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Sequoia

Senior member
Joined
Sep 19, 2010
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841
Reason
PALS
Diagnosis
08/2010
Country
US
State
Washington
City
Seattle
hello every body! I am amazed that the new treatment Dex. has been around for over 14 years! From phase I to now phase III which won't finish until next December.

I am losing muscle in legs, arms, hands, effecting my swallowing, speech, and now breathing. We know how this goes, and it goes fast! If every Pals and Cals were to get on board like the HIV & cancer folks (Komen) did, we would have access to these new treatments now.

So many pals who really need these treatments can't qualify for the drug trial. I no longer can. If we all come out in force and are willing to write a letter to these drug companies, we could make a difference. Especially Biogen who has the big bucks to really do something for us.

Dex appears to be a break through drug. We all believe the ALS community should have the same Compassionate Use availability as was granted by the FDA for Cancer and HIV...

We realize we may need to offer Biogen a significant financial reimbursement and incentive, and ALS-Treat Us Now! will try to raise the funds.

Please help us all live a while longer, so we can see our kids grow up, and have quality of life as well as length of life. These drugs do slow the progression. One of our petitioners has a husband who had been in phase I of the Dex. drug trial and so got access to Dex. for many years. It really slowed the progression down.
 
PALS, We are coming to an era where status quo just won't work for us. Do you want to continue to watch your disease progress-or-do you have enough energy to write just one letter, and maybe a friend/relative writes one letter to Biogen, ... and soon...we have started a "Campaign of Letters."
**Please look at this article** Initiatives to Improve Products for Patients
starting on page 20. It is debating an expedited drug pathway.

http://www.fda.gov/downloads/AboutFDA/ReportsManualsForms/Reports/UCM274464.pdf

The early discovery of a potential breakthrough therapy raises ethical and trial design issues. It is important to gain confidence that the effects seen in the early trials are real, and to understand the safety risks of the new drug. On the other hand, from an ethical standpoint, it is important to make sure that people with serious diseases are getting the best possible therapy. In these situations, the clinical trials for the drug’s development must be compressed and the evidence about its effects gathered in the most efficient manner possible; however, there is not a good understanding in the biomedical community about how to accomplish this.

Our time is now! Raise your voices, and keystrokes,
Why? As Greg M said, "It is the right thing to do."

AveMaria
 
Sequoia,
Can you pass along an address for us to send our letters to Biogen, AND, where do we get a form letter to use, that we can add our 'human side' to?
BTW-the reason I want the Dex drug -- is to add to my ALS cocktail. Besides...Biogen can afford to give it to me.
It's the Right Thing to Do, Biogen! (I think that will be my new mantra::p)
AveMaria
 
Someone asked me an interesting question that I think needs to be answered.......
If some terrible adverse event becomes apparent during the long term data analysis, will the people on compassionate supply, or buying the drug before the study is complete, sue Biogen! Unfortunately the USA has created a culture of litigation. It's the continual law suits and fear of them, that slows down these processes. Everybody including the FDA are protecting there butts.
What is, Treat us now, going to do legally, to protect Biogen against law suits?

One patients slow progression, when not compared with a larger cohort and the subjects on placebo, is purely speculative. It is really upsetting when people use an individual success to push an agenda. It is just not right and it's just such comments that quacks use to support there treatments.
Everything that is being done by treat us now is great, but please don't got down the path of subjective comments. It is damaging to the cause and just a little naive.

Thank you for all the hard work that is being done. I look forward to reading the plan, to protect Biogen from the things that can go wrong and the angry people that may sue them.
 
BTW-the reason I want the Dex drug -- is to add to my ALS cocktail. Besides...Biogen can afford to give it to me.
It's the Right Thing to Do, Biogen! (I think that will be my new mantra::p)
AveMaria

Is that what the Treat US Now mantra going to be -- "<drug company name here> can afford to give me the drug, so I should get it."

The right thing for Biogen to do is complete the trial plan that they've committed to, make sure that their data truly indicates that the drug really does work, and then expedite the approval phase.

It's not Biogen's fault that you developed ALS when you did. It will be their fault if they put an ineffective or, worse yet, dangerous product out on the market.
 
Thank you Thank you Tr. A voice of reason yet again. This whole thing is getting a little scary. Too many people not fully understanding is the recipe for disaster.
 
last week i started a new drug trial that will last a year. at the clinic i mentioned dex (np001) and how some are going to self adminsiter sodium clorit. the clinic administrator pleaded with me not to even consider it (i won,t) she indicated there are only two people really pushing it and we both came up with the same names. when i take new drugs i want to be closely monitored. if more als patients would sign up for trials they could be completed faster instead of being drug out over a long period of time to get enough participants.
 
Here is what the FDA has done for cancer drugs. Cancer Care in the U.S.

Rest assured paying for the treatment, and liability issues will be addressed.
 
My thought was not to push Biogen but more to encourage them to take the lead in the release of the drug and they have the capacity to honor this expanded commitment. The FDA already has this compassionate early access for individuals in place. You have to have your doctor or neurologist on board, because he/she has to monitor you same as the drug trials do.

Here is an article about new cancer drugs...just substitute the word cancer for ALS...

By: Richard Pazdur, M.D

Our staff understands that there are many diverse opinions in the oncology community regarding approval of oncology drugs. The balance between the immediate needs of an individual patient and the assurance of a drug's safety and efficacy for approval will always exist. When appropriate, we have permitted the use of single patient INDs, exemptions to existing protocols, and expanded access programs to allow patient access to promising, yet
unapproved, drugs.

To expedite drug development and our review of applications, FDA has
approved oncology drugs on the basis of a single trial and used a
variety of clinical trial endpoints --- including overall survival
(the time a patient lives before death), progression-free survival
(the time a patient lives without their cancer progressing), and
response rates (tumor size reduction). FDA has taken advantage of
regulatory initiatives for serious and life-threatening diseases, such
as fast-track priority review, and accelerated approval.

http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM079736.pdf

Final Rules for Expanded Access to Investigational Drugs for Treatment Use and Charging for Investigational Drugs
 
Nobody will be asking for anything for free, just asking for early access to a drug that in two trials seems to be effective.And how dangerous can it be, who's going to take it besides ALS patients?
 
Nobody will be asking for anything for free,

One of your erstwhile comrades appears to have just that in mind, if you'll read the other posts in the thread.

just asking for early access to a drug that in two trials seems to be effective.And how dangerous can it be, who's going to take it besides ALS patients?

Please note what I have bolded in the quote above. Until all the data is collected and analyzed, how do you know whether the drug is doing anything? Seriously? Eric Edney claims that his "Eric Is Winning" program stops ALS progression. Should we refer new PALS to that program?

The answer to how we know that Dex works against ALS is the same as for Eric Is Winning: we don't know. Why? Because neither has completed the clinical testing. And multiple anecdotes do not necessarily constitute data.
 
Someone asked me an interesting question that I think needs to be answered.......
If some terrible adverse event becomes apparent during the long term data analysis, will the people on compassionate supply, or buying the drug before the study is complete, sue Biogen! Unfortunately the USA has created a culture of litigation. It's the continual law suits and fear of them, that slows down these processes. Everybody including the FDA are protecting there butts.
What is, Treat us now, going to do legally, to protect Biogen against law suits?

One patients slow progression, when not compared with a larger cohort and the subjects on placebo, is purely speculative. It is really upsetting when people use an individual success to push an agenda. It is just not right and it's just such comments that quacks use to support there treatments.
Everything that is being done by treat us now is great, but please don't got down the path of subjective comments. It is damaging to the cause and just a little naive.

Thank you for all the hard work that is being done. I look forward to reading the plan, to protect Biogen from the things that can go wrong and the angry people that may sue them.

This would be easily handled by having them sign the same consent form with all the disclosures that you must for entry into the trial. The patient must be willing to take additional risks in order to have faster access to drugs. Phase I is a must as is phase II. Perhaps a shorter phase III but how long is long enough. If a patient is willing to take additional risks then make it available to them.
 
Why not you can't hit the ball unless you swing! doing nothing will get you exactly that nothing!
Eric isn't the same as controlled trials
 
One of your erstwhile comrades appears to have just that in mind, if you'll read the other posts in the thread.



Please note what I have bolded in the quote above. Until all the data is collected and analyzed, how do you know whether the drug is doing anything? Seriously? Eric Edney claims that his "Eric Is Winning" program stops ALS progression. Should we refer new PALS to that program?

The answer to how we know that Dex works against ALS is the same as for Eric Is Winning: we don't know. Why? Because neither has completed the clinical testing. And multiple anecdotes do not necessarily constitute data.

Trfogey,

You are right no one should expect anything for free and drug companies have the right and frankly obligation to their investors to not only seek compensation but ensure they are making a profit.

As far as your other question your point is more then fair. I will answer it the best I can and while it may not satisfy your intellectual analysis it is my simplistic perspective.

Phase I safety study is a must. Phase II is a must for additional safety and efficacy. Obviously some side effects and potential side effects may not show up for years. However my feeling is that once, perhaps no later then 6 months in phase III if the drug is showing safety and some potential efficacy impact then make it available as a compassionate care drug. Obviously not yet vetted or approved for off label usage. Have every patient sign a consent and liability wavier. Give the patient the facts, the risks, the limited testing that was completed, the good and bad of side effects etc and possible improvements seen. At the same time patients who sign the wavier and consent form are tracked whether through their Dr or the ALS registry or whatever vehicle needed in order to continue to collect data.

I guess my point is and I stress this is only an opinion, is that a drug trial for ALS should not be expected to meet the same strict criteria as a trial for acne or migrane medication.

At some point a patient with a terminal illness who has been educated on the risks should be given the opportunity to take a drug that has been partially vetted. Colud it cause other issues? yes. Could it kill an ALS patient? possibly. However we all now how the story ends if we do nothing.

Some people may not be willing to take the additional risk and that is OK as well, the point is they had the option.

None of this addresses the cost issue which is obvioulsy the other elephant in the room.
 
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Cost is cost, and companies are in business to make money. Yes they would love to help people, but they are not in business to help alone.

But, offer it 1st, have PALS sign their lives away for liability sake, then offer it at a fair cost. Fair cost of course, is different depending if you're the company or the patient.

If it helps, great.
If it doesn't, well that's the choice you make.

When you're at the casino and you "double down" two things can happen.
1) You lose, and you go home early.
or,
2) You win and use your winnings to stay and play some more, then go home.

We're all going home sooner or later, but some would gamble for the chance to stay and play a little longer.


Food for thought:
Is it the same as asking controversial cryonics companies to freeze everyone with a terminal illness until cures are found?

Keep it real,
Cheers,
Casey
 
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