Thread by @bsw5020

If you talk only of defeat, expect to lose. If you promise great victories without any basis in fact, expect to be proven wrong. If, however, you put in the work each day to change the world, you may just succeed.

Now if you will allow a moment on my soapbox I want to talk ALS.

For decades the fight against ALS languished. Doctors managed a patients death. ALS organizations carved up the community into my patients (read: donors) and yours. Pharma saw ALS as too risky. And the world knew little of our fight. /2

This instilled a language of defeatism into the fight. Doctors told patients at the 1st appointment there was nothing they could do. We expected drug trials would fail and we settled for leaders who pushed us to leave our estates to their org so we could help future patients. /3

That began to change scientifically in 2011 with genetic discoveries, but the language of our fight remained the same: patients were unheard, doctors defeated, progress ephemeral and public support absent. The narrative we bought into: progress will come in 20 years. /4

That narrative was bullshit. The last 2 years showed that.

Let’s start with treatments. Two treatments are coming before FDA soon, numerous trials are coming online in 2021, and the therapy pipeline is diverse and full and growing every day. /5

Let’s turn to the doctors. They pushed for the creation of a 53 center platform trial, they have discovered dozens of new genes, proteins and biomarkers linked to ALS and the best have begun using EAPs and therapies to fight the disease rather than manage our death. /6

Let’s turn to the patients. They have found their voice—real or synthetic—and pushed every part of the system to be built for and with patient input, to be focused on those alive today and to see us as subject matter experts to be heard, respected and valued in this fight. /7

Let’s turn to pharma. Next year alone they are looking to bring small molecule, ASO, AAV, and immunotherapy treatments to trial. They are seeking out patient input, making OLEs standard, expanding eligibility criteria for trials and placing larger bets in ALS. /8

Let’s turn to the public. A 200 member federal ALS caucus and NIH have helped increase research spending by tens of millions. The caucus introduced 2 critical bills, ACT and PPA, to fix flaws in our system that prevent patients from accessing promising therapies now. /9

The caucus will hopefully deliver SSDI reform after Thanksgiving and will continue to lead the fight to make real 21st century solutions to seemingly intractable problems.

There are those who will criticize this thread pointing out that patients are still denied therapies; /10

Pointing out that there still is no cure, that Congress has not passed critical reforms and that 17 people die every day in the US alone from ALS, including @PQuinnfortheWin today.

They are right—the fight rages on. What they miss though is that for the first time ever: /11

The outcome of the fight against ALS is not preordained. For the first time ever this is not a fight about 20 years from now but about today. About therapies that could impact my fight today, pathways to access today and breakthroughs today. /12

It is because patients, caregivers, advocates, doctors, researchers and yes pharma, pushed ahead, demanded better, and caused good trouble.

Now there are those that defend the old system, old vocabulary and tell us to sit down and shut up. /13

To them I say: don’t tell me what I can’t do nor what hope I can hold in my heart; instead join me in doing the work and changing the world. #EndALS /end