatments. So stay calm and focus on the possibilities.

I do not know where you are from, so I cannot suggest any specific place, but I can suggest some. In the USA, FDA-approved HSCT is only available for MS in clinical trials at selected centers (Northwestern University in Chicago, Johns Hopkins). Getting into a trial can be challenging, as they often require recent relapses or MRI activity.

In the case of other countries, HSCT is offered in Russia, Mexico, Israel, and India for MS patients, but quality and safety protocols vary. Russia and Mexico have treated many international MS patients with outcomes similar to clinical trials.

I hope this helps.

Kindly follow up if you have more concerns.

Thank you.