Let's all ask Biogen to Treat Us Now!

[Alyoop]

Thank you Ed and Ted. I am not sure how robust the consent form would be. Till will tell.

The other point I need to stress though , which to me is most important is to stop saying A person has had slow progression therefore it must work. This type of thing is creeping in to the Treat us now push and it should not. Until those results are analysed, its purely subjective. Please stop saying stuff like was said in the opening post of this thread.

 

[Olive13]

ALS treat us now is asking for Biogen to give us the CHOICE to have access to dexpramipexole which *has shown safety and efficacy in the first and second phase of trials.*

Of course there would be paperwork in place just like in the clinical trials for Biogens "protection."

This request for compassionate use is not unheard of or new. *As shown above its been done many times over for cancer treatments.

In regard to wether it works or not,*
I think Casey stated it beautifully above "We're all going home sooner or later, but some would gamble for the chance to stay and play a little longer."

ALS Treat Us Now is just asking for a fair chance! It should be our choice if we want to "gamble" or not.*

 

[trfogey]

The other point I need to stress though , which to me is most important is to stop saying A person has had slow progression therefore it must work. This type of thing is creeping in to the Treat us now push and it should not. Until those results are analysed, its purely subjective. Please stop saying stuff like was said in the opening post of this thread.

Excellent point, Aly. If all it took was one person with a large improvement to justify waiving test protocols and medicating "compassionately", Treat Us Now should have beating down the doors of the Neuralstem trial TedH5 has been working with, not Biogen.

 

[Sequoia]

I think that by the end of Phase II the research team is fairly certain that the drug is safe and effective...if Dex has been around for 14 years, why not cut down the time of approval. They have done that for people with HIV and Cancer....why not ALS?

 

[Olive13]

Just wanted to point out some information that has been analyzed:
"Knopp Biosciences is very pleased to see dexpramipexole entering Phase III development with the initiation of the EMPOWER study," said Michael Bozik, M.D., President and CEO of Knopp. "The promise of dexpramipexole has brought together a motivated multi-national network of investigators and study coordinators committed to determining if dexpramipexole can fulfill its potential as a treatment for patients with ALS."
Dexpramipexole was well-tolerated in three Phase I studies in healthy volunteers who received dexpramipexole in single doses up to 300 mg or multiple doses of up to 150 mg twice daily for four-and-a-half days. Dexpramipexole was also well-tolerated in a two-part Phase II study (CL201) that evaluated about 100 ALS patients treated for up to nine months and in a subsequent Phase II extension study (CL211) that has continued to follow ALS patients for about two additional years. In the first part of CL201, dexpramipexole at 150 mg twice daily showed a trend toward slowing functional decline over a 12-week period compared to placebo. Following re-randomization in the second part of CL201, dexpramipexole at 150 mg twice daily again showed a trend toward slowing functional decline and also showed a trend toward improving survival compared to low dose over a six-month period. In CL201, the joint ranking, which incorporates both function and survival and which is the primary endpoint of the Phase III trial, significantly favored dexpramipexole 150 mg twice daily compared to 25 mg twice daily.
SOURCE: BIOGEN IDEC

 

[AveMaria]

Dear trfogey,
After going back and looking thru a number of your postings on PLM, I understand better why you are unhappy.
You quoted me saying Biogen can afford to give it to me. It's the Right Thing to Do, Biogen! (I think that will be my new mantra
They CAN afford to give it to me, before it reaches market, long after you and I have died, trfogey. Now, if they do not, well I'm double insured, and not with medicare.
trfogey, I have lots of mantra's. THIS is my favorite, "It's the right thing to do." In fact, it's something I try to live by-how about you?
Dear Alyoop,
You said, will the people on compassionate supply, or buying the drug before the study is complete, sue Biogen
We won't sue anymore than I can sue Neuraltus for NP001 drug. As mentioned by other posters, who have a better grasp of this idea than some, paperwork is always in place.

Alyoop said, 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.

Alyoop, I take it, you will not be writing a letter to Biogen, to save your life, I mean your soul.
And trfogey, neither will you. Is that right. Well then, continue on 'doing nothing for the cause,' because 'that's the right thing for YOU to do.'

 

[TedH5]

Excellent point, Aly. If all it took was one person with a large improvement to justify waiving test protocols and medicating "compassionately", Treat Us Now should have beating down the doors of the Neuralstem trial TedH5 has been working with, not Biogen.

No one is suggesting that the criteria is one person, or that the people utilizing the drugs will be under the impression that it is absolutely a cure or guaranteed to slow down progression. Believe I knoq better then anyone that when you are 1 out of 13 to show a difference then you are considered an outlier.

This is not what we are asking to be considered. We are asking for a lowered standards and time table. Lets say a study showed it was able to slow down progression in a 1/3 of the cases and the drug had proven safe, even if the data was early and as you make the drug available to a greater pool the % could go up or down, the point is give patients the option. Not under false pretenses, educate them on the percentages, risks etc.

I stick with my basic tennant that the standard of proof needs to be lowered as compared to non terminal illnesses and conditions.

 

[Alyoop]

Avemaria, please go back and carefully read what I wrote, carefully being the key here. I am not at all against anything treat us now is doing. I wanted to point out that patients can and do sue if things go wrong and that is one of the reasons Biogen and the FDA will have to proceed carefully. That's a truth. Not me being judgmental.

Second point is the sensationalism about an occasional person on the study showing a slowing in progression. If you understood why clinical trials are done and why placebos were used, you would know what I am meaning. Wild sensationalism and claims of dexpramapexoles efficacy before the study results are analyzed, is damaging to Treat us now.

I think it's a fantastic initiative, many people are working hard. It just needs to be careful.

And to close.....how dare you talk about my soul. Don't you EVER do that again. You do not know me, and you have proven that you know very little including the ability to read someones post and understand what you are reading.

I also read that you are taking a pot shot at my knowledge of these things. I am a clinical trial coordinator and research nurse. I have worked on a huge number of clinical trials, oncology, neurology and dermatology. I take the protocols from conception, through the ethics committees, start the study up and run it to completion.
I have seen many drugs from phase 2/3 to market. I have seen many fantastic compounds that showed promise, fail to be statistically significant, when a placebo cohort is involved.
Sadly I have seen drugs show amazing efficacy, get a good market share and end up with terrible long term side effects. These were never evident during the clinical trial. Consent forms can not always stand up to the fury of a subject or there families.

Do I have a different perspective to some of you? Of course I do, which is why I wrote 2 simple words of caution.

For that it seems rather harsh, that a lady would come on here ( a new member) and threaten my soul.

May God forgive her......and I mean that from the bottom of my heart. Ava Maria, I will pray for your judgmental soul.

 

[vlcare]

Ave Maria - I'm hearing frantic in your words. That's understandable. But, truly, we need each other on the Forum. We cannot afford to alienate each other. What happens is that one good punch promotes another--what goes around, comes around.

It is best to formulate a reply, throw it out to the community, and then relish the points of view that surface in the froth. Food for thought is a good term for what occurs here. I would venture to say that 100% agreement on any topic is 100% unlikely.

Keep plugging. And read your posts 3 times before you hit the send button. Your energy will serve you well.

VL

 

[lakelover]

I am lakelover's husband whose has ALS. We all face adversities in life and how we deal with them are unique to who we are. Some come out swinging; some sit back and wait and some choose to just check out permanently (assisted suicide). The ones that come out swinging are willing to take a gamble in order to make a difference. We are already taking a gamble by volunteering for Drug Trials. TedH participated in the stem cell drug trial. How many would be willing to participate in this trial and take the chance he did. Sequoia is participating in the Dex Trial, AveMaria is participating in the NP001 Trial and I am participating in the CytoKentics Trial. We are willing to take a gamble to not only make a difference for us but for people diagnosed in the future. I was told today by one of the doctors overseeing the CytoKentics Trial that there are as many new ALS cases being diagnosed as there are with MS. If some of us are willing to take the chance, sign off on any forms releasing drug companies from any responsibility we should have the right to make this decision. We already know what the outcome for ALS patients is going to be, we just don’t know when. For some it is fast and in others it is slower. No one should question or find fault with a person who wants the opportunity to make the decision they feel is right for them.

I come from a family of fighters. In 1993 my mother was diagnosed with rectum cancer. She just celebrated her 83rd birthday. My older brother is going into his 4th year cancer free from Stage 3B non small cell lung cancer. They are here today because cancer patients took a stand to put pressure on drug companies and the FDA to have early access to drugs. I would rather die fighting this disease with everything I have than sitting around waiting for ALS to ravage my body robbing me of the ability to move, talk, swallow or breathe. I know this is not the right decision for everyone but if I am willing to make whatever sacrifice it takes, then give me the opportunity to do what I think is right for me.

Ralph

 

[trfogey]

Dear trfogey,
After going back and looking thru a number of your postings on PLM, I understand better why you are unhappy.

Really? Do tell. Tell me these dark things that you've been able to glean from posts I wrote over there two years or more ago, since I've written very few over there in the past two years.

You quoted me saying Biogen can afford to give it to me. It's the Right Thing to Do, Biogen! (I think that will be my new mantra

Yes, I did. I usually do quote the person I'm responding to -- it helps avoid confusion.

They CAN afford to give it to me, before it reaches market, long after you and I have died, trfogey.

Biogen can afford to do a lot of things. Including deciding that the market is not favorable for bringing a new drug to market in the United States at this time. In fact, until the particulars of the Patient Protection and Affordable Care Act (aka Obamacare) have been decided and published by the US Dept. of Health and Human Services, most medical vendors are playing their hands close to the vest, so it would not surprise me in the least to see promising new drugs held back for a couple of years until the post-PPACA medical care market stabilizes.

Now, if they do not, well I'm double insured, and not with medicare.

You do know that neither of your insurance companies are obligated to pay for drugs that have not been approved for the purpose intended, don't you? And I seriously doubt that they'll pay for Compassionate Use drugs at all. Finally, as the provisions of PPACA go into effect over the next few years, your insurance coverage might be substantially changed, if not eliminated.

trfogey, I have lots of mantra's. THIS is my favorite, "It's the right thing to do." In fact, it's something I try to live by-how about you?

The right thing to do is to let the approval process run its course. You are, of course, entitled to disagree.

And trfogey, neither will you. Is that right. Well then, continue on 'doing nothing for the cause,' because 'that's the right thing for YOU to do.'

No, I will not be writing a letter to Biogen because I don't agree with the tactic. I also think Biogen should spend its time completing the studies it has underway rather than creating the distraction of opening a new round of negotiations with the FDA to get Compassionate Use approved.

It is possible to disagree without being disagreeable, which is what I have chosen to with this particular issue. It's a shame that you couldn't do the same.

 

[tomby]

Well said Ralph (lakelover)
I was in the ceftriaxone trial, which included a catheter in my jugular and twice daily injections, because it was the only phase 3 trial available. I had to drop out after 5 mos because of a reaction.
Now, although I'm willing, I don't meet the inclusion criteria for any of the current trials.
Fact is, the window when most PALs meet these criteria is pretty short. But, if any of these drugs were to get to market, I'm pretty sure these criteria wouldn't apply. There could be valuable information learned from willing PALs further along in the disease.
I support Treat Us Now because there is room for some activism here. There has been no new drug approved since rilutek 15+ years ago. Let's push some and make some noise.
I believe Biogen is designing a new phase 3 trial - Why not expand the inclusion criteria?
Why not compassionate use at the appropriate time? Let's shake the tree a little and see what falls out. Noise attracts attention, attention attracts investment.

 

[TedH5]

It is possible to disagree without being disagreeable, which is what I have chosen to with this particular issue. It's a shame that you couldn't do the same.

I agree...obviously this disease and the lack of a treatment or cure evoked very passionate debates and responses. That being said I always refer back to BarryG's
quote that "we are all in this together" We can certainly agree to disagree on our various opinions and do so respectfully.

On a side note on the NEALS webinar today with Dr. Bedlack I was suprised to learn how difficult a time they have in finding PALS to participate in trials. I suspect that some of that most likely is a result of inclusion criteria and using the date of symptom onset rather then diagnosis onset as part of the exclusion criteria.

None the less knowing that they said less then 10% of PALS volunteer was startling. Trials are obviously optional and not for everyone but I would hate to think that we are our own worst enemy in the search for a cure.

 

[Shane the Pain]

Dear ALS patients friends and families,

I really am not too worried about the drug companies getting sued by a dieing ALS patient !

What I am worried about is idiots worrying about this kind of crap rather than supporting the idea to Treat Us Now !

Personally I feel my time is very limited and if one of these drugs could improve my condition so I could hang around a while longer and help my wife raise our four children, I would be thrilled !

Fight with us not against us !

Fight On,
Shane the Pain

 

[penney21]

HI SEQUOIA,
I AM A RELATIVE OF SOMEONE THAT HAS ALS. IAM WILLING TO WRITE TO BIOGEN,BUT COULD YOU E-MAIL THE ADDRESS TO ME. SINCE IWORK PART TIME,ICAN DO OTHER THINGS FOR YOU TO HELP WITH THIS PETITION.JUST LET ME KNOW WHAT YOU WANT ME TO DO. SINCE THIS IS AN ELECTION YEAR SHOULD STATE POLITICIANS BE NOTIFIED ABOUT THIS. I LIVE IN R.I.AND WILL HELP IN ANYWAYTHE IS NECESSARY,
THE PETITION IS MOVING TO SLOW.IF CARETAKERS ,FRIENDS.AND RELATIVES TAKE PART IN HELPING TO MOVE THE PETITION.,YOU WOULD HAVE MORE SIGNATURES.PLEASE GET THESE PEOPLE INVOVED NOW.
TIME IS OF THE ESSENCE,IF EVERYONE WAITS TILL THE NET DEX TRIALTHAT,S WASTING TIME.PALS NEED TO BE TREATED NOW,NOT IN 2014. READ DR. BENNET'S STORY.HE DISCOVERED THIS DRUG OVER 14 YEARS AGO.TYPE IN-readthehook.com/.../cover-patented-medicine-trail-money-follows-trail-tears

cathy