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.

Sunday, March 18, 2018

Guest Blogger: It's Crunch Time. Take Your Shot!

It's crunch time for comments on the FDA ALS Guidance.  Time's almost up  Here are thoughts generously provided by a knowledgeable and relentless ALS advocate.  Please read and take your shot!
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Hey. Back to the “Considerations Regarding Food & Drug Administration Review and Regulation of Drugs for the Treatment of Amyotrophic Lateral Sclerosis”. We need help. It would be fantastic if we had an advocacy group taking the lead here but alas…. let’s move on. 

When it comes to drug/ therapy development we are failing. We have two approved drugs to “treat” ALS. One “may” extend life 3 months. One “may” slow down progression (while fleecing everyone involved). There is no question – ALS is a difficult disease to target. However, we are failing before we begin. We are currently using archaic protocols in drug development. The measure of success will never be met because we are using a yardstick that does not correspond to the disease. If we are ever going to make a dent in the utter havoc this disease wreaks on everyone it touches, we MUST change the way we approach it. 
We have one month left to make comments. PLEASE comment on the guidance for industry the FDA has proposed. I am including some suggestions below to get your thoughts rolling. Feel free to cut and paste any combination, should you agree with them. I am also including some complete comments. Change them up, use completely different ones. use them exactly. Whatever works for you. PLEASE speak up, share your thoughts, be heard. 
The FDA must encourage innovative clinical trial designs tailored to the population living with the disease and reflective of the heterogeneous nature of its presentation.  

This disease demands urgency. Use clinical trial designs which reflect the same. 
  • Push for the use of trial designs that realistically represent the number of people living with this disease at one time. 
  • Push for mobile travel sites in to allow a larger number of participants.
  • Push for remote data collection to make it easier on families to participate. 
  • Use historical controls from the PROACT data base vs. placebos. 
  • Widen participant eligibility criteria. 
  • Choose an endpoint other than death... a predesignated drop in FVC or specific point loss of ALSFRS. 
  • Consider taking guidance from people living with this disease.

Please do not accept this guidance as is or we will be having the same conversation in another 50 years. 
This guidance does not reflect the advances in technology, science, or data collection we have made this last century. We must encourage the industry to embrace innovate trial design that correlates to the number of people living with the disease at a given time. Use historical data, mobile trial sites and remote data collection. Change eligibility criteria so more people can participate without compromising quality of life interventions that currently exist like the feeding tube which allows people to maintain hydration and minimize the thickening of secretions. We already ask too much of these families - do NOT ask them to give up the few things we can offer. 
This proposed guidance will do nothing to accelerate the development of viable therapies to treat ALS. The status quo is NOT working and this guidance IS the status quo. We need the FDA to encourage and support innovative trial designs that make sense with a rare and terminal disease with rapid progression. Widen the base of participant eligibility, use mobile trial sites and remote data collection to include people who do not live near trial sites. Use historical controls. encourage accelerated approval. We must change the way this disease is approached because what we have done the past 50 years, what we are doing RIGHT NOW, is not working.
The guidance presented is antiquated, out of date, and demonstrates zero use of updated technology and data collection methods. This is a rare and fatal disease with no significant therapy. Trials used in drug development must reflect the number of people living with this disease at one time - the duration of the disease - the wide geographic locale of those diagnosed. There should be mobile trial sites and remote data collection, the use of historical data vs. placebo arms, eligibility criteria reflective of a savagely progressive disease. Please do not allow this blatant disrespect and disservice to the people from whom we already ask too much.

Click HERE to submit your comments.

Friday, March 16, 2018

You Are Smarter Than That

Over a decade ago we fought hard for a good ALS registry for the US -- a sound epidemiological census of people with ALS that would retain people's data and inform the science.

We fought very hard for it. We got it generous funding.  Perhaps we got too much funding for it.

After all these years and $78,000,000, it has simply not delivered.
  • The Registry has found a maximum of 15,927 people with ALS in the US (in its recent report that reflects 2014 data).  That's a number that nobody believes.  The CDC continues to depict ALS as white guys' disease based on its data.
  • This report was published in October on registry completeness,  It's a smoking gun. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5815913/  Whoa.  The National ALS Registry only found 57% of the people with ALS that smaller studies found.  Even more concerning was that the 43% the Registry didn't find were more people of color, more Hispanic, younger.
  • Look at scientific ALS papers that cite ALS data for the US.  They simply don't use this Registry's data.
  • For over a decade the project has permitted scope creep rather than focus and deliver its primary purpose -- "to better describe the incidence and prevalence of ALS in the United States."  Shoot, it doesn't even gather the data needed to calculate incidence.
  • The CDC can brag about using the Registry as an emailing system for clinical research notification, but in reality only a tiny percentage of enrolling ALS trials are included.  Even the scope creep isn't done well.
ALSA is telling you to ask for another $10,000,000 for this project.  You are being told that it's a success.  You are smarter than that.  

There are many lesser options to ask your legislators to consider --

  • Cease funding this project
  • Use the neurological disease surveillance included in 21st Century Cures to track ALS epidemiology (provided it does not use the same failed processes of the ALS Registry)
  • Decrease funding to well under $5.000,000 to allow basic operation of the ALS Registry
Do not let the tab for this disappointment grow to $88,000,000 as part of the charade that it is a success.  You are smarter than that.  Please speak up to legislators who are drawing up appropriations now.