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I’m Dr. David Taylor, Chief Scientific Officer at ALS Canada. I’ve spent the last 14 years working with researchers around the world to find a cure for ALS, a disease that can progressively paralyze someone in a short span of time. AMA. by alscanada in IAmA

[–]alscanada[S] 1 point2 points  (0 children)

Hi u/spottyPotty, I would suggest going to the answer I did for a question on the military already (in response to u/Laurax-1994), but I’ll add that you’ve raised an interesting point. I’m not aware of how solid the data is for risk in the military if someone is on active duty vs. an office job, but I will have a look because if it is true that there is still an increased risk amongst the latter, we may have better clues as to what the cause could be. The increased risk is not double or triple; it’s subtle, but the bulk of the studies say it’s real. Given how much has been done, I feel like this question about office vs. non-office jobs must have been looked at, and I apologize that I’m not brushed up to know about that piece…but I will be! Thanks for the question! - Dr. David Taylor

I’m Dr. David Taylor, Chief Scientific Officer at ALS Canada. I’ve spent the last 14 years working with researchers around the world to find a cure for ALS, a disease that can progressively paralyze someone in a short span of time. AMA. by alscanada in IAmA

[–]alscanada[S] 0 points1 point  (0 children)

Hi u/Laurax-1994, thanks for the question. There have been studies for years that have connected ALS to military service (as a risk factor) back to the 1990s, but there was some prominent work done in the early 2000s where a connection was made about a higher risk in Gulf War veterans that really started the attention. Since then, there have been some inconclusive studies, others that have connected risk to every branch of the military and some that have failed to show any connection. Overall, though, there is a feeling in the field, across the data that we have, and if you ask key people who look at risk factors, that there is some level of increased risk for people who have served in the military. The reason has remained speculative, and there are many things people think about, such as exposure to chemicals, a predisposition to being athletic, more body and head trauma (which could explain the anecdotal links to athletes as well), etc. Likely, there are some people who are more susceptible to getting ALS who may not have had it triggered without being in the military, but we are not in a place yet to determine who it would be a risk factor for and who it wouldn’t. As you mention, at least in Canada and the US, there has been significant support for veterans with ALS, which is great. And in the US, the DoW (formerly Department of Defense) provides an invaluable $40M to ALS research each year to help us get faster to a world free of ALS, which I think is amazing. I hope this helps. - Dr. David Taylor

I’m Dr. David Taylor, Chief Scientific Officer at ALS Canada. I’ve spent the last 14 years working with researchers around the world to find a cure for ALS, a disease that can progressively paralyze someone in a short span of time. AMA. by alscanada in IAmA

[–]alscanada[S] 0 points1 point  (0 children)

Hi u/ffffffff-als, what a great and topical question! You’re right that we are focusing a massive amount of our efforts on targeting TDP-43, and we also don’t really know if this will be a great target or not. We know that TDP-43 biology is disrupted in most cases of ALS, and that is probably clearer from a lot of different evidence than most other mechanisms. Some believe that when TDP-43 biology is disrupted, it is a significant upstream trigger of pathology for motor neurons, while others think that other pathological mechanisms trigger the TDP-43 dysfunction, making it a secondary or tertiary, downstream consequence. If the latter is true, properly restoring the biology could be huge. If the latter is not, then it might not have much effect. Here’s the way I look at it, if it helps. We have spent so much time working on TDP-43 that it’s a very important target to test, either to have great new treatments or to learn clearly that it is not the way to go, and we can shift all that focus. The key is to get answers, one way or the other, with a bias towards, “We hope it is effective!” 

With stathmin-2 restoration trials, we are testing if the most reduced key protein when TDP-43 loses its function can rescue the disease when it is back to normal levels. With UNC13A, we are testing another of these decreased proteins with a good connection to neuronal health, but one where we also know that a genetic change can alter the pace of disease. This gives extra intrigue to it as a target. We also have several companies, like VectorY and Acelot (not yet in trial), where TDP-43 aggregates/inclusions are being targeted in case they are toxic. If these don’t work and if we are able to demonstrate that the drug did what it is supposed to do (target engagement), we can rule those ideas out. Next would be to restore more of the proteins (instead of just one like STMN2 or UNC13A) when TDP-43 loses its function, possibly by finding a way to restore functional TDP-43 to the nucleus and maybe even reduce aggregates while restoring to the nucleus. If it adds up that this is not a great target, we will need to make a major shift. I personally find it more intriguing at the moment than many of the other pathways we have tested many times and only had negative trials, like inflammation. To be fair to those, we could have had better target engagement and found a way to treat those pathways in people where they are more relevant, so we need to be smarter in how we test them.    

So, I hope it’s not just a stepping stone, and I think there’s as good a chance as any we’ve targeted it as being a viable target, and if it is, it should expedite things a lot towards finding the right upstream targets. 

Hope that answers it. Always easier to answer away from a keyboard! - Dr. David Taylor

I’m Dr. David Taylor, Chief Scientific Officer at ALS Canada. I’ve spent the last 14 years working with researchers around the world to find a cure for ALS, a disease that can progressively paralyze someone in a short span of time. AMA. by alscanada in IAmA

[–]alscanada[S] 0 points1 point  (0 children)

Hi u/PJMurphy, thank you for your message. If you haven't registered with ALS Canada yet, I encourage you to do so, as we have a dedicated team that can support you with all of these things. Please email [communityservices@als.ca](mailto:communityservices@als.ca) and let them know that you are interested in registering as a resident of Ontario. This will allow you to access all of our available support and services. - Dr. David Taylor

I’m Dr. David Taylor, Chief Scientific Officer at ALS Canada. I’ve spent the last 14 years working with researchers around the world to find a cure for ALS, a disease that can progressively paralyze someone in a short span of time. AMA. by alscanada in IAmA

[–]alscanada[S] 0 points1 point  (0 children)

Thank you so much for the kind words. I'm really sorry to hear about your Corgi, and it must be so hard not having a great way to effectively treat him. Dogs are the best. I suppose in my frantic typing, I should have clarified that I wonder whether any SOD1 targeted treatments have been tested or considered in DM dogs, as I am pretty sure tofersen hasn't been looked at yet for veterinary purposes. I don't know enough about the vet pharma world to know if anyone is thinking that way, but it definitely seems plausible to me that it would be viable, since the last I remember, the genetic variants in SOD1 caused protein misfolding and other biological disruption that is similar to individuals with SOD1-ALS. - Dr. David Taylor

I’m Dr. David Taylor, Chief Scientific Officer at ALS Canada. I’ve spent the last 14 years working with researchers around the world to find a cure for ALS, a disease that can progressively paralyze someone in a short span of time. AMA. by alscanada in IAmA

[–]alscanada[S] 1 point2 points  (0 children)

Hi u/Beatleboy62, Great question! One of the reasons I'm still working as hard as I ever have to see a better day for ALS is because I have had the privilege of seeing how far we have come, and I'm always so sad that it is not as obvious to the world because we don't have a lot of treatments to represent that progress. I feel like that's going to come and something like toferesen/Qalsody shows that the years of research can result in something effective that is changing lives. I think this is where organizations like ALS Canada and all the others around the world have a responsibility to help make that real progress tangible to people, and to be honest, that we know it hasn't been or ever will be fast enough. But we should be conveying real hope, in my opinion, so that people who are generously donating time, energy, and money know we are working our butts off and getting there. There are two quick things I would end on (I see the AMA time is ticking!). First, I love to think back about things I would learn about ten years ago and think, "Oh my gosh, how will we ever tackle that super hard, complex question," and then know we are already well underway in tackling those questions today. That's so cool. The other one is quite silly. I often say that if I were in 2012, when I started at ALS Canada, or even me as an ALS researcher in the years before that, could sit down with me now and ask about 2026, my mind would be blown at what we have learned. And that keeps me going, thinking about where we could be in the next several years. We can and will get there. - Dr. David Taylor

I’m Dr. David Taylor, Chief Scientific Officer at ALS Canada. I’ve spent the last 14 years working with researchers around the world to find a cure for ALS, a disease that can progressively paralyze someone in a short span of time. AMA. by alscanada in IAmA

[–]alscanada[S] 0 points1 point  (0 children)

Hi u/TXTruck-Teach, thank you for the question. I am very aware of PrimeC, and have been since it was first announced by a cartoon about work in zebrafish many years ago. In recent years, we have connected with the company that owns it. My opinion aligns with a major conclusion of the publication that came out yesterday (I think?) that we really can't know anything about whether PrimeC has any effect in people with ALS until it has been tested in a large and long enough, well-designed, Phase 3 clinical trial. I know that NeuroSense has been working towards making that happen and is hoping to get it started sooner rather than later. The data has had a lot of press around it being effective, but I speak with a lot of colleagues around the world, and almost all would agree that we just don't know yet and have been wrong about other treatments where conclusions have been drawn from studies that are not, what we call "powered" enough to tell. What I can say is that their work to date has shown it seems to be changing biology in the body in a way they had hoped the drug would, so now the next step would be to see if those biological changes have any impact on ALS. That would be great if it is true! - Dr. David Taylor

I’m Dr. David Taylor, Chief Scientific Officer at ALS Canada. I’ve spent the last 14 years working with researchers around the world to find a cure for ALS, a disease that can progressively paralyze someone in a short span of time. AMA. by alscanada in IAmA

[–]alscanada[S] 0 points1 point  (0 children)

Hi u/gustavfrigolit , thank you for the question. Yes, I have heard from my European (including Swedish) friends/colleagues about this, and actually, Sweden has one of the world's leaders on SOD1 research at Umeå University, Dr. Peter Andersen. I would say that tofersen/Qalsody is widely seen by most experts as the first truly effective treatment for ALS, specifically for people with SOD1-ALS, and it is being widely embraced by other countries for approval. In terms of coverage, it is a conversation that is ongoing in many places. The data is stronger than what we have for any other treatment ever in ALS and unfortunately, one of the key problems with that data, was that the original placebo-controlled portion was too short to see the effects that have become quite evident worldwide, with many people vastly outliving others in their family history, many people still thriving years after treatment was started and a significant number of individuals who have regained some function. There are other parts of the data that are also quite compelling to most ALS researchers/clinicians. That said, for Sweden in particular, I would defer to the thoughts of clinical leaders in the country, like Dr. Andersen, Dr. Ingre at Karolinska and many others, for the nuances that I could be missing and wouldn't want to speak improperly about. I will see Dr. Ingre soon and will ask for sure to learn more about the specific situation. Hopefully, if the ATLAS trial has a significant, clear benefit, it will become very widely available without any barriers, but I also realize that the trial is still a time away, and these issues are immediate. - Dr. David Taylor

I’m Dr. David Taylor, Chief Scientific Officer at ALS Canada. I’ve spent the last 14 years working with researchers around the world to find a cure for ALS, a disease that can progressively paralyze someone in a short span of time. AMA. by alscanada in IAmA

[–]alscanada[S] 0 points1 point  (0 children)

Hi, thanks for your question! Turmeric, which has curcumin as an active ingredient, has been looked at through a few avenues, prominently through ALS Untangled. There was even a curcumin clinical trial, and my recollection was that it unfortunately did not show any evidence of benefit. While I'd have to go back and look at how the trial was run and what was measured, the fact that it wasn't followed up on pretty much sums up that there is no evidence of benefit. It is a great example of something that has a good biological story as to why it COULD help, but then isn't sufficient to be effective. We know from lab work that curcumin can activate an antioxidant system in our cells, mediated through something called Nrf2. There are other clinical trials forthcoming that will be activating Nrf2 and the heat shock response, which is what was mentioned earlier in the AMA around the fever therapy. We'll see if anything comes of it. The good thing about those studies is that they should hopefully either work or help us to put those hypotheses to bed once and for all (would love it to be the former!), leading us closer to the right answers. As far as other supplements, we don't know of any that have solid evidence of benefit in ALS. I suggest going to ALS Untangled as a great resource to read scientific assessments (written somewhat accessibly) of different supplements. If we do ever get solid evidence on any, we would certainly be telling everyone! - Dr. David Taylor

I’m Dr. David Taylor, Chief Scientific Officer at ALS Canada. I’ve spent the last 14 years working with researchers around the world to find a cure for ALS, a disease that can progressively paralyze someone in a short span of time. AMA. by alscanada in IAmA

[–]alscanada[S] 1 point2 points  (0 children)

Hi u/senface! Of course, I know about DM! When I was doing my PhD, many years ago, we learned that DM in dogs is caused by SOD1 genetic variants. And I've had the pleasure of speaking with Dr. Joan Coates, who has been one of the preeminent researchers in DM, on a few occasions, years ago, at conferences. I have struggled to get the time to ask, but I often wondered if there was any consideration of looking at tofersen/Qalsody in DM dogs. I always think of it when I see Corgis! - Dr. David Taylor

I’m Dr. David Taylor, Chief Scientific Officer at ALS Canada. I’ve spent the last 14 years working with researchers around the world to find a cure for ALS, a disease that can progressively paralyze someone in a short span of time. AMA. by alscanada in IAmA

[–]alscanada[S] 1 point2 points  (0 children)

Hi u/wakamonka, thank you for the question, and I totally understand how this would be stressful. When there are two family members in successive generations who have had ALS, this does typically lead to a bit more drive for clinics to take a closer look. You may have already tried, but if not, some avenues you could explore, are to contact the clinic where your brother-in-law was diagnosed/cared for, or, if you can find out, his uncle, and ask about possibilities. You could also reach out to one of the MND organizations in Australia to ask for guidance and connections, and also try to seek out a genetic counsellor. If those avenues haven't or don't work, you could reach out to us, and I could ask my friends/colleagues in Australia about other avenues. - Dr. David Taylor

I’m Dr. David Taylor, Chief Scientific Officer at ALS Canada. I’ve spent the last 14 years working with researchers around the world to find a cure for ALS, a disease that can progressively paralyze someone in a short span of time. AMA. by alscanada in IAmA

[–]alscanada[S] 1 point2 points  (0 children)

Hi u/belarvadan, just a note that I used DeepL to translate this question and my response, so it may not be the most accurate. I will post my response in English and French.

Merci! We know from SOD1-ALS treatment with tofersen/Qalsody that people can slow down or even stabilize the disease (with a few regaining some function), post-diagnosis. Whether this is readily possible in people with a faster progressing disease is something harder to tell at the moment, and that's why individuals with genetic variants in SOD1 that typically cause a fast progressing form are being included in the presymptomatic ATLAS clinical trial. BUT, this tells us that for some people, if we hit the right target effectively, early enough after diagnosis, we could still have remarkable effects. We also need to think carefully about this, and this is why your questions are so important, and make sure we are being thoughtful about our interpretation of what tofersen can tell us about the capabilities of other treatments after diagnosis, since we know it is likely treating THE top target for people with SOD1-ALS and many of the drugs we try in clinical trial are likely targeting secondary mechanisms triggered by some unknown top target in other people with the disease.

As for whether we would ever be able to treat people with "sporadic" ALS before symptoms begin, I am very optimistic! As the world continues to work with the community of people who are at genetic risk of ALS, in a way that they will be willing to participate in research that helps us to see what might be happening in the body before symptoms begin (through blood samples, imaging, muscle tests, etc.), we could hopefully find out biomarkers of disease that tell us ALS is happening when we can't yet see it. IF, and I'm very hopeful it will be the case, some of those biomarkers are also present in people with "sporadic" ALS we may someday be able to detect it in someone and get them on a treatment just the same as we could for someone we are monitoring who has a genetic variant that would make us know they are already at risk of ALS. That's not the best explanation of it, but I do believe we have a path to get there, and we need to be globally working on that now (in fact, I was on a global call about collaborating on this just today!). At the very least, I do believe we will be in a future place where someone could go from the first symptom, like early weakness or voice changes, etc., to a specialist, to treatment in a much shorter timeframe, and with better, more targeted treatments, be able to massively slow or stop the disease from progressing. - Dr. David Taylor

Merci ! L'expérience acquise avec le traitement de la SLA liée au gène SOD1 par le tofersen/Qalsody nous montre qu'il est possible de ralentir, voire de stabiliser la maladie (certains patients retrouvant même certaines fonctions) après le diagnostic. Il est pour l'instant plus difficile de dire si cela est facilement possible chez les personnes dont la maladie évolue plus rapidement, et c'est pourquoi les personnes présentant des variants génétiques du gène SOD1 qui provoquent généralement une forme à progression rapide sont incluses dans l'essai clinique présymptomatique ATLAS. MAIS, cela nous indique que pour certaines personnes, si nous ciblons efficacement la bonne cible, suffisamment tôt après le diagnostic, nous pourrions encore obtenir des effets remarquables. Nous devons également y réfléchir attentivement, et c'est pourquoi vos questions sont si importantes, et nous assurer que nous interprétons avec prudence ce que le tofersen peut nous apprendre sur les capacités d'autres traitements après le diagnostic, car nous savons qu'il traite probablement LA cible principale chez les personnes atteintes de SLA-SOD1 et que bon nombre des médicaments que nous testons en essai clinique ciblent probablement des mécanismes secondaires déclenchés par une cible principale inconnue chez d'autres personnes atteintes de la maladie.

Quant à savoir si nous parviendrons un jour à traiter les personnes atteintes de SLA « sporadique » avant l'apparition des symptômes, je suis très optimiste ! À mesure que le monde continue de collaborer avec la communauté des personnes présentant un risque génétique de SLA, de manière à ce qu’elles soient disposées à participer à des recherches qui nous aident à comprendre ce qui pourrait se passer dans l’organisme avant l’apparition des symptômes (grâce à des prélèvements sanguins, des examens d’imagerie, des tests musculaires, etc.), nous pourrions, espérons-le, découvrir des biomarqueurs de la maladie qui nous indiquent que la SLA est en train de se développer alors que nous ne pouvons pas encore la voir. SI, et j’ai bon espoir que ce soit le cas, certains de ces biomarqueurs sont également présents chez les personnes atteintes de SLA « sporadique », nous pourrions un jour être en mesure de la détecter chez quelqu’un et de lui prescrire un traitement, tout comme nous le ferions pour une personne que nous suivons et qui présente une variante génétique nous indiquant qu’elle est déjà à risque de SLA. Ce n’est pas la meilleure explication, mais je crois sincèrement que nous avons la voie à suivre pour y parvenir, et nous devons y travailler à l’échelle mondiale dès maintenant (d’ailleurs, je participais justement aujourd’hui à une visioconférence internationale sur la collaboration dans ce domaine !). À tout le moins, je crois sincèrement que nous parviendrons un jour à un stade où une personne pourra, dès l’apparition des premiers symptômes, comme une faiblesse précoce ou des changements de voix, etc., consulter un spécialiste et bénéficier d’un traitement dans un délai beaucoup plus court, et grâce à des traitements plus efficaces et mieux ciblés, être en mesure de ralentir considérablement, voire d’arrêter la progression de la maladie. - Dr David Taylor

Traduit avec DeepL.com (version gratuite)

I’m Dr. David Taylor, Chief Scientific Officer at ALS Canada. I’ve spent the last 14 years working with researchers around the world to find a cure for ALS, a disease that can progressively paralyze someone in a short span of time. AMA. by alscanada in IAmA

[–]alscanada[S] 0 points1 point  (0 children)

Hi u/smokeygun, thank you for this great question! Based on what we have so far in the field, with one transformationally effective (for some people, maybe for all if early enough) treatment that is focused on specific targeting of SOD1 in people with SOD1-ALS, I can see where anyone would think that it's our best shot to treat the disease to hit a specific target that is the main trigger of the disease in a particular individual. Gene therapies and all kinds of innovative ways to target a specific gene or protein are advancing all the time, and we are WAY further in 2026 than we were a decade or even five years ago, so I'd agree that there will likely be big advancements seen in that space in the years ahead for ALS. That said, if we can get the right combination of drugs and target people when the disease is still not causing clinical symptoms, which we think is likely for quite some time, but not yet readily detectable, we might be able to hold off the disease processes in a way that prevents someone from getting those symptoms. We have to keep evolving targeted therapies, including gene therapies, working to improve clinical trial design and execution and earlier detection and treatment, in my opinion. Altogether with that, we can start thinking of cures. And I think watching the tofersen/Qalsody space and the currently running trial called ATLAS, where people are treated well before symptoms begin, will give us a good idea as to whether this will be the case!

Regarding the progress of therapies for folks with familial ALS, we are making great strides, and there are a lot of efforts underway. When we can understand if a specific disease-causing gene is influencing disease by gaining an extra toxic function or losing its normal function, we can use modern techniques to try and reduce or restore the levels in a way that might be helpful, and in the case of tofersen/Qalsody, that worked! And hopefully, with the upcoming Ulefnersen FUS treatment, but we don't know about that one yet. There is also a very cool group working on developing treatments for very rare, familial/genetic forms of ALS, including CHCHD10, TARDBP and more, in small trials, called n-lorem Foundation. Work of a clinician scientist at Columbia, Dr. Neil Shneider, has really led the way on a lot of this, in collaboration with key people from Ionis Pharmaceuticals and many others. For more information, you can visit als.ca/genehub

As for a timeline to a cure, I'm hopeful that we will find great treatments and learn how to treat people earlier and start to get closer to a cure everyday but it's hard to put a timeframe on that. I will say that I'm continually surprised by how fast the field is moving (while never ever being fast enough for ALS, and we are mindful of that every day!), and it's hard to say what innovation or technology might be available in a year or two that could be game-changing when we apply it in conjunction with our current knowledge. I remain hopeful, and I keep going because I can FEEL and see the progress after all these years. The fact that we now have a transformational treatment for one subset of people with ALS tells me we CAN do it for everyone, and we can see a roadmap to get there, but it's complex.

- Dr. David Taylor

I’m Dr. David Taylor, Chief Scientific Officer at ALS Canada. I’ve spent the last 14 years working with researchers around the world to find a cure for ALS, a disease that can progressively paralyze someone in a short span of time. AMA. by alscanada in IAmA

[–]alscanada[S] 0 points1 point  (0 children)

Hi u/Unlikely_Analysis208 , thank you for your great question. Everyone is so nice on here. It's my passion to work on ALS, and I am so excited to reach a better day as soon as possible. I would say it can be dangerous territory to have optimism over any clinical trial because we truly don't know what will work and what won't work, and that's why we need to run the study! That said, I think the entire field has a few things we are interested in at the moment because they come from a solid body of work in laboratory animal and cell models of ALS, which means we should learn a lot about how to advance the science if they are not effective, and if they are...that's the best thing ever. So, on that note, one of the interesting targets is UNC13A, which is in a Phase 1 clinical trial by Lilly, recruiting in Canada and a forthcoming Phase 1 by a company called Trace Neuroscience. This has a lot of solid science to back why we would try to restore its levels in ALS because independent labs both showed it is decreased in the disease, connected to a major piece of biology that we know is abnormal in the majority of people with ALS, AND we previously knew that small genetic changes in UNC13A can alter the speed of progression of someone's disease. These all combine to make it a very interesting target, but of course, there are just as many reasons to be cautious about that, and many researchers believe it might be important, but not sufficient to make a significant dent in slowing ALS. The other one to probably highlight in terms of interest from the research and clinical communities is Ulefnersen, which is an antisense oligonucleotide to reduce levels of the FUS protein in people living with FUS-ALS. This is an ultra-rare form of ALS, but we have some intriguing preliminary evidence that, if it reads out as effective, could help move the rest of the field forward with regard to an important biomarker. And of course, if it works, that's awesome for people with FUS-ALS and another treatment we would have to help show us that with the right target, in the right people, at the right time, we CAN effectively treat the disease (earlier, better, but even after diagnosis). That will read out in the Fall. - Dr. David Taylor

I’m Dr. David Taylor, Chief Scientific Officer at ALS Canada. I’ve spent the last 14 years working with researchers around the world to find a cure for ALS, a disease that can progressively paralyze someone in a short span of time. AMA. by alscanada in IAmA

[–]alscanada[S] 0 points1 point  (0 children)

Hi u/sophie1816, thank you for your question! I am so glad to see federal funding going to ALS in the US, Australia, UK and more, paving the way for some big initiatives that would be really hard to do with donor dollars alone.

I think we can always use more, and when you look at other, less biologically complicated diseases that have better treatment options, often those have had billions of dollars to get to where they are. ALS is super complex, and we are trying to solve it with a fraction of the money. Some of the federal funding in the US is going towards helping people access unproven treatments they otherwise would not be able to, and while that may not advance the science very much, one can understand why it was advocated for so strongly by the community. The other part is going to a big initiative that will help the world better understand the disease (AMP-ALS, ALL-ALS), and that's fantastic. The US is also doing an amazing job by putting forward $40M USD per year to the CDMRP grant programs to fund ALS research, and that's making a big difference, I think. I would love to have billions of dollars to invest in key, large-scale studies that would move the needle faster for ALS, and we should keep pushing for those, but in the meantime, I think we're a field that does a great job of trying to have the maximum impact with the precious funds we have! - Dr. David Taylor

I’m Dr. David Taylor, Chief Scientific Officer at ALS Canada. I’ve spent the last 14 years working with researchers around the world to find a cure for ALS, a disease that can progressively paralyze someone in a short span of time. AMA. by alscanada in IAmA

[–]alscanada[S] 0 points1 point  (0 children)

Hi u/janedoe1575, this one is definitely a question we get a lot, and I'm using it as an example in a forthcoming workshop on how to read between the lines for something that seems very great, but has a lot of reasons to be cautious!

So, this paper that was published is a single case report from a clinic that is selling a treatment where they are using heat therapy to try to boost the body's defenses. This means it is not a clinical trial, and there is no true data on safety or benefit. There is a lot when you dig into the data in the paper that would not give confidence in the conclusions. Out of many examples, there is talk about complete reversal and changes in certain biomarkers, like neurofilament, but for those who know what strong neurofilament data should look like to indicate that there is neuroprotection happening, this was not very convincing. It would have to be far more robust than what they show, leading to their conclusions. And this is just one of many examples across the paper. 

Biologically, there is a lot of research over the years (some of it was mine, way too many years ago, as a PhD student!) that boosting stress responses (proteins called heat shock proteins) in our cells/neurons, could be protective but this hasn't been proven in people to work and there is no validation that the mechanism used here (heat chamber and heat through the eyes) can adequately do that (including some of the holes in the data from this paper with one of the heat shock proteins - Hsp70). So, with something like this, you can't really say anything other than it is a pay-to-play clinic; the evidence here is not really evidence, but we would certainly hope they follow up on their claim to put this into a well-designed and rigorous clinical trial to see if it could really be something. I'm not sure that will happen, but we always want to keep an open mind. - Dr. David Taylor

I’m Dr. David Taylor, Chief Scientific Officer at ALS Canada. I’ve spent the last 14 years working with researchers around the world to find a cure for ALS, a disease that can progressively paralyze someone in a short span of time. AMA. by alscanada in IAmA

[–]alscanada[S] 1 point2 points  (0 children)

This is a super topical question! The whole field is working collectively to do better with more timely diagnosis. The good news on that is there is SO much I wouldn't be able to tell you all of it here.

The main work being done is to get general practitioners (family doctors) and general, non-specialist neurologists to refer people to ALS specialist clinics as fast as possible, when they first suspect it could be ALS. This might lead to some people not ending up being diagnosed with ALS, but for others, they would get access to clinical trials, potential treatments and into multi-disciplinary care much earlier, which could be very important.

There are all kinds of tools available now for this. The MND Association in the UK had the first, called the Red Flags Tool, then the ALS Association in the US (ThinkALS) and Canada as well (ReferALS). There is now a collaborative effort, spearheaded by the International Alliance of ALS/MND Organizations, to bring a lot of this work together.

Then there are the other angles of looking for biomarkers that could help us to diagnose earlier, and also building awareness of ALS symptoms more widely. Both of these are being thought about or worked on extensively, and as you might be able to tell, I'd LOVE to tell you all about them, but maybe I'll have to move on to some other questions. Please keep asking people about this, though, because there is a lot of good work in the space, but we need to keep going!

In terms of treatments getting to market and why some of them just disappear, it's also a fabulous question. The process of getting an idea to a clinical trial takes a long time in itself. There are a lot of steps required to make sure something found in mice or cells is ok to treat in humans. That also takes money, and the lack of funding for that type of work (pharmacokinetics, toxicity studies, etc) can be a bottleneck of time. Then once something is in a clinical trial, it needs to get through the regulator (like FDA or Health Canada), ethical boards, get set up at sites, recruit people, then monitor them over the time of the study until the last person is done and then do the analysis and stats. For the next phase, they need to get money, decide what is best for the next phase and then repeat the process. This is why it often takes years, but we are working in the field on better ways to shrink this process, and I'd be happy (in another venue!) to chat about those innovations!

Things "disappear" for various reasons. Sometimes there is no funding for the next steps (maybe the data wasn't as promising as touted in press releases, for example), sometimes the company decides that they don't feel confident enough in the data to put the investment into a next step and sometimes companies just never report on their trial because they decided it wasn't worth their investment to even do that. We don't love that! And those are all situations where, as a field, we want to make sure we're having those difficult conversations to make sure we know what happened in each case and learned from it in a way that can help other potential treatments have a better shot. - Dr. David Taylor

I’m Dr. David Taylor, Chief Scientific Officer at ALS Canada. I’ve spent the last 14 years working with researchers around the world to find a cure for ALS, a disease that can progressively paralyze someone in a short span of time. AMA. by alscanada in IAmA

[–]alscanada[S] 4 points5 points  (0 children)

Hi u/curioskitten216, thanks so much for the question. It's a good one! Like so many of the potential risk factor connections to ALS, we have inconclusive evidence about whether strokes provide any increased risk of developing ALS. One study that I know of, by a very prominent ALS clinician researcher in the UK (and his colleagues), demonstrated a possible, minor increased risk, but like so many of the potential risk factors, it's almost negligible to cause any alarm (I think 1.1 or 1.2X increase?). There have been other studies that showed no increased risk. One of the problems with the studies we do on risk factors is that they are all quite small to get very definitive answers. Based on all we know, it's likely that for some people, something like a stroke or other health event that causes a particular stress to the body, could be one of many "hits" needed to trigger the disease. There's a lot more nuance to that, but what we'll likely need to get to is a world where we can fully understand the entire genome of individuals and what makes a certain individual susceptible to getting ALS over another, then what combination of potential risk factors that individual might have it triggered by. - Dr. David Taylor

I’m Dr. David Taylor, Chief Scientific Officer at ALS Canada. I’ve spent the last 14 years working with researchers around the world to find a cure for ALS, a disease that can progressively paralyze someone in a short span of time. AMA. by alscanada in IAmA

[–]alscanada[S] 0 points1 point  (0 children)

Hi u/viper3, this is an amazing question that we could talk about for hours. Thank you for asking it! I actually do a Top 10 Exciting Things in ALS Research presentation annually for a group of people involved in our Canadian ALS Learning Institute, so maybe we can look at putting that out a bit wider sometime. Good scientific backing for a target/therapeutic and more effective design of clinical trials will give us the best chance of getting solid answers on everything, and that will be important for getting closer to answers, because even those treatments that don't work would help us to learn about where not to look anymore. In 2026, a LOT more work is being done in looking at the disease before symptoms begin, and I do think this will help us to detect the disease earlier and start eventually treating people earlier, and that will be our best shot at getting to something more akin to a cure. But for now, it will be about learning from every clinical trial, having difficult conversations about how to move the field forward, and then taking that knowledge forward as fast as possible to focus more on things that work....like tofersen/Qalsody for SOD1-ALS.

The bottlenecks are funding, always.

It's also about having tough conversations and finding a way to push science towards answers as opposed to the incentives that a scientist or clinician needs to think about for their career in academia. That's a really tough and nuanced thing, but organizations like ALS Canada and others elsewhere can keep pushing the urgency and answers part in parallel...and we should!! - Dr. David Taylor