Stay on the trial?

[lpcarr]

My husband has been on the Ceftriaxone trial for 15 months. He is a slow progressor but definitely getting weaker. Using a walker now and getting his braces this week. Very slow eater and very fatigued. He wants to go off the trial. He has an appt to have his hickman taken out in 3 days. since he has had no side effects he is convinced he has the placebo. I want him to stay on but I know it is his decision. Even his neuro said he has " gone to the moon and back" for the sake of data for the trial. What are others feeling who are on the trial? What if he goes off and starts a more rapid progression?
Watching the decline is so hard. Thanks for any input. PAM

 

[TedH5]

Wow that is a tough decision. I would never pretend to know the right answer because it is such a personal decision and there really is no right or wrong answer. My only advice is for you and your husband to sit down and have an open, honest and very candid discussion. Allow him to express himself and do not be afraid to share your thoughts, concerns, fears etc. I think that if you both engage in a very open dialogue the two of you will clearly see what is the best decision for your circumstances and most importantly for your husbands comfort and happiness.

Best of luck, I know you both will make the right decision for the two of you!

 

[trfogey]

As TedH5 said, it's a very tough decision. That's one of the unfortunate things about the structure of drug trials with regard to ALS. From what I can tell, most people sign up in the hope of receiving the trial drug, not to be part of the control group. When they come to the conclusion, rightly or wrongly, that they didn't get the drug, they are ready to stop the trial -- and who can blame them?

 

[rcharlton]

How much time is left in the trial?

A big thank you to your husband for his sacrifices to date.

 

[Sequoia]

I am on the Dex trial and I know what you mean about being tired...I have to fly to fresno and stay with relatives and the whole thing exhausts me. But I will do it for as long as I can..but if it gets ti be too much...then I will consider.But I think that the trial folks will then just have the blood drawn at home...but I am just taking pills...I want to do this for the research also...to be a part of discovering a drug that will help pals!

 

[HelenL]

That is a hard decision to make, but a couple of questions I would ask myself... even if he's on the placebo, when does the trial end and will all patients get the medicine at that point? Is there another trial coming up that he may qualify for that may work better for him?

If the trial is really intrusive, and he's not getting any benefit for himself, then the only benefit would be the data collected for the future... or the potential medicine at the end of the trial.

Good luck in whatever you decide to do, you have to do what's right for your own situation.

 

[momap53]

Thanks to your husband for participating in the trial for over the requested one year mark. I understand from my clinic that the Ceftriaxone Trial will be ending next December 2012. It does take some time and effort to maintain everyday. I'm always frustrated with keeping the Hickman dressing dry when showering. I hope that I'll be able to continue for at least the full year as I've had to take a couple of breaks already due to side effects. Best of luck to you with your decision. Let us know how it goes

 

[Sequoia]

The ALS Treat Us Now Steering committee, is initiating a Pilot 1 initiative to allow early access to Dexpramipexole (and/or Cytokinetics CK-2017357).

We invite 10-20 PALS who want Early access to these two drugs to join in this Pilot-1 initiative.

Our goal will be to have early access granted by March 31 2012.

If you are interested in participating and helping, please send us an email with your response to these 4 questions, ...or post your response on this thread.


1-You are willing to spend 1 hour per week, most if not all the time will be on your computer, IPad etc.

2- You are willing to send us your email address and are willing to join a Google group set up to facilitate our communication with other Pilot-1 volunteers.

3- You understand that Early Access probably means you will have to buy the treatment from the Drug company. There is no free lunch. You are NOT committing to purchase, only saying you understand there will most likely be a cost to PALS.

4- You have a working relationship with a Neurologist.

Please post your response and comments/questions on this thread or send your response, or questions to: [email protected]

After 1 week from today we will assume all interested have replied....We will cap at 20 for each treatment,....we intend to have a Pilot 2 if necessary.




Sincerely, Ed for the ALS Treat us Now! Steering committee (We are Barbara, Diane, Ed, Larry, Laura, Sharon, Ted).

 

[cubcake]

The ALS Treat Us Now Steering committee, is initiating a Pilot 1 initiative to allow early access to Dexpramipexole (and/or Cytokinetics CK-2017357).

We invite 10-20 PALS who want Early access to these two drugs to join in this Pilot-1 initiative.

Our goal will be to have early access granted by March 31 2012.

If you are interested in participating and helping, please send us an email with your response to these 4 questions, ...or post your response on this thread.


1-You are willing to spend 1 hour per week, most if not all the time will be on your computer, IPad etc.

2- You are willing to send us your email address and are willing to join a Google group set up to facilitate our communication with other Pilot-1 volunteers.

3- You understand that Early Access probably means you will have to buy the treatment from the Drug company. There is no free lunch. You are NOT committing to purchase, only saying you understand there will most likely be a cost to PALS.

4- You have a working relationship with a Neurologist.

Please post your response and comments/questions on this thread or send your response, or questions to: [email protected]

After 1 week from today we will assume all interested have replied....We will cap at 20 for each treatment,....we intend to have a Pilot 2 if necessary.




Sincerely, Ed for the ALS Treat us Now! Steering committee (We are Barbara, Diane, Ed, Larry, Laura, Sharon, Ted).

Sequoia - I tried to use this email and it is undeliverable. Can you please correct? Thank you!

 

[edjohnson]

To raise another area for discussion:
- How about asking every trial site doc and staff to only sign up for handling trials where the Drug company promises a best effort for allowing all participants access to the drug...PALS pay a very heavy emotional price to participate they should be first in line to have early access...of course the PALS has to pay market rates.
- And how about pressing the FDA to allow an ALS advocate to attend all drug trial reviews...the voice of the patient...sign a confidential agreement and help the FDA accelerate access.

 

[Sequoia]

Sorry about the email...i think if you dont have window's live it doesnt work....so just pm me and i will forward to the steering committee. Thanks!

 

[Sequoia]

Everybody, we have almost reached the 20 for the Pilot 1...If you want to be on the Pilot 2.... PM me with your real name and your email address and I will put you on the list. This is great pals and cals....we are on the move!

 

[lpcarr]

thanks to so many who have responded. I never realized what it meant to
other pals that many are participating in these trials. My husband has decided to
stay on the ceftriaxone trial for a while longer. The pilot sounds good to me also.
It is hard for him to decide what to do. He struggles with it and so do I. What if
a different drug made him sick with side effects. Thats why he thinks this is placebo, but who knows? The discovery of this forum has given me great comfort and good advice.
So many mirror each other in feelings and physical symptoms. Thanks to all who are
here. My husband and I look forward to learning so much more from all of you. I will keep updating as we go along.

 

[Sequoia]

GUESS WHAT? WE DO NOT HAVE 20 FOLKS IN THE PILOT 1 group, some opted out. So if you are interested email us (go back 3 posts for the email)


I HAVE A DREAM: that one day these drugs will be available for everyone who suffers from ALS, so that we can live longer to see a CURE

MAKE MY DREAM COME TRUE: get involved!
__________________

 

[Sequoia]

Our 8 key points: to the FDA and Drug companies

1-Include access to the treatment after trial for all trial participants...
Use this promise to encourage more trial participation. Payment would be responsibility of
Treatment company and PALS (Patients with ALS)...If supply is limited then set up a lottery,. ..
or a process similar to that used for selecting patients for organ transplants.
Strongly encourage the ALS association and other groups to help with funding early access

2-Encourage monthly monitoring and reporting during Phase II and III. As soon as efficacy
becomes apparent during Phase II begin taking the necessary manufacturing steps to have
available supply for those PALS willing to sign off on accepting potential risk. Assure that
pharmaceutical manufacturers understand the Public Relations benefit from supporting
early access. PALS will have to arrange funding for access to early treatment.

3-As Phase III proceeds agree to declaring efficacy when early data shows unequivocally
that patients are demonstrating significant improvement. Prepare for, and allow expanded
access to all PALS whose doctors confirm they are able to benefit PALS will have to arrange
funding for access to early treatment.

4-Off label drug treatments and non drug treatments ( the current Creatine trial, or
Tamoxifen trial are examples..) should have a shorter timetable for Phase II and III.
As soon as monthly monitoring shows efficacy allow full access at market price; insurance
companies encouraged to grant coverage pending statistically valid outcomes. If Trials end with no
positive results than Insurance company can deny payments.

5-Consider establishing a Committee of Experts who will meet every 3 months to
assess risk, reward and recommend Best Treatment Protocol to PALS and Neurologists...a "Best
Protocol for treatment at this time"

6- ALSTDI and the ALS Association help us draft a "Best Practices" document encompassing
the above 5 principles. The document is sent to stake holders asking for endorsement and
compliance.

7- Conduct a research study to establish the cost benefit for Insurance companies, Medicare
and PALS by providing early treatment. Treatment that will delay the onset of expensive surgical
and other life support expenses. Use the results to educate all stake holders on the societal benefit
of early treatment.

8- Establish an education and awareness campaign to help society understand that the many
promising treatments now in trial stages suggests that a new era has finally arrived!
We are transitioning from a "Survive till early death, to a Survive for the Cure" The ALS community
focus should now tip towards funding and support for early treatments that will slow progression.
This new focus should include empirical data to vividly demonstrate the many deaths that occur
while waiting for final approval and market availability. We need to find a way to extrapolate and
dramatize that impact.

We are trying to move a mountain: transitioning from handing out wheelchairs to getting early access so we live long enough to see a cure.
We need all the help we can get, so if you are available contact us!

THANK YOU- from the ALS-Treat Us Now! Steering Committee