An opinion article published on Mar. 19 discusses the ongoing challenges in developing precision drugs for amyotrophic lateral sclerosis (ALS), highlighting that economic barriers, rather than scientific ones, may be slowing progress. The author, who is living with ALS and participated in an expanded access program, points to a recent Government Accountability Office report that reviewed the impact of the Act for ALS, a law passed four years ago with $500 million in government funding.
The topic is significant because ALS remains a fatal disease affecting thousands of Americans each year, with most patients dying within two to five years of diagnosis. Despite increased funding and legislative support, only a small fraction of patients have benefited from experimental treatments under current programs.
According to the article, much of the Act for ALS funding has been used to provide experimental drugs to about 750 patients through expanded access programs—just 2.3% of those eligible—with costs averaging $167,000 per patient. The author notes that there was no requirement for sponsors to submit early efficacy data before providing these 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," the article states.
The author argues that ALS is not a single disease but varies widely among patients at both clinical and molecular levels. This diversity means that targeted or precision therapies are needed rather than one-size-fits-all solutions. Recent research has shown promise for such approaches: Biogen’s Qalsody was approved by the Food and Drug Administration in 2023 for a specific genetic subtype and has demonstrated positive results in slowing or partially reversing progression.
However, as therapies become more specialized, funding gaps between academic discovery and industry development—the so-called "valley of death"—are expected to widen. The article suggests adapting small business innovation research (SBIR) grants specifically for ALS could help bridge this gap by supporting high-risk early-stage research tailored to patient needs.
In conclusion, the author calls on Congress to address these economic barriers when considering renewal or updates to the Act for ALS later this year: "Patients don’t need false hope. We need drugs that work. Addressing the valley of death could save thousands of lives."
