Something needs to be done !

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JamesMcC

Distinguished member
Joined
Feb 2, 2009
Messages
106
Reason
PALS
Diagnosis
01/2009
Country
US
State
idaho
City
V
I know different organizations are doing their best for pals and als awareness but its not good enough.I'm very p''''ed off by the drug companies and their greed.While ALSTDI has a promising drug and not one of them care about it cause it wont make them $$$$$.Its disgusting ! 5 months and nothing,theyre not interested.WE NEED TO ACT!
CALS/PALS AND FRIENDS OF THEM NEED TO EMAIL,WRITE,PUBLISH whatever it takes to get peoples attn and let them know what these greedy drug companies are "not" doing.

Email your friends, with 30000 of us and 10x that with friends 300,000 people have a voice.
Email your local senator,congressman or whoever represents you.

Email your Als ambassadors .where are they?I don't see them on TV like Michael J Fox.Email them.I am so so mad.

Email Oprah or whoever you want.(i did a month ago,no response)

Email your fav news channel


Publish something in your local newspaper re Drug Cos and their lack of......anything!

EMAIL YOUR PRESIDENT! DO IT!

Go on every Als forum and have them act.
We have to do something in mass numbers.It's all very well saying you will......DO IT!

SHAME ON THEM! LETS PUT THEM TO SHAME!
James
 
Do something
Thankyou
 
John McCarty, PhD
Director of Therapeutic Investigation
ALS Therapy Development Institute
Rank: Administration
Groups: Administration , Member

Joined: 1/15/2009
Posts: 2,096
Location: Cambridge , MA
Hi Everyone –

I’m reposting this same reply that I’ve put in a related thread: http://www.als.net/forum/Default.aspx?g=posts&t=47309

It’s great that everyone is sharing the excitement we as researchers feel on the promise for this specific potential therapeutic: one of some 40 or so projects that we have currently ongoing and is giving the best indications of efficacy we have seen to date. I do need, however, to convey – as Dr. Perrin indicated in that panel discussion on translating therapies to patients in these critical diseases (see link at http://www.als.net/articles/articleDetail.asp?articleID=5351 ) - that this process can be complex.

First, there are the challenges of bringing forth drugs through the clinical trial processes so that drugs can be used safely and clinicians know how to optimally dose to yield efficacy. For example, it is worth reiterating what has been mentioned on this forum previously that the average new drug requires nearly 70 trials before finally getting initial approval and such can requires thousands of patients to step up to the plate and make the sacrifice of participating in trials and can cost tens if not hundreds of millions of dollars. The primary goal of such exercises is learning not just if there can be efficacy by how to optimally and predictably use a drug. Obviously, therefore, for us to meet our mission of getting drugs to PALS as quickly as possible – which will require working towards FDA approval – we need to work diligently to engage the companies that have experience necessary to work with this complex, protein-based biologic. Steve has that as a priority and we will update with specifics as we can and I can only encourage folks to call in to our next Research Update Conference call scheduled for July 16th where Steve will update as much as he can.

Secondly, we have not put this particular drug through quite a number of mouse studies and used a large number of mice simply because we want to repeat an already statistically-significant result. This is a complex potential therapeutic that is not appropriate or available whatsoever at this point to be taken by folks outside of a clinical trial. In short – this is not an already approved drug or even one which is that well understood and could in no way be accessed by patients privately. Despite our depth of experience in testing therapeutics in the ALS mouse model, having performed more such studies than any other research organization – we are still challenged by understanding fully how to effectively dose this material across the genders and to maximize potential benefit. It is anything but a straightforward proto-drug and will require engaging as experienced as possible partners if we are going to be able to bring this to patients in clinical trials for the ultimate validation in humans.

Folks should please understand that in order for us to meet our mission effectively, we need to work with some discretion and I will not necessarily be responding to various requests for specific information on this topic. Our mission to the ALS community is the same now as they have been at the onset and are wholly guided by our commitment to bring truly effective therapeutics to ALS patients as quickly as possible. Our philosophy is, as always, to update and share information as much as possible and, in comparison to any other organization whether it be commercial or academic, we have well demonstrated such. We obviously will publish information on these studies as we can and when we can within the primary, crucial constraints of meeting our mission. Anyone who wants to help our mission can do so in a wide variety of manners including helping our fund-raisers gather the resources to fuel our research as well as well as restraining from perpetuating un-substantiated conjectures or rumors on this specific, potential therapeutic: such could disrupt the complex path that must be successfully navigated if it is to even get the chance of being tested in ALS patients.


--------------------------------------------------------------------------------
John McCarty, PhD
Director of Therapeutic Investigation
ALS Therapy Development Institute
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