February, 2021
I recently watched a fascinating seminar on recent advances on the fight against this wretched disease. The seminar, which can be found at this link https://youtu.be/p9e9wV6Pw-Q contains highlights of the 11th Annual California ALS Research Summit. I personally love to nerd out on something that is going to kill me, but that’s just my thing.
The semnar was put together by the awesome team at the Golden West Chapter of the ALS Association. This is an incredible organization that has provided direct assistance to me and many other pALS, so I’m going to give them this shout out and urge you to give them all of your money.
Most of what I discuss here is from memory from a mind untrained in neurology or any other scientific discipline beyond intro to biology in college. So caveat lector, you have been warned. If I get something wrong you can publicly shame me in the comments.
One of the big innovations is in clinical trial design. Borrowing from cancer research, certain drug trials are now being conducted under a platform regimen. If I understand this correctly it means that the exclusion criteria, placebo control group, and other aspects of the effort can be shared by a variety of different drug candidates. This reduces cost and time for each trial. What’s not to like? The first platform is underway with a few trials ongoing.
The Answer ALS project was discussed. This effort is led by the Packard Institute at Johns Hopkins, named for a classmate of mine who succumbed to this wretched disease while in his 40s. The project has two major tasks. First it has built a database of, among other things, blood chemistry and genetic information (including from yours truly) of over a thousand pALS which is available to researchers. Second, and this is really wild, it will derive stem cells from the blood samples from a pALS with degenerating motor neurons and use the stem cells to make motor neurons that are duplicates of the degenerating motor neurons. Yep, you heard that right. Make duplicate motor neurons from blood so you can perform a biopsy. Check out answerals.org if you don’t believe me.
The seminar also presented the work of three post-Docs at the bleeding edge of motor neuron research, each of which were a recipient of an award given at the seminar. All discussed activities at the sub cellular level occurring in degenerating motor neurons. Two focused on certain proteins that clump in the cytoplasm and cause cell toxicity. The third went right over my head.
There were a number of other cool segments. California’s latest nobel laureate in chemistry was featured in a short video about CRISPR gene splicing technology hinting at genetic solutions to motor neuron degeneration. There was a video of a Google project to speed up communication by tracking facial movements. I can imagine the fun when your favorite four letter word is mapped to a facial feature that later develops a twitch.
There’s so much more to cover but I’m afraid that my brain fog may be coming in. Notwithstanding, I do enjoy sharing these advances against this wretched disease because they give us both something we can never lose: Hope.
Embrace The Suck 
Bob, thank you for continuing to keep all of us informed but also for your incredible sense of humor. Even in the most trying of times you have a way to make us laugh. I hope you continue to keep us up-to-speed and know we are out there and supporting you all the way. Laura (Walker) Preston
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Thanks for the update, Bob! Good stuff.
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You got a lot more out of intro to biology than I did! Thanks for this summary. And more thanks for your attitude, your sense of humor, and your indomitable spirit–they’re truly awesome.
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Thanks so much. My mother was recently diagnosed and things are progressing really rapidly for her. Like you, I derive some sort of comfort from “geeking out” and really trying to learn about ALS, maybe because it’s something I CAN do. There is so much that is out of our control, and as a caregiver/family member I guess I am desperate to latch on to those little things that I can do something about, like expanding my knowledge of finding Mom an eyegaze device and helping her learn how to use it.
I really appreciated your post on hospice care, too. Mom is also in hospice, but unfortunately that means we don’t have access to clinic care at an ALS clinic — she is in Nevada, on standard Medicare and I guess once you are in hospice, nothing else is covered. I’m guessing MediCal is covering the gap for you here.
I was so glad Julie sent around the link to your blog. Thank you so, so much for sharing your journey to help people better understand what people with ALS are going through.
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