aHSCT Gets a Boost in US With NMSS Recommendations

Ed Tobias avatar

by Ed Tobias |

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Is the United States a step closer to approving a form of stem cell transplantation as a treatment for multiple sclerosis? I believe it may be.

That’s because the National Multiple Sclerosis Society (NMSS) has slightly changed its view of autologous hematopoietic stem cell transplantation, or aHSCT.

aHSCT involves removing a patient’s own stem cells from the blood or bone marrow, using chemotherapy to kill off most of the immune system, and then injecting the stem cells back into the patient. Those stem cells regenerate the immune system, hopefully without any of the rogue immune cells that attack the nervous system and create MS symptoms.

New NMSS recommendations

In the past, the National MS Society has failed to make recommendations about how any form of stem cell treatment should be used, at least as far as my research can determine. Now, in an article in JAMA Neurology, a group of neurologists, including members of the NMSS National Medical Advisory Committee, are outlining parts of a stem cell protocol.

The NMSS is now being specific about who might be a candidate for aHSCT:

“The National Multiple Sclerosis Society believes that AHSCT may be a useful treatment option for people with relapsing multiple sclerosis who demonstrate substantial breakthrough disease activity (ie, new inflammatory central nervous system lesions and/or clinical relapses) despite treatment with high-efficacy disease-modifying therapy or have contraindications to high-efficacy disease-modifying therapies.”

That language seems more aggressive than what the NMSS wrote about an Italian study published earlier this year. In that commentary, the society suggested the procedure “might be appropriate” for people with secondary progressive MS, and it suggested further study with a larger group:

“These results suggest that HSCT might be appropriate in a subgroup of people with SPMS that have significant inflammatory activity as measured by MRI. Further study in larger numbers are needed to understand who among those with secondary progressive MS might benefit from HSCT.”

The new guidelines clearly outline which people with relapsing-remitting MS might benefit. They are younger than 50 and have lived with an MS diagnosis for less than 10 years.

The authors recommend that stem cell transplantation be performed in medical centers with “substantial experience and expertise.” A follow-up regime is proposed, along with the creation of a single database to track people who undergo the procedure. But the article cautions that more research is needed to establish best practices for handling the stem cells and other technical processes.

What if you don’t want to wait?

Some hospitals and clinics in Russia, Mexico, and other locations offer aHSCT to MS patients at a cost of about $50,000. aHSCT also is provided to some MS patients through the U.K’s National Health Service. A list of aHSCT facilities worldwide is available at www.hsctstopsms.com.

What about a trial?

The authors of the JAMA Neurology article suggest a clinical trial may be the place for someone who wants their MS treated with a stem cell transplant right now. A well-known trial by Dr. Richard Burt shut down about a year ago. However, a trial called BEAT-MS has been enrolling patients at several locations in the U.S. You can find information about other studies at clinicaltrials.gov.

The authors note that joining a trial “would assure the patient of quality care with an acceptable protocol and provide the personal satisfaction of knowing they are contributing to answering a very important question for people with MS.”

That sounds like a pretty good suggestion to me since the U.S. likely has a long way to go before aHSCT for MS becomes common here, even with the NMSS’ backing. But it’s encouraging that finally it is gaining some traction.

You’re invited to follow my personal blog at www.themswire.com.


 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.


Su Metcalfe avatar

Su Metcalfe

Hello Ed,

Thank you for this article - being from the UK I had assumed the US is already using aHSCT. I have strong interest having developed through to clinical stage biomimetic "stem cells" : they are safe with compelling demonstration of efficacy with partial reversal of established paralysis in mouse models.

The biomimetic simply acts to release a stem cell growth factor called "LIF" : LIF has three functions in MS (i) to switch off the autoimmune attack against myelin; (ii) to repair myelin; (iii) to protect nerves. Delivery is by nanoparticles of FDA-approved gel targeted to lymphocytes that act as a trojan horse to deliver the LIF to sites of inflammation (MS lesions). After 10 days the particles have fully degraded to carbon dioxide and water.

Safety is confirmed (mice) and the particle platform has been through clinical trial, as also LIF, again safe.

So - if "the powers that be" are starting to recognise the value of stem cells - alive or as synthetic biomimetics - your article advances important awareness including the need to treat early. Thank you.

Ed Tobias avatar

Ed Tobias

Thanks for your comments, Su.

There's great interest here in HSCT in the US by people with MS. So, I'll be watching your clinical trials with the hope that they'll prove the safety and efficacy of your biometric stem cells in the patient population.

As for treating early...I've always believed in hitting MS fast and hitting it hard.



Jill Halloran avatar

Jill Halloran

Hi Ed,
I’ve done a lot of research into aHSCT. I am currently scheduled to go overseas for the procedure next year. In my research, I realized that the BEAT-MS trial here in the US is using a myeloablative protocol vs non-myeloablative, which seems to be the more standard offering overseas (and also what Dr Burt offered in his trial). Why do you think they chose this protocol? From my limited understanding, the mortality rate is much higher with myeloablative and it wipes out the entire immune system, even parts that are not effected by auto-immune diseases. Plus, the recovery period is much longer. I’m worried that their decision to use this protocol for the trial will potentially set the US back on acceptance of aHSCT. I would love to hear your thoughts or to most certainly correct me if my understanding is incorrect.

Ed Tobias avatar

Ed Tobias

Thanks for sharing this info, Jill. I wasn't aware that BEAT-MS was following a different protocol and, not being a medical professional, I have no idea why this would be so. I'll check around and see what I can discover.



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