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, May 27, 2014

Is "Right To Try" Right Right Now?

This is an extremely important discussion that affects everyone with ALS.

There are some new state laws intended to give people with untreatable, terminal conditions with a right to try experimental treatments.  The "right to try" name is awesome.  The concept makes sense to people who have a disease worse than any side-effect of any experiment.  The intentions are wonderful.

But I keep remembering my grandmother's words that the road to hell is paved with good intentions.  And if we provide experimental treatments to people with ALS, can we also advance science from their experiences?

I hope that the discussion will continue and grow in depth.  Already we have seen polarizing arguments based on emotion and lung power rather than on thoughtful consideration.

We clearly have a status quo that does not work for people with ALS.  We have failed.  We need to do better.

Here is the text of the legislation that recently passed in Arizona --
http://legiscan.com/AZ/text/HCR2005/2014

It's short and sweet.


“Right to Try” is a great name, but the devil is in the details.  Some of the details concern me a lot are --

  • What constitutes an “investigational drug?”
  • People with ALS are very vulnerable to hucksters, and unfortunately, some of those hucksters are even physicians.  Would this legislation permit anyone with a physician’s license (even a plastic surgeon or dermatologist) to make RTT therapy recommendations for a terminal patient with a complex neurological condition like ALS?
  • Do I understand correctly that the Colorado legislation allows the drug developer to charge patients for the investigational product? How do we prevent a system that excludes dying people who happen to be of limited means.  Will it be “Right to Try” or “Right to Buy?”
  • Attracting people to clinical trials is constant challenge in the fight against ALS.  How in the world would you ever get clinical trial volunteers (who have to agree to the coin-flip of a placebo)  if they had slam-dunk access to the real drug in a Right-to-Try program?
  • There are Expanded Access Programs that provide investigational drugs to people with ALS, and data are not tracked or reported.  We lose valuable clues both to ALS and to these experimental treatments when programs permit data to be lost.  My impression is that there is no requirement or incentive for drug developers to retain or report data on RTT participants.  If that is correct, it seems to me that we lose yet another opportunity to advance the science.  Patients not only have a right to try, they also have a right to have their data used constructively to help the next person.


Let the conversations continue. Please!  And let's come up with something that has both short-term and long-term benefits.

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