Nikki J
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Schmidhogan
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Nikki: can you translate what this means practically for any of us.
MaxEidswick
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>Genervon has received both fast track and orphan drug designations for GM 604 in the treatment of ALS. Genervon has submitted the results of these trials to the FDA for guidance on how to make GM6 available as a therapeutic ALS and PD drug.
this sounds ground-breaking!
this sounds ground-breaking!
GregK
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Most of the results are beyond my understanding, but this extract seems pretty clear (I bolded the important bits):
.... about time we saw something positive.
ALS Clinical Data: The clinical data ALSFRS-R and FVC scores:
1.) GM604 significantly reduced the decline in ALSFRS-R versus the historical control, p=0.0047. 7 out of 8 treated patients had their ALS disease progression slowed or stopped at week 12 after initial six doses of GM604.
2.) 5 out of 7 treated patients had their forced air capacity (FVC) disease progression slowed down or reversed at week 12 when comparing with historical placebo. At week 12 FVC of mean value of placebo group is -11.5% (lower capacity is not good) and mean value of treated group is -4.7%.
ALS Compassionate Use: An ALS patient was first diagnosed with the disease in Q1 2005 and by Q3 2008 was quadriplegic and on a ventilator. After a six-dose treatment of GM604 under an approved single patient compassionate-use IND, the patient’s swallowing volume increased in two weeks to 20cc from a baseline of 10cc. Five weeks after treatment, the patient consumed 240cc of water in 20-25cc bursts without leakage.
1.) GM604 significantly reduced the decline in ALSFRS-R versus the historical control, p=0.0047. 7 out of 8 treated patients had their ALS disease progression slowed or stopped at week 12 after initial six doses of GM604.
2.) 5 out of 7 treated patients had their forced air capacity (FVC) disease progression slowed down or reversed at week 12 when comparing with historical placebo. At week 12 FVC of mean value of placebo group is -11.5% (lower capacity is not good) and mean value of treated group is -4.7%.
ALS Compassionate Use: An ALS patient was first diagnosed with the disease in Q1 2005 and by Q3 2008 was quadriplegic and on a ventilator. After a six-dose treatment of GM604 under an approved single patient compassionate-use IND, the patient’s swallowing volume increased in two weeks to 20cc from a baseline of 10cc. Five weeks after treatment, the patient consumed 240cc of water in 20-25cc bursts without leakage.
.... about time we saw something positive.
Nikki J
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As Greg points out some really encouraging results but from a VERY small trial. I think genervon has funding troubles unfortunately. I hope these results will get some attention and some cash. My takeaway is to keep an eye on it. If there is another trial I would try to get into it, if there is any compassionate use you qualify for I would try that too. It seemed intriguing from the start I had hoped my sister could get into the trial in 2013 but she did not qualify
MaxEidswick
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>My takeaway is to keep an eye on it.
I have asked in ...
I have asked in ...
PattyR
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This sounds very promising -- and their website is fascinating. I emailed them asking about next step time frames and possible west coast sites. Maybe we should all start calling the FDA and asking for action on this!
MaxEidswick
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>Maybe we should all start calling the FDA and asking for action on this!
how do we formally petition them? Congress people? We need a central advocate, any ideas?
how do we formally petition them? Congress people? We need a central advocate, any ideas?
Graybeard
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Welcome to Heartbreak Hotel, Patty Webfoot! Max and I were born in Ashland, years apart. I graduated high school up the road in Springfield.
Max, I know just the person for this, a former CALS who last summer made the trek to CONgress for ALS, and who wrote the letter to VA getting prompt action on the van subsidy. Jean Gronewald is not very computer savvy, but I'll try to get her on here.
Max, I know just the person for this, a former CALS who last summer made the trek to CONgress for ALS, and who wrote the letter to VA getting prompt action on the van subsidy. Jean Gronewald is not very computer savvy, but I'll try to get her on here.
MaxEidswick
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>Max, I know just the person for this, a former CALS who last summer made the trek to CONgress for ALS
good, I was there too ... need someone hungry for fast political capital
good, I was there too ... need someone hungry for fast political capital
Graybeard
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Jean is not in it for herself at all. She's pure compassion. She's off to Colo Springs tomorrow, and then Hawaii for a week after that.
The Genervon site lists the societal cost of ALS at $450 Billion per year, far more than the more common diseases listed.
The Genervon site lists the societal cost of ALS at $450 Billion per year, far more than the more common diseases listed.
Graybeard
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OK, Jean has joined the Forum, but not posted yet. She promises to do it after her much deserved week in Hawaii. This will be her first attempt with joining a Forum. She will be a great asset.
Jgronewald
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The introduction by Graybeard is daunting. Fighting the VA and the FDA are two different animals. I agree with the comment that most of the info is over my head except for the results, which are very encouraging. My husband had bulbar onset, so the swallow and respiratory improvements caught my eye.
I belong to the Evergreen chapter in Washington State, and we support Dr. John Ravitz at UC San Diego. I will put in calls to our director for some advice on how to bombard the right people for a broader trial.
All of you PALS are near and dear to my heart. I'll start digging when I get back from Hawaii. My first time there and my first trip after losing my best half with the exception of flying down to Socal to meet Graybeard in the flesh.
I belong to the Evergreen chapter in Washington State, and we support Dr. John Ravitz at UC San Diego. I will put in calls to our director for some advice on how to bombard the right people for a broader trial.
All of you PALS are near and dear to my heart. I'll start digging when I get back from Hawaii. My first time there and my first trip after losing my best half with the exception of flying down to Socal to meet Graybeard in the flesh.
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