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23 Seriously Ill MS Patients Recover After 'Breakthrough' Stem Cell Treatment (telegraph.co.uk)

Posted by BeauHD on from the groundbreaking-research dept.

schwit1 quotes a report from The Telegraph: Multiple sclerosis patients who were severely disabled are walking, working and even downhill skiing again following a breakthrough therapy which completely destroys, then rebuilds, the immune system. The trial, which is the first in the world to show complete long-term remission from the debilitating disease has been hailed by experts as "exciting" "unprecedented," and "close to curative." Although it is unclear what causes MS it is thought that the immune system attacks the protective coating which surrounds nerve cells in the brain and spinal cord leading to inflammation, pain, disability and in severe cases, early death. [The new technique, which is a treatment usually used to fight leukemia, involves using chemotherapy to entirely eradicate the damaged immune system, before rebooting it with a transfusion of bone marrow cells. Out of the 24 patients who were given the treatment at least seven years ago, the majority have seen significant improvements. 70 per cent of patients saw a complete stop to the progression of the disease, while 40 per cent saw a reversal in symptoms such as vision loss, muscle weakness and balance loss.] Last week, it was reported that a wheelchair-bound stroke victim was able to walk again after an "unprecedented" stem cell trial at Stanford.

44 of 74 comments (clear)

  1. Implications for other immune system disorders? by Ann+O'Nymous-Coward · · Score: 1

    Wouldn't this possibly also have implications for fighting other disorders where the immune system attacks the body?

    1. Re:Implications for other immune system disorders? by ColdWetDog · · Score: 2

      Yep, all you have to do is risk total immune system, liver and other important organ failure. It's done with leukemia all of the time.

      That's the rub. It's a pretty drastic 'cure'. They picked people who would, in all likelihood die or be greatly debilitated by their disease and tested it on them. The interesting point is that it may be a general reboot of the immune system, good for many immune system diseases (MS, Chron's, ulcerative colitis, Rheumatoid arthritis and yes, Lupus).

      But it's going to be a while before you can do this in your local community hospital.

      --
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    2. Re:Implications for other immune system disorders? by SuricouRaven · · Score: 1

      It might, but the side effects are brutal - the treatment killed one patient and landed another five in intensive care. This is a treatment you'd have to be desperate to try. Perhaps further refinement can make it safer.

    3. Re:Implications for other immune system disorders? by Anonymous Coward · · Score: 3, Informative

      Yes. There have been a few small trials, and occasional single patient case reports, of the use of this treatment for a variety of conditions.

      I saw it used about 15 years ago, for a young person with severe Crohn's disease. They had already had all their large bowel and about half their small bowel removed, and was needing about 2 surgeries a year for complications of the disease. The treatment-related infections were unbelievable (afterall, they had no immune system, and their entire intestine was basically falling apart and letting contents spill into their bloodstream). However, they survived, and the Crohn's disease simply stopped.

      Of course, there was lots of existing damage, and several surgeries were needed to deal with the scarring that developed as all the bowel healed up over the next year or so - but the disease was effectively cured.

      There have been a few experimental treatments for type I diabetes - the idea being that if you can stop the continuing attack on the pancreas islet cells, they may be able to regenerate. It seems to have worked in the few cases where it has been tried. Similarly, there are a number of reports of this treatment for a wide variety of autoimmune conditions: lupus, systemic sclerosis, rheumatoid arthritis.

      What is not really known is how long-term the effects will be. In most cases, the transplant performed is "autologous" - the stem cells are harvested from the patient's own bone marrow and preserved. Then the patient receives high dose chemotherapy possibly with whole body radiotherapy. Then their stem cells are then reimplanted by IV injection. The idea is that a new immune system gets reconstituted from the stem cells.

      There is then a period of several years while the immune system matures (one of the reason why childhood diseases are so different to adult diseases, a child's immune system is not fully developed and is significantly weaker in some ways and has had little time to develop experience). The problem is that the triggers which cause the immune system to incorrectly recognise host tissue as foreign are not well known, although genetics are known to play a part. So, if you are re-implanting the patient's own immune stem cells, is there not a risk that the same disease could recur, because the same unknown trigger sets it off again? Maybe. The number of patients treated is too few, and the duration of follow-up is too short to know with any confidence.

      There is scope for donor bone marrow stem cells to be used (so called allogeneic transplant). However, there are potentially issues with rejection (termed graft versus host disease), and long term immune suppression may be needed to keep this in check. The dose of chemotherapy/radiotherapy needed may also be higher, as any original immune system activity left, can swiftly wipe out the fragile donor cells.

      The other issue is that the high doses of chemotherapy/radiotherapy can have long term side effects. High risk of cancer in future life is expected. Infertility, loss of intellectual performance, nerve damge, kidney damage, liver damage, are all common side effects.

    4. Re:Implications for other immune system disorders? by bill_mcgonigle · · Score: 1, Insightful

      But it's going to be a while before you can do this in your local community hospital.

      Yep, better to die than to take the risk of dying. Current FDA body-count estimate is up to 2 Nazi Holocausts - when people believe they can achieve risk avoidance, they completely fail at risk management.

      --
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    5. Re:Implications for other immune system disorders? by jblues · · Score: 1

      A while back Slashdot reported that the same process was used to cure AIDS. A person with co-existing Leukemia was treated for that and recovered from both conditions.

      --
      If it acquires resources on instantiation like a duck, then its a shared_ptr<Duck>
    6. Re:Implications for other immune system disorders? by sjames · · Score: 2

      I'm not about to claim the FDA is reasonable or even thinking. However, those who are largely unaffected between flare ups may prefer to live with it rather than risk death.

      A big problem is the FDA, composed primarily of people who do not have a chronic condition, wants to decide for patients what risk is too high rather than just making sure the patient knows what the risk actually is and how much benefit they can expect from it.

    7. Re:Implications for other immune system disorders? by sjames · · Score: 1

      Actually, since the patient receives his own harvested marrow, rejection is not one of the risks.

    8. Re:Implications for other immune system disorders? by hackwrench · · Score: 1

      Begging the question. You have said nothing to indicate that it won't quickly progress to one injection and you're done. not saying that's what's going to happen or I'd be begging the question just as much as you are, just illustrating the situation by stating the opposite in the same terms. So I'm just pretending to beg the question.

  2. Before the inevitable comments by ERJ · · Score: 4, Informative

    These were bone marrow stem cells, not embryonic stem cells.

    1. Re:Before the inevitable comments by SuricouRaven · · Score: 3, Insightful

      It's been getting some coverage in the right-wing media, where it's being gleefully spun as proving that embryonic cells are useless and only dumb liberals would waste their money on research with them.

      Everything is politics. The nature of the hyper-partisan nature of US political culture: There is nothing that cannot be spun into supporting one of the two major factions. Sometimes both spin it to their own ends.

    2. Re:Before the inevitable comments by quantaman · · Score: 1

      These were bone marrow stem cells, not embryonic stem cells.

      Where did the bone marrow stem cells come from?

      The treatment was done 13 years ago, I didn't think they were gene editing back then so I assume they come from donors? Does that mean they require immunosuppressant drugs?

      --
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    3. Re:Before the inevitable comments by ShanghaiBill · · Score: 1

      I assume they come from donors? Does that mean they require immunosuppressant drugs?

      Immunosuppressant drugs should not be needed. The patient's original immune system is wiped out. The new immune system is regenerated from stem cells, and even if they are from donors, as the new immune system matures it will learn to recognize the patient's cellular surfaces as "self". So their should be no rejection.

    4. Re:Before the inevitable comments by CrimsonAvenger · · Score: 2

      Where did the bone marrow stem cells come from?

      The treatment was done 13 years ago, I didn't think they were gene editing back then so I assume they come from donors? Does that mean they require immunosuppressant drugs?

      Bone marrow transplant survivor. If it requires immunosuppressant drugs, then there isn't a good enough match, probably. I had a very good match, and George (my new bone marrow) and I have gotten along just fine.

      --

      "I do not agree with what you say, but I will defend to the death your right to say it"
    5. Re:Before the inevitable comments by chispito · · Score: 1

      Which part of everything did you fail to understand?

      The part where he provided a source for the fringe, inconsequential commentary he repeated.

      --
      The Daddy casts sleep on the Baby. The Baby resists!
    6. Re:Before the inevitable comments by rgmoore · · Score: 1

      The treatment was done 13 years ago, I didn't think they were gene editing back then so I assume they come from donors? Does that mean they require immunosuppressant drugs?

      They do come from donors, and immunosuppressive drugs are not required. Transplants of tissue from living donors like bone marrow is very different from tissue from deceased donors like hearts and lungs. With transplants from deceased donors, the pool of donors is small and there's very little time to choose a recipient before the organ goes bad. In practice, that means it isn't always a very good tissue match, and it's usually necessary to give the recipient immunosuppressive drugs to avoid rejection.

      With a transplant from a live donor, the pool of donors is larger- much larger in the case of hematopoetic stem cells or bone marrow, which grow back completely- and the tissue will keep indefinitely. That gives doctors plenty of time to search for the closest possible tissue type match, so the recipient and donor are generally perfect or nearly perfect matches. They won't even try to do the transplant unless there's a very close match.

      --

      There's no point in questioning authority if you aren't going to listen to the answers.

    7. Re:Before the inevitable comments by MichaelSmith · · Score: 1

      My father in law died when chemotherapy for cancer killed his bone marrow, and thus, his immune system. Maybe it would be a good idea to take samples of marrow stem cells before doing something which could kill the marrow entirely, so that the marrow could be re-booted, so to speak in the case of disaster.

      --
      http://michaelsmith.id.au
    8. Re: Before the inevitable comments by jmac_the_man · · Score: 1

      Yeah yeah. Republicans hate us and want us to die. We know, dude.

    9. Re:Before the inevitable comments by SuricouRaven · · Score: 2

      Here's one: http://onenewsnow.com/science-...
      ---
      OneNewsNow asked why adult stem cell successes are not generally known. According to Prentice, it's partly because the emphasis has been on human embryo research.

      “We kept hearing how embryonic stem cells were going to cure, as one person put it, 'all known maladies,'” he says. “Embryonic stem cells haven't helped a single person - and in decades of research they haven't helped that many laboratory mice. Usually they just grow and make tumors."
      ---
      ONN is very right-leaning, but it's not really fringe. It's run by the AFA, a quite large and influential organisation.

  3. Let's hope it works by NotInHere · · Score: 1

    Let's hope it works, as for the usual stem cell treatments this is as helpful as the treatment itself.

  4. Re:23/24 improved. Number 24 died. by techno-vampire · · Score: 1

    My sister was diagnosed with MS over fifteen years ago, so I know more about it than most people need to. When you have an episode, various functions degrade, such as vision and so-on. When you go into remission, some, but not all of the functions return, meaning that you're on a gradual downward slope. Judging from what was written, not all of the patients who experienced a stop in the progression got any function back.

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  5. Re:23/24 improved. Number 24 died. by Anonymous Coward · · Score: 1

    I have MS, while my condition is nowhere near as serious as the people in the study (I'll never be able to ride a bike, but I can usually walk fine without a cane on level ground, sometimes even in a straight line), if someone came to me and said that there's a ~4% chance I'd die, but a ~35% chance my mental deterioration can be reversed (per the study 35% experienced an improvement) and ~60% chance I would stop getting worse, I would hop onboard pretty damn quick.

    Before I jump on this, though, I'd want to know more about the side effects other than risk of death. Getting better then dying of a cold the week after would be some serious flowers for algernon shit.

  6. If it cures MS by MrKaos · · Score: 5, Funny

    does it mean it stops Windows 10 from installing?

    --
    My ism, it's full of beliefs.
    1. Re:If it cures MS by vux984 · · Score: 2

      I came here to confirm someone would have posted this.
      You did not disappoint.

    2. Re:If it cures MS by MrKaos · · Score: 1, Funny

      I tried to resist - honestly I did!

      On a more serious note, I do have some vicarious experiences of friends who have suffered this disease and I hope that this turns out to be a cure for them and their Windows 10 problems. shit, I did it again, help me!!

      --
      My ism, it's full of beliefs.
  7. Multiple sclerosis by Livius · · Score: 1

    So it's not about computers that have had Microsoft malware forced on them.

  8. Re:23/24 improved. Number 24 died. by wickerprints · · Score: 1

    Stopping disease progression in MS does not automatically mean that a patient regains some amount of functioning that was lost to the disease. It simply means that the disease does not get worse, not that the patient is healed of the damage. Some cases of MS do not have acute phases with subsequent remission at all; and of those that do exhibit remission, sometimes it is not complete.

    Therefore, one should not expect those percentages to add, since some patients would be counted in multiple categories.

  9. Re:23/24 improved. Number 24 died. by Guy+Harris · · Score: 1

    "And 70 per cent of patients saw a complete stop to the progression of the disease, while 40 per cent saw a reversal in symptoms such as vision loss, muscle weakness and balance loss."

    Also, 70 + 40 = 110, WTF?

    An article in the Toronto Star about this indicates that, of the 24 patients, "seven saw their symptoms deteriorate even after the transplant", although "this progression levelled off after about two years, Freedman said." (24-7/24)*100 ~= 70, so 70 percent apparently had no progression of symptoms after the treatment.

    As for the 40%, presumably 30% saw a stop in the progression of the disease but no reversal of symptoms, 40% saw a reversal, and 30% saw no immediate stop although it appears that the progression at least leveled off after 2 years.

  10. Common... by jopsen · · Score: 2

    Yep, better to die than to take the risk of dying...

    Many immune deceases Chron's, UC, etc. aren't likely going to kill you. Full experimental immune system reboot might be a bit risky for those patients :)

    - when people believe they can achieve risk avoidance, they completely fail at risk management

    That argument goes both ways... take the question of whether or not colonoscopy is useful for screening cancer.

    Avoiding or seeking to minimize risk is not easy... Please tell me what level of risk one should accept for diagnosis and treatment of annoying but largely manageable deceases like Chron's, UC, etc. ?
    1/300 for major complication and possible disability? Or 1/10 ? or 1/10000 ?

    It's super hard as a patient to know whether or not you should let a doctor do a procedure. I largely figure they aren't affected by financial incentives, yet, doctors can't help but be affected by the mindset of their specialization.

    1. Re:Common... by ShanghaiBill · · Score: 4, Insightful

      Please tell me what level of risk one should accept for diagnosis and treatment of annoying but largely manageable deceases like Chron's, UC, etc?

      It should be the level of risk acceptable to the informed and consenting individual patient, not the level of risk acceptable to a government bureaucrat. Many of these diseases may not be directly fatal, but they can destroy a person's quality of life to the point they feel their life is not worth living, so they may be willing to take a substantial risk for a cure. Nobody should have the right to veto that decision.

    2. Re:Common... by Anonymous Coward · · Score: 1

      Please tell me what level of risk one should accept for diagnosis and treatment of annoying but largely manageable deceases like Chron's, UC, etc?

      It should be the level of risk acceptable to the informed and consenting individual patient, not the level of risk acceptable to a government bureaucrat. Many of these diseases may not be directly fatal, but they can destroy a person's quality of life to the point they feel their life is not worth living, so they may be willing to take a substantial risk for a cure. Nobody should have the right to veto that decision.

      THIS THIS a billion times! THIS!

      If something like this could cure my top 1 diabetes and I would never have to listen to another asshole trying to tell me I got type one from eating at mc donalds too much or eating too much candy as a kid... yes it would be worth risking dying!

    3. Re:Common... by ArylAkamov · · Score: 1

      That argument goes both ways... take the question of whether or not colonoscopy is useful for screening cancer.

      It works great when they fuck up and perforate your colon, leaving you in the ICU for two months, then booting you out with a bill just shy of one million dollars.

    4. Re:Common... by hackwrench · · Score: 1

      Whenever someone trots out the "informed" pony, I think along the lines of, in cases like this: To be really informed is to know enough to be able to perform the operation yourself AND insure a positive outcome. I don't care how the best outcome is obtained as long as it is the best outcome available. Even the judgement of someone who aced statistical analysis might be preferable.

  11. Weird math by NotQuiteReal · · Score: 1

    Why say things like "70 percent" and "40 percent" for such a small group (of 24).

    Are we to believe 16.8 of the 24 had their symptoms stop and 9.6 had reversals?

    --
    This issue is a bit more complicated than you think.
    1. Re:Weird math by M0j0_j0j0 · · Score: 2

      But of course! Hence the name "Multiple" Sclerosis.

  12. Organ transplants? by Applehu+Akbar · · Score: 3, Interesting

    Organ transplant patients have to spend the rest of their lives gobbling immunosuppressive drugs. Could this technique be used to remove and reboot the immune system, including in the replacement organ, for such patients?

  13. Re:Wait, wait! by Anonymous Coward · · Score: 1

    Nice strawman, pretty much no Republican has been against stem cell research itself. The opposition is source of embryonic stem cells, a la aborted fetuses, which as mentioned above, is not the type of stem cells used in this treatment.

  14. Stephen Hawking by Anonymous Coward · · Score: 1

    what about Stephen Hawking

  15. Not in the blurb by Anonymous Coward · · Score: 1

    1) Embryonic stem cells are never used anymore, these trials were conducted by harvesting bone marrow from each patient and growing their own stem cells from that. 2) The Ottawa researchers stated that this treatment was useful for about 15% of patients. Other factors included age of patient and length of time they had the disease: older patients were selected out, and patients who had the disease for decades (not just 1 or 2 years) would not have benefited from this because the long term damage of the disease is not repairable. You can stop the disease at any time (and that's what this does), but there is no going back from long term damage. Also in the blurb it said something about Stanford. This trial was not done in the US, it was done in Ottawa, Canada.

  16. Probably not by Okian+Warrior · · Score: 2

    Organ transplant patients have to spend the rest of their lives gobbling immunosuppressive drugs. Could this technique be used to remove and reboot the immune system, including in the replacement organ, for such patients?

    I believe the answer to your question is "no", although I'm not a biologist.

    Cells have a specific molecule, the "major histocompatibility complex", which lives in the cell wall and presents bits of broken-down proteins to the outside. The immune system checks these proteins to determine the health of the cell and to determine whether a response is needed.

    Each MHC complex also has its own particular code, and the immune system of the body is trained to recognize these codes as "self" and not trigger a response to the MHC itself. This happens in the thymus, where developing T-cells are culled if they show any response to the "self" MHC codes. There are about 16 total code variables per MHC molecule, and some individual variables can have 600 or so variations. If two people are not identical twins, then their different MHC proteins would trigger an autoimmune response.

    If you remove and reboot the immune system, the thymus will still be making cells that expect the "self" MHC codes, and will show a response to foreign cells. Most of the cells in the body would present "self" MHC molecules, and the transplants would still present a "non-self" molecule.

    Unless you take some type of further step, such as adjusting the Thymus or cleansing the transplanted organ of MHC molecules, you'll still get a response when you reboot the immune system.

    1. Re:Probably not by AmiMoJo · · Score: 1

      Thanks for that really informative post. May I trouble you with another question?

      How about auto immune diseases like Reiter's Syndrome / Lupus, and how about Chronic Fatigue Syndrome? I'd be willing to try almost anything to improve these conditions, but there isn't much research or many trials.

      --
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  17. Use measles to reboot the immune system? by TDDPirate · · Score: 1

    Another possible way to reboot the immune system is to infect the patient with measles.
    One of the effects of this disease is to erase all information accumulated by the immune system and force it to relearn how to make antibodies for everything.
    Hopefully, it won't learn how to attack its host's body.

    I wrote a blog article about the need to treat information embodied in the patient's body: http://www.zak.co.il/tddpirate...

  18. Dear reader ain't gettin' any younger, you know by Impy+the+Impiuos+Imp · · Score: 1

    > unprecedented stem cell MS cure
    > unprecedented stem cell paralysis cure

    Unprecedented penis growth and functionality cure?

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  19. Heh heh by wkwilley2 · · Score: 1

    "Why are we not funding this?" - Peter Griffin

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