Edaravone feedback on its effectiveness

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SEAMUS

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Hi first time on this forum. Seamus from Ireland diagnosed1year but symptoms about 4 years, 49 married 4 kids. What I would like to ask people is about Edaravone. It has been in use in the US since 2017. It is not available in Europe yet but there is a large clinical trial this year on the oral version of it. The clinical trial here in Europe called ADORE is looking at oral EDARAVONE in tablet form 3 times daily. They have conducted a phase 1/2 trial looking at safety which had similar side effects and it also came back with 60/80% bioavailability which means no drug holiday period and 2.8 times more drug administered per month. Here in Ireland they are recruiting early this year but the exact protocol has not been finalized. It sounds simple that if your getting 2.8 times the drug will it have 2.8 times the effect who knows but hopefully it might increase its potency. Can people who have used it pass on what they think of it ,does it work , does it make a difference, in other words is it worth trying. I also suffer from Asthma does that make a difference ? Any info you could pass on would be very helpful . Best wishes and keep fighting.
Seamus
 

lgelb

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Welcome, Seamus, while sorry you are here.

Unfortunately, most drugs have a tipping point where going past a certain blood level does nothing more to help, and/or the side effects of that more effective dose make that dose unworkable for too many people to recommend or market.

Whether the oral form will have better, worse or equal efficacy to the IV form in terms of its optimal dose is not yet clear. Bioavailability (blood levels) do not equate to slowing progression.

Note that the Phase I trials for this oral form ended years ago but no Phase III trial timeline, as would be needed for marketing, has been announced. Funding and staff may be issues.

People with asthma are more subject to sulfite sensitivity, which can occasionally happen with Radicava, so you would just need to be cautious during initial dosing.

There is a search box at the top right of this and other pages, where you can type in edaravone or Radicava, and there is a thread here.

Best,
Laurie
 
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affected

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I think that if anyone is still using this it is well worth hearing where they are at with effectiveness of it now :)

Welcome Seamus!
 

diagnosed2016

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We are still on it. Started first course Oct 2017 and still going. It doesn’t stop progression but he’s still here, so....
 

Wilson2009

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I started in Oct 2017 as well. As measured using the ALSFRS-R, I have had no progression. Prior to starting Edaravone, I had lost at least two points on the ALSFRS-R since my diagnosis in January 2017.

However, I have noticed continued deterioration over the course of the past year. My FVC was at 98% during the last pulmonary visit. However, my FVC had been testing over 100% before the last test. I can tell that my speech has deteriorated over the past year, but not to the point where I can't be understood by repeating slowly.

As I have said before, I have no idea if Edaravone is stopping my progression, but I am not about to stop taking it to find out.
 

sassy

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My husband has been taking radicava since August 2018 and has not had any progression. Although, he has lost more muscle mass in one leg. Like others- we are unsure if his slow progression is related. His next clinic visit is in two weeks and we will see if his dr concurs.

Sassy
 

adrivtham

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The experience we have with edaravone was that at the beginning of the illness it appeared that it helped because my husband's speech seemed better but then he went down very fast. It varies so much from patient to patient. I
 

Bestfriends14

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My husband has been on Edaravone since October 2017. When he was diagnosed in May of 2017, is ALSFR was 45; today it is 43. We do think that it has helped slow progression (it was never meant to stop progression), and for that we are grateful. However, the next clinic appointment is in April and I do think there will be a reduction in the ALSFR score. My husband now has double drop foot, weakness in his left pinky and ring finger and no longer walks outside but uses his scooter.

For whatever time Edaravone has given my husband, I do think it has been more than would be without it. Since starting Edaravone, we have managed to travel all over the world and I do not think that would have been possible without it.
 

SEAMUS

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Thanks very much for your information, it is very helpful and I would be very grateful if you keep it coming. There is another trial starting here were they are looking at antiviral drugs which might be worth looking at called Lighthouse 2. It has shown some promise but I think its only in patients with a certain mutated gene. I am lucky enough to attend a top Neurologist and researcher and precision medicine definitely seems the way forward. As in the right drug for the right patient at the right time, is how she explains it. I hope this helps keep up the fight
Seamus :
 

Gorby

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Glad to hear they have made progress on the oral formulation. I believe this is the study Seamus is talking about:
 
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diagnosed2016

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Interesting that those who started early on after being diagnosed have had little progression on the als scale. We have definitely had more or a drop in score since starting but were also 2 years in
 

SEAMUS

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Hi thanks for info, so the feedback seems to be generally positive to a certain degree with regard to Edaravone. Is it that its more effective in slow progressors? Does anyone have anything to say about it?
The more I read about trials the more it makes sense to stratify PALS into cohorts and not cherry pick all the still healthy ones only. Allow most people to enter trials and take baselines values to place them into cohorts and at least see what works for who and what doesn't or look at different doses according to disease progression etc. I feel so fed up with people telling me to keep fighting but what do we fight with, we need quicker better accessed clinical trials and outcomes better understood. I would rather find out something doesn't work for me suck it up and move on to the next thing than be refused entry into a trial. I read on ALS news 60% of us are ineligible for clinical trials, how does that compare with other diseases?
Anyway forgive the rant thank you for the info please keep it coming We don't have any form of Edaravone legally available here in Europe so we really don't have anything to go on only what we hear from the USA and the clinical trials.
Best wishes and KEEP FIGHTING
Seamus;)
 

Nikki J

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The issue of clinical trials is very complicated. The trials are of course not for the participants but to prove that the drug works or doesn’t in the most efficient manner. I think the brutal truth is probably that even if something works it isn’t going to do much for the most advanced PALS. The more diversity in your group too the bigger and longer the trial will need to be

That said, the doctors are looking at trials too trying to make them better. Things that are talked about - the need to use biomarkers as much as possible. If we can find something measurable ( like blood sugar for a diabetic) it would be much more efficient. They also talk about using biomarkers to pick participants for therapies. If a drug targets inflammation it makes sense to have participants with biomarkers for inflammation. A more extreme example is gene therapy. They are recruiting for sod1 and c9 gene blocking therapies one of the entry entry criteria for each is having that targeted mutation.

My doctor spoke last fall at a meeting about a new kind of trial. Her proposal is to have a trial with multi arms and one control group. The arms would look at different therapies and there would be the possibility of moving people into another arm if their treatment arm failed. She said this had been used in cancer trials somewhere. It would be complicated to set up and would need cooperation among the various sponsors but would move more potential therapies more quickly.

The Edarovone studies only showed benefit in a certain group - early ( within 2 years) with decent function and good breathing but progressing fairly quickly. The original more inclusive trial failed and it was only because they anslyzed subgroups that they did another trial with only that subgroup and showed benefit. The big question remains will it help other groups such as naturally slow progressing PALS. Have you talked this over with Professor Hardiman who I suspect is your doctor?
 

Kristina1

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I did 6 cycles of Radicava and then discontinued by choice. I did not feel it was helping me, and, whether by coincidence or not, my progression actually sped up during that time period. I was also having bad migraines (a known potential side effect). While it seems to help some PALS, it was not a good match for me.
 

SEAMUS

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Hi Folks thanks for information. I think Nikki is right it is a case of different horses for different courses. As my Neurologist who is Professor Hardiman says she thinks precision medicine is the way forward. THE RIGHT DRUG, FOR THE RIGHT
PATIENT, IN THE RIGHT DOSE, AT THE
RIGHT TIME. So absolutely we need Biomarkers but also imaging and cognitive behavior could be used. She is the lead investigator for the oral edaravone trial here in Ireland. They have not published the entry protocol yet but it is supposed to start soon I will keep info updated on it and hopefully it will answer questions on its efficacy and with who.
Thanks again, best wishes and KEEP FIGHTING
Seamus
 
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