ALS Advocacy

ALS Advocacy
Lou Gehrig's Disease - Motor Neuron Disease - Amyotrophic Lateral Sclerosis
Thought it had been cured by now? Still no known cause. Still no cure. Still quickly fatal. Still outrageous.

Thursday, July 21, 2011

Wow ... The Patients Are Taking Matters Into Their Own Hands

And they're making a big difference.

Right now there are some very interesting clinical trials being conducted for ALS. Over the years there have been hundreds of such "promising" trials, always with the quotation marks around "promising," and the disappointments and failures have been heartbreaking. This year it seems different. There are anecdotal reports of improvements in one trial. There is another that is a Phase III trial. Both are being led by some of the finest neurologists throughout the country. After all of the recent buzz and hope of finally removing the quotation marks from "promising," one fact remains -- we'll not know if these drugs will live up to the promise until the clinical trial seats are filled and the trials are completed.

One would think that patients with an untreatable, terminal disease would be knocking down the doors to get to these clinical trials. One would think. It seems that the process for getting information to ALS patients regarding clinical trials is horribly flawed. Many rely on their neurologists for information and guidance, yet often trials aren't introduced to patients and when they ask, the physician may not be familiar with the possibilities. Many rely on their trusted ALS organizations for information and guidance, yet we find a melange of outdated and incomplete information on their websites.

In the last few weeks, we have seen patients enrolled in trials taking matters into their own hands with some impressive results. Any patient who is enrolled in a clinical trial can be highly motivated to get the other seats in that trial filled so that it can move ahead. We see patients contacting others via social networking to encourage and inform regarding trials they are in. We see patients and caregivers asking trusted ALS organizations to speak up to patients regarding enrolling trials (and there have been some results). We see patients contributing to information online so that their peers can understand the relative advantages and disadvantages of trials. We even saw one patient who had traveled away from home for his monthly clinical trial infusion who dropped into a support group meeting in the clinical trial city. He found peers who were not aware of what was happening in their own back yard. One patient published a map online to show his peers all of the cities offering his trial. Facebook friends are posting information on walls wherever people with ALS and families might visit, and patients who do not qualify for trials are being a huge help to spread the word to others who might. Patients are posting their data at http://www.patientslikeme.com/ so that others may learn.

The system for spreading clinical trial information is broken. Some say there is no system. There is no excuse for a patient who qualifies for a clinical trial to be unaware of the possibility. Patients are leading the way right now. We hope that organizations and clinics and physicians will follow their lead and step it up a notch or ten!

5 comments:

  1. Will this be remembered as "The ALS Summer?"

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  2. Many brilliant ALS minds are tech savy. It's time to show the associations, clinics and medical community the e-patients revolution. We are all about making information available for patients to save patients' lives.
    Let Patients Rule!

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  3. I want to be on a phase III clinical trial now! I am being seen by ALS Clinic in Phila. I will go around the world for a "promising" trial. No one has informed me of any. Just, "this is your social worker. We'll see you in three months to check your deterioration." makes me angry! Where shall I turn next? Stacey
    Amiga.delmundo@yahoo.com

    <3

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  4. Stacey,
    There is a phase III Dexpramipexole trial that has a lot of patient interest and is currently recruiting -- http://www.clinicaltrials.gov/ct2/show/NCT01281189?term=dexpramipexole+amyotrophic&rank=3 It looks like there are two locations in Philly - One at Drexel is recruiting, one at ALS Center that is not yet recruiting. I suggest contacting ALSclinicaltrials@biogenidec.com (Biogen Idec is the pharma company sponsoring the trial) to get more information and to get in touch with the right person in Philadelphia.

    Another trial that is Phase II but has been getting a lot of buzz among patients is NP-001 (Neuraltus). http://www.clinicaltrials.gov/ct2/show/NCT01281631?term=amyotrophic+neuraltus&rank=1 It doesn't show a site in Penna, but, there is one not far from you in NYC and they have travel assistance available. Principal investigator on NYC is Hiroshi Mitsumoto, MD 212-305-8148. The NP-001 trial is open to patients up to 36 months from onset which is a wider window than some of the other trials.

    I'm not a healthcare professional, so please consult with the folks at the trial sites and with your doc for their advice. Also, there are other trials recruiting if you look at www.clinicaltrials.gov .

    I hope this might help. The system for getting clinical trial info to patients sure isn't working well.

    God bless.

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  5. Thank you! I spoke to someone at Columbia. Its too far I guess. They didn't offer travel assistance and 3 consec days of train travel for 6 months is too much HOWEVER I am to be screened for the Dex trial in a couple of weeks. Thank you!

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