Four years ago, Congress passed the Act for ALS and authorized $500 million in government funding to accelerate treatments for a disease that kills most patients in two to five years. Recently, the Government Accountability Office issued a much-awaited report on the law’s impact—and painted a complicated picture.
While the report lauded several scientific collaborations enabled by the Act for ALS, it found that much of the funding is being used to provide 750 patients with experimental drugs through expanded access programs. The problem? Besides the small number of patients benefiting (2.3% of those eligible) and the exorbitant per-patient cost ($167,000), there was no requirement for sponsors to submit early data indicating the potential efficacy of the drugs. NIH officials in charge of the program said this was a deliberate choice, telling GAO that “it was unclear whether NIH would have had any grants to fund if the review process included a review of efficacy.” One treatment, trehalose, went on to fail in late-stage trials; the jury is still out on the others.
As a person living with ALS who participated in one of these programs, I believe that patients deserve better. When the Act for ALS comes up for renewal later this year, Congress must address why so few promising drugs are available for expanded access. It must address ALS drug development’s “valley of death.”
ALS is a devastating condition that often strikes at random and robs those afflicted of their ability to move, speak, and breathe while keeping the mind intact. The lifetime risk is 1 in 400—similar to that of multiple sclerosis; 800,000 Americans alive today are predicted to develop ALS in their lifetimes.
I was 32 years old and recently married when I was first diagnosed. I was in excellent health. But then I started slurring my words after a drink. Soon, typing was tiring. After genetic tests revealed nothing and doctors ruled everything else out, I heard the three letters: ALS. I crumpled and cried.
When Congress passed the Act for ALS with bipartisan support, it gave me and the 33,000 other Americans living with ALS reason for hope. Coming seven years after the Ice Bucket Challenge raised over $115M for research, the law aimed to speed up new treatments by enabling faster FDA approvals, improving data collection, and creating compassionate use programs. But what the law’s drafters didn’t anticipate was that so many drugs would continue to fail.
After a demoralizing period punctuated by the withdrawal of one of the few marketed therapies for ALS, investment in new biotechs, state-backed collaborative initiatives, and buzz at BIO2025 suggest a new day in drug development for one of medicine’s most intractable diseases.
As I moved from despair to determination and began building a research effort to accelerate drug development, I learned that ALS is not a single condition. Like cancer, it varies from patient to patient in age of onset and patterns of progression. Some succumb in months; others endure years. In my case, alongside muscle weakness, I developed an atypical neurogenic stutter that doctors still can’t fully explain.
Over the past few years, scientists have found that these differences extend to the molecular level. This helps to explain why roughly 99% of ALS drug candidates fail and why so few truly promising drugs are available for expanded access. A therapy that helps one subgroup of patients may have no effect in another. This means that we may never find a single cure for ALS, just as there isn’t a single cure for cancer.
This evolving understanding suggests that the bigger barrier to progress over the coming decade may not be scientific; it might be economic—and failing to address this in “Act for ALS 2.0” risks maintaining the broken status quo.
If anyone thought that the current “one size fits all” approach to ALS drug development was working, data collected in 2025 indicate that what we really need is a basket of targeted, or precision, drugs.
In January, researchers at the Washington University School of Medicine showed that Biogen’s Qalsody—a precision medicine approved by the FDA in 2023 for 1–2% of patients with a particular genetic subtype—can significantly slow and, in some cases, even partially reverse ALS progression. Other papers showed how advances in AI, stem cell modeling, and molecular profiling will enable similar precision treatments, including for sporadic ALS, the disease’s most common and least understood form.
The U.S. Congress greenlit a historic $315 million in federal ALS research funding for 2026 amid Rare Disease Month, spotlighting biotech progress like VectorY Therapeutics’ first patient dosing in its TDP-43-targeting PIONEER-ALS trial and EverythingALS’ pharma consortia driving biomarker innovations and trial alignment.
As ALS moves into the precision medicine era, an ALS-optimized SBIR program in Act for ALS 2.0 could unleash a torrent of breakthroughs currently trapped in academic labs, bring thousands of new people and millions of dollars in private investment into the field, allow the field to benefit from the current AI revolution and greatly expand the quality and quantity of drugs reaching the clinic—enabling more effective expanded access programs for patients.
The GAO report should be a call to action. Patients don’t need false hope. We need drugs that work. Addressing the valley of death could save thousands of lives.
