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.

Tuesday, January 15, 2013

Which Out-of-Control Train Will He Board?


ALS has been compared to a ride on an out-of-control downhill train.  You can't stop it.  You are a passenger.  You do your best to cope.  The destination is never good.  No two people with ALS are on exactly the same train.

Some people's ALS speeding ride lasts only a few months.  Others are on a different itinerary and their downhill slide lasts for years.

You can't tell the timetable or itinerary of a case of ALS by taking a snapshot of a person with the disease, especially when the passenger has just stepped on the train.  Snapshots of passengers don't tell anything about the downhill slope ahead that the train will take.


When people are chosen for ALS clinical trials, the selection often focuses on people who have not been on their trains for long, without regard for the vastly different trains.

Do we somehow stack the clinical-trial deck by the way patients are chosen for ALS trials by time stamps on their tickets?



Every day scientists learn more about those trains -- the genetic variations, the potential biomarkers, the itineraries themselves.  Do ALS trials contain and follow sufficient diversity of trains?







2 comments:

  1. Couldn't have said it better than this ... but it seems to me that although this is so well known, nothing has been done to address it in over 100 years (at least no significant progress).

    Maybe this FEB 4-5 FDA Public Meeting will help progress in this area --> https://www.federalregister.gov/articles/2013/01/15/2013-00607/creating-an-alternative-approval-pathway-for-certain-drugs-intended-to-address-unmet-medical-need?utm_campaign=subscription+mailing+list&utm_medium=email&utm_source=federalregister.gov

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    1. Because nothing has been done to address subpopulations in #ALS c.t.s, it seems to me that we're two steps behind in what the FDA might be able and willing to do to expedite approvals for subpopulations.

      The spectrum of conditions that is ALS has been described by ALS scientists for over a decade, yet c.t. cohorts are filled based on when the ticket was stamped.

      Perhaps the FDA willingness to move based on strategic subpopulations will finally get our ALS organizations and scientists to smarter c.t. designs? I'm not a scientist, but I sure am familiar with the various out-of-control trains.

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