As a kid, I wanted to be an astronaut or a baseball player.
When I realized I was prone to motion sickness, I backed off the astronaut plan. Later, I learned I couldn’t hit a curveball.
Today, at 65, after a career in media, I have a new plan: to be among the first to recover from ALS, amyotrophic lateral sclerosis, which has an average survival rate of two to five years. I’m in year two.
I feel optimistic. Every week I read about potential breakthrough drugs. “Novel ALS therapy a game-changer,” declares one. “Treatment Helps Some Patients Improve,” says another. And so on.
However, our U.S. drug development system is slow and dysfunctional. Without systemic fixes, today’s promising ALS drugs will take 10 years to reach sick people like me.
For 35,000 Americans who have ALS, that’s essentially a death sentence.
Happily, there is a plan to speed up the system.
It’s called ACT for ALS, a bill pending in the House. An earlier effort like this streamlined the process of getting new treatments for 700-plus ALS patients, with results still pending.
That’s good news, but the funding for that effort — about $100 million yearly — is a fraction of what’s needed.
Five years ago, we ended the COVID crisis by investing $18 billion in just a few months. We saved untold millions of lives, calling the effort Operation Warp Speed.
Where is Operation Warp Speed for ALS?
There are more than 180 companies and more than 200 drugs in the ALS category pipeline, according to DelveInsight, a pharmaceutical market research firm. Virtually all of them are starving for investment.
They are stuck in a system that favors big, slow-moving pharmaceutical companies and stymies smaller, more nimble researchers. That’s the most harmful dysfunction right now.
The chief science officer at a small, cutting-edge research firm knows this all too well. He shared his frustration in personal correspondence with me, recounting 15 grant applications related to ALS that were all rejected after being reviewed by “key opinion leaders.”
These individuals — unknown outside the medical establishment — are paid by big pharmaceutical companies to review new drugs in development, and we’ve known for years that they tend to overlook drugs created by small companies.
In 2014, a watchdog at UC San Francisco found these opinion leaders are biased toward favorable conclusions about drugs developed by Big Pharma. That same year, the Los Angeles Times reported about their dark role in the opioid epidemic: Even as the lights flashed red, key opinion leaders promoted their patrons’ addictive drugs, which adversely affected 1 in 3 adults in the U.S.
Nature Reviews summed it up in 2021, saying key opinion leaders offer “opinion-based influence … rather than innovation and knowledge.”
Dismantling the problematic system is long overdue. Let’s put those small companies on a fair and level playing field with Big Pharma. Let’s do what the U.S. has long prided itself on doing: Champion the tenacious underdogs.
The Trump administration, for all its flaws, could play a positive role. The iconoclastic director of the National Institutes of Health, Jay Bhattacharya, has emphasized “encouraging different perspectives.” That’s the opposite of the current playbook.
Marty Makary, commissioner of the Food and Drug Administration (reportedly now on the ropes), declared on March 18 that the agency would speed the shift to human- and data-centric drug development pipelines, saying, “It’s faster, it’s more effective … and it’s better prediction.”
Although I can’t take credit for Makary’s direction, I said the same thing in a recent op-ed.
The spirit of attempting difficult things makes me feel like a kid again. Back then, whether I was anticipating an Apollo mission or the next battle between Willie Mays and Sandy Koufax, I was always looking forward to something.
My current heroes are not astronauts or hall-of-famers. They are ALS patients like me, willing to try investigative drugs, even as they realize there are no guarantees.
When I watched the live-streamed ACT for ALS news conference hosted by one of the bill’s sponsors, I was amazed by Brian Wallach and Dan Tate. Both have been living for nine years with their ALS diagnosis. Both are raising money for ALS research.
I feel like yelling, “Go team!”
At the event, Rep. Morgan Griffith (R-Va.), chairman of the House Subcommittee on Health, said something that made me smile big: “There is a belief that we may now have people who survive ALS.”
As another of my boyhood heroes, Hank Aaron, said: “Whether I was in a slump or feeling badly or having trouble off the field, my motto was always to keep swinging.”
As a country, let’s keep swinging at ALS, even if it is one of those darn curveballs.
Kevin J. Morrison is a consultant for nonprofit and other private-sector organizations and a senior producing director at Stanford University.
