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23 Seriously Ill MS Patients Recover After 'Breakthrough' Stem Cell Treatment (telegraph.co.uk)
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.
These were bone marrow stem cells, not embryonic stem cells.
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.
Faster! Faster! Faster would be better!
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.
does it mean it stops Windows 10 from installing?
My ism, it's full of beliefs.
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.
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?
But of course! Hence the name "Multiple" Sclerosis.
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.
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.