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apollick

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This just came in from the MDA - ALS Division: As it is encouraging for those diagnosed in the future - I am praying that the success of these trials speeds up.

Trial participants receiving the highest dose of CK-2017357 showed symptom improvement in the first stage of an ongoing phase 2 trial of this experimental drug
In the first stage of an ongoing phase 2 clinical trial to test the experimental therapy CK-2017357 in amyotrophic lateral sclerosis (ALS), the drug was found to be safe and well-tolerated.
Participants who received the highest dose showed improved scores on tests that measure motor and breathing function, muscle strength and fatigue.
CK-2017357 is designed to work by increasing muscle sensitivity to calcium, which in turn is expected to increase skeletal muscle force and improve muscle function.
The drug is the lead therapeutic candidate for ALS from South San Francisco biotechnology company Cytokinetics, and was granted “orphan drug” status by the U.S. Food and Drug Administration (FDA) in March 2010. (Orphan drug status provides financial incentives for the development of drugs for rare diseases.)
The phase 2 trial results were reported Nov. 30, 2011, at the 22nd International Symposium on ALS/MND in Sydney, Australia, by Jeremy Shefner, professor and chair of the department of neurology at Upstate Medical University, State University of New York in Syracuse. (Shefner directs the MDA/ALS Center at that institution, but MDA is not involved with this study.)
About the new findings
Shefner reported on Part A of the phase 2 trial, in which 24 people with ALS who were not taking riluzole were randomly assigned to one of four groups in which they received daily oral doses of either a placebo or CK-2017357 at a dose of 125, 250 or 375 milligrams.
Clinical assessments took place at scheduled intervals during the two-week treatment phase, and again one week after the final dose.
Investigators found dose-dependent levels of the drug in the blood. Four out of five people who were treated with the highest dose of the drug reported overall improvement. Three out of the five improved by at least one point on the ALS Functional Rating Scale-Revised (ALSFRS-R), a validated ratings scale used by physicians to assess symptom progression in individuals with ALS.
Although dizziness was a reported side effect in those taking CK-2017357, it tended to be mild, and lessened or stopped with continued treatment over the two-week dosing period. The drug was well-tolerated at all dose levels, and blood levels of the drug increased with the higher doses.
For more, read Cytokinetics’ press release: Cytokinetics Announces Positive Results from Phase 2 Clinical Trial Evaluating CK-2017357 in Patients with Amyotrophic Lateral Sclerosis.
Analysis from the trial still is ongoing, and Cytokinetics expects to report additional results at a later date.
CK-2017357 testing continues
Cytokinetics already has moved on to Part B of the phase 2 trial, in which CK-2017357 is being tested in people with ALS who are taking riluzole (brand name Rilutek). Riluzole is the only drug approved by the FDA for treatment of ALS.
For more see CK-2017357 now being tested in combination with Riluzole. Or, read Cytokinetics' Nov. 1, 2011, press release.
For details on participating in the trial, and for contact information, see A Study to Evaluate the Effects of Multiple Doses of CK-2017357 in Patients With ALS; or enter NCT01378676 into the search box at ClinicalTrials.gov.
 

tdamess

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sounds good do you know what CK-2017357 is excatley
 

KissJ

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Can I ask a dumb question? Is the Dex trial and this trial the same or different trials?
 

susanf

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Kiss, not dumb, I don't know either
 

apollick

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I just received this email over the weekend, they are moving and progressing and as I stated before I wish it was faster.

Congress Approves ALS Funding!

Dear Anne,

Great news! Just a few minutes ago, Congress approved legislation that provides an additional $6.4 million for the ALS Research Program (ALSRP) at the Department of Defense and almost $6 million more for the National ALS Registry. In a year when Congress sought to significantly cut or eliminate programs throughout the federal government, it is clear that finding the cause, treatment and cure for ALS is a priority for Congress. These victories would not have happened without the outreach of people with ALS and their families throughout the year. Your efforts to tell the ALS story once again have made a difference and are why both of these critical research programs have received vital funding for 2012.

Thank you to everyone who reached out to Congress!

The funding for the Registry and ALSRP was included as part of a larger spending bill that also funds nine of the 12 annual appropriations bills. President Obama is expected to sign the legislation into law.
 

Sequoia

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Just in case NP001 is not made available to PALS under early access, we, 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]

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

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.
 

Shane the Pain

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Hi Ya'll,

I went to clinicaltrials.gov and found out that people like me on a VPAP machine 24/7
are not eligible for this drug trial ! Oh the horror !

So Sequoia I am willing to join your Pilot 1 program and I meet with your four requirements.
If you need more information from me, send me a private message and I will respond.

Always great to hear good news !

Fight On,
Shane the Pain
 

Sequoia

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This is great....we now have 2 recruits and 18 more to go for our Pilot 1 access to drugs!
 

Sequoia

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I have posted this in other places so we now have about 7 recruits for the Pilot 1....we will keep this open until we have 20.
Some people are having trouble with our email...SO DO PM ME WITH YOUR EMAIL ADDRESS! I think if you dont have WIndow's Live it doesnt work, so just PM me here....
 

HelenL

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That's great Sequoia, thank you on behalf of all of us for the work you and Ed are doing to get this going!
 

pearshoot

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participated in this test at un of ks med cnt. the test was sucessful, safe, just a little dizzy which made me gab a lot. some thought i was on a cheap drunk but no hangover. the only strength test i could see the results of was hand squeeze (objective test) left hand went from 11lbs to 19 lbs, right hand from 43lbs to 50.6lbs. the pushy, pully tests (subjective test) i have no information, the neuro said her tests were not subjective of course i disagreed. don't know what level of drug i got but sure it was not a placebo effect. certainly glad i participated. go to the als clinic the end of the month. will be tested for another drug trial as i am keeping my name on a permenent roster. we live 185 miles from the clinic but this is whats importment to me.
 

Sequoia

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Wrong email address....the ALS-Treat Us Now! steering committee apologizes!

We will keep the Pilot One open until we have 20 folks.
 
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sadiemae

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There is a "Do It Yourself" trial going on with Sodium Chlorite, which is supposedly the main ingredient in NP001. My husband is going to start it next week, as soon as the stuff arrives. PM me if you want more info.
 

Sequoia

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We have reached the 20 for the Pilot 1...so if others are interested please PM me and I will put you on a list for Pilot 2. Thanks for all of you are participating in this! We may be able to have influence on how fast the FDA will give compassionate access to these promising drugs.
 
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