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.

Monday, October 29, 2012

Empowered Patients Hold The Key

People dealing with ALS often feel like passengers on an out-of-control train.  That is changing.  The train is still out-of-control, but patients are starting to find their way to the locomotive.

It's Q4 -- that time of year when people wait for publications and announcements of ALS clinical studies.  The big global ALS MND conference in December is a target date for researchers. In the past, patients and families have been forced to wait patiently on the out-of-control train for news.  Clinical trial data are top secret.  People die waiting for information.  Valuable data are in a secret folder marked, "Don't Open 'Til December ... (Maybe This Year, Maybe Next, We'll See)."

The following was posted by Jamie Heywood in the ALS forum at --

Dear ALS Community, 
PLM has been working to refine the tools we used in 2010 to show lithium was ineffective. We are continuously refining our ability to predict progression in ALS and to improve our understanding of the disease.  The purpose is to give you better ways of measuring your health outcomes and eventually show, in real-time, what is working and what’s not.  We have recently prepared a publication to talk about what it means for the collective ALS community to share this kind of data, specifically on the development process for therapies like NP001 and KNS-760704 (Dexpramipexole) as well as off-label or experimental use of treatments like lithium and sodium chlorite. 
Our goal in issuing this publication is to encourage a discussion and debate as to the best way to address the complex medical, ethical, and scientific issues surrounding the collection and sharing of such data. We would appreciate your feedback and thoughts.  This is still not peer-reviewed work, although we are submitting it to one of the major scientific journals.  There is still time for your voice as patients to be added. 
Below is a link to the paper, but here is a summary of our results:
These are based on the PLM members that reported using these treatments, which are detailed in the paper.  Note for reference ALS patients on PLM decline at an average of roughly 1 FRS point per month:
  • Lithium:
  •  as we reported before, has no effect on ALS progression.
  • Sodium Chlorite:
  •   we have an 80% confidence that it is worsening ALS-FRS progression in ALS patients and potentially by quite a bit.  We estimate the most likely result to be an worsening of the average FRS points/month of +0.69 after correcting for a placebo effect.
  • NP001:
  •  we have a 75% confidence that it is slowing the rate of decline of the ALS-FRS progression by more than 20% over the 4-month trial period.  We estimate the most likely result to be a slowing of the average FRS points/month of 0.41 for the combined treatment arms in the study.
  • KNS-760704 (Dexpramipexole):
  •  we have a 64% confidence that it is slowing the rate of decline of the ALS-FRS progression by more than 20% over the 12-month analysis period.  We estimate the most likely result to be a slowing of the average FRS points/month of  0.27 FRS point per month for the treatment arm of the study. 
Please note that the error bars on our data are large (which is why we keep asking you for more data).  Therefore, it’s possible that even if our PLM population represents what actually happens in the trial, the effect could be much larger or smaller.  It’s also possible that our group is different than the rest of the patients in the study. We do not encourage anyone to make any treatment decisions based on these preliminary scientific findings.  As always, you should consult your doctor whenever considering changing treatments.
The full paper can be downloaded here:
It’s been six years since we launched our ALS community and every person who has stopped here along the way has contributed to where we are on this journey.  We thank you for that.  Please tell us what you think and advise us on how to use this approach to serve you.
Thank You

People with ALS don't have time to wait until some December to make their decisions.  They are forced to play their lousy cards the best way they can every day.  Information and facts are the best antidote to those lousy cards.  Information like Jamie describes above is simply empowering.

For those dealing with ALS, please post your data at ... regardless of what you are or aren't trying.  It will enlighten you and other patients.  It will get you all closer to getting control of that train.

Please unlock your data from those secret clinical trial and medical files.  Please share. You own your data.  Please google "patient-reported outcomes" to learn more about the difference you can make.

You hold the key.

1 comment:

  1. Even though a lot of NP001 trial patients posted data at patientslikeme, it would have been so much more powerful today if more had. Ditto with Dexpramipexole, DPS, etc. Information is power. Patients own their own information. Patients can own that slice of power if they choose to.