Old 07-07-2018, 05:51 PM #1 (permalink)
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Wink SOD1

Thanks Nikki,
I have been following all the gene blocking trials for sod1.
I myself is sod1 h47r (a very very rare variant). All the research is going on with sod1 G93A mutations. My concern is that if it works for sod1 G93A, will it work for other sod1 variants as well.
Thanks



Search for antisense therapy, biogen, gene silencing. Antisense is being used for SMA as I said. It is in hopeful looking trials for HD and SOD1

My neurologist has consistently said it represents the best hope for my family. In fact she says don’t worry about the kids and is optimistic for my currently asymptomatic cousins who are all older now than our youngest onset age

It makes sense to me that if our ALS is caused by a mutant gene blocking the function that causes the damage should be the answer[/QUOTE]
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Old 07-07-2018, 05:58 PM #2 (permalink)
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Default Re: Hi all. TIANDB

The sod1 trial is not limited to a single variant. I know people in it with different ones. Did you try for it? Or do you not qualify? I see you are motor neuron disease other?

Are you talking about the mouse model used?
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Old 07-10-2018, 08:03 PM #3 (permalink)
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Default Re: Hi all. TIANDB

Its good to know that different variants of SOD1 are participating.
My neurologist wanted me to participate in a trial at Northwestern University Chicago, which is to collect blood from FALS for identifying different genes, which i did.
Haven't really participated in any gene blocking trial yet.
If anybody is interested to partipicate, its NCT00821132.
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Old 07-10-2018, 08:13 PM #4 (permalink)
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Default Re: SOD1

I moved these posts Ash because I want to talk to you but not hijack the other thread.

Sometimes people mask their location for privacy but if you are really in Boston where are you being seen that they are not discussing trials with you?

Even if you don’t currently qualify I am amazed they haven’t discussed the human SOD1 trial with you
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Old 07-11-2018, 11:50 PM #5 (permalink)
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Default Re: SOD1

We moved to Dallas from Boston couple of years back, and i was diagnosed at UT Southwestern Dallas.
I don't think they are having any SOD1 trials here.
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Old 07-12-2018, 04:52 AM #6 (permalink)
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Default Re: SOD1

You might not be eligible ( other mnd?) and it has waiting lists but people traveled for it and expenses were paid last I heard.

Even if your diagnosis doesn’t qualify you I am surprised you were not told that this is happening.

My doctor has been telling me about the equivalent therapy that is almost to trial in C9 right along.
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Old 07-16-2018, 12:27 AM #7 (permalink)
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Default Re: SOD1

Nikki,
I'm not pursuing to participate in any clinical trial right now, don't wan't to go through this hassle, although hoping and praying for any treatment/cure soon.

My focus is my young kids, trying to make everyday as normal as it can be for my kids while struggling to walk with progressively weakening leg muscles.

I am the youngest one (onset at 40) hit by this disease among all the known family members going back five generations.
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Old 07-16-2018, 06:28 PM #8 (permalink)
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Default Re: SOD1

Some SOD 1 News.. Mice/Rat Related but looks promising.

Quote:
Now, new research led by Washington University School of Medicine in St. Louis indicates an investigational therapy for an inherited form of ALS extends survival and reverses signs of neuromuscular damage in mice and rats. The findings, published July 16 in The Journal of Clinical Investigation, have led to a phase one/two clinical trial to investigate whether the drug could benefit people with ALS whose disease is caused by mutations in a gene called SOD1.

"This drug had an impressive effect in mice and rats with just one or two doses," said Timothy Miller, MD, Ph.D., the David Clayson Professor of Neurology at Washington University. "We don't know yet if this works in people, but we're very hopeful. We've completed the first phase of safety testing, and now we're working on finding the right dose."

About 10 percent of ALS cases are inherited. Of those, about a fifth are caused by mutations in SOD1. Such mutations cause the SOD1 protein to be overly active, which suggests that reducing protein levels might help ALS patients with SOD1 mutations.

Patients with ALS have few options for treatment. Only two drugs have been approved by the Food and Drug Administration (FDA) for ALS, and both only modestly slow the course of the disease.

In collaboration with Ionis Pharmaceuticals, Miller and colleagues tested DNA-based compounds that block the body from making SOD1 protein.

Miller and colleagues at Ionis tested two such compounds—known as antisense oligonucleotides, or oligos for short—in mice and rats. The animals were genetically modified to carry a mutated form of the human SOD1 gene. By a few months old, such animals start having trouble walking and feeding themselves.

Mice were given an anti-SOD1 oligo or a placebo at day 50, and a second dose about six weeks later. The mice that received the active drug maintained their weight 26 days longer and lived 37 days longer than those given the placebo, an increase in life span of 22 percent.

As a comparison, the researchers also tested the treatment in rats. The rats that received an active oligo fared much better than the ones that received the placebo. They maintained their weight more than nine weeks longer and survived eight to nine weeks longer.
https://medicalxpress.com/news/2018-...-survival.html
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Old 07-17-2018, 01:28 AM #9 (permalink)
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Default Re: SOD1

Ash, I would encourage you to meet with a coordinator and learn more, for a more informed decision about the potential benefits, risks and logistics of any trial for which you're eligible. The treatments under test certainly have some promise, and your kids surely would want you to explore every plausible road to more years at home. It doesn't take much time to send an email or do a meeting.

TIANB, please don't paste long blocks of text, especially press releases. You can always summarize and provide a link.

Best,
Laurie
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