[Owen]

For me, Radicava falls into the same category as Riluzole- marginally beneficial. My wife has taken Riluzole basically since diagnosis, but decided against Radicava because the cons (needing a port, needing to have someone come to the house to do infusions daily for weeks) outweighed the pros (slowed progression by ~3 months). With a liquid form that can be administered easily by any caregiver, that changes the calculation significantly. Unless it’s prohibitively expensive, or not covered by insurance, it would make sense to add it to the regimen.

[Owen]

I would recommend talking to your doctor about trying Tizanidine (Zanaflex). My wife takes 4mg at bedtime, and another 4mg in the middle of the night when she wakes up to go to the bathroom. I would also look at either OTC sleeping pills (e.g. Aleve PM, melatonin), or prescription pills like Ambien- something to help him stay asleep even though he’s having spasms. Lastly, you might also want to consider trying something like Xanax or another b<span class=”aCOpRe”>enzodiazepine. My wife takes them occasionally to deal with anxiety, but they have the added benefit of helping her sleep very soundly, and she wakes feeling relaxed and rested. I haven’t seen much about Xanax and ALS, but considering how much it seems to help her at night, I think Xanax could merit being suggested by doctors.</span>

If we’re talking about cramps and not just spasticity, you can try a potassium supplement, and/or magnesium and Vitamin E.

[Owen]

My wife uses weed butter (a high CBD/low THC flower infused into butter), and we’ve come to think of it as a medical necessity. The days she takes it versus the days she doesn’t are noticeable, and it greatly helps with her stiffness and spasticity

[Owen]

The hardest part for me is that we’re young for ALS. My wife and I are both 39- so on top of seeing all your dreams crushed and dealing with the grief, I’m working full time, we have a 5 year old son, and now I’m a full time caregiver and paperwork jockey. I suppose we’re fortunate in a way that I can work from home, but there are definitely days when I don’t know how much longer I can keep this up.

I’ve found the ALS resources to be pretty lacking, but then again, what I’d really like is someone to live with us full time and take care of half of this! My biggest lesson learned is to accept help when offered. It took a while to get to that point, but family and friends have been generous with their time when they can, and that’s been helpful.

[Owen]

I personally would choose the Healey platform trial if it’s available to you. I’m hoping my wife can get enrolled at our local site. It seems to have a few benefits: 1) the platform reviewed something like 40 possible options, and picked the most promising 4-5 to test, so you’re already starting from a more advanced position, 2) they will be testing multiple drugs at once, so there is a higher likelihood patients might receive something beneficial, and 3) I firmly believe that if/when the ALS nut is ever cracked, it’s going to be through a cocktail of drugs, not a single magical bullet, so I’m hoping a breakthrough like that occurs here.

[Owen]

We bought a used Dodge Caravan that had been converted already by Braun. It has the middle row removed, anchors in the floor for the chair, an automatic sliding door, and an automatic ramp that folds out. All in all a very simple, but practical and functional solution. We don’t have a lift, but a ramp seems much easier to me. I’m not sure what other solutions would be available given that power chairs typically weigh 300-400 lbs. You might be able to attach a small trailer or platform off the tow hitch of your existing car (if you have one), but that has its own challenges.

New or slightly used accessible vans run $40k-$50k+ we found, so we ended up buying an older model with low mileage through a private sale from the widow of a former ALS patient (our local ALS chapter connected us).

[Owen]

Here’s an ALS Untangled article you might want to read: https://www.tandfonline.com/doi/full/10.3109/21678421.2015.1088707

It’s in regard to this supplement (https://www.lifevantage.com/us-en/shop/protandim-nrf2), which also contains Ashwagandha. It sounds pretty similar to your Dad’s supplement. It got a promising (and rare!) “A” rating on mechanism, but it hasn’t really been studied so the ALS untangled team wasn’t able to recommend it (they did recommend it for further study however).

I’d be interested in learning more too if anyone has any thoughts.

[Owen]

Hi Igor,

You might try contacting the ALS Therapy Development Institute. They have a form you can fill out, but I also spoke with Dileep there (contact info below), who could help put you in touch with the right people. I think the problem you’re going to face is that most organizations either a) rely on grant money for specific investigations, or b) have already programmed the funds they do have. You could try reaching out to some of the more well-known ALS doctors in the US. I emailed Dr. Richard Bedlack ([email protected]) out of the blue about a topic, and he responded to me.

I like the idea of crowdsourcing research- I haven’t heard of something like that done before. But I suspect there could be a lot of complications with essentially starting up a grant-making organization.

Dileep Dadlani

Development Director – Southeast Region

ALS Therapy Development Institute

[email protected]

Office    617.441.7283

[Owen]

Here’s my thought on this subject. From what I’ve read, and what my wife’s doctor at Johns Hopkins said, mouse models are really of minimal use. I think they’re typically used to justify moving on the human trials, but they’re of such little use that it’s an unnecessary delay more than anything else. We still know so little about ALS, but we know enough that we should be able to make some educated guesses about drugs or types of drugs with plausible mechanisms. From there, they should do a safety study and then quickly move to Phase 2. The one thing ALS patients don’t have is time, and they have very little to lose by trying something new.

I personally think more research needs to be done on trying drugs in combination. I think it’s fairly unlikely we’ll ever discover a miracle drug that cures ALS. I think it’s much more likely we could find a cocktail of drugs that either a) slows the progression way down, or b) turns the illness from terminal to chronic, but manageable.

[Owen]

Hi Danielle- My first and main question is, have you noticed any improvement?

[Owen]

Thanks Chuck! Anyone have any experience with any of the orgs listed here? https://iamals.org/encyclopedia-of-als-organizations/

[Owen]

Thanks Jonathan- that definitely helped! I never knew you could expand the contacts list in the studies. Fingers crossed the trial is able to start soon!

[Owen]

Jonathan- could you explain what you mean by “virtually standing in line?” I haven’t seen a link anywhere to register or otherwise get in line. We’re in DC, and close to UMD and JHU, both of which will be participating in the platform trial too.

[Owen]

Where is the trial taking place, and who contacted you about participating? My wife is still early in her progression, so it would be great to get her involved with any studies taking place.

[Owen]

Hi all- I’d be interested in hearing an update on whether or not Ibudilast has helped at all. The Phase 3 trail has been pending for a while now.

I’d also be interested in hearing if anyone knows the dose and ratio AMX 0035 used between TUDCA and sodium phenylbutyrate (i.e. how many mg of TUDCA, how many mg of sod phenyl), and where you could buy sodium phenylbutyrate in the US online?

[Owen]

My wife has been working for the past year, but is just about to go on short-term disability, and then that might be it after that. She gets tired more easily and driving (which she does a lot of) is becoming more of a challenge, but most of the issues have been around using a computer, as she’s lost most of the use of one hand, and part of the other.

I would say the biggest challenge has been around reasonable accommodations under ADA. Despite submitting the request and doctor letter in October 2019, her work has been dragging their feet/fighting her on basic accommodations like speech-to-text software and flexible work hours/additional telecommuting. It’s been a pretty disappointing process, and my wife is going on STD out of frustration as much as anything. My recommendation to others would be to start the process as early as you possibly can, and shoot to secure accommodations such as: Nuance Dragon Professional (speech recognition software‎), a flexible work schedule and/or additional work from home days for when you’re tired or have medical appointments, handicapped parking (if you have the tag) and possibly a device like an iPad if you move around a lot for work. And if you’re not already signed up for short-term and long-term disability insurance, do so as soon as possible at your work’s next open enrollment event.

[Owen]

In addition to what’s in the ALSUntangled review, here is what makes me skeptical about RCH4: if you’re developing a treatment for ALS, you’re either doing it to make money, or you’re doing it because you’re passionate about solving ALS (or some combination of the two). In either case, your objective is to get your treatment in the hands of as many doctors as possible so they can prescribe it to their patients. The way you do that is through clinical trials, peer-reviewed journal publications, and ultimately, FDA/regulatory approval. They claim they don’t have the funding to conduct clinical trials-which is plausible-but I find it exceedingly hard to believe that the medical community as a whole would choose not to investigate further if there was even a slim chance RCH4 is effective.

[Owen]

Just based on phase and study completion date, I’d put these five up at the top of the list for potential approval:

• NurOwn: https://alsnewstoday.com/nurown/; https://clinicaltrials.gov/ct2/show/NCT03280056 (study completion date: Oct 2020/Dec 2020)
• Arimocolomal – genetic only: https://alsnewstoday.com/arimoclomol-orph-001/; https://clinicaltrials.gov/ct2/show/NCT03491462 (study completion date: Dec 2020/Jan 2021)
• Tofersen (BIIB067) – genetic only: https://alsnewstoday.com/ionis-sod1-rx-biib067/; https://clinicaltrials.gov/ct2/show/NCT02623699 (study completion date: May 29, 2020)
• Ibudilast: https://alsnewstoday.com/2019/06/07/enrollment-starting-soon-in-phase-2b-3-trial-of-ibudilast-for-als/; https://clinicaltrials.gov/ct2/show/NCT04057898 (study completion date Dec 2021)
• TUDCA: https://alsnewstoday.com/tudca-tauroursodeoxycholic-acid/; https://clinicaltrials.gov/ct2/show/NCT03800524 (study completion date: June 2021)

In terms of the ones I’m most excited about, I’d say NurOwn and Ibudilast. There’s also a Phase III trial of ultra-high dose methylcobalamin (B12) in Japan that should be publishing results in the next six months: https://clinicaltrials.gov/ct2/show/NCT03548311

[Owen]

Hi all- I’m a new member, and thought I would piggyback off of this conversation. My wife was diagnosed with ALS recently, and I have been conducting research in a number of areas to try and wrap my head around the massive amount of information on ALS. One area I was looking into was supplements, and whether or not there is anything promising in this area. Below is a dump of research I’ve done on supplements. I’d be interested in hearing from people here 1) if anything on this list has been recommended to you by an ALS-focused neurologist, 2) if anyone has any personal experience with anything on this list and could comment on perceived benefits and side effects, and 3) if anyone has guidance from doctors or research studies on dosing levels. Thanks!

<u>Vitamins and supplements</u>

• Good overall summary: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4712627/
• Good overall summary of metabolic treatments: https://www.frontiersin.org/articles/10.3389/fnins.2016.00611/full
• Big list of ALS supplements: https://alstuttu.org/wiki/index.php?title=Information_on_nutritional_supplements_people_with_ALS_have_been_taking
• Human studies with promising results:
  • Vitamin D & E: https://alsworldwide.org/care-and-support/article/supplements-and-vitamins
  • Methylcobalamin (Vitamin B12) – high dose of 25-50mg: http://informahealthcare.com/doi/pdfplus/10.3109/21678421.2015.1070574
  • L-carnitine: https://www.ncbi.nlm.nih.gov/pubmed/23421600
  • L-Serine: https://brainchemistrylabs.org/lserine
  • Tauroursodeoxycholic acid (TUDCA): https://alsnewstoday.com/tudca-tauroursodeoxycholic-acid/
• Mouse models with promising results
  • Ginseng: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3659610/
  • Extract of ginkgo biloba (EGb761): https://link.springer.com/article/10.1385/JMN:17:1:89
  • Catechin in green tea (Epigallocatechin gallate): https://www.ncbi.nlm.nih.gov/pubmed/16356650; https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3585739/
  • Alpha-Lipoic acid – specifically R(+)Alpha lipoic acid: https://www.ncbi.nlm.nih.gov/pubmed/29842900
  • Nicotinamide mononucleotide (NMN): https://alsnewstoday.com/2018/01/19/study-shows-symptoms-of-mice-with-als-improve-after-they-are-treated-with-enzyme/
  • Ashwagandha (SOD 1 variant only): https://www.ncbi.nlm.nih.gov/pubmed/30134145
  • Deanna protocol: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4111621/; http://informahealthcare.com/doi/pdf/10.3109/21678421.2013.788405
  • Cannabinoids: https://www.researchgate.net/publication/329447341_Effects_of_cannabinoids_in_Amyotrophic_Lateral_Sclerosis_ALS_murine_models_A_systematic_review_and_meta-analysis
• Mixed (but not necessarily negative) results
  • Protandim – potentially sketchy but may have some promise: http://www.tandfonline.com/doi/pdf/10.3109/21678421.2015.1088707
  • Theracurcumin – oral: https://www.tandfonline.com/doi/pdf/10.1080/21678421.2018.1440738?needAccess=true