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.
The U.S. Congress approved recently new federal research funding for amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, for a total of $315 million, the highest level of government funding ever for this disease research. Given February is Rare Disease Month, the timing could not be more appropriate. ALS affects approximately 30,000 to 31,000 people in the U.S.
ALS is a particularly cruel neurodegenerative condition, rapidly progressive and ultimately fatal, where the motor neurons responsible for controlling voluntary muscles are attacked, but cognitive functions remain intact. The disease has complex biology and patient variability with late diagnosis, confounded by irreversible neural damage, complicating treatment, said Dr. Olga Uspenskaya, Chief Medical Officer at VectorY Therapeutics.
“If you can slow or halt ALS even modestly, you’re not just changing a market, you’re changing the trajectory of life for almost every ALS patient,” Uspenskaya said.
Pharma and EverythingALS Collaboration
It took two years for Indu Navar’s husband to get an ALS diagnosis, after trying multiple physicians and practices, she said. Navar is the CEO and founder of the Peter Cohen Foundation, named after her spouse who died of the disease in 2019; the foundation operates as EverythingALS. It is a patient-focused non-profit that harnesses technological innovations and data science “to support efforts -- from care to cure -- for people with ALS, by offering a open-data platform for direct engagement with patients, caregivers, researchers and drug companies.”
EverythingALS has evolved to connect patients with drug developers, Navar said. Pharma partnerships, such as with VectorY, are important so drugmakers understand patients’ lived ALS experiences, she added. “We need to be very open about what we are going through so we can actually fix it,” she said. The collaboration can mitigate development bottlenecks, such as strict inclusion criteria that can exclude potential patients.
Almost two dozen pharma companies are part of an EverythingALS consortia, VectorY included. In addition to frequent virtual meetings with patients and researchers, there is an annual event where the group aligns on the ALS research and treatment roadmap for the next year.
EverythingALS has sought an FDA stamp of approval for granular speech metrics as clinical trial outcomes; the purpose is to link speech improvements to clinically meaningful outcomes, Navar said.
The organization has also commenced a monthly expert talk called HopeUntangled, in partnership with ALSUntangled. The website scientifically reviews alternative and off-label ALS treatments “to facilitate more informed decision-making.” The partnership aims to consider this research and guide patients on “what’s real and not real” in terms of medical alternatives, Navar said.
Optimism for Future Development
In terms of specific ALS pathology targets, VectorY is focused on the common pathological characteristic of TDP-43 proteinopathy. This disrupts multiple essential cellular processes and is now recognized as a central driver of modern neuron degeneration in up to 97% of patients, Uspenskaya said.
VectorY recently announced its first patient dosed in its Phase I/II PIONEER-ALS trial evaluating VTx-002, a vectorized antibody targeting TDP-43 pathology in ALS patients.
BioSpace reported in September 2025 on the new wave of ALS therapies in development, after a period befallen by the withdrawal of one of the scant marketed therapies, and a slew of trial failures.
The optimism is warranted, Uspenskaya said, considering “advances in genetics, biomarkers and multiomic technologies have revealed that additional factors influence ALS-onset phenotypes and progression rates and patterns.” This new clarity lends the field much higher confidence in developing better treatments, she added, with the potential to detect disease earlier, design biomarker-guided and genetically informed trials and develop therapies that target the specific molecular failure driving ALS.
You can hear more about this topic on Thursday’s Denatured podcast episode.
