Arthur T Knackerbracket has found the following story:
By obliterating the broken immune systems of patients with severe forms of multiple sclerosis, then sowing fresh, defect-free systems with transplanted stem cells, researchers can thwart the degenerative autoimmune disease—but it comes at a price.
In a small phase II trial of 24 MS patients, the treatment halted or reversed the disease in 70 percent of patients for three years after the transplant. Eight patients saw that improvement last for seven and a half years, researchers report in the Lancet. This means that some of those patients went from being wheelchair-bound to walking and being active again. But to reach that success, many suffered through severe side effects, such as life threatening infections and organ damage from toxicity brought on by the aggressive chemotherapy required to annihilate the body’s immune system. One patient died from complications of the treatment, which represents a four percent fatality rate.
Moreover, while the risks may be worthwhile to some patients with rapidly progressing forms of MS—a small percentage of MS patients—the researchers also caution that the trial was small and did not include a control group.
“Larger clinical trials will be important to confirm these results,” study coauthor Mark Freedman of University of Ottawa said in a statement. “Since this is an aggressive treatment, the potential benefits should be weighed against the risks of serious complications associated with [this stem cell transplant], and this treatment should only be offered in specialist centres experienced both in multiple sclerosis treatment and stem cell therapy, or as part of a clinical trial,” he added.
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Dr. Lowe, from In The Pipeline, wrote Parkinson's As An Autoimmune Disease: More Evidence:
For many complex diseases, you'll find that there are a couple of hypotheses floating around them that are hard to prove and hard to disprove: one is that they're actually caused by some (as yet unrecognized) infectious agent, and the other is that that they're actually an autoimmune/inflammatory disorder. You can also recognize that these two can have features in common, as seen in something like Guillian-Barré syndrome, where a (usually innocuous and often hardly noticed) viral infection or other stimulus can lead to a sudden autoimmune crisis. A whole list of conditions have had such explanations attached to them, more or less persuasively: Alzheimer's, obesity, various forms of arthritis (with little doubt on the autoimmune side), diabetes (Type I, certainly, but even Type II), multiple sclerosis, Parkinson's, and more. Those links lead mainly to autoimmune explanations, but infectious-agent hypotheses are found quite easily as well, and going back many years.
A new paper adds what might be strong evidence to the Parkinson's explanation. It's been known for some time that there's an association between the disease and MHC (major histocompatibility complex) alleles although (at the same time) having another autoimmune disease doesn't seem to raise the risk for Parkinson's itself. That's interesting, in that the brain has mostly been thought of as an "immunoprivileged" compartment, but it's also been increasingly clear that this doctrine is not as solid as it might be.
From the research article:
Approximately 40% of the participants with Parkinson's disease in our cohort exhibited immune responses to α-syn epitopes, and these responses may reflect variations in disease progression or environmental factors. The fraction of patients who display these responses in classic autoimmune disorders such as type-1 diabetes, rheumatoid arthritis and multiple sclerosis is often around 20–50%. As with type-1 diabetes, which features epitopes that are derived from both preproinsulin and additional proteins, it may be that epitopes related to Parkinson's disease are derived from α-syn and additional proteins.
In short, the researchers found that the immune system in patients with Parkinson's disease can recognize the protein associated with it and induce a response that will kill neurons. If Parkinson's disease is autoimmune, then current therapies for other autoimmune diseases may also be relevant for Parkinson's disease.
Research Article: https://www.nature.com/nature/journal/v546/n7660/full/nature22815.html
https://en.wikipedia.org/wiki/Parkinson%27s_disease
https://en.wikipedia.org/wiki/Autoimmune_disease
Previous discussion of Multiple Sclerosis treatment: https://soylentnews.org/article.pl?sid=16/06/13/1038232
Stem cell transplant 'game changer' for MS patients
Doctors say a stem cell transplant could be a "game changer" for many patients with multiple sclerosis. Results from an international trial show that it was able to stop the disease and improve symptoms. It involves wiping out a patient's immune system using cancer drugs and then rebooting it with a stem cell transplant.
Louise Willetts, 36, from Rotherham, is now symptom-free and told me: "It feels like a miracle." A total of 100,000 people in the UK have MS, which attacks nerves in the brain and spinal cord.
There are just a few problems, however: The experimental procedure is under scrutiny from regulators, the experiment's web site may have overstated the effectiveness of the not-yet-proven treatment, and patients have to foot the bill. Oh, and no one has seen the study yet.
[...] The results reported in the BBC piece are just the preliminary findings. And that leaves a number of questions still unanswered — are these results permanent? What are the risks? Who isn't suited to have their immune system wiped out through aggressive chemo?
The U.S. Food and Drug Administration (FDA) has also flagged some serious issues in the study's protocol. If that sounds boring and bureaucratic, think of it this way: for a few months, the lead investigator somehow forgot to report a number of nasty side effects of the treatment, including chest infection and the worsening of conditions as diverse as vertigo, narcolepsy, stuttering, and hyperglycemia, among others.
One thing we know for sure? It's real expensive. The BBC noted it cost patients £30,000 ($42,000) to receive the experimental treatment, but biomedical scientist and science writer Paul Knoepfler, who has been following the trial since last year, says it ran some patients between $100,000 and $200,000.
Related: Low Vitamin-D Genes Linked to Multiple Sclerosis
Scientists Identify Potential Inhibitors of Cancer Metastasis and MS
Risky Stem Cell Treatment Reverses MS in 70% of Patients in Small Study
(Score: 2) by GreatAuntAnesthesia on Monday June 13 2016, @04:24PM
It's great because it looks like real progress to a reliable treatment for Multiple Sclerosis.
However it's also great because it is presented with all the caution and disclaimers that an early, small scale trial of a new potential treatment should be.
Give it a few days though, I'm sure we'll see "Boffins Cure MS! 100% Guaranteed!" headlines soon enough. Shortly followed by "MS cure-hoax run by deathmongers!" from the same publications when their attention is drawn to the "4% fatality" part of the article.
Also, I hope the name of the person who died on the trial is remembered. They may have been looking for a cure for their own sake (or maybe not, I don't know) but they ended up making a sacrifice for science that will almost certainly benefit others. I wonder if news of the fatality will affect patient recruitment for the next round of trials.
(Score: 0) by Anonymous Coward on Monday June 13 2016, @05:08PM
But they have no control group. Somehow they reason that this issue can be dealt with by having a long follow up:
I don't really understand the connection they are making between follow up and control group there. Anyway, these results are pretty much the same as they found reported previously:
http://www.thelancet.com/journals/lancet/article/PIIS0140-6736%2816%2930169-6/abstract [thelancet.com]
So 20-30% of the patients progressed in the literature search and current dataset. This paper is a great example of the scientific surprise two-step:
http://andrewgelman.com/2014/08/01/scientific-surprise-two-step/ [andrewgelman.com]
(Score: 2) by ikanreed on Monday June 13 2016, @05:33PM
People always talk about death-mongering like it's this trivial pursuit.
But have you actually ever gone to a bazzar and tried to sell death? It's a rough market, no demand at all even though so many people need it. And then big government comes in and is like "What exactly are you selling here?" and "You know that assisted suicide is illegal, right?" and "I don't care if the handcuffs are too tight".
(Score: 0) by Anonymous Coward on Monday June 13 2016, @06:46PM
But have you actually ever gone to a bazzar and tried to sell death? It's a rough market, no demand at all even though so many people need it.
http://foreignpolicy.com/2015/03/06/the-death-merchants-do-abu-dhabi/ [foreignpolicy.com]
https://www.irinnews.org/maps-and-graphics/2016/04/27/boom-time-british-arms-exports-saudi-arabia [irinnews.org]
http://www.ipsnews.net/2015/05/u-s-hosts-arms-bazaar-at-white-house-arab-summit/ [ipsnews.net]
http://www.independent.co.uk/voices/the-night-manager-got-it-wrong-about-illegal-weapons-trading-roper-would-be-a-legitimate-arms-dealer-a6944291.html [independent.co.uk]
No demand at all? And it's not all big governments involved. There are small arms companies too.
(Score: 2) by GreatAuntAnesthesia on Monday June 13 2016, @06:55PM
That's why you always bring plenty of cake, then you can offer people a choice: Cake or death.
(Score: 0) by Anonymous Coward on Monday June 13 2016, @07:00PM
There have been other small scale potential treatments too:
http://www.scientificamerican.com/article/could-multiple-sclerosis-begin-in-the-gut/ [scientificamerican.com]
http://www.abc.net.au/catalyst/stories/3572695.htm [abc.net.au]
(Score: 0) by Anonymous Coward on Monday June 13 2016, @11:20PM
http://terrywahls.com/ [terrywahls.com]
http://terrywahls.blogspot.com/ [blogspot.com]
"For four years, secondary progressive multiple sclerosis confined Dr. Terry Wahls to a tilt-recline wheelchair. But by using Functional Medicine to create the Wahls Protocol, Dr. Wahls has transformed her health and body: now she walks easily without a cane and commutes by bicycle. She did this even though, Multiple Sclerosis has no known cure. ... Dr. Wahls began studying the latest research on autoimmune disease and brain biology, and decided to get her vitamins, minerals, antioxidants, and essential fatty acids from the food she ate rather than pills and supplements. Dr. Wahls adopted the nutrient-rich paleo diet, gradually refining and integrating it into a regimen of neuromuscular stimulation. ...
As with heart disease reoccuring after a stent or bypass without changing eating habits, perhaps after this "Cure" the same bad health habits will disable the immune system again?
http://www.diseaseproof.com/archives/cardiovascular-disease-angioplasty-and-stent-placement-is-worthless.html [diseaseproof.com]
(Score: 4, Informative) by Joe on Monday June 13 2016, @04:43PM
Since I know many will not look past TFS, the stem cells from the treatment were hematopoietic stem cells derived from the bone marrow of the patients.
The therapy is basically: collect patient hematopoietic stem cells, suppress the immune system and kill all the T cells, then transplant the hematopoietic stem cells back into the patient. Patients will be immunosuppressed until their hematopoietic stem cells can produce a functioning immune system again.
Another study used a similar strategy, but used a weaker immunoablative therapy and were less selective about how they obtained the hematopoietic stem cells so disease-causing T cells likely contaminated the transplant and caused problems later.
http://www.ncbi.nlm.nih.gov/pubmed/20697363 [nih.gov]
- Joe
(Score: 0) by Anonymous Coward on Monday June 13 2016, @05:25PM
What prevents the stem cells from once again producing bad T-Cells once reinserted?
(Score: 2) by Joe on Monday June 13 2016, @06:17PM
That depends on how the disease developed in the first place:
If the disease was initiated by an infection that "looked" (molecular mimicry) similar to the brain, then the individual would have to be re-infected and have a "bad" T cell (that cross-reacts with brain tissue) respond.
If the autoimmune disease is only the result of defective self tolerance, then the patient will still produce self-reactive T cells (defective central tolerance) or be unable to properly control unwanted inflammation (defective peripheral tolerance). Defective self tolerance would take a while, depending on the patient's luck, to induce disease by itself (or the patients wouldn't have made it to as old as they are).
Multiple sclerosis is an autoimmune disease of the brain, so a breakdown of immune privilege would've occurred at sometime in the patients' history. If immune privilege is maintained, then "bad" T cells either shouldn't get past the blood brain barrier or maintain proper function in the brain.
https://en.wikipedia.org/wiki/Autoimmune_disease#Molecular_mimicry [wikipedia.org]
https://en.wikipedia.org/wiki/Central_tolerance [wikipedia.org]
https://en.wikipedia.org/wiki/Peripheral_tolerance [wikipedia.org]
https://en.wikipedia.org/wiki/Immune_privilege#Central_Nervous_System [wikipedia.org]
- Joe
(Score: 0) by Anonymous Coward on Monday June 13 2016, @06:26PM
Wow, thank you for that.
(Score: 2) by Zinho on Monday June 13 2016, @05:06PM
the researchers also caution that the trial was small and did not include a control group
How exactly do you define a control group for a trial like this? Healthy patients who undergo the procedure without MS symptoms? (risky) MS patients who get the immunoablation and no therapeutic stem cell replacement afterwards? (death sentence) MS patients who go into the clinic and pretend to receive the therapy, but instead do not? (how could you possibly be convincing?)
It seems to me that the only safe, ethical strategy would be to keep some MS patients under observation but untreated for the time of the trial. Am I missing something here? Medical experiment designers, please weigh in and correct my ignorance on this.
"Space Exploration is not endless circles in low earth orbit." -Buzz Aldrin
(Score: 0) by Anonymous Coward on Monday June 13 2016, @05:11PM
Truth is that people with advanced MS don't really have much to lose for trying pretty much anything.
(Score: 2) by VLM on Monday June 13 2016, @05:23PM
some MS patients under observation but untreated for the time of the trial
I was going to snarky reply that's what they do with all of them, but I googled a bit and for better and worse there's been a huge amount of pharmaceutical work done since the 90s. So the problem might be finding someone not already under drug treatment plans #6 or #21 or #53.
There's no reason to assume this treatment wouldn't work as a new step in the existing treatments, so there's that complicated chronological aspect.
So its actually crazier, to make sure the effects aren't just a side effect of treatment #48 you'd have to use recently diagnosed untreated patients.
(Score: 2) by frojack on Monday June 13 2016, @09:00PM
Most medical procedures have natural controls (those not treated), in addition to any controls for statistical purposes.
For a new pill, where is it safe enough (for some values of "enough") to give some a placebo, and others the treatment that is what is done.
But for high risk treatments, as you point out, there is no rational control that can get only part of the treatment, so the controls become those not being treated, and those being treated by conventional means,
But none of them are actually part of the study. They are just statistically matched after the fact from records. All indications are that they didn't even bother to do THAT, because they already know the outcome prognosis of no-treatment, as well as the other existing treatments.
Its not a perfect control, and there probably is no ethical way to have a perfect control in this situation.
Killing off one's immune system and restarting it is done for other diseases. Its not totally new. The risk level has been established by now.
No, you are mistaken. I've always had this sig.
(Score: 0) by Anonymous Coward on Monday June 13 2016, @07:24PM
Reverse Microsoft? That would be great! I'd also like a vaccination against Windows 10.
(Score: 0) by Anonymous Coward on Tuesday June 14 2016, @02:46AM
Fuck off. The illness is no joke.
(Score: 2) by Marand on Tuesday June 14 2016, @03:44AM
Fuck off. The illness is no joke.
So true. Windows 10 has been rapidly infecting users for the past year, it's practically pandemic. I'd say that's pretty damn serious.