The Tone-Deaf Won't Defeat ALS

For decades some people with ALS and caregivers have worked hard to nudge and push the fight in new directions.  Organizations and the FDA often appear to be listening, but in reality, they have been tone-deaf to ideas that will require big changes.  They are confident to stay their course, and besides, the annoying people demanding change eventually fade away with this quickly fatal disease.

Yesterday I went through transcripts of the February, 25, 2013, FDA hearing on ALS.  What a complete disappointment that none of the stakeholders in that room delivered on any of the ideas that people with ALS and their caregivers presented there.  Nothing.  That's tone-deaf.  That's a waste of time, money, energy, and, yes, lives.

"Stay the course" is nuts.  The failed status quo must go.  The tone-deafness must end.

A group of thoughtful, unfunded, grassroots people with ALS and those who care about them have found their voice thanks to social media.  Below is an announcement that they issued for your consideration.  They deserve to be heard by people who can actually grasp what they are saying and who are capable of change.

Members of the  ALS patient community are pleased to announce the creation of a new organization, Hope NOW for ALS, (www.HopenowforALS.org )  The  mission of this grassroots organization, comprised of people with ALS (PALS) and their families and friends,  is “to expedite access to promising investigational drugs for the current generation of PALS through the FDA’s Accelerated Approval Program (AAP) and other pathways, and to speed up ALS drug development by promoting improved alternative and humane designs for clinical trials.”   Hope NOW for ALS is hosting the “ALS Rally for Faster Drug Approval” in Washington D.C. on May 11 and hundreds are expected to attend from across the country. www.meetup.com/ALS-Faster-Drug-approval/events/221529700/ 

ALS, also known as Lou Gehrig’s Disease, is a fatal disease for which there is currently no cure and no effective treatment to stop the progression.  The average life expectancy after diagnosis is two to five years.  Some live longer but about half will pass away in under three years.  The genesis for Hope NOW for ALS was a petition, www.change.org/ALSMedication, started in January calling for “accelerated approval” of a promising new ALS drug called GM604.  Through the campaign, many in the patient community became aware of the outdated and broken drug approval system based on the 1962   “gold standard” or double blind randomized controlled trial (RCT) protocol required for new drugs to be approved.  This 53-year-old protocol requires Phase I, II, III trials that take 10 to 15 years and require tens of millions of dollars.  Many promising drugs never make it to Phase III due to the prohibitive cost, or because they don’t meet stringent FDA criteria (P=.05) that often dismisses data even when they demonstrate a drug is effective on a subset of the population.  The current generation of people with ALS has almost no hope for survival unless treatments become available much faster through the Accelerated Approval Program (AAP) adopted in 1992 as a result of AIDS-HIV activism.  AAP was designed to speed up drug approvals for serious conditions that have an unmet medical need.  Still, not a single drug for ALS has moved through the AAP process.  Hope NOW for ALS intends to change that. 

Nick Grillo, Jehad Majed, Eric Valor, and Linda Clark

Email:  info@HopenowforALS.org