Request for advice regarding best treatment for C9

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Neil J

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I was wondering what is considered the current Best treatment for C9orf72? I was identified as having this condition in January 2022, and my neurologist is trying to get me into a LAM – 002 trial, but wonder if some alternate treatment that hopefully is currently available without entering a trial that may be better for treating this condition.
 

Nikki J

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If I qualified I would do the trial. I don’t so besides riluzole I think the things I take that mostly impact c9 are theracurmin for inflammation which is a big issue in c9 tudca for er stress and metformin which reduced c9 protein in the lab ( all these either at my neurologist’s recommendation or with her full approval)

the other issue is whether there will be an aso trial soon. The next part of WAVE is supposed to have US sites but I don’t know when and biogen will be reporting phase 1 within a couple of months. If it is positive there should be a bigger trial enrolling. I haven’t heard the same positive buzz in the patient community though as I did with phase 1 tofersen for sod1 so I am worried.

are you at UMiami? They can’t tell you trial data and don’t know the outcome anyway but they might respond to a question about should I wait? It would take a while for the next round of biogen even if it happens so if you are progressing fairly quickly waiting isn’t worth it and it would be very competitive to get in
 

Neil J

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My ALS clinic is the holy cross hospital in Fort Lauderdale Florida. Thank you for your drug and supplement recommendations. Next week I'll ask my neurologist to add metformin to the drugs I'm taking for ALS.
 

Nikki J

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remember any of the things I listed except Riluzole would disqualify you from the trial as they ate considered experimental for ALS
 

GXTrex

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remember any of the things I listed except Riluzole would disqualify you from the trial as they ate considered experimental for ALS
Cant you stop the experimental treatment like one month before the first trial appointment? There usually are limits how long you need to be off of something to participate in a trial
 

Nikki J

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Correct it is usually 30 days but op is considering a currently recruiting trial
 

GXTrex

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Do they schedule you for an appointment right away or is there usually a waiting list?
 

Nikki J

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if they are actively enrolling which I believe this trial is you would get first available appointment with the screener. This obviously depends on the clinic but it isn’t like getting an appointment for a regular clinic visit. I certainly wouldn’t count on 30 days in this situation especially as this is a very small trial.
 

steve613

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Hi Neil and others
Nikki as always is the source of the latest and most accurate information.
we are enrolled in the LAM 002 trial out of Mass General in Boston, which is still actively enrolling. Holy Cross in Fort Lauderdale was going to be a site but in the end dropped out. Our travel expenses are reimbursed at $100 per visit, which doesn't go very far to cover airfare, hotels and ground transportation. We favored this trial because the randomization is 2:1 drug to placebo in the first 12 weeks, and everyone gets study drug in the second 12 weeks. The duration is 6 months, so hopefully the WAVE study or the biogen study for C9 patients will be available later this year when we complete the LAM 002 trial.
The only ALS drugs permitted in the trial are riluzole and radicava, at least until AMX 0035 is approved by the FDA. We were taking TUDCA/sodium phenylbutyrate and had to stop it 30 days before enrollment in the trial, and were not permitted metformin either (since it was not for diabetes).
 

lgelb

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Phase 3 for tofersen failed, possibly because the patients were not early enough and maybe dosing was not long enough. So now there is an early access program that docs can refer patients to, in an effort to build up some better data.
 

Nikki J

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To avoid confusion tofersen is the aso for sod1 not c9 which is the mutation under discussion here. Although tofersen did not meet their primary endpoint there were some positive findings which is why there is an eap while biogen talks to the fda. Because of the things they saw in the trial biogen has continued with the ATLAS trial which is designed to treat carriers ahead of symptoms based on biomarkers to see if onset can be delayed or prevented.

the phase 1trial for biogen‘s c9 aso is due to report out soon. I am nervous about it as there hasn’t been positive buzz in the FALS community as there was with tofersen. It is true people are under an NDA but they were with tofersen as well
 

lgelb

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I'm sorry -- should not have imposed sod1 on c9. The c9 compound is tadnersen (also known as BIIB 078 or IONIS C9Rx). Biogen has an option to acquire it from Ionis. I did notice that the record for the extension trial, NCT04288856, was updated in February, which is hopeful that they had some efficacy signal in the original trial, or they could have canceled the extension. Also, Ionis' Jan. JP Morgan presentation still listed a readout this year, which if they knew data were negative, they might not have listed. But you are right, no buzz as yet.
 
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