Working to improve the fight for therapies for amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig’s disease) has been like a cruel version of 1950s game show “Beat the Clock,” in which grassroots advocates like me and many others manage to do everything that today’s version of Bud Collyer asks, even those last-minute wrinkles that seem impossible. We jump through hoops and figure out solutions and do it all according to the rules as the clock ticks down. We’ve heard applause from the audience. But we’ve never been given the prize.
We’ve been played.
I’ve been an advocate for better public policies and clinical research for ALS since my mom died from this beast in 1997. It was outrageous that her prognosis then was largely the same that Lou Gehrig faced in 1939, and is all the more outrageous today. ALS is still cruel, difficult, quickly fatal, and has no cure.
I’ve tried to do the right things since losing my mom: read, study, be informed. Talk to people who know processes and systems and can influence change. Think creatively. Go to meetings. Speak out. Find like-minded individuals and move the system in new directions.
Decades into the struggle for effective therapies, I’ve found myself among an incredibly talented and thoughtful group of people living with ALS and their caregivers who want to facilitate smart, practical, and needed change in how therapies for this devastating disease are developed and approved.
We thought we were close over the past couple years — close to more humane trials and faster approvals for ALS therapies, and to better access to investigational therapies for those dying from ALS.
In the past few weeks, though, we’ve learned we’re anything but close.
Clinical trial and regulatory processes are broken. When it’s time to translate theories about faster approvals into practice, nothing happens. The ALS community is in the same position today that Lou and Eleanor Gehrig faced in 1939 and my family faced in 1997. What’s more, there is a frustrating shroud of secrecy about what is really holding up approvals or access to investigational therapies that gives cover to the parties involved — physicians, researchers, regulators, industry, and sometimes even ALS advocacy organizations.
People with ALS and their caregivers want humane trials that get to the scientific truth more quickly, and have worked hard to get the Food and Drug Administration’s guidance for industry for developing ALS therapies to set out a plan to do just that.
Where is the FDA that nodded politely while we testified at its ALS hearing about urgency and flexibility? Americans dying of ALS now have to wait while the company has moved on to Canada and Europe for faster approval possibilities.
All of the meetings ALS families and advocates have had with the FDA and all of the encouragement its regulators offered ring hollow when the agency offers no information to the people most affected by its decision, and sponsors are hesitant to share it for fear of annoying the FDA, meaning we get only rumors and the occasional informative news report.
It appears to me and to others that FDA officials wanted Amylyx’s study to knock the agency’s socks off. But in all those encouraging FDA meetings, agency officials forgot to tell ALS families and advocates that, in addition to the trial hitting its endpoints, knocking socks off was also going to be required, and the socks clause certainly wasn’t included in the agency’s ALS guidance for industry.
In addition to faster, better paths to drug approval, people with ALS, their caregivers, and other advocates have been working to improve access to investigational therapies for people dying from ALS who can’t get into clinical trials. The FDA’s expanded access pathway was designed for a deadly disease like ALS — at least that’s the theory. Family members, physicians, and advocates talk and talk with sponsors over hours of meetings. Sponsors’ representatives nod — just like the FDA does — when asked about smart implementation of expanded access programs as part of their clinical research efforts.
Families and advocates do their homework. We know the rules about expanded access better than some sponsors do, and patiently counter concerns about cost or trial disruption. Sponsors’ representatives nod some more and schedule more Zoom meetings, but then don’t deliver expanded access in meaningful ways for people with ALS. A friend of mine with ALS put it this way: If they are just humoring dying people with no intention of changing anything, then I have better ways to spend my last months.
Think of an ALS clock: The day before being diagnosed with ALS, a 35-year-old has a life expectancy of around 40 years. On the day of diagnosis, that suddenly compresses to three or four years. This temporal shift should cause a sense of urgency, but it doesn’t, at least not for drug sponsors or the FDA, who I fear are using it to their advantage: The disruptors with ALS today will soon be dead, and the people who are nodding affirmatively but not acting may be just waiting them out.
The ALS community is tired of being played. Physicians, researchers, regulators, those in the pharmaceutical and biotech industries, and others need to show a fraction of the courage people with ALS display every day. It’s time to step up and deliver.
We’ve caught on to those polite nods. We won’t be played again.
Mary Catherine Collet is an advocate for better clinical research and public policy for those dealing with ALS. She volunteers for several organizations and blogs about ALS at ALS Advocacy.