aHSCT for MS Patients—The Italian Study & the BEAT-MS Trial

aHSCT for MS Patients—The Italian Study & the BEAT-MS Trial

A recent study led in Italy was organized to investigate the question whether autologous hematopoietic stem cell transplantation (aHSCT) can actually induce durable multiple sclerosis (MS) disease remission in patients with MS by analyzing long-term outcomes post-transplant in a large cohort of MS patients. The Italian-based researchers discovered that of the 210 MS patients actually receiving the aHSCT procedure, that is a stem cell (regenerative) procedure with cells from their own blood, nearly 70% of the total group experienced no degradation in their disability ten years after.  This study suggests that this form of regenerative therapy may introduce longer-term benefits for at least some patients with challenging MS cases.

What is MS?

A neurological disorder triggered by a misguided immune system that attacks the body’s own myelin—that is the protective sheath surrounding nerve fibers located in the spine and brain. Symptoms range from muscle weakness and numbness to challenges with balance, coordination and even walking. The MS Society suggests that approximately 85% of those with MS first have a form associated with relapsing-remitting but most ultimately and unfortunately transition to more dangerous and debilitating progressive forms of the condition.

The Treatment

In this regenerative therapy approach, the MS patient is treated with stem cells produced from their own bone marrow, which happen to represent the fundamental elements of the immune system. With a mission to essentially “reboot” an off kilter immune system during the procedure, the physician and care team actually remove step cells from the MS patients’ blood and in the process they employ chemotherapy drugs to essentially take out the present immune system. This ,of course, comes with its own set of symptoms and issues.

Thereafter, the stem cells produced via autologous process are infused into the MS patient with a goal that their immune system actually rebuilds into one quite different than the one before. The procedure is not trivial, with lengthy hospital stay, sizeable expense and long periods where patients are immunocompromised.  

TrialSite has been communicating with MS patients that see real potential for this approach. With a price tag of about $60,000 in places like Mexico and Russia, patients are mortgaging their homes to get this treatment. People are in desperate condition when the condition degrades, and, in fact, there are stories of people hoping for death for a Christmas gift. A tragic situation, these patients need newfound hope that perhaps an “Operation Warp Speed” for MS could bring.


In this study, 71% of the patients experienced relapsing-remitting MS, a common condition for those suffering from this horrific condition. As reported in WebMD, Bruce Bebo, executive vice-president of research programs for the National Multiple Sclerosis Society, shared “that the actual results of this study for MS patients was “pretty dynamic.”   

Three patients died post the transplant, although no one died past 2007.

Now, according to Bebo, not involved in this study, some issues should be discussed. For example, the patients weren’t participants that investigated stem cell transplants against current standard of care-based MS medication. As those individuals went through their transplants in Italy between 1997 and 2019, the results aren’t apples to apples when asking how does the regenerative therapy compare against medication.

Given the cost and effort associated with the regenerative therapy undertaking, Bebo raises questions about how would MS patients be methodically screened and assessed to determine if they are a good candidate for aHSCT.  Not s small matter, suggests Bebo, as he also conveyed that this procedure cannot be reversed. Medication, on the other hand, can be stopped.

Not Proof

While many MS patients must be excited by the large percentage of patients that sustained well past 10 years, Dr. Alexander Rae-Grant, a neurologist and member of the American Academy of Neurology reported that the study, while compelling, “Doesn’t prove [stem cell transplant]is better than the standard treatments we currently have.” But the longer-term data represents promise. But Dr. Rae-Grant asks the same question—when to pick one procedure over the other?  

Perhaps those patients that are younger yet facing relapsing-remitting MS, facing a degrading condition despite medication—could they be ideal regenerative therapy candidates?


The BEAT-MS Trial (NCT04047628) represents a multi-center prospective rater masked (blinded) randomized controlled trial of 156 participants, comparing the treatment of Autologous Hematopoietic Stem Cell Transplantation (aHSCT) to the treatment of what’s known as “Best Available Therapy’ or “BAT” for treatment-resistant MS. Participants are randomized at a 1 to 1 ration and all participants are followed for 72 months post randomization.

The study started during the end of 2019 and runs through the estimated primary completion date of December 2025. The final study completion is October 2028.

Sponsored by the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH), collaborators include the Immune Tolerance Network (ITN), the Blood and Marrow Transplant Clinical Trials Network, contract research organization PPD and Rho Federal Systems. 

A number of endpoints can be reviewed here

What are the Trial Sites Involved?

In America the following sites are involved (by state or country)

LocationTrial Site CenterPrincipal Investigator(s)
CaliforniaStanford Multiple Sclerosis CenterPrincipal Investigator: Jeffrey Dunn, MD, FAAN
ColoradoRocky Mountain Multiple Sclerosis Center, University of Colorado School of MedicinePrincipal Investigator John R. Corboy, MD
MassachusettsMultiple Sclerosis Center, University of Massachusetts Memorial Medical Center,Principal Investigator: Carolina Lonete, MD, PhD
MinnesotaUniversity of Minnesota Multiple Sclerosis CenterPrincipal Investigator: Flavia Nelson, MDRochester, MN,  Principal Investigator: B. Mark Keegan, MD,FRCPC
MissouriJohn L. Trotter Multiple Sclerosis Center, Washington University School of Medicine in St. Louis; PrincipalJohn L. Trotter Multiple Sclerosis Center, Washington University School of Medicine in St. Louis;Principal Investigator: Gregory Wu, MD, PhDPrincipal Investigator: Gregory Wu, MD, PhD
New YorkBaird Multiple Sclerosis (MS) Center, Kaleida Health, Buffalo, Principal Investigator: Bianca Weinstock-Guttman, PI; Corinne Goldsmith Dickinson Center for Multiple Sclerosis at Mount Sinai, Principal Investigator: Aaron Miller, MD; Rochester Multiple Sclerosis Center, University of RochesterPrincipal Investigator: Corinne Goldsmith Dickinson Center for Multiple Sclerosis at Mount Sinai, PrincipalPI; Corinne Goldsmith Dickinson Center for Multiple Sclerosis at Mount Sinai, PrincipalPrincipal Investigator: Andrew D. Goodman, MD
North CarolinaNeurology, Duke University Medical Center
OhioMellen Center for Multiple Sclerosis Treatment and Research, Cleveland Clinic,Principal Investigator: Jeffrey A. Cohen, MD
OregonMultiple Sclerosis Center, Oregon Health & ScienceUniversity, Principal Investigator: Yadav Vijayshree, MD,MCR,FANA,FAAN
PennsylvaniaPenn Comprehensive MS Center, Hospital of the University of PennsylvaniaPrincipal Investigator: Amit Bar-Or, MD,FRCP,FAAN,FANA
TexasUniversity of Texas Southwestern Medical Center: Division of Multiple Sclerosis and Neuroimmunology; Maxine Mesigner Multiple Sclerosis Comprehensive Care Center, Baylor College of Medicine Medical CenterPrincipal Investigator: George J. Hutton, MD
VirginiaVirginia Commonwealth University Multiple Sclerosis Treatment and Research CenterPrincipal Investigator: Unsong Oh, MD
WashingtonClinical Research Division, Fred Hutchinson Cancer Research Center; Multiple Sclerosis Center, Swedish Neuroscience Institute, Multiple Sclerosis Center at Northwest HospitalPrincipal Investigator: George E. Georges, MDPrincipal Investigator: James D. Bowen, MDPrincipal Investigator: Annette Wundes, MD
United KingdomImperial College Healthcare NHS TrustPrincipal Investigator: Paolo A. Muraro, MD,PhD

Patients Must Be Aware

As mentioned, TrialSite reports that patients seek aHSCT out of the country and end up paying between $50,000 to $75,000 for a treatmemt that can range from $150,000 to $200,000 and payers may not cover the procedure. These procedures cannot be done at clinics that describe themselves as “stem cell clinics,” which tend to be non-regulated operations that at times can have brush-ins with regulators and the law.

Call to Action: Check out the Jan. 2020 edition of Neurology for the source.  TrialSite will start investigating various studies for MS patients and connecting MS patients with research minded, dedicated physicians and evaluating various drug development pipelines. MS patients: if you have had negative experience with a trial or a clinic, please send us the information.


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  1. I am MS patient with PPMS. I would like to see a discovery for remyelination treatment so I can get my life back. Then maybe that will also cure my spasticity, neuropathy, MS Hug, and neurogenic bladder. It would be so nice to be able to walk again for more than a couple of feet without having to sit down. Every time I have an exacerbation, I get a little bit worse, as I am never able to return back to my former baseline, even though I do physical therapy exercises every day. Yes, I do get occasional exacerbations, even though I have PPMS.

  2. Diagnosed with MS 18 years. At that time I was unaware of aHSCT. That is very sad. Such aHSCT could be very effective then and my life and health would have been different now. Unfortunately it’s too late now as I am secundary progressive.
    I really hope it will be possible in 5 years or so to have a new myeline sheet around the axons and the damaged axons will be repaired. That instead of decline of my health it is the opposite: knowing that things will be better in the future, that my body starts improving. There is no need for me to run a marathon, but simple things as a walk through the park together with my wife or take the bicycle for a small tour, that would be really really magnificent.
    A good remyelination therapy, available as soon as possible, that is what I hope for. So please accelerate or fast track promising research in this area.

  3. I have had MS 2001 gone way down hill. My biggest thing is these drug companies are all making the same thing basically. Go 2 steps further…a cure!! Companies honestly don’t want to find a cure. Then they can’t keep charging thousands of dollars for drugs we have to take every month, or 2x a year.

  4. Ok, I have a definite problem with these companies that do trials too. I have been turned down by several trials due to the fact that I cannot walk unassisted. Why wouldn’t these companies want to try trials with people in a wheelchair!! Why?