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Which ALS-related science, research, etc. topic is on your radar right now?
Many news articles are being published with updates on new ALS research, studies, treatment, and even lifestyle (diet, quality of life, etc.)
What are the “hot topics” that you are following? Have any stories sparked your interest lately?
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46 Replies
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Crispr and C9orf72 research and development by Biogen.
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angela
how do you pee
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CNM-Au8 at the 30 mg dosage. It’s showing very promising results in a clinical trial. I like that it’s in the planning stage for Phase 3 and hopefully would succeed there and then be available to patients within a reasonable time period.
Coya 302. It sounds promising but it’s very, very early in its clinical testing.
Mark
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PTC-857, CARDINALS. I might participate in it, but I can’t start Relyvrio if I do. So if I started tomorrow, it would be 8 months before I could start Relyvrio. I am on Radacava ORS for a month or so now, and have a prescription for Relyvrio going through the Insurance rigamarole right now. I do think the science checks out on that one. So do I delay? That is the question of the day for me? If I delay and get on PTC-857 and not a placebo, that’s 2 meds and then after the first phase 8 months out, I’m in the open label phase and can start Relyvrio then. Which would put me on 3 drugs.
Life is full of decisions that one just doesn’t know the result in the end.
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Joined A Phase 2a Study of TPN-101 in Patients with C9ORF72 .
Had to promise not to use any of the other drugs. Fair enough. They only promised a slight delay in symptoms.
maybe TPN-101 will turn out to no better but I feel like maybe I’m helping the science move forward.
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Gene therapy is on my radar
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Afternoon All,
I’m scheduled to start PTC-857 at the end of April. I’m currently on Relyvrio and Riluzole. I’m blessed that my wife’s company insurance covers both products. While I also decided to turn my truck back over to the bank, so that I could afford the co-payments. Kaiser, also, approved me to get a 3rd opinion outside their network, and that’s whom I’m participating in the clinical study through.As far as delaying, ALS is a beast with no known good outcomes and if you can participate in any new studies that could slow it down, then participate! Don’t get me wrong everyday, my body reminds me the beast is still here, so I’m trying to slow it down as much as possible!
Looking forward to engaging with you guys in the future!
God bless,
Melton
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My husband has been accepted to a clinical trial at Cedars Sinai for stem cell injections. He will receive injections in the portion of his brain that controls his left hand. We are excited at the prospect of what this treatment can provide for ALS in the future.
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Awesome news. Please keep us updated! Thank you
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Yes, awesome news. And, I believe stem cell/gene therapy treatments will be the best option that will lead us to an ALS cure.
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@L Hagman,
That’s great news! Please let us know how your husband is doing and what you think of the trial.
Amanda -
@L Hagman,
That’s great news! Please let us know how your husband is doing and what you think of the trial.
Amanda-
Thank you. It’s for me though not my husband. I will let you know how things go.
Maryann
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Maryann, I’m sorry. I miss read the “who” part.
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No worries. Thank you
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WVE-004 by Wave Life Sciences for C9 ALS & FTD ASO tech.
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The new Healy regimen F
with targeting tfp43
joan
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Joan
How do you feel on regimen f. I take riluzole, radicava and relyvrio and I started regimen f 4/25. Do you notice any changes with regimen f?
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Maryann, I’m curious… did you encounter any difficulty in having your health insurance pay for you to take all 3 Rs? ( riluzole, radicava and relyvrio)
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The Riluzole was approved right away. Radicava was approved with 2 or 3 weeks. Relyvrio my insurance denied and I received it for free through Amylyxx manufacturer while the appeal process took place. It took a few months for it to ultimately be approved.
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Personally I’m following Tofersen because it targets SOD1 ALS. I’ve applied for early access to the treatments. In the clinical trials it has shown to significantly reduce the neurofilament light chains that are in spinal fluid prior to and when clinically significant symptoms of ALS develop. Toferson is administered intrathecally and is supposed to replace the mutated portion of the SOD1 gene so that it no longer produces the “bad” protein that cannot perform it’s job and damages the nerves. I’m not a doctor or a scientist so I may not have all the science and technical terms exactly right, but this is my general understanding after reading, researching, reading more, and participating in the pre-fALS study for 12 years. I think in cases of ALS that are genetically linked, each mutation will require a different treatment correcting that mutation. Unfortunately that only accounts for 10% of ALS cases.
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All of them. I maintain an exhaustive list of potential ALS meds on weekly basis.
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Great job, Svarog, in compiling this list! It’s very encouraging that there are quite a number of experimental medications that have shown some success in trials. I’m also encouraged in looking at your chart to see that studies to find effective treatments for ALS are ongoing throughout the world.
Mark
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Svarog, that is an impressive collection of charts! Are you the sole creator, or is this a collaboration?
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Thanks, I am the sole creator
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@Svarog – Thanks for this incredible resource. I’m curious, how easy or difficult would it be to figure out how many molecules/drugs were also tested in vitro on motor neurons grown from ALS patients’ iPSC lines during a trial? My guess is the answer is near zero, but I’m curious if we can confirm.
It’s no secret that drugs that work in mouse models rarely (if ever) work in humans with ALS. One reason for this is that mouse models aren’t representative of the >80% of patients with no known genetic predisposition. This helps explain why the success rate of the ALS drug development pipeline is less than a third of most other conditions (3% vs 13%). It’s like we keep testing drugs for liver cancer on patients with breast, lung, and pancreatic cancer and wonder why they don’t work. Here’s a white paper I wrote with more details.
With this in mind, I’ve been investigating whether testing drugs on motor neurons grown from ALS patients’ stem cells (iPSCs) could provide a better approach, and am trying to lobby ALSA, NIH and others for more funding and attention to this. As part of this advocacy, I’d love to show definitively that most clinical trials aren’t yet using iPSC lines to identify subpopulations for whom a drug may work — and that this therefore represents a major opportunity.
Is this something you can potentially help with?
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From what I read the use of iPSC lines is mainly discussed in terms of drug discovery and not so much in the context of clinical trials (e.g. for disease heterogeneity consideration purposes or for deriving scientifically justified eligibility criteria). Also, iPSC-based drug discovery / repurposing seems to be a growing trend. Some examples that I have found are:
- Ropinirole, Retigabine, Bosutinib: New ALS Drug Candidate Developed Using iPSCs
- CRISPR/Cas9 screen in human iPSC-derived cortical neurons identifies NEK6 as a novel disease modifier of C9orf72 poly(PR) toxicity
- Effect of complement C3 inhibition with pegcetacoplan in an iPSC-derived ALS neuromuscular junction model of neuroinflammation
Also, there are some interesting results from clinicaltrials.gov: iPSC | ALS
From my point of view, your proposal of using iPSC lines to identify subpopulations for whom a drug may work could make sense. I just don’t know how willing would the trial sponsors be to do this up front as opposed to using this approach to better understand the drug efficacy results in a post-hoc analysis, thereby motivating potential follow-up trials.
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I’m hoping and praying that everyone’s hot topic is successful.
In the meantime, my question is “why aren’t we as a group pushing for a follow up to the January 9, 2023 ALS News article on the Chinese herb patch Ji Wu Li (JWL)?” As others have noted in their comments, “I’m no scientist” but, the article hints at being less costly, states that the effect in slowing progression is significant, and it states that “Overall, the ingredients of every single herb of JWL have effects on almost all known mechanisms of ALS”.
I might be only reading into this what I want to hear but, I’d like to have this as an option to hopefully save a point on progression while I wait for something else to come along.
If I am way off base with this comment I apologize but, it seems like just about every ALS News Today article since January 9th has referenced this article as a “learn more” reading. Are they trying to tell us to start pushing for commercial availability?
Thanks,
Luke
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I’d also like to know what’s going on with Ji Wu Li. I’m wondering if the drug companies are doing the same thing they did with medical cannabis. They make a synthetic, less effective form, then make huge profits.
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I contacted a Chinese medicine expert and shared that article with her. She made the herb combination into a tea that I drink 3x a day. I don’t have scientific evidence, but I’ve found it to be a positive homeopathic addition to my regimen.
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Hi Bernie, Somehow I missed your input on the Chinese herbal tea til just now. I’m glad you were able to get something going on that – congratulations. Does it make sense for me to ask you for the contact information for the person you source your tea from? I’m thinking maybe I should try to source some via mail order from your person rather than trying to find someone here in Altoona, Pennsylvania. Please advise.
Thank you,
Luke
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Did anybody see the article in ALS News Today, titled
<h1 class=”bio-article-content-heading bio-type-display–large”>MSC transplants found to extend survival by 4 years in ALS: Analysis</h1>
<p class=”bio-article-content-lede bio-type-display–small”>The first paragraph reads as follows:</p>
“Transplants using mesenchymal stromal cells or MSCs — a type of stem cell found in bone marrow — delivered into the spinal canal can significantly extend survival in people with amyotrophic lateral sclerosis (ALS) compared with what would be expected based on their clinical characteristics, a new analysis of trials from the early 2000s found. ”Here’s a link to the article, which contains a link to the study.
Now, I have to say, I have mixed emotions here. On one hand kudos to the research, and essentially reinforcing what NurOwn is about. On the other hand, I’m pissed. Studies from the 2000s? Following patients for 20 years? Somebody should have been shouting this from the rooftops 15+ years ago. How many people have died, and will die, based on a lack of urgency? Perhaps I’m being too critical. I don’t know. But as someone who has been at an FRS of zero, and paralyzed for approaching 3.5 years, I’m angry at the pace.
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Mike — I join you in the “mixed emotions” response regarding the analysis of 15-20 year old studies with MSC transplants. Sounds great and important and promising, and WTF with the pace of follow-up? What’s being done, or what can we do, towards turning this into available treatment? These are not rhetorical questions — I’d like to know how to find answers and take steps!
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More interested in the current state of that research. Is it now available for use or is more study needed?
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I take Riluzole, Radicava, RELYVRIO and 2 weeks ago I started Healey Platform regimen f (Abbv 7262) – hoping I am on the drug and not the placebo but it’s 3 to 1 so I’m hopeful. I notice I am more wobbly when I take the Radicava 10 days out if the month.
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Maryann, I have noticed when I am taking the Radicava that I am a little more wobbly than normal.
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Lynn,
I feel wobbly when I take the radicava. I keep a journal each month and my movement is worse during the 10 days on cycle
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Hi! I am “new to the club” having been diagnosed in Dec 2022. I tried Riluzole but it affected my liver so they took me off. I am participating in a trial – MN-166 obviously don’t know whether I have the placebo or actual drug. I have been on Radicava since Jan and have just started taking Relyvrio. It’s all I can do to get the Relyvrio down and keep it down. I’m not a listerine fan so I’ve been putting a peppermint candy in my mouth as soon as I take it to try and help with taste. Does anyone have any suggestions – or something that works for them?
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Lynn
I mix relyvrio with warm water. Mix it really well and let it sit 15 min or so to dissolve, then drink it down with a straw. It’s tolerable if it’s mixed well.
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I mix with tap water stir well drink as fast as I can. Then eat 2 or 3 hershey kisses. The chocolate cleans most of the after taste. I wipe my lips to remove the taste then brush my teeth. No real issues and no after taste
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business essay writer top for me.
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Tofersen/Qalsody. My husband is SOD1. He cannot participate in clinical trials because of recurrence of prostate cancer.
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I am following the first large clinical trial of an NAD+ elevating supplement – nicotinamide riboside. This trial of 380 participants was initiated in 2020 in Norway and results are expected later this year. A small pilot study published in 2019 was very encouraging, as selected patients demonstrated significant responses to this treatment.
Clinical trial (ongoing): https://www.clinicaltrials.gov/study/NCT04562831
Pilot study: https://www.tandfonline.com/eprint/5j6sIsrMDYd37hjpNghx/full
Following the 2019 study and after discussions with the scientists involved, we started my wife on “Basis” (nicotinamide riboside + pterostilbene). She has been taking it for 5 years now and her progression has essentially stopped. I realize this is anecdotal, but for some patients it is theoretically possible, if the causative defect of the neurodegeneration was indeed related to low NAD+. Ceratain SARM1 mutations (below) would fall into this class. We are currently having my wife’s exome sequenced to see if anything interesting pops up.
SARM1 and ALS: https://molecularneurodegeneration.biomedcentral.com/articles/10.1186/s13024-021-00511-x -
I’ve read that Tudca seems to be listed in supplements many people take. After reading the description, it seems to treat Liver, Gallbladder, gut and kidney problems. Can anyone explain how that would help ALS?
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TUDCA is a molecule that helps to protect motor neurons by camouflaging a stress chemical released within the motor neuron that starts a process that results in cell death.
TUDCA may act by:
-Increasing glucose-induced insulin release via the cAMP/PKA pathway, increases insulin sensitivity
-Relieving endoplasmic reticulum (ER) stress. The ER makes sure proteins are folded properly
-Reducing programmed cell death (apoptosis) in healthy cells. TUDCA prevents the molecule BAX from reaching the mitochondria. BAX causes mitochondria to release cytochrome C, which causes enzymes (caspases) to initiate apoptosis
-Inactivating Bcl-2-associated death promoter (BAD), a molecule involved in apoptosis
-Removing toxic bile acids from the liver and preventing them from damaging liver cells
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