Adoption of bone marrow transplantation as a MS treatment is diffusing widely. #MSBlog #MSResearch
“My recent post on bone marrow transplantation as a treatment for MS generated a large amount of discussion. You may find the following Brazilian guidelines of the use of BMT in MSers of interest. The Brazilians are not alone in their adoption of BMT as a treatment for MS; several centres in Europe, Canada and the USA offer BMT as a treatment for MS.”
The consensus on hematopoietic stem cell transplantation (HSCT) in autoimmune diseases provides an indication of HSCT in patients with progressive MS unresponsive to conventional therapy and EDSS between 3.0 and 6.0. The forms of the disease that might benefit from transplantation are: relapsing-remitting, primary or secondary progressive, and the “malignant” form, provided there is evidence of inflammatory activity at the time of transplant indication. The patients should be transplanted preferably in centers with experience in this procedure. Table 1 summarizes the recommendations (modified below).
“The following is an update on the survey I ran regarding whether or not MSers would be prepared to take the risk of having a BMT. Could these results be correct? Over 50% of MSers are prepared to take the risks of BMT. Only 28% have said no. If these result are correct I have seriously underestimated the risk MSers are prepared to take in the hope of a potential cure for their disease.”
“Maybe I need to survey neurologists about the same issue? At a personal level I think BMT is too risky as a treatment for MS. In the light of these results, maybe I should reconsider my position?”
“Maybe I need to survey neurologists about the same issue? At a personal level I think BMT is too risky as a treatment for MS. In the light of these results, maybe I should reconsider my position?”
“Please have your say!”
Dr G. Many MS'ers are prepared to take risks with drugs such as Tysabri – which on this blog you have said has a risk factor of 1 in 1311 of contracting PML. Someone contracting PML is likely to suffer serious disability or die. Also Tysabri does not offer a cure, and it's effects on progressive MS are limited or yet unknown. Yet it is used more and more frequently as MS'ers weigh up the risk vs benefit.I have also seen in this blog that you agree the risks of HSCT are less than 1% death at reputable hospitals, and has around a 80% success rate for halting MS. (I'm surprised you use the word 'cure', I think halt or put into remission would be more appropriate.) Why is the neurology community still surprised that the approach of 'keeping someone alive and slowing down disability' vs a higher risk for 'complete remission' is the preferred option? The next step is for neurologists to start seriously considering this treatment in the UK, and considering it early on in the disease cycle to give people the best chance of nipping this disease in bud. Before someone mentions Alemtuzumab, again what is the effect on long progressive disease? where as HSCT has (but lower success rate) been shown to halt the progression of SPMS and PPMS.
Prof G,I don't think it's about the risk some MSers are prepared to take. The issue is how soul destroying MS is. It totally transforms your life and the losses are immeasurable. The fact that it gets worse and worse puts it in a league with only a few other diseases. Whatever you guys say, this disease kills in the end and it's a grim death. The chance of a treatment that might stop the disease is something many of us would grab. I'm not a risk-taker by nature, but am not prepared to watch myself be eaten away by this disease and the impact on my family. BMT may bring the risk of death, but in reality the life I loved died a decade ago with the diagnosis of MS. The oath doctors take should not be 'do no harm' but 'do not let suffer'. MS is a disease which causes suffering ( physically and emotionally and we should a choice of treatments,including high risk treatments.
Prof G would you class yourself as a BMT laggard then?
If I had highly aggressive disease I would take the chance with HSCT over all other treatments if it was available and being performed at a reputable facility.
1% risk of death is fine by me. But the problem is it is impossible to know whether the mortality is actually 0.5-1% or 20% in the centre you are referring to at least in the country I'm living (HSCT is available here as long as you can afford it).
MSers are desperate people willing to subject themselves to all manner of rubbish just to keep hope alive. You can go to a young adult oncology clinic to see what length they'll go to just to survive. And why not? They've been dealt a very cruel set of cards by life.But where does it stop, Prof G? MS remains incurable. These new DMTs are not the certifiable cure you're making them out to be; well, at the moment it's too soon to be sure.You cannot cure MS when you don't know why it's happening. Lemtrada is not an official cure. This is something you very much skirt around, I think.
Are there any centres in the UK offering BMT for people with MS?
I think when people talk about HSCT/BMT they need to look at the specifics rather than pidgeon holing it all together. Autologous non-myeloablative cyclophosphamide HSCT or myeloablative BEAM which are both very different chemo regiments that carry different risks.The question that needs to be asked to neurologists is chemo offering a chance of remission vs life long drugs that will only slow down progression. The fact that people in the UK are prepared to pay thousands of pounds to travel to Russia/America/India/Germany to get this treatment, why don't the neurologists/haematologists/oncologists in the UK review the data and offer this treatment and let the patients decide?
People have already started doing HSCT, but as you say most of the time the patient has to pay for it themselves.http://www.ms-uk.org/HSCTOne patient (Stella) from the above link had her procedure done in London. Unfortunately for her she lost some of her toes. This goes to show that the facility is of the upmost importance.Chicago (Northwestern University) is in a Phase 3 trial now. They have had no complications or deaths and if it is approved would be a wonderful option for those in the U.S. Britain seems to be a laggert. http://clinicaltrials.gov/ct2/show/NCT00273364
Stella had the procedure done in Nottingham. The facility is top quality, but there is no way that the medical staff can predict in advance who will and who won't get a life threatening infection, even with isolation units and careful nursing. The strong chemo that is needed to ablate your immune system also weakens your gastro intestinal tract, allowing bacteria in that wouldn't normally be a problem, but with no immune system, can set of infections. The transplant would need to be done in the haemo oncology unit- the neurology dept. just isn't geared up for it. Not every town in the country has a unit that can do a HSCT- they may have a haematology dept, but that doesn't mean they can do a transplant. For a start, you need an apherisis machine to harvest the stem cells. Blood cancer patients sometimes have to travel 100's of miles for their transplants, so MS patients would have to join them in the queue for the beds in the transplant unit
Prof G, are you a pro-medicine type of guy in general? For example, if you have cold to you rush to the medicine cabinet for some Night Nurse or do you sweat it out holistically? I'd love to know.
He is definitely the pro-medicine type. And the type that gets man flu. https://www.youtube.com/watch?v=VbmbMSrsZVQ
Then gets on plane to Australia and becomes a biological weapon:-)
Short answer, yes, you should imho reconsider. You would get fewer suicides if people felt they had more options and a little more hope. You've reported the odds of remaining mobile into old age. My biggest fear is lying helplessly conscious in a nursing home.
Any comment from Prof G or Mouse doctor? This is a hot topic for many MS'ers and their insight would be interesting.
I'm at the CIS stage so would be premature but it is something I will do if I go on to develop MS. Its something I've researched into and weighed up the cost and risk. My discussions with my neuro about this have sadly been met with scorn. I do think neuro's underestimate the psychological impact of living with a disease such as MS. If having HSCT would likely halt progression and give me back my future I would do it in a heartbeat fully knowing I would be risking my life in the process. The risk after all is small and the potential gain is great.The medical profession needs to wake up and actually listen to what people with MS are saying, or should that be screaming.
Unfortunately it appears that this does not necessarily stop or prevent progression. Know a girl who underwent HSCT ten years ago and pretty early in the course of her disease, and she went 9 years very well, nearly forgotten about her MS, but succumbed into the progression later. :(But I agree that HSCT looks like only possibility for a cure available and worth the risk.
Perhaps for these more aggressive treatments to have a chance the diagnosis has to be made as early as possible. Seems that there are still too many unknowns.Here in the UK a lot of people with a barn door CIS will often get relatively little by way of investigation, told little about what the diagnosis could be and are left to re-present when they have another clinical episode. Strikes me as a way to miss a lot of potential damage. I've no doubt this country generally remains quite backward when it comes to diagnosis and treatment. When Brazil has a more pro-active outlook on the disease you know it probably not being treated in a world class way in the UK by a long stretch. It would seem that treatment here isn't even average by western standards.Out of interest, did the person you know have very aggressive disease to start off with, given that she had HSCT?
Yes, she had a severe MS from the beginning. With many relapses during the first year, so she decided to go on HSCT and then felt well for many years until began to deteriorate a year or so ago. Don't know how much time were wasted between the onset and HSCT, can ask by a chance. But don't think this was more then 2 years.
I don't know what did you mean by more proactive look and don't know how the health system works in the Brazil either, but I suppose that like in Russia, mentioned above, you are almost all alone with your disease there. Unless you got plenty of cash. I don't think that things differs much between developing countries. And in the UK you at least have the access to highly effective treatments (Gilenia, Tysabri) through a insurance system.