ALS Research

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New member
Nov 30, 2020
Lost a loved one

First father died three years ago from ALS (U of Michigan diagnosis), his brother died the year prior from ALS (U of Iowa diagnosis), their grandfather died from ALS (Mayo Clinic diagnosis). I haven't kept up as I ought as regards new or emerging therapies, I recall when Dad was diagnosed there wasn't even much thought given to familiar ALS.

I'm sure there are participants here who actively review new/emerging research and perhaps could share credible sites? Neither I nor my brothers are fixated on the liklihood of diagnosis, but we're all approaching the age where symptoms with Dad began to show. Are there any interventions with promise?

Many thanks;

Short answer. Yes.

what is your family mutation? Surely U of M offered testing?

there is a ton of genetic research. Sod1 has a therapy in phase 3 that has showed a lot of promise. I expect there will be a prevention trial next year probably early next year for documented carriers

a similar therapy is in late phase 1 for c9. If all goes well eventually there will be carrier trials for that too. Other c9 therapies are expected to go to trial soon.

you and your brothers can help. There are several studies of genetic carriers searching for early biomarkers so preventive therapy can be given in a timely fashion
None of us have been tested -- Dad insisted on managing his relationship with UofM without family involvement (after we got him there for a workup). Genetic testing advances continually, and frankly I've put it off waiting for greater discrimination in the results, but I am willing to participate in any credible effort or research to advance the knowledge.

If you've any guidance/referral I would be glad to follow up and speak to my brothers.
Is there any possibility he was tested? The executor of his estate can get medical records. Of the three big presymptomatic studies 2 require knowing the family mutation. ( not your status ).

the two most common mutations in the US are c9 and SOD1. If your family is European descent and onset age is over 50 c9 is likely ( c9 strikes younger too but sod1‘s most common variants usually hit by midforties).

if your mutation is not known the study that would be appropriate is DIALS at Mass General in Boston and Wash U in St Louis. They will test for all known mutations. You can learn the results or not. They do not pay travel but confirmed mutation carriers can do other studies at MGH that do help with costs. I will pm the coordinator’s email

the biomarker data is critical to presymptomatic intervention so if you are able participating would be valuable. I think you would find it rewarding too
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