HSCT makes the recommended list


Barts-MS rose-tinted-odometer: ★★★★★

Good news for people with MS living in the US. The National Multiple Sclerosis Society is acknowledging that autologous hematopoietic stem cell transplant (AHSCT) is an effective treatment for MS as is recommending  AHSCT a useful treatment option for pwMS who have substantial breakthrough disease activity despite treatment with high-efficacy DMTs or have contraindications to high-efficacy disease-modifying therapies. The acknowledge that pwMS younger than 50 years with shorter durations of disease (<10 years) have the most to gain from AHSCT. 

The big question is will insurers and national funders pay for HSCT in the US based on this recommendation or will they still need FDA approval? 

The good news for pwMS living in the UK is that the NHS already covers the cost of HSCT and MS is on the list of approved autoimmune diseases for treatment with HSCT. The problem in the UK is not necessarily the access to the treatment, but to get risk-averse neurologists to refer pwMS for the procedure or am I wrong?

Miller et al. Autologous Hematopoietic Stem Cell Transplant in Multiple Sclerosis Recommendations of the National Multiple Sclerosis Society. JAMA Neurol. Published online October 26, 2020. doi:10.1001/jamaneurol.2020.4025

Importance:  Autologous hematopoietic stem cell transplant (AHSCT) for multiple sclerosis has gained increasing interest in recent years. Despite the availability of many US Food and Drug Administration–approved disease-modifying therapies, some patients do not respond adequately and others may have very early aggressive disease that prompts consideration of alternative, highly effective, long-lasting therapy. The National Medical Advisory Committee of the National Multiple Sclerosis Society has reviewed recent literature on AHSCT for the purpose of making recommendations about its use based on current knowledge, as well as pointing out areas of controversy and issues requiring further research.

Observations:  Studies on AHSCT have repeatedly demonstrated high efficacy and a durable outcome in people with relapsing multiple sclerosis. Recent studies have shown considerable improvement in the safety of the procedure, with much lower mortality rates than were reported earlier. Consensus is emerging about the characteristics of the best candidates for the procedure. Questions remain about the ideal protocol, particularly about the best conditioning regimen to be used to kill immune cells. Larger randomized clinical trials are needed to address the question of whether AHSCT has advantages over the most efficacious disease-modifying agents currently available. One such trial (Best Available Therapy Versus Autologous Hematopoietic Stem Cell Transplant for Multiple Sclerosis [BEAT-MS) is currently in progress.

Conclusions and Relevance:  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. The best candidates are likely people younger than 50 years with shorter durations of disease (<10 years). The procedure should only be performed at centers with substantial experience and expertise. Ideally, recipients of the procedure should be entered into a single database, and further research is needed to establish ideal cell mobilization and immune-conditioning regimens.

CoI: multiple

Twitter: @gavinGiovannoni  Medium: @gavin_24211

About the author

Prof G

Professor of Neurology, Barts & The London. MS & Preventive Neurology thinker, blogger, runner, vegetable gardener, husband, father, cook and wine & food lover.


  • Hey Prof G,

    What percentage of people continue to progress following HSCT?

    Also, any idea why the post HSCT might be more aggressive?

  • MS docs must surely rank among the most conservative in their Rx approach. I could be more blunt.

    1 Neuroaxonal loss begins early. This much we know. We also know that cortical loss begins EARLY based on 7T data and so forth. And yet the JAMA article talks about an ideal candidate for AHSCT being < 10 years (what is the rationale for someone WC bound in 6 months if the 10 yr rule is applied, is beyond me) in disease duration and having 'aggressive' disease. We define aggressive disease – well, everyone has their own definition. Relapses usually figure in the lexicon – but we ALSO know that the tip of the iceberg is probably cortical disease. So, why the delay in Rx initiation ? And spinal cord disease might have to be classified differently since the real estate that needs to be affected is pretty limited. What of cord disease ? Do those unfortunate patient cohort get AHCST as well ?

    2 We also know that in acute MS lesions, we may lose up to 11 K axons (NEJM article by Trapp et al., 1988) and yet we wait for 'aggressive MS' model to show up. We have MRI volumetric data that we rarely, if ever, apply to approach Rx options. Why is that ? The volumetric data is the closest to perfection, if applied cleverly but we rely on lesion size, volume, and other descriptors made by human eyes. Why ? The algorithms don't work ?

    3 We are like firefighters waiting patiently next door, for the house to burn down. And then we move in for the kill.

    4 In other words, publications and garnering public interest and Altmetric scores is terrific for getting grants but what of the poor soul who is 16 and is crawling on all fours ?

  • I don’t understand why HSCT is not offered as an option to all patients. I am still RRMS and my ms is slowly progressing. Since I’m young and have relatively low disease burden, why not do the hsct now when the odds are in my favor. Why waste brain on dmts with tones of side effects while at best slowing ms and at worst doing nothing.

    Could you explain this? Is hsct not effective in people with lower disease activity? What’s the rationale?

  • “The problem in the UK is not necessarily the access to the treatment, but to get risk-averse neurologists to refer pwMS for the procedure or am I wrong?”

    Based on the advice on the MS Trust website, i would say this is wrong

    “However, treatment is becoming available through the NHS at a very small number of centres, but the number of people who are accepted for treatment is extremely small.”

    Prof G – can you share your real world experience based on the number of referrals / acceptance from patients treated by members of the Barts team? The Trust seems to suggest that HSCT is only a viable treatment option for aggressive forms of MS or following failure on tysabri / lemtrada. Chances are by the time you have reached and then failed on one of those drugs, you have already sustained a reasonable degree of damage

    • Their are criteria attached to (internal) re-embursement and this means that other options are typically examined first

  • Im normally quiet impressed with this blog but this is a very dissapointing post. Are you really that blind to the issues around hsct ? The problems are unreal when trying to get better treatment in the uk.

    1. Finding a neurologist that even knows it exists you then have to convince them that it is available on the nhs! My previous neuro thought it was only available on trials and he used to work at barts !
    2. Once you find a neuro that knows its available which will either take you months and months on the nhs refferal system or hundreads of pounds and months privately you then have to convince them to refer you for it!
    3 once you have convinced your neuro that you should be treated with it you then have months and months of waiting for another panel of neuros that have likely never met you to decide if you are worthy of having the treatment even if you meet the requirements set by NICE! i know right crazy thought isnt it that all the while we are waiting to see if somehow by some miracle we qualify for life changing treatment and a loads of diffrent doctors and proffesors decide that we are that we will still have months and months of waiting after they decide that we are by which point some of us will go so far downhill that its less likely to be effective anyway ! Its a joke ! An absolute joke! Do something about it ? make a change you have asked this question so many times i remember reading a post from a questionaire asked a couple years back asking almost this exact same question i dont understand how this is still a question and nothing has been done to make it nore widely available! Wake up people from the uk are going abroad to get this treatment due to the sheer waiting time to get treatment with it here in the uk which we have a right too but somehow still get denied ! Otherwise what is the point in paying tax and national insurance ? Its fine saying its all paid for in the uk or its free but realisticly its the carrott being dangled ! Its the promise never followed through in the uk. Im 29 i was diagnosed 7 months ago by a careless neurologist that done nothing but throw me on the scrap heap. He left me with no hope for my future and suicidal crying my eyes out in a hospital bed completely alone surrounded by people with active covid even though i had just recovered from it. I didnt take in what he said in the 30 seconds he spent telling me that i have ms when the ward nurses asked him to see me before i left hospital after three days of steroids guess what his answer was ? No he wouldnt when i had my phone follow up app guess what he spoke over me didnt answer any questions and wouldnt give me a fixed relapsing or progressive diagnosis and wouldnt discuss treatment even though after leaving hospital i had continued to worsen to the point i had to give up my dream job ! How the hell are we supposed to get better treatment when some of us have to battle to even get heard from ? I have had to pay out of my savings to see a neuro that actually understands the disease because i was and am still going downhill so quickly. Ive now been diagnosed for 7 months and have yet to start or be offered treatment in that time ive gone from numb to achey to weak to the point i struggle to walk around the house i wonder how bad i would get if i had to wait to be reffered for hsct ? The problem in the uk is not just the neuro willing to refer its the waiting time to even get to the neuro that will !

  • The rationale I believe is the risk-versus effectiveness trade-off. You are young and doing relatively well as you said. Why shall you risk it to do HSCT with a 1% chance of mortality? If statistics is our guidance, 1 in every 100 people die from complications. If there are 2 million with people living with MS and all undertake HSCT that means around 20 000 deaths. MS is a disabling disease but not fatal so only those that life is significantly affected in the short run should take this risk

    • hi nic i dont know if you were replying to my comment above but i am definitely not doing ‘well’ i have had to give up a dream job and have worsened to the point where walking around the house is hard. if 1 in 100 die from complications of hsct i wonder how many on the course my disease is taking will die due to ms complications ? how were your 30s? because it looks likely i wont be able to walk in mine. in no world should that risk be anyones to decide on other than the person suffering this awful disease if i want to be administered treatment with a 1 in 100 chance of killing me or a much higher chance of stopping me from becoming more unwell that should be my decision not someone who can go home and forget i exist.

      • Hi Anon, have you considered Alemtuzumab? Its just as good as HSCT and much safer. I too progressed very quickly after diagnosis and found walking from living room to kitchen tiring. But 2 years post alemtuzumab can walk 5 to 6 km if pushed! L.O.L. I also opted to see a private neuro to avoid the 3 month wait on NHS. Who straight away took me into his NHS clinic and put me 2 weeks after on tecfidera. After 8 months I had a lession on my MR and no fuss offered me HSCT or Alemtuzumab. He was wise enough to know b therapies are not the full solution. I don’t want to mention names of people without their consent but London ms clinics tend to be the best. Or you can always book a private appointment with Prof g? I think you should knock HSCT on the head and go for intermittent treatment like Ocrelizumab. Then take HSCT when available. Also London Bridge offer the treatment privately but costs £80000. But definitely don’t wait around! Any treatment is better than no treatment.

    • Where are you getting that statistic from? My understanding was globally it is more like 1 in 250, still a risk no doubt, and not one to be taken lightly. But the inset on any of the DMDs do not exactly make for an ‘easy read’. The risk v’s benefit is a very case specific discussion.

  • Unfortunately, as with most things on the NHS it appears we are geared towards crisis response and very little in the way of preventative medicine. The sad reality I have found to be true is money talks, after 8 years of dealing with ‘carpal tunnel’ that never went away, and suddenly got worse, I went private and paid for apt with consultant neuro and x2 mri’s and ms was confirmed. I was referred back into the nhs to discuss drug options. It’s unfair I was able to ‘jump’ the queue because I had paid. Still I waited months for my apt, with no input from ms nurses/ anyone. I read everything I could find and had weighed pro/con and decided that the risk was my own to take and I wanted to pursue ahsct as firstline response. I met criteria where in evidence suggests I would benefit most from it, I had active liaisons,thirties, and felt things moving fast. My prognosis in accordance with some of the advise on this blog would not be good, spinal liaisons, lingering symptoms, bowel and bladder issues previously put down to pregnancy/labour. I was also JC++++

    My nuero initially misquoted the mortality rate and threatened ‘coming home in a box’, but later agreed ahsct could well be the future, he advised that he had referred 6 ppl to London MDT and none were approved. For myself tysabri was not an option, ocrevus had a 6+ month wait list and at that time lemtrada was suspended for safety concerns. Yet I couldn’t be referred to MDT as I had not tried and failed the drugs that I could not actually access in a timely way. I paid (again) to be referred privately to MDT and saw London based nuero. Again more muddled thinking, I encountered a neurologist who told me I should be more afraid of death via hsct, than cancer risk with ocrevus-London mortality rate quoted. The haematologist, was firmly of view that ahsct could be used as a first line response in certain cases, though privately and not on the nhs. Back, forwards, money wasted, TIME wasted… I took a cancellation abroad and am now 9 months post. But because I went abroad I have not been able to access haematology 🙈 the one I saw I paid for off course… they system is broke. The ppl approved at the MDT at which my case was discussed are still waiting to have any date for any movement, covid has exacerbated this brought it to a standstill.
    I am not a hsct zealot, the treatment is tough as is recovery, and we don’t know what we don’t know about longer term implications, it is a decision that needs to be taken after much consideration and dependent on individual circumstance, but in this country us brainless ms’ers don’t seem to be afforded any autonomy in this regard.

  • I should add that 6months after diagnosis and 2 weeks before I went abroad for this treatment I received a letter advising that the funding panel I my locality had agreed I would be approved for high efficacy drugs, goodness knows how long from being approved to actually starting a drug would have taken🧐

  • Pharma is not going to be pleased. I’ll wager that they will round up the old guard to raise the red flag warning of the danger regarding aHSCT.

  • I think this is a great step in a positive direction, but I would want it to be an available option to anyone with MS who is informed of the risks (even first line). The cost savings is clear, as are the rough risk vs. reward. Let patients make the choice. I went abroad for HSCT first-line and just got back last week (USA). My Neuro was generally supportive, as was my hematologist, but he (neuro) agreed there was no way I could get HSCT done first line in the US, not without an exceptionally bad case of MS (let alone get insurance coverage). My hematologist said the only thing holding him back from doing more HSCT for MS are referrals from neuros. This makes me wish I could have gotten that referral and been covered in the US. Maybe for patients in the future.

    Thanks to this blog, I had a good understanding of the potential risks that even if Ocrevus or Mavenclad “worked” and I was NEDA, it was possible (and more likely) to have accelerated BVL. I explained this perspective and my Neuro understood – I am excited to continue follow up and am hoping for the best, and hope I may be a case study to sway local decision making in my healthcare system. Thank you to everyone at the Barts MS team for putting this information out there, it has been supremely helpful.

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