Disease progression halted in rat model of Lou Gehrig's disease

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I'm not a mouse, but this sounds promising nonetheless!
I do have a question for you research-saavy types here; if TDP-43 was first 'discovered' in 2006, is a 2011 study like the one shown in the attached article fairly representative of the timeline for medical research/testing?

5 years or 50 years, I hope this latest testing leads to more potential treatment options for those of us who don't eat cheese and run a theme park in Orlando!
 
It can take 10 years for a drug to get to market.

Sure wish I was a mouse or rat -- lots of options for them to be cured! :roll:
 
Roll on the research for a cure for sporadic Als!
 
Pray for a cure!
 
How about we do something about getting compassionate access to drugs on a faster track! We, the ALS-Treat Us Now! Steering Committee are dedicated to do just that! We need many signatures on our petition. And we could use some volunteers to help us out.

Please sign our online petition: **removed email/address harvesting**

THANKS!
 
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Jeff Lang, Lobster:
The average time to market is about twelve years, counting only successful drugs . . . a new molecule has about a one in fivethousand chance of making it. The actual lenght of time will depend on complications that are discovered, et alia. There are parts of the process that can not be made to develop more rapidly.
 
SO IF IM CORRECT ON WHAT I READ THEY USED doxycycline TO SUPPRESS THE GENE AND doxycycline IS A ANTIBOTIC USED IN THE TREATMENT OF LYME DISEASE.
 
Not following you clearly here, zac111. Who is the "they" that used doxycycline to suppress a gene? And where and when did they do it?
 
neurons. Compared with NEF-tTA/TDP-43M337V rats (Figure 2), ChAT–tTA-9/TDP-43M337V rats displayed a more severe loss of motor neurons, but displayed a similar progression of paralysis phenotype (Figure 6). We determined whether disease progression could be halted by stopping mutant TDP-43 expression after disease onset in ChAT–tTA-9/TDP-43M337V rats. When Dox was withdrawn from ChAT–tTA-9/TDP-43M337V rats at the age of 60 days (Figure 6A), the rats developed an early paralysis phenotype by 70 days of age and reached disease end stages by 78 days of age (Figure 6, C–F). Dox was thus withdrawn from rats that were 60 days old and was added back 12 days later, so that mutant TDP-43 was expressed in the rats from 63 to 77 days of age (Figure 6B and Figure 7B). We refer to restoring Dox as a treatment. Dox-untreated and Dox-treated rats all developed paralysis at comparable speeds before Dox produced an effect on disease gene expression (Figure 7C). Soft food was provided to rats with paralysis. While the disease rapidly progressed to end stages in Dox-untreated rats, the disease stopped progressing at paralysis stages in Dox-treated rats (Figure 7, C and D). Within a few days, Dox-treated rats began to gain body weight and to gain partial recovery of motor activity (Figure 7, C and D). Dox-untreated rats were terminated at disease end stages (by age of 80 days), and Dox-treated rats were terminated at 90 days of age.

In Dox-treated transgenic rats, H&E staining of gastrocnemius revealed remodeled motor units: some muscle fibers with centered nuclei, muscle fibers of varied sizes, and absence of atrophied muscle fibers (Figure 7, E–G, and T). Histochemistry for nonspecific esterase (Figure 7, H–J) and ATPase (Figure 7, K–M) showed that muscle cells of the same types, but with varied sizes, were grouped, suggesting a remodeling of motor units. During functional recovery, muscle fibers increased in size (Figure 7U). Toluidine blue staining revealed that most degenerated motor axons were cleared in the ventral roots (Figure 7, N–P). Interestingly, stereological cell counting revealed that the number of spinal motor neurons (L3–L5) between Dox-treated and Dox-untreated transgenic rats was similar (Figure 7V). These findings suggest that degenerating motor neurons cannot be rescued and that survived motor units can be remodeled.
 
SO IF IM CORRECT ON WHAT I READ THEY USED doxycycline TO SUPPRESS THE GENE AND doxycycline IS A ANTIBOTIC USED IN THE TREATMENT OF LYME DISEASE.

If only it could be this simple...I will bring this article to our next appointment with our ALS/Neuro
 
OK, so the material zac111 is quoting comes from the original article, which can be viewed here.

glimmer, it's very likely not this simple. There have been several compounds that tested well in the ALS mice that haven't worked in humans, including a chemical cousin of doxycycline called minocycline. Minocycline actually caused PALS' conditions to get worse, while it made ALS mice get better. Quite frankly, the wee beasties require very careful handling to provide usable and transferrable data.

ALS Research Roundup January 2008 | MDA/ALS Newsmagazine

A final note for those of you beating the Treat Us Now drum: the harmful effects of minocycline on PALS were discovered in the Phase 3 trials. The Phase 1 and 2 trials appeared to show safety and some efficacy, but the end result was that minocycline was harmful to PALS. That's one big reason why many of the top researchers and clinicians don't support short-circuiting the clinical trials process.

When the Phase 2 trials appeared to show some efficacy from minocycline, an unknown number of PALS decided to go "off-label" with minocycline, since it was already an FDA-approved drug. We'll never know how many of those PALS shortened their days on this earth because they rolled the dice and lost.
 
Trfogey is 100% correct. After I miracuously showed improvements and my doctors were shocked and suprised I said to them I know this is a safety study but from what I read it showed promising results in mice and rats. My Dr.'s response was he is not even sure why they use mice and rats as so many things that work well in mice and rats have zero effect on people. So until it shows promise in people the prudent thing to do is take a wait and see attitude.
 
Rats and mice are easy to come by, cheep to keep and hardly anyone cares when they are locked in cages their whole life and then die nasty deaths. Beagles and chimps are hard to come by, expensive to keep and most everyone cares when they live their whole lives in small cages and die nasty deaths. I totally agree about wait and see.
 
Thanks Trfogey and TedH5 for elaborating and your info. I understand what you are saying. I'm always looking for bits of hope to hold onto so I appreciate the clarification :) I am amazed at how much info I have read in the short time we have had since being confronted with this disease. There are so many degrees and angles and opinions about als, it's good to know I can come here to research with those who really know, thanks and ((hugs)) to you all!
 
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