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, May 29, 2014

More Qs For The Right-To-Try Discussions

The op-ed in USAToday leads us to believe that Neuralstem is full-speed-ahead with a Colorado Right-To-Try stem-cell program for people with ALS.

http://www.usatoday.com/story/opinion/2014/05/28/drugs-fda-approval-right-try-legislation-column/9692735/

The discussion is making us all think.

There are some thoughts and questions that I would like to add to the discourse --

  • Is anyone willing to commit to conscientious and open data collection for any people with ALS who enter a Neuralstem RTT program?  Colorado law does not have any data requirements that I am aware of.  We need to raise that bar voluntarily lest these programs be no better than the stem cell programs in Mexico and China that thrive on anecdotes rather than trustworthy data on all patients.
  • It would be very enlightening for us to see a specific head-on comparison of what Neuralstem and PALS would have to do in an FDA Compassionate-Use program versus a Colorado Right-to-Try program.  Also interesting is the question of whether any people with ALS had applied for Neuralstem compassionate use prior to this Right-to-Try push.
  • The USAToday op-ed again cites the truly miraculous outcome of Ted Harada who participated in the Neuralstem clinical trial.  We know that ALS is a spectrum of diseases.  Is anyone willing and able to provide a profile of Ted's ALS, e.g. known genes, onset details, pre-trial trajectory, etc.?  I would think that people with ALS that most closely resembles Ted's ALS would have the best roll of the dice in a Neuralstem Right-To-Try program. 
Thanks to all who are helping the conversation continue. I hope that it helps us figure out ways to move ahead more quickly and still advance the science for all with ALS.


No comments:

Post a Comment