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Author Topic: Als craze  (Read 133 times)

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Online Roman

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Als craze
« on: July 07, 2014, 08:15:02 AM »
Good morning all I've been on anxiety zone for approx 4 months and can't help but notice the epidemic of als fears, myself included, that ppl have been having. Instead of discussing symptoms I would like to know if the is any promising research being done that will help ppl who have it in the near future.
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Offline 3r1cR0c9

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Re: Als craze
« Reply #1 on: July 07, 2014, 08:40:41 AM »
been down this road and yes i believe so. i've done research & stem cell is a little promising though it's not yet considered a cure for now but the results of the experiments made were really promising so let's hope & pray that someday they find a cure for it and soon :)
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Online Roman

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Re: Als craze
« Reply #2 on: July 07, 2014, 08:50:46 AM »
Thank you for responding, I too have heard of promising stem cell research, notably the treatment given to a rabbi in Israel. However I don't know how long it lasted or his long term status. There was also a man on my local news here in boston who had  end stage als and through a soy supplement he can talk and swallow again and gained 20 lbs. I think the federal govt. spending on als is appalling only 39 million dollars is earmarked for als research the rest is private donations.
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Offline Niceguy237

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Re: Als craze
« Reply #3 on: July 07, 2014, 09:11:09 AM »
Yes there is promising research going on with stem cells as a treatment but it's still every stages.  Even more promising is the transforming of A** patients skin cells into stem cells which can then be transformed into motor neurons and other nerve cells to study the disease biology and therapeutic intervention on cells actually containing specific patient mutations.  This adds a new element to the research as previous the reliance on an animal model containing one particular mutation (SOD1), didn't necessarily recapitulate all of the disease biology of the multitude of other mutations causing the disease symptoms.  Since it's such a heterogenous disease - this approach is likely to yield semi personalized treatments for clusters of patients with similar gene mutations.  This is very promising but still in it's infancy.

The challenges with A** research and treatment:
1) Biology is not completely understood making the disease a risky investment for big pharma.  They have almost pulled out of researching the disease and are relying on small biotech and academia to do the tough work before they will invest
2) Disease heterogeneity creates problems in terms of understanding biology and setting up trials
3) research is significantly underfunded given the challenges above - I heard govt spends $5 billion/yr on cancer research and no where near that is spent on A** or any neurodegenerative disease. 
4) drugging the CNS creates challenges as well as it is sometimes difficult to get any therapeutic to spread throughout CNS

I think it'll be like MS where there were no treatments until the biology was better understood and someone found something that worked very well against a broad target (neuro inflammation and autoimmunity of nervous system).  There is no "cure" for MS but there are like 10 approved treatments which can slow symptoms in many patients and allow patients to live better quality lives. 

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Online Roman

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Re: Als craze
« Reply #4 on: July 07, 2014, 09:51:59 AM »
Thanks for the info nice guy. I remember reading about a new discovery I think it's what your talking about, als in a dish? Where the animal model will not be the used that often in the future. I agree that because there are so many genetic mutations that it makes a cure difficult but I think, like ms, it will be manageable and ppl will live longer with a better quality of life.
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Offline Niceguy237

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Re: Als craze
« Reply #5 on: July 07, 2014, 12:08:19 PM »
The technology is called iPS - induced pluripotent stem cells.  The great advantage is that they can be induced from a non invasive patient skin biopsy and then turned into cells that are usually not able to be harvested via a brain biopsy.  The challenge with neurodegenerative diseases is that you just can't biopsy a brain or spinal cord.  This is a big difference why cancers are so treatable today compared to neurodegenerative diseases.  With cancer you can easily biopsy or get at the malignancy (in the vast majority of cancers).  This has allowed for deep understanding of disease biology and personalized approaches for specific genetic presentations. 

iPS is still a young technology but I think it will allow for more high quality drug candidates to move into trials especially when combined with animal models and the postmortem work that is done as well on patients and the current stem cell trials in patients.   Still can't put a timetable on when better therapies will actually be available because it is still hard to find things to work in humans.  but these new technologies along with widespread genetic sequencing and what seems to be the potential for increased funding will only help get better treatments into the clinic faster and increase the technical likelihood for success across the spectrum of neurodegenerative disorders including A**
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