ALS (Lou Gehrig's disease) drug approval by FDA

[LadyStanley]

ALS drug wins FDA approval despite questionable data

A much-debated drug for Lou Gehrig’s disease won U.S. approval Thursday, a long-sought victory for patients that is likely to renew questions about the scientific rigor behind government reviews of experimental medicines. The Food and Drug Administration approved the drug from Amylyx...

news.yahoo.com

Science marches forward

Click to read more...

 

[adsfan]

ALS drug wins FDA approval despite questionable data

A much-debated drug for Lou Gehrig’s disease won U.S. approval Thursday, a long-sought victory for patients that is likely to renew questions about the scientific rigor behind government reviews of experimental medicines. The Food and Drug Administration approved the drug from Amylyx...

news.yahoo.com

Science marches forward

It is amazing how little progress has been made on ALS since 1939.

A professor at my old school got ALS. After a year, he was in an electric wheelchair like Steven Hawking. The second year he couldn't hold his head up. There was no third year. He was a big man, he looked like a pro football player, like a defensive lineman. That disease just destroyed him,

I think that anybody with ALS will try anything.

Does anybody know why Steven Hawking was able to live 50 years with ALS?

 

[JMCx4]

... Does anybody know why Steven Hawking was able to live 50 years with ALS?

You aren't the first to ponder that. See this NBC News story from the time of his death ...

“His survival is longer than most,” said Dr. Jeffrey Elliott, chief of the neuromuscular disorders section at the University of Texas Southwestern Medical Center. “I think part of his longevity may have been because he had a slowly progressive form. Probably it was also due to the exclusive nursing and medical care that he received.” ...

People diagnosed when they are young, as Hawking was (21 years of age), live for longer for reasons that are not well understood. It may be that a different cause of the disease shows up in young adults.

“One type of ALS is caused by a change in gene A, another by gene B. There are different causes, also, for sporadic ALS,” said Elliott. ...

 

[LadyStanley]

The Ice Bucket Challenge wasn't just for social media. It helped fund a new ALS drug

In 2014, it was hard to miss the ALS Ice Bucket Challenge that set out to raise money to fund research for the disease. And it paid off. A new treatment was funded by $2.2 million of the funds raised.

www.npr.org

Awesome that phenomenon resulted in new drug.

 

[beowulf]

It is amazing how little progress has been made on ALS since 1939.

A professor at my old school got ALS. After a year, he was in an electric wheelchair like Steven Hawking. The second year he couldn't hold his head up. There was no third year. He was a big man, he looked like a pro football player, like a defensive lineman. That disease just destroyed him,

I think that anybody with ALS will try anything.

Does anybody know why Steven Hawking was able to live 50 years with ALS?

There are different kinds of ALS and while it is deadly pretty fast to most as 80% die within 2-5 years of diagnoses. Some 20% can live a lot longer as the disease seems to affect them as a slower pace and this 20% can live for over 10 years, sometimes much more, but why exactly is still not really understood.

 

[TheTotalPackage]

It is amazing how little progress has been made on ALS since 1939.

A professor at my old school got ALS. After a year, he was in an electric wheelchair like Steven Hawking. The second year he couldn't hold his head up. There was no third year. He was a big man, he looked like a pro football player, like a defensive lineman. That disease just destroyed him,

I think that anybody with ALS will try anything.

Does anybody know why Steven Hawking was able to live 50 years with ALS?

I had an uncle who got ALS. Typical old-school European who was strong/thick-headed, tough as nails, his handshake was like shaking a brick. Then ALS wilted him to mush. Toughest part was that everything was fine upstairs -- just that the motions of his body wouldn't follow. Really sad to see.

It's unfortunate, as you say, that after all these years very little advancement has been made, and that a diagnosis of ALS is still a death sentence with a limited time to it.

 

[BigBadBruins7708]

It is amazing how little progress has been made on ALS since 1939.

A professor at my old school got ALS. After a year, he was in an electric wheelchair like Steven Hawking. The second year he couldn't hold his head up. There was no third year. He was a big man, he looked like a pro football player, like a defensive lineman. That disease just destroyed him,

I think that anybody with ALS will try anything.

Does anybody know why Steven Hawking was able to live 50 years with ALS?

There's "classic" ALS which is the quicker death version and there's PLS which progresses much slower and doesn't attack the heart/breathing as quickly.

My grandfather had PLS and had it for 15 years before he passed from it

 

[Carey Chant]

Why is the FDA approving it if it has questionable data? Shouldn't it take years of testing before it's approved?

 

[JMCx4]

Why is the FDA approving it if it has questionable data? Shouldn't it take years of testing before it's approved?

On this rare occasion, the U.S. FDA showed they have a heart. Increasing public pressure over the last few years no doubt softened them some, too, especially when faced with a chance to change the course of a deadly incurable disease that carries a high level of recognition.

 

[beowulf]

On this rare occasion, the U.S. FDA showed they have a heart. Increasing public pressure over the last few years no doubt softened them some, too, especially when faced with a chance to change the course of a deadly incurable disease that carries a high level of recognition.

This pretty much. People with this kind of disease know they have a death sentence so trying something that shows a lot of promise is worth it since they are likely going to suffer and die soon anyways.