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Update us on the research and trials you are in!!
Hello pALS and pre-fALS members! Lets post some updates about the study, research and clinical trials you are in. What are you involved with (which trial/study), What have you learned? How do you feel? Would you recommend this to other pALS? What was the selection criteria?
Please share you experiences and keep us updated!
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49 Replies
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I’m currently exploring clinical trials and would love insights from other pALS. I have familial ALS but genetic testing was negative. Is anyone enrolling in the Healey Platform or COMBAT ALS trials?
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Hi Sandra,
I’m in the Healey trials for the drug Pridopidine. This trial of the 4 they are currently running is the only open to enrollment at this time. I started the trial med one week ago. I liked the Healey trials for 2 reasons. 1) the trial is 24 weeks and if you remain in the trial at that time, you will get the actual medication if you were on the placebo at that point. 2) the odds of actually getting the medication vs placebo are highly in your favor, 75% chance of getting the trial medication.
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Scott, I opted for the Healey trial for those reasons. I’m also in regimen D and have been participating for just over 2 months. I haven’t had any side effects, and my progression continues to be slow. I find the weekly webinars helpful and appreciate hearing from various research partners.
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I just completed Healy Platform for CMN Au8. Now I am taking open label for 1 year. I think I was receiving placebo for 6 months because my ALS declined rapidly. Either placebo, or the drug didn’t work for me.
I slao did the Cromylyn trial, but quit early because it is an inhaled powder and i experienced severe choking.
I wanted to contribute to research but it’s a lot of time commitment. Living on ALS time, that is an important consideration. I’m nearing the 2 year since diagnosis window, so will probably not qualify any longer. It is so great for the future if you can participate!
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Can you say more about the time commitment for clinical trials? Do you mean the length of the trials or the time it takes to participate. I’ve asked to be screened for the Healey Platform, opting for that 6-mo trial instead of a one-year phase 3 trial for COMBAT-ALS (Ibudilast). I’m not sure how long I’ll be eligible for trials – I’ve heard 2 or 3 yrs from symptoms or diagnosis.
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Glad you are starting platform. I live 2 hours from my research clinic so that makes it a time commitment for me. Then when I get there it is 1-2 hour visit. I hope you get drug instead of placebo! Good luck!!
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My advice for anyone interested in a clinical trial is to read the full details. These can be viewed on the US Clinical trials register and include the inclusion & exclusion criteria. Don’t just take your clinician’s word. Thanks goodness I didn’t!
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Hi All,
I’ve just completed Healy Platform, pridopidine. I’ve gone on open label extension. My ALS isn’t progressing much and in one area, breathing, has improved. Though I don’t know if I was on active drug or placebo, I think I may have been getting the drug. Now I’m getting the active drug. I highly recommend the platform. The experience of regular check-ups was very positive.
Tom Hehir
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Glad to hear of your positive experience, Tom. I’m going next week for Healey Platform screening.
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I am soon to complete the 6 month Healey Platform Trial, and I’ll begin open label, the actual drug, in September. I’ve been on Zilucoplan, which is an injection I give myself each morning. My doctor thinks I might be on the drug itself and not a placebo, since my ALS has not been rapidly progressing. She did say that since I am also taking Riluzole and Radacava, there is no way of knowing which of these drugs, or all of them, are contributing to the fact that I’ve basically been stable for the past 6+ months. The time commitment for this trial hasn’t been too hard to manage. I have to go for an in-person visit either every month or ever 2 months. When I don’t have to go in, the Research coordinator calls me and asks a number of questions as to how I’m doing. It has been fine and I feel that if it can even help me a little bit, it’s worth the effort and time involved.
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Great to hear about your experience in the Healey trial. I hope to complete the screening next week and get started.
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Carolyn, Thanks for your update. My biggest concern is that, if I remain stable in six months as this is all my open label allows, will I be allowed to continue on the drug? It seems the FDA takes a long time to approve drugs and time is something we don’t have.
Tom
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Tom,
Possibly my regimen A drug, the Zilucoplan allows for a longer open label extension. I actually asked about this at my recent research appointment and I was told I could be on it for 1 year, for the open label. I wanted to know if after the year is over, whether or not I could extend that time. The coordinator told me that it’s possible I could be given an extension, but if I can’t, I could go through the screening process again and randomly be assigned to another regimen. Since I have remained slow progressing and stable in some areas, I would most definitely reapply to the platform.
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Hi Amanda, I (someone) wasn’t quick enough to get me onto cohort 1 of the Wave Life Sciences WVE-04. However cohort 2 will be starting in October and Prof Shaw at SITranS Sheffield, UK has told me it will be running there and she’ll let me know as soon as it starts. I’ve stopped Metformin & TUDCA as I need have stopped 1 month before first dose. Acceptance criteria include SVC at or above 50. Check European trials database for locations. US site does not include this data at present.
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I’ve been in the Healey Platform trial on regiment C (CNM-Au8). I finished my 24 weeks in April and have been the open label, active drug for 16 weeks. It has not changed my progression at all. I have sporadic, limb onset(legs first), slow progression. I can’t say this treatment has worked for me, but it might for others. I will finish the open label in hopes that it does start working….
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I’m in the Biogen Biib 78 Extension trial. My ALS has slowed but I’m still progressing.
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Since I’ve had ALS for 12 years I’m usually not eligible for drug studies. I am involved with the following research efforts:
ALSTDI precision medicine program
Everything ALS speech study
NHI rare disease COVID-19 study
Mass Hospital NQ keyboard study
Purdue University breathing and speech study
ALS Community research study
I may not be able to get involved in drug trials but I have found a way to contribute.
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Paul, I’m 5 1/2 years since my diagnosis so, like you I’m not eligible for drug trials. But like you I’ve found observational studies to participate in. Makes me happy to think that I’m contributing in some way. My list is a little shorter than yours, but includes a couple of particularly interesting studies designed to investigate disease progression:
University of Florida – swallowing study
Mass General – Beiwe smart phone app
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I’m presently in the Cromolyn trial… no noticeable improvement, but no side effects.
<p class=”xmsonormal”>I have been offered inclusion in the Apellis (APL2-ALS-206) study: https://clinicaltrials.gov/ct2/show/NCT04579666</p>
<p class=”xmsonormal”>I received my genome sequence and there are no defects, so I have guarded optimism for this C3 Inhibitor study.
Does anyone have any information on the drug Pegcetacoplan?</p>
<p class=”xmsonormal”></p> -
I live near Pittsburgh and go to the Allegheny General Hospital
Als clinic. I have tried to get into trials for 2 years. In the beginning I was told I wasn’t sick enough then I found the Healy trials and signed up for them and COVID hit and all the trials were put on hold. No I am over 3 years since onset of symptoms and not eligible for them anymore. So for me trials would have to be made more inclusive.
Jim Olejar-
Jim
the amx0035 is in a phase 3 trial Phoenix and is applying for expanded access also which you could qualify for potentially
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I’m not in any trials, metformin is suppose to help my kind of als but did not engage with the trial too far to drive from Miami so looked up what it was made of and found out it is Goat’s Rue, a plant so ordered the herb and it helped so then I looked into the als drug amx0035 found out it was made of two drugs one cost $500 and ounce and the other Tudca i ordered from amazon, So those two actually stopped the symptoms of als, no twitches. So it started in my right foot on the right side, but these two have stopped the als. I wish I would have known sooner< I would be taking them.
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Hi Judy I also started taking the Pheburane and TUDCA combination (Amx0035), About 2 1/2 months ago. I take 500 mg of TUDCA and 3 g of the PB twice a day. I have not noticed that the twitching in my legs has stopped. In fact sometimes it’s ‘worse’ although that’s on days of my workouts and a fair bit of physical activity.
I guess it’s also hard to figure out if it’s helping or not, unless you actually see a reduction in symptoms. How do you actually know if it’s slowing the disease?? My hope is the combination of all I do is actually slowing things down waiting for that magic pill one day 🙂
My symptoms began two years ago and my official diagnosis was a year and a half ago. I started right away on the riluzole and Keppra for muscle cramping. Most of my issues present in my hands. I went right away for stem cell therapy and did this twice over the course of four months. I have not continued with that, as it is expensive, and there was some scientific evidence to indicate this may not be as effective as originally thought. Therefore put my resources into other things
My regime today is as follows:
I walk an hour each day, I have a personal trainer once a week and do weight training three days a week, and yoga on three other days a week. I spend one hour everyday in my hyperbaric oxygen chamber breathing 97% concentrated oxygen. I have the BeMer EMF Mat to promote circulation and relaxation. I lay on that every day for 16 minutes, and sleep on it once a week. Five nights a week I sit for 30 minutes in my infrared sauna at 140°. I take a variety of supplements including reservitol MetForman, CoQ10, vitamin D, TA 65, calcium and a multivitamin. I have an amazing family and friend community and remain positive and thankful every day, Honestly this is probably the best medicine of all of the things I’ve listed!
At my one year check up, I had only lost two points on the functional scale, so I was pretty excited. Is this a result of what I’m doing? Or is it a result of a slow progressing disease in my case? I have no idea! I just think that keeping my body as healthy as possible it will have the best chance to fight this horrible disease.
If others have any tips on their day today regime I would be happy to hear 🙂
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where do you live and what kind of als do you have
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Hi Judy, I’ve been taking Metformin & TUDCA for about a year. Then I had to stop two weeks ago to get on a clinical trial. Now my wife & I have noticed my ALS progressing more quickly.
Metformin free on the NHS, TUDCA from Amazon.
David
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where did yours start
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do you have familial 9c orf72?
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where do you live
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Mine started with right foot drop. I have c9fALS & live in Northumberland, UK.
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What is the NHS? Is that a European health institute? My 23 year old son was recently diagnosed with ALS and am looking for any help I can get for him.
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> What is the NHS?
Given that David is a UK citizen, I guess it is the National Health Service. The publicly funded healthcare system in UK.
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> so then I looked into the als drug amx0035 found out it was made of two drugs one cost $500 and ounce and the other Tudca i ordered from amazon
At my job, patent officers often said us that it is impossible to patent an existing product for two reasons:
1. A patent is about a process (akin to a recipe), not about a new usage.
2. A patent is about something new, so it is impossible to patent something that exists already.But they added that it was possible to patent a combination of two existing products, at the condition it is a novel, never described combination.
So the patent is written as:
This patent describes the combination of two products, such and such, with an application in this domain-
Yes indeed it is. After WW2 the Labour government delivered on its promises. The National Health Service has provided healthcare free to all in the UK ever since. We pay a percentage of income through our employers or as self- employed. This is ring-fenced for health care and funds the NHS. The Conservative governments in recent years have gradually tried to privatise elements of the service, but the NHS remains a highly rated public asset. Contrary to right leaning free market proponents, it remains an efficient and effective model. In contrast, our elderly care is now almost totally privatised and is extremely inefficient and costly. I know – I was CEO of a not-for-profit care provider and my sister a senior NHS doctor.
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https://thesocialmedwork.com/ketas-ibudilast ask your Dr for Prescription
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I am a participant in the HEALEY Platform Trial: Regimen D (Pridopidine), which I enrolled in early July. Of course, it’s hard to know if I am on placebo or active drug, but I’ve been experiencing some symptoms that may well be side effects of active drug. Side effects have included some nausea, lightheadedness, and extreme dry mouth. There is some time commitment: I have been going in for appointments about every 4 weeks (each lasts about 3 hours, and include blood draws, EKGs, some strength tests, PFT, etc.). In addition, there is an at-home speech test done 2 times/week utilizing their app, and I just had an in home PFT using a take home device and an app. All in all, I am grateful to be able to participate as it provides a sense of participation in the search for a cure. I’m realistic about the chances for a breakthrough, but participating with the community in the effort is rewarding by itself.
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I learned last week that I was accepted and assigned to regimen D. I go this week for my baseline appt. Like you, I’m excited to be a part of the study regardless of whether I receive Pridopidine or the placebo.
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Hi Eric and Sandra,
I just finished my Pridopidine trail and have moved to open label extension. I have not experienced side effects. I found the visits to be helpful in that I could discuss my ALS with the various team members. I also like the thought that I am participating in a community effort.
Tom
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My screening visit for the Wave FOCUS C-9 WVE-004 trial is sheduled for 30 Sep and first dose 11 Oct.
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I am in the second week of Healey Platform B, drug Verdepirstat, at Vanderbilt hospital research. Not on the full dosage until next week, but haven’t noticed any side effects. This is a 6 month trial with actual drug available after that time, if I’m not already taking it.
I am in my third year since right leg foot drop started my search for answers and 1 1/2 years from ALS diagnosis. Stairs are difficult but walking with my rollator and leg brace is ok. Balance is awful. Hands somewhat affected . I feel very fortunate that breathing, swallowing, and speech have not been an issue. Look forward to reading the forums- not just for information, but the positive attitudes that so many Pals inspire us with. Thank you and keep up the good fight!!
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Hi Amanda,
My wife (Vanessa) has been on a trial drug called RCH4. She has been on it for over 5 years now. Her ALSFR-S score has frozen at 45 after stabilizing when she started taking RCH4. The drug is struggling to get financial backing to get to FDA trials which is a shame but has recently been recognized by the FDA and given Orphan Drug designation. Hopefully, things move forward soon. My wife has SOD1 Familial and is the only remaining triplet after her sisters and mother passed very quickly after getting the disease. Here is a link to how well she is still walking. https://youtu.be/goobRa-3b7c
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You wait for a bus for hours, then two come along.
I start dosing in the Wave WVE-004 trial next Monday. And then read in ALS News Today about another genetic therapy targetting c9orf72 ALS being developed at SITtraN where I will be next week.
So I may well meet Guillaume Hautbergue, PhD, and find out more.
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I was just informed yesterday that Phase III Trial of AMX0035 is expected to start within a month in Europe (not certain, but expected).
I was asked by my doctor would I consider participating. However, I don’t know what would be the best decision –> if I choose to participate I can’t start with TUDCA now and there’s a chance of getting a placebo during the trial. So in that case a full year without any meaningful therapy.
Alternatively, I could ignore the trial and just take TUDCA on my own, perhaps with ibudilast (ketas) if I can obtain it via SocialMedwork. I’m in an early stage of ALS. Does anyone have a suggestion what to do?
As for the 2nd component of AMX0035 (SPB), although it is available in NL I can’t get a prescription from my doctor for it (as it is not proven for ALS). So without prescription is both unavailable and too expensive for me.
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I turned down the opportunity to participate in the TUDCA trial here in the UK for the very same reasons you outlined. I import the TUDCA from the US as it appears to be a better grade and value. I imported Ibubilast directly from Japan. I only take a low dose 10mg as a background medication because unlike the other trials I can take it in conjunction with other supplements/meds. Anecdotally, I would say that when I stopped taking the 10mg of Ibubilast deterioration felt like it speeded up. I can’t say the same for TUDCA but take it anyway at 500mg per day. I have also felt that liposomal Gluthatione (not all brands seem to be the same) has also helped. I would also like to point out that oxidative stress is a much talked about factor that may be implicated in ALS. However, if you overload the system with anti oxidants then you may be exposing yourself to reductive stress instead. I have found that I have probably made things worse at differnt points by taking too many anti oxidants and I am now at a point where I think I am going to get the balance right. The self experiment continues. It seems such a shame that I have to do this on my own without any support from the medical profession. I have asked for specific help and they do nothing once you have been diagnosed other than see you every six months to document your decline. I can’t help but feel angry if I think about it.
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I also I’m not doing a trial as I want to continue with all the other aspects of my care that I too have figured out for myself. I agree with you… You are diagnosed, patted on the head and told to get your affairs in order. Every six months they check my functional scale and clap their hands that I’ve only declined two points in one year. I will those say there is amazing support in the ALS society and incredible people to help along the way. But That’s not really my goal. My goal is to slow halt or reverse this nasty disease.
I am doing the AMX 0035 protocol- the Tudca and the phebrurane. It is very expensive but I am applying for some funding through our fair Pharma care system in Canada. In conjunction with this I do take the riluzole. My daily regime includes 20 minutes on a BeMer mat for improved circulation, an hour in my hyperbaric oxygen chamber, half an hour in an infrared sauna (135 degrees), yoga with some strength training and Walking my dog. 16 months ago I had stem cell therapy over a three week period.
I am sharing this because I think it’s helpful to see what other people are doing. Is it helping? Who knows! And there’s no way to tell if the AMEX 0035 is responsible for how good I feel or if it’s everything else I’m doing. I believe like the other person mentioned, it’s just a personal experiment of trying Different things and seeing how you feel.
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I’m usually not allowed to participate in drug trials because of my time since first onset but I am involved in a number if studies.
1. Everything ALS speech study where I record my speech weekly
2. Purdue University’s breathing study where I use an EMST breathing device
3. Mass General Hospital swallowing study
4. Mass General Hospital activity study where I wear watch type device to monitor my movement
5. ALSTDI where I record my voice and take the ALSFRS survey monthly
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I am unable to get into any drug trials. My ALS score is too low. I’m currently at 26/27 varies from month to month. I’m always excluded because of my score or I’ve had symptoms too long. I got my ALS diagnosis in Oct 2020 and symptoms started in 2018.
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