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, July 27, 2021

We'll Have What They're Having

My message for the House Energy Committee Health Subcommittee Hearing coming up on Thursday, July 29, 2021.

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Everyone reading this letter already knows the urgency that ALS demands.

 

ALS has become an orphaned orphan disease.  That’s the worst.  

 

Ever since we lost Mom to ALS in 1997 I’ve written letters and testified to the FDA and written more letters and visited legislators.  We’re still an orphaned orphan disease.  

 

FDA

 

We want the same FDA that oncology has.  They have Project Facilitate to help with Expanded Access.  We don’t.  Why not?  

 

You can find 106 enrolling interventional trials for ALS at clinicaltrials.gov.  How many Expanded Access Programs are there?  Two.  And one of them is highly restricted.  Just two.

 

Something is not right here.  Other diseases have this Expanded Access path that also allows rigorous science to proceed while dying people can get access to therapies.  They even have an 800 number to help.  We in the world of ALS seem to get bogged down in opaque processes that move at the speed of concrete.  

 

NIH

 

We want the same NIH that other diseases have, too.

 

Sure, we appreciate the grants that the NIH makes toward a patchwork of ALS research.  But what about the expertise of the NIH that can make trials smarter and more accessible?  What about the knowledge of the NIH on how to do biorepositories better and more cost effectively than anyone else?  We need the NIH to become engaged with ALS for more than passing out grant money.  Please, we need the NIH that is known for the science that goes on at its headquarters for so many other diseases.

 

 

Is there any question as to why those dealing with ALS and their families feel that they have been orphaned?  We need for our federal agencies simply to do for ALS what they have done for other diseases.

 

That’s all.

 

Thank you.

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