Hype or not?

[konagirl]

We are new to this world, as you all were at one point. I am embarrassed to say, but before the letters ALS were mentioned to us by a neurosurgeon, and I began researching, I had never really even heard of it other than the ice bucket challenge.

Now, I see articles all the time, hear news snippets, hear stories of others, not just on here but just in normal daily life. It seems like ALS is everywhere. Is it just that I have awareness now, so I am paying attention? Or is there actually more out there in the public now about this horrible disease? For those of you who have been battling this for a longer time, have there always been so many clinical trial options and a full pipeline? Or is there really that much new research surfacing?

 

[Bob R]

We are new to this world, as you all were at one point. I am embarrassed to say, but before the letters ALS were mentioned to us by a neurosurgeon, and I began researching, I had never really even heard of it other than the ice bucket challenge.

Now, I see articles all the time, hear news snippets, hear stories of others, not just on here but just in normal daily life. It seems like ALS is everywhere. Is it just that I have awareness now, so I am paying attention? Or is there actually more out there in the public now about this horrible disease? For those of you who have been battling this for a longer time, have there always been so many clinical trial options and a full pipeline? Or is there really that much new research surfacing?

There are many trials underway. Check out https://clinicaltrials.gov/ search for als. Unfortunately, there doesn't seem to be any urgency to any of it. A trial can take 2 - 3 years in preparation, trial, and results. Then it's time for phase II. And then, even after phase III, usually he final phase, years more before the FDA approves anything.

Edavarone, a drug approved in Japan for als, is not available in the U S. And Masitinib, another promising drug, is under consideration for release in Europe, but not here. I'm sure I'll get criticism for this, but the problem, I believe is that our FDA is controlled by big pharma companies, whose goal is to male a profit, not cure diseases.

 

[Nikki J]

It is some of both I think. There has been a step up in research findings plus a boost to getting trials going from the Ice Bucket
But ALS has been here and trials have been going on.

I think the underlying science has advanced certainly in genetics but also in other areas. That is helping. We are probably close to getting a couple of biomarkers which will be huge. If anyone has an opportunity for a biomarker study please consider it. A decent biomarker would both speed diagnosis but also make clinical trials faster and less expensive.

 

[GregK]

There is much more awareness now, following the Ice Bucket Challange.

And there seem to be many more trials going on, most likely for the same reason.

Trials take forever; choose your favorite conspiracy. My guess is the cause is twofold: the FDA is a typical beauracracy and the drug companies have to leap backwards thru hoops trying to avoid lawsuits. Daytime television is clogged with tort lawsuits over drugs and medical devices.

In Europe if you lose a lawsuit you have to pay the other sides costs. Makes nuisance suits less attractive.

 

[konagirl]

Nikki, we have talked with our Dr about Biomarker testing and it is something we are going to do soon.

Please excuse my ignorance, I am learning as much as I can and trying to wrap my brain around all of this.....but if a drug is a approved in another country, and not here, can we go to that other country and get our hands on it still?

 

[GregK]

I believe that depends on the country.

Mexico seems pretty much open but there are residency issues elsewhere.