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 26, 2012

Let The Data Speak

Let the patients speak.  Better yet, encourage the patients to speak.

Yesterday we heard of a "promising" new treatment that is being tested in a clinical trial for safety on 12 patients.  We heard words like "exciting" and "compelling," but we didn't get any specifics.  It will be years (if ever) before we see the efficacy data to support those words of promise.  The words were music to the ears of those of us who are not scientists, but how much more meaningful would they have been if we could have seen some data voluntarily supplied by people in the trial.

Whether people with ALS are in a formal clinical trial or in a do-it-yourself trial, why not encourage them all to share their data in public place where others can benefit from insights into the data?  I know, it's just not done that way.

Why not?
  • In a double-blind, placebo-controlled trial, it's really a purer environment if the patients are clueless.  I get that. But this is ALS, a disease like no other.  Do both the patients in the trial and those on the sidelines not have an ethical right to know more?
  • If a couple of patients start to feel better, that could cause a placebo effect of lots of patients feeling better.  I get that, but this is ALS, a disease like no other.  If placebo can save these folks' lives, then we'll be quite happy.
  • If a couple of patients get a rash, that could cause everybody to start itching.  I get that, but this is ALS, a disease like no other.  People who put up with the things that ALS throws at them aren't likely to be deterred by side effects if the end goal of life is being met.
  • If the trial data aren't living up to the promise, it could scare people (including investors) away.  I get that, but this is ALS, a disease like no other.  It's the ultimate in knowing when to hold 'em and knowing when to fold 'em... in many ways.  
  • Lay people don't know how to interpret the data. I get that, but this is ALS, a disease like no other.  There are some very smart people who get ALS. 
  • There are all kinds of privacy concerns. I get that, but this is ALS, a disease like no other.  Data can be shared online ( http://www.patientslikeme.com/ comes to mind) under a creative screen name.  Encourage each person with ALS to share data and let each decide whether there are personal privacy concerns.
We struggle with regulatory systems that are slow and very protective.  Perhaps one of the keys to helping people with ALS is already in the hands of the people.  Perhaps all stakeholders should encourage the data to speak by encouraging the patients to share data.

Please let the conversation begin.

2 comments:

  1. Great writing. I agree that there is no side effect that is worse than ALS. The old saying that, "we want to make sure what we are doing is safe for ALS patients," is maddening. I'm smart, I can make choices, I can weigh options. Let me decide. What ever side effect I might experience can't be any worse than being in a wheelchair, living out a dying existence, "trying to make the best of it," while waiting for a treatment.

    Then there is the added bonus of having ALS for well past the 2 year criteria that most clinical trials require. When a clinical trial takes 5 years to complete, what the heck is in it for PALS who have the good fortune of living longer that the clinical trial criteria? Nothing. PALS might be dead by the time even phase 2 is completed. Is that safer than any side effect?

    Give me a break, please.

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  2. "First do no harm," takes on a new meaning when the disease itself is the ultimate harm.

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