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  • Understanding ALS research

    Posted by Amanda on May 9, 2019 at 10:14 pm

    I’ve been a school psychologist for nearly 15 years. Prior to that I spend time working in the counseling field. In both careers, I’ve was trained in crisis response/management and mental health. Additionally,  I have been volunteering for ALS medical research since 2009, when my family realized that we had a genetic mutation association with ALS. There was one study completed at the University of Washington in St. Louis on my family. At that point we had lost about 5 family members and had several more diagnosed. Although I have been  involved in several research studies and I have been reading  about ALS research for years, I often times find some of the articles to be overly technical and I don’t always understand the implications or medical writings. I recently came across this website and I thought they did a great job of explaining ALS research and various aspects and types of studies. If you are interested have a look and share your thoughts.

    Dagmar replied 5 years ago 3 Members · 2 Replies
  • 2 Replies
  • Jean-Pierre Le Rouzic

    May 10, 2019 at 10:49 am

    I am sorry for your loss Amanda.

    A few observations on this text you linked:

    * SOD1 mutations cases are only a few percent of all cases. In fact ALS is very diverse disease, in UK they do not use this word, they use “motor neuron diseases” and I think that it is much more accurate. But most diseases of the Central Nervous System share many features, so it might be that there is no ALS, SMA, Down, FTD, Alzheimer, Parkinson, etc… but proteinopathies. A good step in this direction is for example

    * Having diseases constantly subdivided in new sub-categories (for ALS there might be dozens of sub-types) is very interesting to the pharmaceutical industry who might sell “precision drugs” that are very costly but it is counter-productive for patients as it further delay the availability of drugs. Having common research on several proteinopathies (such as TDP-43) would be much more in our interest.

    * I think the sentence “In the case of ALS, researchers have been able to convert pluripotent stem cells derived from skin/blood into becoming motor neurons” is not accurate, it might have been done in-vitro but that means nothing for pALS.

    * In overall I think this site focus on stem cells is wrong. We do not know why motor neurons die, or even if the root cause is motor neuron are dying, so it is a long stretch to assume that infusing other cells in the CNS would magically solve the problem. And stem cells therapies are usually very inefficient, only a few percent of the cells are living for a few months, meaning the healing effect is feeble and do not last (see Nurown).

  • Dagmar

    May 10, 2019 at 11:51 am

    I agree with you Jean-Pierre in that there are too many sub-divisions of diseases; case in point – – ALS.

    It is confusing for not only patients but the public; especially when it comes to awareness and fundraising campaigns. ALS? MND? Lou Gehrig’s disease? – – people wonder exactly which disease they are contributing to. In the end, they are the same.

    There is an interesting trend that I’ve been noticing, to group (ALS, MS, Parkinson’s, etc.) under the umbrella term: Rare Diseases. This term is beginning to be used more and more for advocacy and funding purposes. We’ll have to see where it goes.


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